Download Medicines in Development Cancer

2014 REPORT
MEDICINES IN DEVELOPMENT
Cancer
PRESENTED BY AMERICA’S BIOPHARMACEUTICAL RESEARCH COMPANIES
Nearly 800 Medicines and Vaccines in Clinical
Testing For Cancer Offer New Hope to Patients
Medicines in Development for
Cancer
Application
Submitted
Phase III
Phase II
Phase I
98
87
78
73
56
L
Ca ung
nc
er
Ly
mp
ho
ma
Ov
a
Ca rian
nc
er
S
k
Ca in
nc
er
Br
Ca east
nc
er
Le
uk
em
ia
48
Some medicines are listed in more than one
category.
In recent decades, great progress has
been made in the fight against cancer.
Advances in molecular and genomic
research have unveiled complexities
and changed the perception of cancer,
which we now know is more than 200
unique diseases. Continued research
has expanded our knowledge of how
cancer develops and how to target
medicines for specific cancer types,
which has resulted in more effective
therapies for patients.
America’s biopharmaceutical companies
are responding to the needs of cancer
patients, working to develop more and
better treatments. Researchers are now
exploring new high-tech weapons to
fight the disease as well as ways to use
existing medicines, either alone or in
combination with other therapies, to
treat various cancers. The 771 medicines
and vaccines are either in clinical trials
or awaiting review by the U.S. Food and
Drug Administration (FDA) and include:
A cancer diagnosis once meant almost
certain death but with new therapies,
better testing, earlier diagnosis and
follow-up care, patients are often able
to live longer with a better quality of
life. According to the American Cancer
Society (ACS), the number of cancer
survivors living in the United States has
increased from 3 million in 1971 to 13.7
million in 2012.
• A monoclonal antibody for colorectal
cancer that targets a cell surface
protein that plays a role in tumor
growth and metastases;
Although great progress has been made
and continues to be made against
cancer, it remains the second leading
cause of death in the United States,
behind heart disease, accounting
for nearly 1 of every 4 deaths. The
ACS estimates that in 2014, more
than 1.6 million new cancer cases will
be diagnosed and nearly 600,000
Americans will die from cancer,
approximately 1,600 people per day.
Contents
Medicines and Vaccines in
Development to Treat Cancer ...............2
Advances in Cancer Treatment .............3
Value in Cancer Care
in the United States ............................5
Research Collaboration: Public-Private
Partnerships ...................................... 6
Facts About Cancer in the
United States .................................... 8
Medicines and Vaccines in
Development Chart ........................... 12
The Drug Discovery, Development
and Approval Process ...................... 103
Key Issues
• A monoclonal antibody for non-small-cell lung cancer that
blocks a signaling pathway allowing for immune responses
that can recognize and destroy cancer cells;
• A medicine that targets and disrupts a signaling pathway
associated with tumor growth in stomach cancer.
The development of new therapies would not be possible
without the patients who volunteer to participate in clinical
trials. Currently, there are 3,137 active clinical trials for
cancer in the United States. Of those, 1,824 are now seeking
volunteers to participate or have not yet started recruiting
patients; the other 1,313 are ongoing but are not currently
recruiting new patients.*
The progress made against cancer has resulted in better
treatments that help extend lives, improve patients’ quality
of life, and increase productivity contributing to economic
growth. The hundreds of medicines being developed are
our best hope for continuing that progress and lessening the
burden of cancer. However, researching and developing new
medicines remains a risky investment and a lengthy process.
On average, it costs $1.2 billion and takes between 10–15
years to bring a new, effective medicine to patients. Despite
those risks, America’s biopharmaceutical research companies
are continuing their efforts to develop effective new therapies
for the prevention and treatment of cancer.
Medicines and Vaccines in
Development to Treat Cancer
Many of the medicines in the pipeline today are using
novel approaches to attack cancer at the molecular level.
Approximately 80 percent of cancer pipeline drugs are
potentially first-in-class treatments, according to the Tufts
Center for the Study of Drug Development. Among the
771 medicines and vaccines in development are potential
treatments for:
Colorectal Cancer—A humanized monoclonal antibody in
development for metastatic colorectal cancer targets the cell
surface protein “endosialin,” which is expressed on cells that
are part of the tumor blood vessel structure. In preclinical
studies, blocking the function of endosialin inhibited tumor
growth and metastases.
Liver Cancer—A medicine in development for hepatocellular
carcinoma (liver cancer) is a small molecule kinase inhibitor
designed to selectively block transforming growth factor-beta
(TGF-beta) signaling. The overexpression of TGF-beta in cells
may enhance tumor growth and intensify metastases.
Source: www.clinicaltrials.gov, search performed 9/25/2014. Search criteria: cancer, United States, Phase 0, 1, 2, 3; industry only; excluding unknown status.
*
2
Medicines in Development
Cancer 2014
Key Issues
Lung Cancer—A fully human monoclonal antibody in
development for non-small-cell lung cancer targets the
PD-1 (programmed death-1) checkpoint receptor which is
expressed on activated T-cells. Cancer cells may exploit
the checkpoint pathway to hide from the immune system
and protect the tumor. Blocking this pathway may allow
for immune responses that recognize and destroy cancer
cells. Another medicine which blocks the PD-1 receptor was
recently approved by FDA for the treatment of malignant
melanoma.
Pancreatic Cancer—In malignant solid tumors, oxygen levels
are often low (called tumor hypoxia) relative to oxygen levels
in healthy tissue. Tumor hypoxia is associated with tumor
progression, metastases, and resistance to chemotherapy
and radiation treatment. A medicine in development is
activated when it reaches the hypoxic region of the tumor
environment where it eventually kills tumor cells in its vicinity.
The medicine is also being tested in patients with soft tissue
sarcoma.
Stomach Cancer—A monoclonal antibody in testing for
stomach cancer with high MET (mesenchymal-epithelial
transition factor) expression is designed to inhibit hepatocyte
growth factor (HGF)/scatter factor from binding to the
c-MET receptor. The HGF/c-Met signaling pathway is
thought to play a role in tumor growth and metastases in
many cancers, including stomach cancer. An exploratory
biomarker analysis found that the addition of the medicine to
chemotherapy improved median overall survival in patients
with tumors that expressed high levels of MET protein.
“There is no question that when we talk
about turning the tide against cancer,
the most exciting opportunities,
the new opportunities in fact, are
understanding the biology and
applying that biology to new treatments.
We are certainly at a turning point.”
—J. Leonard Lichtenfeld, M.D., American Cancer Society
Advances in Cancer Treatment
As we continue to advance cancer treatment, progress can
be seen in declining death rates from cancer and increases in
cancer survivors. According to the ACS, the death rate from
cancer has been steadily declining since the 1970s—declining
more than 15 percent in the period from 2000-2011 alone.
Five-year survival rates have increased 39 percent across all
cancer types since 1975, with 2 out of 3 people diagnosed
with cancer surviving at least five years.
To sustain this progress in an environment of increasing
pressure to contain health care costs, it is important to
understand the valuable role new medicines play in improving
patient outcomes. According to the ACS, 83 percent of
survival gains are attributable to new treatments, including
medicines. Some key advances in treatment include:
Personalized Medicines—Personalized medicines are
therapeutics that are tailored to individual patients based
on certain biologic and physiologic characteristics, such
as genetic makeup. Patients can be identified by their
susceptibility to a certain disease or how they will likely
respond to a specific therapy. By targeting therapies to
patients most likely to benefit, personalized medicines
represent an important tool, as they may reduce the use of
Medicines in Development
Cancer 2014
3
Key Issues
HOW CANCER COULD ECHO
HIV SUCCESS
Innovations in cancer treatment could echo the
success we have seen in HIV/AIDS treatment.
Advances in medicines for HIV/AIDS have
helped lower the death rate by 83 percent since
the disease was first recognized in 1981. Prior to
1995, when the first antiretroviral medicine was
approved by the FDA, an HIV diagnosis was
a death sentence. Now, thanks to medicines
developed by biopharmaceutical scientists and
their research partners, it is a chronic condition
with manageable costs, and patients are able
to reach nearly full life expectancy.
unnecessary and often costly treatments or procedures in
patients who are unlikely to benefit from them. Innovative
personalized medicines make up 12 to 50 percent of the
pipeline for all diseases, according to the Tufts Center for the
Study of Drug Development.
4
Targeted Therapies—Targeted therapies are designed to
interfere with specific molecular targets involved in cancer
cell growth or progression, whereas traditional chemotherapy
acts against all rapidly dividing cells, including healthy ones.
Several targeted therapies have been approved for use by
the FDA. According to IMS Health, the role of targeted
therapies in oncology treatment has grown from 11 percent in
2003 to 46 percent in 2013.
Therapeutic Cancer Vaccines—In the late 1990s, scientists
began experimenting with new vaccines that could harness
the power of the immune system to fight cancer rather
than to prevent it. One type of therapeutic vaccine, called
autologous cellular immunotherapy, takes a patient’s own
immune cells and reprograms them to attack the cancer. The
first and only therapeutic cancer vaccine was approved by
the FDA in 2010 to treat metastatic prostate cancer.
Preventive Vaccines—Preventive vaccines target viruses or
other infectious agents that can cause or contribute to the
development of cancer. They are intended to prevent people
from being infected with those viruses and agents so that
they do not develop cancer. For example, nearly all cases of
cervical cancer can be attributed to human papillomavirus
(HPV), with two HPV subtypes (types 16 and 18) alone
causing 70 percent of cervical cancer cases. The FDA has
Medicines in Development
Cancer 2014
“We used to think HIV costs would
overwhelm us…but we figured it out
and let drug development progress…
similarly, cancer care will evolve.”
—Ira Klein, M.D., M.B.A., FACP, Aetna
approved vaccines to prevent infection by HPV types 16 and
18 and vaccines to prevent hepatitis B virus, which can cause
liver cancer.
Value in Cancer Care in the
United States
New cancer medicines bring tremendous value to cancer
patients and the U.S. health care system. Medicines are
an important part of cancer treatment but represent just a
small portion of all cancer costs. When compared to total
Medicines in Development By Disease and Phase
Some medicines are listed in more than one category.
Bladder Cancer
Brain Cancer
Breast Cancer
Colorectal Cancer
Head/Neck Cancer
Hematological Malignancies
Kidney Cancer
Leukemia
Liver Cancer
Lung Cancer
Lymphoma
Myeloma
Ovarian Cancer
Pancreatic Cancer
Prostate Cancer
Sarcoma
Skin Cancer
Solid Tumors
Stomach Cancer
Other Cancers
Unspecified Cancer
Medicines in Development
Cancer 2014
Application
Submitted
Phase III
18
47
73
Phase II
46
19
Phase I
63
24
87
37
98
78
33
48
39
45
11
56
213
24
36
53
5
health care spending, cancer care represents an even smaller
portion of costs.
• Spending on cancer medicines represents less than
1 percent of all health care spending for all diseases
—$25.9 billion out of $2,800 billion.1
• Cancer medicines represent 20 percent of total spending
on cancer care.2
• Between 1988 and 2000, 23 million years of life were
saved due to cancer treatment advances. During that
time, the United States realized $1.9 trillion in value due
to improved productivity, extended life, and other factors
resulting from improved cancer treatment.3
• On average, privately insured cancer patients pay
out-of-pocket more than 20 percent of their total
prescription drug costs, compared to 4 percent of
inpatient hospital care and 7 percent of outpatient care.4
Sources: 1. IMS Institute for Healthcare Informatics, “Declining Medicine
Use and Costs: For Better or Worse? A Review of the Use of Medicines in
the United States in 2012.” 2. K. Fitch et al, “Benefit Designs for High Cost
Medical Conditions,” Milliman Research Report. 3. Lakdawalla et al, “An
Economic Evaluation of the War on Cancer,” Journal of Health Economics.
4. PhRMA analysis based on Medical Expenditure Panel Survey (MEPS).
Research Collaboration:
Public-Private Partnerships
As scientists probe deeper into the causes and signs of cancer,
the amount of information has increased exponentially, and
making sense of all the data is a colossal undertaking that no
single individual or institution can handle alone.
As a result, biopharmaceutical research companies are
working together and with academic medical centers,
government, and non-profit organizations to share, organize
and make sense of huge volumes of information. Below are
examples of collaboration to advance progress against cancer.
The Cancer Genome Atlas (TCGA) is a project to create a
catalog (or atlas) of genomic changes associated with specific
types of tumors to improve the prevention, diagnosis, and
treatment of cancer through the collection and analysis of
tissue samples. Sponsored by the National Cancer Institute
and the National Human Genome Research Institute, the TCGA
network includes scientists, bioinformaticians, bioethicists,
doctors, nurses, advocates, and others. TCGA is currently
focused on 32 tumor types, including nine rare cancers.
6
Medicines in Development
Cancer 2014
Lung-MAP (Lung Cancer Master Protocol) is a
multi-drug, multi-arm, biomarker-driven clinical trial focused
on developing new treatments for lung cancer. The trial
will recruit patients with advanced squamous cell lung
cancer, which represents approximately 25 percent of all
lung cancers, from more than 500 medical centers in the
United States. Unlike traditional clinical trials, Lung-MAP
uses advanced molecular screening to match patients
to an investigational treatment targeted to the genetic
alterations driving their cancer. Lung-MAP is a joint effort by
government, biopharmaceutical companies, patient advocacy
groups, and research institutions designed to improve clinical
trial efficiency and make new targeted therapies in lung
cancer available as quickly and safely as possible.
I-SPY 2 is a research collaboration among the Biomarkers
Consortium, the FDA, the U.S. National Institutes of Health,
and biopharmaceutical companies. I-SPY 2 uses genetic
and biological markers from individual patient tumors and
adaptive clinical trial design to develop new breast cancer
medicines quicker by identifying which ones are most
effective in which patients and at a reduced cost. I-SPY 2 is
focused on women with newly diagnosed, locally-advanced
breast cancer and will test whether adding investigational
drugs to standard chemotherapy is better than standard
chemotherapy alone prior to having surgery.
The CEO Roundtable on Cancer is committed to improving
the health of employees and making progress against
cancer. The Roundtable is comprised of executives from
diverse American corporations, nonprofit organizations
fighting cancer, and National Cancer Institute-designated
cancer centers. Programs initiated by the Roundtable
include the CEO Cancer Gold Standard, a workplace-based
wellness accreditation program that provides a framework
for employers to lower the risk of cancer, detect it early,
and ensure access to high-quality care. The Life Sciences
Consortium brings together leading biopharmaceutical
research companies focused on cancer in collaboration to
effectively deliver new medicines to prevent and treat cancer
and continue the emerging understanding of cancer biology.
RESEARCHING CANCER MEDICINES: SETBACKS AND STEPPING STONES
With the many advances made in cancer research, there are also setbacks. Researchers are discovering that
cancer is actually a set of more than 200 extremely complex diseases. Finding medicines that effectively
treat each one is an immense challenge. The path to new oncology drugs is littered with so-called failures
that do not make it through human clinical trials to approval.
Although it is very disappointing to see a promising drug candidate cut from the pipeline, researchers
learn from every setback and build upon each one of them to achieve success with future research and get
effective therapies to patients.
A new PhRMA report looks at setbacks and approvals in melanoma, brain cancer and lung cancer between
1998 and 2014:
• Researchers have had recent successes in treating melanoma with the approval of seven new
medicines, building on 96 medicines that didn’t make it through clinical trials.
• There has been progress against some subsets of lung cancer, but the disease remains deadly.
Ten lung cancer medicines were approved, while another 167 potential medicines failed in
clinical testing.
• Brain cancer continues to be a difficult cancer to treat despite concentrated research. There were
three approvals and 75 unsuccessful candidate medicines.
Although these setbacks are valuable to researchers, they are also an indication of the difficulty in
developing medicines to treat cancer. These setbacks serve as a reminder that to make progress, we need
a public policy framework that supports the drug development process as well as the promising science so
that important innovations can reach patients.
Medicines in Development
Cancer 2014
7
Facts
Facts About Cancer in the United States
Overview
•
Cancer is the second leading cause of death by disease—
nearly 1 of every 4 deaths—in the United States,
exceeded only by heart disease.1
•
Men have slightly less than a 1 in 2 lifetime risk of
developing cancer; for women, the risk is a little more
than 1 in 3. Approximately 77 percent of all cancers are
diagnosed in people ages 55 and older.1
•
•
•
•
•
•
•
8
•
Pancreatic cancer is the fourth leading cause of cancer
death. The average survival rate from pancreatic cancer
is low, in part because it is difficult to detect early. It is
most frequently diagnosed among people aged 75-84.2
•
This year alone, more than 1.6 million new cancer cases
are expected to be diagnosed, and an estimated 585,720
people are expected to die of cancer.1
Stomach cancer accounts for 1.3 percent of all new
cancer cases and mostly affects older people with an
average age of diagnosis at 69. Approximately 60
percent of people diagnosed with stomach cancer each
year are age 65 or older.2
•
Approximately 13.7 million Americans with a history of
cancer were alive in January 2012.1 The 5-year survival
rate for all cancers diagnosed between 2004 and 2010 is
66 percent, up from 49 percent in the mid-70s.2
Liver cancer and intrahepatic bile duct cancer is the
seventh leading cause of cancer death. It is also more
common among Asian/Pacific Islander and American
Indian/Alaska Native populations.2
•
In 2014, an estimated 10,450 new cases of cancer will be
diagnosed and 1,350 cancer deaths will occur in children
ages 0-14. Among adolescents ages 15-19, 5,330 new
cases will be diagnosed and 610 deaths will occur.1
•
The most common cancers among children are
acute lymphoblastic leukemia (26 percent), brain and
central nervous system (CNS) cancers (21 percent),
neuroblastoma (7 percent), and non-Hodgkin lymphoma
(6 percent).1
•
Among adolescents the most common cancers are
Hodgkin lymphoma (15 percent), thyroid carcinoma
(11 percent), brain and CNS cancers (10 percent), and
testicular germ cell tumors (8 percent).1
Lung cancer is the leading cause of cancer death in
both men and women. An estimated 159,260 deaths,
accounting for about 27 percent of all cancer deaths, are
expected to occur in 2014. From 2006 to 2010, rates
decreased 2.9 percent per year in men and 1.4 percent
per year in women.1
Colorectal cancer is the second leading cause of cancer
death and accounts for 8.2 percent of all new cancer
cases. It is more common in men than in women, and
more common in African Americans.2
Prostate cancer is the second leading cancer cause of
death in men with nearly 30,000 deaths estimated this
year. Prostate cancer death rates have been decreasing
since the early 1990s in men of all races/ethnicities,
though they remain more than twice as high in African
Americans as in any other ethnicity.1
Costs
•
An estimated 40,430 breast cancer deaths are expected
in 2014, with men accounting for 430 of those. Breast
cancer is the second cause of cancer death in women.
From 2006 to 2010, rates decreased 3.0 percent per
year in women under age 50 and 1.8 percent per year
in women 50 and older. The decrease in breast cancer
death rates represents improvements in early detection
and treatment, and possibly decreased incidence.1
Although extraordinary advances are being made
against cancer, the disease remains a major health care
challenge and a huge financial burden, both nationally
and internationally.3
•
This year nearly 300,000 new cases of gastrointestinal
cancer will be diagnosed and nearly 150,000 deaths will
occur in the United States.2
The National Institutes of Health (NIH) estimates the
overall costs for cancer in 2009 at $216.6 billion:
$86.6 billion for direct medical costs (total of all health
expenditures) and $130 billion for indirect mortality costs
(cost of lost productivity due to premature death).3
•
Globally, 12.9 million new cancer cases cost $286 billion
in 2009. In 2030, the 21.5 million projected new cancer
cases will cost the world economy $458 billion.3
Medicines in Development
Cancer 2014
Facts
New Cancer Cases/Deaths 1
Cancer Type
Estimated 2014 New Cases
Estimated 2014 Deaths
All Sites ........................................................... 1,665,540 ............................................................. 585,720
Male ....................................................... 855,220 ..............................................................310,010
Female .................................................... 810,320 .............................................................. 275,710
Acute Lymphocytic Leukemia ................................... 6,020 .................................................................1,440
Male ........................................................... 3,140 ................................................................... 810
Female ....................................................... 2,880 ................................................................... 630
Acute Myeloid Leukemia .........................................18,860 ...............................................................10,460
Male ..........................................................11,530 .................................................................6,010
Female ........................................................ 7,330 ................................................................ 4,450
Bladder Cancer ......................................................74,690 ............................................................... 15,580
Male ........................................................ 56,390 .................................................................11,170
Female ......................................................18,300 .................................................................4,410
Brain Cancer (and other nervous system) ...................23,380 ............................................................... 14,320
Male ......................................................... 12,820 ................................................................ 8,090
Female ...................................................... 10,560 ................................................................ 6,230
Breast Cancer ......................................................235,030 .............................................................. 40,430
Male .......................................................... 2,360 ................................................................... 430
Female .................................................... 232,670 .............................................................. 40,000
Cervical and Uterine Cancers .................................. 64,990 ................................................................12,610
Male ................................................................— ......................................................................—
Female ..................................................... 64,990 ................................................................12,610
Chronic Lymphocytic Leukemia .................................15,720 ................................................................ 4,600
Male ........................................................... 9,100 ................................................................ 2,800
Female ....................................................... 6,620 .................................................................1,800
Chronic Myeloid Leukemia ....................................... 5,980 ................................................................... 810
Male ........................................................... 3,130 ................................................................... 550
Female ....................................................... 2,850 ................................................................... 260
table continued on next page
Medicines in Development
Cancer 2014
9
Facts
New Cancer Cases/Deaths 1 (continued)
Cancer Type
Estimated 2014 New Cases
Estimated 2014 Deaths
Colorectal Cancer ................................................. 136,830 ............................................................... 50,310
Male ......................................................... 71,830 ............................................................... 26,270
Female ..................................................... 65,000 .............................................................. 24,040
Esophageal Cancer .................................................. 18,170 ............................................................... 15,450
Male .........................................................14,660 ............................................................... 12,450
Female ........................................................ 3,510 ................................................................ 3,000
Kidney Cancer .......................................................63,920 ............................................................... 13,860
Male ......................................................... 39,140 ................................................................ 8,900
Female ......................................................24,780 ................................................................ 4,960
Leukemia (all types) ................................................52,380 .............................................................. 24,090
Male ......................................................... 30,100 ...............................................................14,040
Female ......................................................22,280 ............................................................... 10,050
Liver Cancer (and bile duct) ..................................... 33,190 .............................................................. 23,000
Male ........................................................ 24,600 ............................................................... 15,870
Female ....................................................... 8,590 ..................................................................7,130
Lung Cancer (and bronchus)................................... 224,210 ............................................................. 159,260
Male ....................................................... 116,000 .............................................................. 86,930
Female .....................................................108,210 ............................................................... 72,330
Lymphoma (all types) ..............................................79,990 ................................................................20,170
Male ........................................................ 43,340 ................................................................ 11,140
Female ..................................................... 36,650 ................................................................ 9,030
Melanoma-Skin Cancer ........................................... 76,100 ................................................................. 9,710
Male ........................................................ 43,890 .................................................................6,470
Female ...................................................... 32,210 .................................................................3,240
Myeloma ..............................................................24,050 ............................................................... 11,090
Male ......................................................... 13,500 ..................................................................6,110
Female ...................................................... 10,550 ................................................................ 4,980
Non-Hodgkin Lymphoma ....................................... 70,800 ...............................................................18,990
Male .........................................................38,270 ............................................................... 10,470
Female ......................................................32,530 .................................................................8,520
table continued on next page
10
Medicines in Development
Cancer 2014
Facts
New Cancer Cases/Deaths 1 (continued)
Cancer Type
Estimated 2014 New Cases
Estimated 2014 Deaths
Ovarian Cancer ...................................................... 21,980 ............................................................... 14,270
Male ................................................................— ......................................................................—
Female ...................................................... 21,980 ............................................................... 14,270
Pancreatic Cancer ................................................. 46,420 ...............................................................39,590
Male .........................................................23,530 ................................................................20,170
Female ......................................................22,890 ............................................................... 19,420
Prostate Cancer ...................................................233,000 .............................................................. 29,480
Male .......................................................233,000 .............................................................. 29,480
Female .............................................................— ......................................................................—
Stomach Cancer ..................................................... 22,220 ...............................................................10,990
Male ..........................................................13,730 .................................................................6,720
Female ....................................................... 8,490 .................................................................4,270
Thyroid Cancer ..................................................... 62,980 .................................................................1,890
Male ..........................................................15,190 ................................................................... 830
Female ...................................................... 47,790 .................................................................1,060
Sources:
1.
Cancer Facts & Figures 2014, American Cancer Society,
www.cancer.org
2. National Cancer Institute, www.cancer.gov
3. AACR Cancer Progress Report 2014, American Association
for Cancer Research, www.cancerprogressreport.org
Medicines in Development
Cancer 2014
11
Medicines and Vaccines in Development for Cancer
Bladder Cancer
Product Name
Sponsor
Indication
Development Phase*
ABI-009
(nanoparticle albumin-bound
mTOR inhibitor)
Aadi
Pacific Palisades, CA
non-muscle invasive bladder cancer
Phase I/II
ALT-801
(immunotherapy-fusion protein)
Altor BioScience
Miramar, FL
advanced bladder cancer, non-muscle
invasive bladder cancer
Phase II
www.altorbioscience.com
ALT-803
(IL-15 superagonist complex)
Altor BioScience
Miramar, FL
non-muscle invasive bladder cancer
(see also hematological, myeloma,
skin)
Phase I/II
www.altorbioscience.com
apatorsen
(Hsp27 inhibitor)
OncoGenex Pharmaceuticals
Bothell, WA
metastatic bladder cancer
(see also lung, pancreatic, prostate)
Phase II
www.oncogenex.com
apaziquone
(DNA synthesis inhibitor)
Spectrum Pharmaceuticals
Henderson, NV
non-muscle invasive bladder cancer
(Fast Track)
Phase III
www.sppirx.com
ASG-15ME
(antibody drug conjugate)
Agensys
Santa Monica, CA
Seattle Genetics
Bothell, WA
bladder cancer
Phase I
www.agensys.com
www.seattlegenetics.com
CG0070
(oncolytic virus therapy)
Cold Genesys
Newport Beach, CA
non-muscle invasive bladder cancer
Phase II/III
www.coldgenesys.net
CV-301
(targeted immunotherapy)
Bavarian Nordic
Mountain View, CA
bladder cancer
(see also breast, colorectal)
Phase II completed
www.bavarian-nordic.com
Cynviloq™
paclitaxel polymeric micelle
for injection
Sorrento Therapeutics
San Diego, CA
bladder cancer
(see also breast, lung, ovarian,
pancreatic)
Phase II
www.sorrentotherapeutics.com
Cyramza®
ramucirumab
Lilly
Indianapolis, IN
bladder cancer
(see also breast, colorectal, liver, lung,
ovarian, solid tumors, stomach)
Phase II
www.lilly.com
instiladrin
(gene therapy)
FKD Therapies
Kuopio, Finland
superficial bladder cancer
Phase II
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
bladder cancer
(see also breast, head/neck,
hematological, kidney, lung,
myeloma, stomach)
Phase I
www.merck.com
*For more information about a specific medicine or company in this report, please use the website provided.
12
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancer
Bladder Cancer
Product Name
Sponsor
Indication
Development Phase*
MAGE-A3 immunotherapeutic
(recombinant antigen-specific
cancer immunotherapeutic)
GlaxoSmithKline
Research Triangle Park, NC
bladder cancer
Phase II
www.gsk.com
mocetinostat
(HDAC inhibitor)
Mirati Therapeutics
San Diego, CA
bladder cancer
(see also hematological, lymphoma)
Phase II
www.mirati.com
mycobacterium cell wall-DNA
complex (MCNA)
Bioniche Life Sciences
Belleville, Canada
refractory non-muscle invasive
bladder cancer
Phase II
www.bioniche.com
RG7446
(anti-PD-L1 mAb)
Genentech
South San Francisco, CA
metastatic urothelial bladder cancer
(Breakthrough Therapy)
(see also kidney, lung, skin)
Phase II
www.gene.com
TMX-101
(TLR7 agonist)
Telormedix
Bioggio, Switzerland
bladder cancer
Phase II
www.telormedix.com
vesigenurtacel-L
(cancer immunotherapy)
Heat Biologics
Durham, NC
bladder cancer
Phae I/II
www.heatbio.com
Product Name
Sponsor
Indication
Development Phase
2B3-101
(doxorubicin liposomal)
to-BBB technologies
Leiden, Netherlands
brain metastases, recurrent malignant
glioma
Phase II
www.tobbb.com
AbbVie
North Chicago, IL
glioblastoma multiforme
(see also solid tumors)
Phase I
www.abbvie.com
ADU-623
(bivalent Listeria-based
immunotherapy)
Aduro Biotech
Berkeley, CA
glioblastoma
Phase I
www.adurobiotech.com
aldoxorubicin
(delivery-enhanced doxorubicin)
CytRx
Los Angeles, CA
glioblastoma multiforme
(see also lung, sarcoma)
Phase II
www.cytrx.com
AMG 595
(tubulin inhibitor)
Amgen
Thousand Oaks, CA
advanced malignant glioma,
anaplastic astrocytomas,
glioblastoma multiforme
Phase I
www.amgen.com
ANG1005
(targeted taxane derivative)
Angiochem
Montreal, Canada
brain metastases, malignant glioma
(Fast Track)
Phase II
www.angiochem.com
Brain Cancer
ORPHAN DRUG
ABT-414
(antibody drug conjugate)
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
13
Medicines and Vaccines in Development for Cancer
Brain Cancer
Product Name
Sponsor
Indication
Development Phase
antineoplaston
Burzynski Research Institute
Houston, TX
glioma (combination therapy)
ORPHAN DRUG
Phase II
www.burzynskiresearch.com
Avastin®
bevacizumab
Genentech
South San Francisco, CA
glioblastoma multiforme (1st-line)
(see also lung, ovarian, other)
Phase III completed
www.gene.com
Azedra®
iobenguane I-131
Progenics Pharmaceuticals
Tarrytown, NY
paraganglioma, malignant
pheochromocytoma
(Fast Track)
Phase II
www.progenics.com
BGJ398
(FGF-R kinase inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
glioblastoma
(see also hematological, liver, solid
tumors)
Phase II
www.novartis.com
cancer gene therapy
(AdV-tK)
Advantagene
Auburndale, MA
anaplastic astrocytoma,
glioblastoma, malignant glioma,
recurrent ependymoma
(see also pancreatic, prostate)
Phase I/II
www.advantagene.com
CPP-1X
(eflornithine)
Cancer Prevention Pharmaceuticals
Tucson, AZ
neuroblastoma
(see also colorectal)
Phase II
www.canprevent.com
crenolanib
(PDGFR inhibitor)
AROG Pharmaceuticals
Dallas, TX
glioma (pediatric)
(see also stomach)
Phase I
www.arogpharma.com
CTO
(multiple oncokinase inhibitor)
Tactical Therapeutics
New York, NY
glioma (combination therapy)
(Fast Track)
Phase II
www.tacticaltherapeutics.com
DCVax®-L
dendritic cell vaccine
Northwest Biotherapeutics
Bethesda, MD
glioblastoma multiforme
Phase III
www.nwbio.com
DM-CHOC-PEN
DEKK-TEC
New Orleans, LA
brain tumors
Phase II
www.dekk-tec.com
DNX-2401
(cell death stimulant)
DNAtrix
Houston, TX
recurrent glioblastoma
(Fast Track)
Phase I
www.dnatrix.com
E7080
(multi-targeted kinase inhibitor)
Eisai
Woodcliff Lake, NJ
glioma
(see also liver, lung, skin, other)
Phase II
www.eisai.com
EGFRvIII CAR
(chimeric antigen receptor)
Kite Pharma
Santa Monica, CA
National Cancer Institute
Bethesda, MD
glioblastoma
Phase I/II
www.kitepharma.com
ERC-1671
(cancer vaccine)
Epitopoietic Research
Les Isnes, Belgium
glioblastoma, gliosarcoma
Phase II
www.ercbelgium.com
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
14
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancer
Brain Cancer
Product Name
Sponsor
Indication
Development Phase
G-202
(calcium-transporting ATPase
inhibitor)
GenSpera
San Antonio, TX
glioblastoma multiforme
(see also liver, prostate)
Phase II
www.genspera.com
galunisertib
(TGF-beta1 inhibitor)
Lilly
Indianapolis, IN
glioblastoma, glioma
(see also liver, pancreatic)
Phase II
www.lilly.com
heat shock protein vaccine
ORPHAN DRUG
Agenus
Lexington, MA
recurrent glioblastoma multiforme,
newly diagnosed glioma
(see also skin)
Phase II
www.agenusbio.com
ICT-107
(dendritic cell-based vaccine)
ImmunoCellular Therapeutics
Calabasas, CA
glioblastoma (1st-line)
Phase II
www.imuc.com
ICT-121
(dendritic cell vaccine)
ImmunoCellular Therapeutics
Calabasas, CA
recurrent glioblastoma
Phase I
www.imuc.com
KPT-330
(XPO1 inhibitor)
Karyopharm Therapeutics
Newton, MA
glioblastoma multiforme
(see also leukemia, prostate, solid
tumors, other)
Phase II
www.karyopharm.com
KX02
Kinex Pharmaceuticals
Buffalo, NY
glioma
Phase I
www.kinexpharma.com
LDE225
(SMO protein inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
medulloblastoma
(see also skin, solid tumors)
Phase II
www.novartis.com
macitentan
Actelion Pharmaceuticals
South San Francisco, CA
glioblastoma
Phase I
www.actelion.com
mibefradil
(t-type calcium channel
antagonist)
Cavion
Charlottesville, VA
recurrent high-grade glioma
Phase I
www.cavion.com
mipsagargin
(ATPase inhibitor)
GenSpera
San Antonio, TX
glioblastoma multiforme
(see also liver, prostate)
Phase II
www.genspera.com
MM-398
(irinotecan nanotherapeutic)
Merrimack Pharmaceuticals
Cambridge, MA
glioma
(see also colorectal, pancreatic)
Phase III
www.merrimackpharma.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
glioblastoma
(see also colorectal, head/neck,
hematological, kidney, liver, lung,
lymphoma, skin, solid tumors)
Phase II
www.bms.com
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
15
Medicines and Vaccines in Development for Cancer
Brain Cancer
Product Name
Sponsor
Indication
Development Phase
nivolumab + Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
glioblastoma
(see also colorectal, head/neck,
hematological, kidney, lung, skin)
Phase II
www.bms.com
PEG-SN38
(angiogenesis inhibitor)
Belrose Pharma
Princeton, NJ
pediatric neuroblastoma
(see also breast, solid tumors)
Phase I
www.belrosepharma.com
PLX3397
(Fms/Kit/Flt3-ITD inhibitor)
Daiichi Sankyo
Parsippany, NJ
Plexxikon
Berkeley, CA
glioblastoma
(see also leukemia, skin)
Phase II
www.dsi.com
www.plexxicon.com
RG7666
(PI3K inhibitor)
Genentech
South San Francisco, CA
glioblastoma (2nd-line)
Phase I
www.gene.com
rindopepimut
(EGFRvIII antagonist)
Celldex Therapeutics
Hampton, NJ
glioblastoma (1st-line)
(Fast Track)
----------------------------------------recurrent glioblastoma
(combination therapy)
(Fast Track)
Phase III
www.celldex.com
----------------------------------------Phase II
www.celldex.com
SL-701
(cancer immunotherapy)
Stemline Therapeutics
New York, NY
glioblastoma multiforme
Phase I/II
www.stemline.com
Toca 511 & Toca FC
(flucytosine gene therapy)
Tocagen
San Diego, CA
high-grade gliomas
Phase I/II
www.tocagen.com
TPI 287
(microtubule stabilizing taxoid)
Cortice Biosciences
New York, NY
recurrent glioblastoma,
neuroblastoma
----------------------------------------breast cancer brain metastases,
secondary brain metastases
Phase II
www.corticebio.com
----------------------------------------Phase I
www.corticebio.com
TRC105
(ENG protein inhibitor)
TRACON Pharmaceuticals
San Diego, CA
glioblastoma multiforme
(see also kidney, sarcoma)
Phase II
www.traconpharma.com
TSC
(trans sodium crocetinate)
Diffusion Pharmaceuticals
Charlottesville, VA
glioblastoma
Phase I/II
www.diffusionpharma.com
TVI-Brain-1
(personalized cancer
immunotherapy)
TVAX Biomedical
Lenexa, KS
late-stage glioma
Phase II completed
www.tvaxbiomedical.com
VAL-083
DelMar Pharmaceuticals
Vancouver, Canada
glioblastoma
Phase I/II
www.delmarpharma.com
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
16
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancer
Brain Cancer
Product Name
Sponsor
Indication
Development Phase
VB-111
(vascular targeting system)
VBL Therapeutics
Or Yehuda, Israel
recurrent glioblastoma multiforme
(Fast Track)
(see also ovarian, other)
Phase I/II
www.vblrx.com
AbbVie
North Chicago, IL
brain metastases
(see also breast, lung)
Phase I/II
www.abbvie.com
Product Name
Sponsor
Indication
Development Phase
abemaciclib
(CDK 4/6 inhibitor)
Lilly
Indianapolis, IN
hormone receptor positive,
HER2-negative breast cancer
(see also lung, lymphoma)
----------------------------------------metastatic breast cancer
Phase III
www.lilly.com
ORPHAN DRUG
veliparib
Breast Cancer
----------------------------------------Phase II
www.lilly.com
Abraxane®
paclitaxel protein-bound particles
for injectable suspension,
albumin-bound
Celgene
Summit, NJ
triple-negative breast cancer
(1st-line)
(see also pancreatic)
Phase III
www.celgene.com
ADI-PEG 20
(PEG arginine deiminase)
Polaris Pharmaceuticals
San Diego, CA
HER2 negative breast cancer
(see also leukemia, liver, lung,
lymphoma, skin)
Phase I
www.polarispharma.com
Ad-RTS-IL-12
(interleukin-12 gene therapy)
ZIOPHARM Oncology
Boston, MA
breast cancer
(see also skin)
Phase II
www.ziopharm.com
AE37
(immunotherapeutic)
Antigen Express
Worcester, MA
HER2/neu positive breast cancer
(see also prostate)
Phase II
www.antigenexpress.com
Afinitor®
everolimus
Novartis Pharmaceuticals
East Hanover, NJ
HER2-positive breast cancer
(1st-line), HER2-positive breast cancer
(2nd/3rd-line)
(see also lung, lymphoma, stomach)
Phase III
www.novartis.com
ARN-810
(selective estrogen receptor
degrader)
Seragon Pharmaceuticals
San Diego, CA
breast cancer
Phase I
www.seragonpharm.com
ARRY-380/ONT-380
(HER2 inhibitor)
Array BioPharma
Boulder, CO
Oncothyreon
Seattle, WA
breast cancer
Phase I
www.arraybiopharma.com
www.oncothyreon.com
Medicines in Development
Cancer 2014
17
Medicines and Vaccines in Development for Cancer
Breast Cancer
Product Name
Sponsor
Indication
Development Phase
AV-203
(ERBB-3 receptor antagonist)
AVEO Oncology
Cambridge, MA
early breast cancer
Phase I completed
www.aveooncology.com
AZD5363
(AKT kinase inhibitor)
AstraZeneca
Wilmington, DE
breast cancer
Phase II
www.astrazeneca.com
bavituximab
Peregrine Pharmaceuticals
Tustin, CA
HER2-negative breast cancer
(see also liver, lung, skin, other)
Phase I
www.peregrineinc.com
BBI608
(cancer stemness kinase inhibitor)
Boston Biomedical
Cambridge, MA
breast cancer
(see also colorectal, lung, ovarian,
skin, stomach)
Phase I/II
www.bostonbiomedical.com
BKM120
(PI3K inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
metastatic breast cancer
(ER-positive AI resistant/mTOR naïve)
(see also solid tumors)
----------------------------------------metastatic breast cancer
(ER-positive post AI and mTOR inhibitor)
Phase III
www.novartis.com
----------------------------------------Phase II
www.novartis.com
BMN 673
(PARP inhibitor)
BioMarin Pharmaceutical
Novato, CA
metastatic breast cancer
Phase III
www.bmrn.com
BZL-101
(glycolysis inhibitor)
Bionovo
Emeryville, CA
breast cancer
Phase I
www.bionovo.com
CV-301
(targeted immunotherapy)
Bavarian Nordic
Mountain View, CA
breast cancer
(see also bladder, colorectal)
Phase II completed
www.bavarian-nordic.com
Cynviloq™
paclitaxel polymeric micelle
for injection
Sorrento Therapeutics
San Diego, CA
metastatic breast cancer
(see also bladder, lung, ovarian,
pancreatic)
Phase III
www.sorrentotherapeutics.com
Cyramza®
ramucirumab
Lilly
Indianapolis, IN
breast cancer (combination therapy)
(see also bladder, colorectal, liver,
lung, ovarian, solid tumors, stomach)
Phase II
www.lilly.com
eniluracil
Adherex Technologies
Research Triangle Park, NC
breast cancer
Phase II completed
www.adherex.com
ENMD-2076
(aurora A/angiogenic kinase
inhibitor)
CASI Pharmaceuticals
Rockville, MD
triple-negative breast cancer
(see also ovarian, sarcoma)
Phase II
www.casipharmaceuticals.com
entinostat
(HDAC inhibitor)
Syndax Pharmaceuticals
Waltham, MA
HR-positive breast cancer
(Breakthrough Therapy)
Phase III
www.syndax.com
18
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancer
Breast Cancer
Product Name
Sponsor
Indication
Development Phase
etirinotecan pegol
(PEGylated irinotecan)
Nektar Therapeutics
San Francisco, CA
metastatic breast cancer
(Fast Track)
(see also colorectal, ovarian)
Phase III
www.nektar.com
ganetespib
(Hsp90 inhibitor)
Synta Pharmaceuticals
Lexington, MA
breast cancer
(see also leukemia, lung, ovarian)
Phase II
www.syntapharma.com
GDC-0980
(mTOR/PI3K inhibitor)
Genentech
South San Francisco, CA
breast cancer (combination therapy)
Phase II
www.gene.com
glembatumumab vedotin
(antibody drug conjugate)
CellDex Therapeutics
Hampton, NJ
triple negative breast cancer
(Fast Track)
(see also skin)
Phase II
www.celldex.com
GTx-024/GTx-027
(SARM)
GTx
Memphis, TN
androgen receptor-positive breast
cancer
Phase II
www.gtxinc.com
Halaven®
eribulin
Eisai
Woodcliff Lake, NJ
HER2-negative breast cancer
(1st-line, 2nd-line)
(see also lung, sarcoma)
Phase III
www.eisai.com
HER2 cancer immunotherapy
(AVX901)
AlphaVax
Research Triangle Park, NC
Duke University
Durham, NC
late-stage breast cancer
Phase I/II
www.alphavax.com
indoximod
(IDO pathway inhibitor)
NewLink Genetics
Ames, IA
metastatic HER2-negative breast
cancer
(see also prostate)
Phase II
www.newlinkgenetics.com
ipatasertib
(GDC-0068)
(Akt inhibitor)
Array BioPharma
Boulder, CO
Genentech
South San Francisco, CA
(see also prostate, stomach)
Phase II
www.arraybiopharma.com
www.gene.com
Jakafi®
ruxolitinib
Incyte
Wilmington, DE
breast cancer
(see also colorectal, lung, pancreatic,
other)
Phase I
www.incyte.com
Kadcyla®
ado-trastuzumab emtansine
Genentech
South San Francisco, CA
HER2-positive breast cancer
(1st-line), HER2-positive breast
cancer (adjuvant), HER2-positive
breast cancer (adjuvant combination
therapy), HER2-positive breast cancer
(neoadjuvant combination therapy)
(see also stomach)
Phase III
www.gene.com
KD019
(TKI inhibitor)
Kadmon Pharmaceuticals
Warrendale, PA
breast cancer
(see also lung)
Phase I/II
www.kadmon.com
Medicines in Development
Cancer 2014
19
Medicines and Vaccines in Development for Cancer
Breast Cancer
Product Name
Sponsor
Indication
Development Phase
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
breast cancer
(see also bladder, head/neck,
hematological, kidney, lung,
myeloma, stomach)
Phase I
www.merck.com
LCL161
(IAP inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
breast cancer
Phase II
www.novartis.com
LEE011
(CDK4/6 inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
breast cancer (1st-line)
(see also solid tumors)
Phase III
www.novartis.com
LFA102
(anti-prolactin receptor mAb)
Novartis Pharmaceuticals
East Hanover, NJ
XOMA
Berkeley, CA
prolactin receptor-positive metastatic
breast cancer
(see also prostate)
Phase I
www.novartis.com
www.xoma.com
lucitanib
(FGFR/VEGFR/PDGFR inhibitor)
Clovis Oncology
Boulder, CO
breast cancer
(see also lung)
Phase II
www.clovisoncology.com
LY2606368
(Chk1 inhibitor)
Lilly
Indianapolis, IN
BRCA 1/2 mutation breast cancer,
triple-negative breast cancer
(see also ovarian, solid tumors)
Phase II
www.lilly.com
margetuximab
(anti-HER2 mAb)
MacroGenics
Rockville, MD
metastatic breast cancer
(see also solid tumors, other)
Phase II
www.macrogenics.com
MEDI-573
(anti-IGF mAb)
MedImmune
Gaithersburg, MD
metastatic breast cancer
Phase II
www.medimmune.com
mifepristone
Corcept Therapeutics
Menlo Park, CA
triple-negative breast cancer
(combination therapy)
Phase I
www.corcept.com
MLN0128
(TORC1/TORC2 inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
metastatic breast cancer
(see also hematological, solid tumors)
Phase II
www.millennium.com
MM-121
(ErbB-3 receptor antagonist)
Merrimack Pharmaceuticals
Cambridge, MA
breast cancer
(see also lung, ovarian)
Phase II
www.merrimackpharma.com
MM-302
(HER2-targeted nanotherapeutic)
Merrimack Pharmaceuticals
Cambridge, MA
advanced HER2-positive breast cancer
Phase I
www.merrimackpharma.com
MVA-BN-HER2
(immunotherapy vaccine)
Bavarian Nordic
Mountain View, CA
breast cancer
Phase I/II
www.bavarian-nordic.com
Myocet™
doxorubicin liposomal
Sopherion Therapeutics
East Brunswick, NJ
metastatic breast cancer (1st-line)
(Fast Track)
Phase III
www.sopherion.com
20
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancer
Breast Cancer
Product Name
Sponsor
Indication
Development Phase
NeuVax™
nelipepimut-S
Galena Biopharma
Portland, OR
node positive, HER2-positive breast
cancer
(see also stomach)
----------------------------------------node-positive & negative
HER2-positive breast cancer
(combination therapy)
----------------------------------------node-positive & negative
HER2-positive breast cancer,
breast cancer (neoadjuvant)
Phase III
www.galenabiopharma.com
----------------------------------------Phase II
www.galenabiopharma.com
----------------------------------------Phase I completed
www.galenabiopharma.com
Nexavar®
sorafenib
Bayer HealthCare Pharmaceuticals
Whippany, NJ
breast cancer
(see also kidney)
Phase III
www.bayerpharma.com
niraparib
(PARP inhibitor)
TESARO
Waltham, MA
BRCA-positive breast cancer
(see also ovarian, sarcoma)
Phase III
www.tesarobio.com
olaparib
(PARP inhibitor)
AstraZeneca
Wilmington, DE
breast cancer (adjuvant), metastatic
breast cancer
(see also ovarian, stomach)
Phase III
www.astrazeneca.com
palbociclib
(CDK4/6 inhibitor)
Pfizer
New York, NY
advanced breast cancer (1st-line)
(Breakthrough Therapy)
(see also colorectal)
----------------------------------------high risk early breast cancer,
recurrent advanced breast cancer
application submitted
www.pfizer.com
----------------------------------------Phase III
www.pfizer.com
patritumab
(anti-HER3 antibody)
Daiichi Sankyo
Parsippany, NJ
breast cancer
(see also lung)
Phase II
www.dsi.com
PB272
(neratinib)
Puma Biotechnology
Los Angeles, CA
metastatic breast cancer
(see also solid tumors)
----------------------------------------metastatic breast cancer with
brain metastases, breast cancer
(neoadjuvant)
Phase III
www.pumabiotechnology.com
----------------------------------------Phase II
www.pumabiotechnology.com
PEG-SN38
(angiogenesis inhibitor)
Belrose Pharma
Princeton, NJ
metastatic breast cancer
(see also brain, solid tumors)
Phase II
www.belrosepharma.com
Perjeta®
pertuzumab
Genentech
South San Francisco, CA
HER2-positive metastatic breast
cancer (2nd-line), HER2-positive
breast cancer (adjuvant)
(see also stomach)
Phase III
www.gene.com
PF-05280014
(trastuzumab biosimilar)
Pfizer
New York, NY
metastatic breast cancer
Phase III
www.pfizer.com
Medicines in Development
Cancer 2014
21
Medicines and Vaccines in Development for Cancer
Breast Cancer
Product Name
Sponsor
Indication
Development Phase
pictrelisib
(PI3K inhibitor)
Genentech
South San Francisco, CA
estrogen receptor-positive metastatic
breast cancer, metastatic
HER2-negative/hormone
receptor-positive breast cancer
(see also lung)
Phase II
www.gene.com
PM01183
(marine-derived alkylating agent)
Pharma Mar
Madrid, Spain
metastatic breast cancer
(see also leukemia)
Phase II
www.pharmamar.com
RAD1901
(SERM modulator)
Radius Health
Waltham, MA
brain metastases from breast cancer
Phase I
www.radiuspharm.com
reparixin
(IL-8A/B receptor antagonist)
Dompe
Milan, Italy
early-stage breast cancer
Phase II
www.dompe.com
----------------------------------------Phase I
www.dompe.com
----------------------------------------meatastatic breast cancer
SGN LIV1A
(anti-LIV-1 mAb)
Seattle Genetics
Bothell, WA
metastatic breast cancer (2nd-line)
Phase I
www.seattlegenetics.com
taselisib
(PI3K inhibitor)
Genentech
South San Francisco, CA
HER2-negative metastatic breast
cancer, hormone receptor-positive
breast cancer
(see also solid tumors)
Phase I
www.gene.com
ThermoDox®
doxorubicin liposomal
Celsion
Lawrenceville, NJ
breast cancer
(see also colorectal, liver)
Phase II
www.celsion.com
topotecan liposomal
Spectrum Pharmaceuticals
Henderson, NV
breast cancer
(see also lung)
Phase I
www.sppirx.com
TPIV100
(cancer immunotherapy)
TapImmune
Seattle, WA
breast cancer
Phase I
www.tapimmune.com
trastuzumab biosimilar
Actavis
Dublin, Ireland
Amgen
Thousand Oaks, CA
breast cancer
Phase III
www.actavis.com
www.amgen.com
Tykerb®
lapatinib
GlaxoSmithKline
Research Triangle Park, NC
breast cancer (neoadjuvant, adjuvant)
Phase III
www.gsk.com
veliparib
AbbVie
North Chicago, IL
BRCA-deficient breast cancer,
triple negative breast cancer
(neoadjuvant)
(see also brain, lung)
Phase III
www.abbvie.com
22
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Breast Cancer
Product Name
Sponsor
Indication
Development Phase
WT1 immunotherapeutic
(antigen-specific cancer
immunotherapeutic)
GlaxoSmithKline
Research Triangle Park, NC
breast cancer
Phase II
www.gsk.com
Xgeva®
denosumab
Amgen
Thousand Oaks, CA
delay or prevention of bone
metastases in breast cancer
(see also lung)
Phase III
www.amgen.com
Xtandi®
enzalutamide
(androgen receptor inhibitor)
Astellas Pharma US
Northbrook, IL
Medivation
San Francisco, CA
breast cancer
(see also prostate)
Phase II
www.astellas.com
www.medivation.com
Zytiga®
abiraterone acetate
Janssen Research & Development
Raritan, NJ
metastatic estrogen receptor-positive
HER2-negative breast cancer in
postmenopausal patients
Phase II
www.janssenrnd.com
Product Name
Sponsor
Indication
Development Phase
abituzumab
(anti-integrin mAb)
EMD Serono
Rockland, MA
colorectal cancer
(see also prostate)
Phase II
www.emdserono.com
AD-p53 gene therapy
MultiVir
San Diego, CA
colorectal cancer
Phase I completed
www.multivir.com
BBI608
(cancer stemness kinase inhibitor)
Boston Biomedical
Cambridge, MA
colorectal cancer
(see also breast, lung, ovarian, skin,
stomach)
----------------------------------------colorectal cancer
(combination therapy)
Phase III
www.bostonbiomedical.com
Colorectal Cancer
----------------------------------------Phase II
www.bostonbiomedical.com
birinapant
(apoptosis stimulator)
TetraLogic Pharmaceuticals
Malvern, PA
colorectal cancer
(combination therapy)
(see also hematological, ovarian)
Phase I/II completed
www.tetralogicpharm.com
CEA cancer immunotherapy
(AVX701)
AlphaVax
Research Triangle Park, NC
Duke University
Durham, NC
late-stage colorectal cancer
Phase I/II
www.alphavax.com
CEP-32496
(BRAF kinase inhibitor)
Teva Pharmaceutical
Frazier, PA
metastatic colorectal cancer
(see also skin, solid tumors)
Phase II
www.tevapharm.com
Medicines in Development
Cancer 2014
23
Medicines and Vaccines in Development for Cancers
Colorectal Cancer
Product Name
Sponsor
Indication
Development Phase
CPP-1X/sulindac
(eflornithine/sulindac)
Cancer Prevention Pharmaceuticals
Tucson, AZ
colorectal cancer
(risk reduction)
(see also brain)
Phase III
www.canprevent.com
CPX-1
(irinotecan/floxuridine liposomal)
Celator Pharmaceuticals
Ewing, NJ
colorectal cancer
Phase II
www.celatorpharma.com
CRLX101
(camptothecin
nanopharmaceutical)
Cerulean Pharma
Cambridge, MA
late-stage rectal cancer
Phase I/II
www.ceruleanrx.com
CV-301
(cancer immunotherapy)
BN ImmunoTherapeutics
Mountain View, CA
colorectal cancer
(see also bladder, breast)
Phase II
www.bavarian-nordic.com
Cyramza™
ramucirumab
Lilly
Indianapolis, IN
colorectal cancer
(see also bladder, breast, liver, lung,
ovarian, solid tumors, stomach)
Phase III
www.lilly.com
E7820
(integrin alpha2 inhibitor)
Eisai
Woodcliff Lake, NJ
colorectal cancer
Phase II
www.eisai.com
encapsulated cell therapy
Rogosin Institute
New York, NY
colorectal cancer
Phase II
www.rogosin.org
ensituximab
(neoplasm antigen inhibitor)
Precision Biologics
Dallas, TX
advanced colorectal cancer
(see also pancreatic)
Phase II
www.precision-biologics.com
ETBX-011
(vector cancer vaccine)
Etubics
Seattle, WA
colorectal cancer
Phase II
www.etubics.com
etirinotecan pegol
(PEGylated irinotecan)
Nektar Therapeutics
San Francisco, CA
colorectal cancer (2nd-line)
(see also breast, ovarian)
Phase II/III
www.nektar.com
FANG™ Vaccine
genetically modified autologous
tumor cell vaccine
Gradalis
Dallas, TX
colorectal cancer
(see also ovarian, skin, solid tumors)
Phase II
www.gradalisinc.com
IMMU-130
(antibody drug conjugate)
Immunomedics
Morris Plains, NJ
metastatic colorectal cancer
Phase II
www.immunomedics.com
Imprime PGG®
intravenous immunostimulant
Biothera
Eagan, MN
metastatic colorectal cancer (3rd-line)
(see also leukemia, lung, lymphoma,
pancreatic)
Phase III
www.biothera.com
Jakafi®
ruxolitinib
Incyte
Wilmington, DE
colorectal cancer
(see also breast, lung, pancreatic,
other)
Phase I
www.incyte.com
24
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Colorectal Cancer
Product Name
Sponsor
Indication
Development Phase
KD018
(Chinese botanical formulation)
Kadmon Pharmaceuticals
Warrendale, PA
colorectal cancer
(see also liver)
Phase I/II
www.kadmon.com
KD032
(Ras antagonist)
Kadmon Pharmaceuticals
Warrendale, PA
colorectal cancer
(see also lung, pancreatic)
Phase I
www.kadmon.com
labetuzumab-SN-38
(IMMU-130)
Immunomedics
Morris Plains, NJ
metastatic colorectal cancer
Phase II
www.immunomedics.com
LGX818
(BRAF inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
BRAF-mutant metastatic colorectal
cancer (combination therapy)
(see also skin, solid tumors)
Phase I/II
www.novartis.com
linaclotide
Actavis
Dublin, Ireland
Ironwood Pharmaceuticals
Cambridge, MA
colon cancer prevention
Phase I
www.ironwoodpharma.com
Mekinist® trametinib +
Tafinlar® dabrafenib +
panitumumab combination
GlaxoSmithKline
Research Triangle Park, NC
colorectal cancer
Phase II
www.gsk.com
MM-398
(irinotecan nanotherapeutic)
Merrimack Pharmaceuticals
Cambridge, MA
colorectal cancer
(see also brain, pancreatic)
Phase III
www.merrimackpharma.com
MORAb-004
(CD248 antigen inhibitor)
Eisai
Woodcliff Lake, CA
colorectal cancer
(see also lung, sarcoma, skin)
Phase II
www.eisai.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
microsatellite instability
(MSI)-positive colon cancer
(see also brain, head/neck,
hematological, kidney, liver, lung,
lymphoma, skin, solid tumors)
Phase II
www.bms.com
nivolumab + Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
MSI-positive colon cancer
(see also brain, head/neck,
hematological, kidney, lung, skin)
Phase II
www.bms.com
OncoVAX®
cancer immunotherapy
Vaccinogen
Frederick, MD
stage II colon cancer
Phase III
www.vaccinogeninc.com
palbociclib
(CDK4/6 inhibitor)
Pfizer
New York, NY
colorectal cancer
(see also breast)
Phase I
www.pfizer.com
pelareorep
Oncolytics Biotech
Calgary, Canada
KRAS mutant-positive metastatic
colorectal cancer
(see also head/neck, lung, myeloma,
ovarian, pancreatic, skin, solid tumors,
other)
Phase I
www.oncolyticsbiotech.com
Medicines in Development
Cancer 2014
25
Medicines and Vaccines in Development for Cancers
Colorectal Cancer
Product Name
Sponsor
Indication
Development Phase
pexastimogene devacirepvec
(genetically-engineered oncolytic
vaccinia virus)
StillaJen Biotherapeutics
San Francisco, CA
treatment refractory colorectal cancer
(see also kidney, liver)
Phase II
www.sillajen.com
PF-05212384
(PI3K-mTOR inhibitor)
Pfizer
New York, NY
colorectal cancer (3rd-line)
Phase II
www.pfizer.com
RG7221
(Ang2-VEGF Mab)
Genentech
South San Francisco, CA
colorectal cancer
Phase II
www.gene.com
RG7597
(anti-HER3/EGFR DAF mAb)
Genentech
South San Francisco, CA
colorectal cancer
(see also head/neck)
Phase II
www.gene.com
RRx-001
(free radical stimulant)
RadioRx
Mountain View, CA
metastatic colorectal cancer
(see also lymphoma, solid tumors)
Phase II
www.radiorx.com
simtuzumab
Gilead Sciences
Foster City, CA
colorectal cancer
(see also hematological)
Phase II
www.gilead.com
SM04755
(Wnt inhibitor)
Samumed
San Diego, CA
advanced colorectal cancer
(see also liver, pancreatic, stomach)
Phase I
www.samumed.com
Stivarga®
regorafenib
Bayer HealthCare Pharmaceuticals
Whippany, NJ
colorectal cancer with resected
liver metastases
(see also kidney, liver, solid tumors)
Phase III
www.bayerpharma.com
Sym004
(anti-EGFR mAb)
EMD Serono
Rockland, MA
colorectal cancer
(see also head/neck, solid tumors)
Phase II
www.emdserono.com
TAS-102
(tipiracil/trifluridine)
Taiho Oncology
Princeton, NJ
colorectal cancer
Phase III
www. taihooncology.com
ThermoDox®
doxorubicin liposomal
Celsion
Lawrenceville, NJ
colorectal metastases
(see also breast, liver)
Phase II
www.celsion.com
vemurafenib
(BRAF inhibitor)
Daiichi Sankyo
Parsippany, NJ
colorectal cancer
Phase II
www.dsi.com
Xilonix™
monoclonal (IgG1k) antibody
XBiotech
Austin, TX
colorectal cancer in patients with
cachexia, colorectal cancer
(Fast Track)
Phase III
www.xbiotech.com
26
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Head/Neck Cancer
Product Name
Sponsor
Indication
Development Phase
Ad-IL-24
(interleukin-24 gene therapy)
MultiVir
San Diego, CA
head and neck cancer
Phase II
www.multivir.com
ADXS-HPV
(live, attenuated Listeria
monocytogenes (Lm)-based
immunotherapy vaccine)
Advaxis
Princeton, NJ
head and neck cancer
(see also other)
Phase I/II
www.advaxis.com
AZD6738
(ATR serine/threonine kinase
inhibitor)
AstraZeneca
Wilmington, DE
head and neck cancer
(see also leukemia)
Phase I
www.astrazeneca.com
GC4419
(superoxide dismutase modulator)
Galera Therapeutics
Malvern, PA
squamous cell carcinoma of the
head and neck
Phase I
www.galeratx.com
Gilotrif®
afatinib
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
squamous cell carcinoma of the
head and neck
Phase III
www.boehringer-ingelheim.com
GL-0817
(cancer immunotherapy)
Gliknik
Baltimore, MD
squamous cell cancer of the oral
cavity
Phase II
www.gliknik.com
GL-ONC1
(oncolytic virus immunomodulator)
Genelux
San Diego, CA
head and neck cancer
(see also lung, solid tumors, other)
Phase I
www.genelux.com
HF10
(oncolytic virus immunomodulator)
Takara Bio
Shiga, Japan
head and neck cancer
Phase I
www.takara-bio.com
INO-3112
(cancer therapeutic vaccine)
Inovio Pharmaceuticals
Plymouth Meeting, PA
head and neck cancer caused by
HPV types 16/18
(see also other)
Phase I
www.inovio.com
IRX-2
(immunostimulant)
IRX Therapeutics
New York, NY
head and neck cancer
(Fast Track)
Phase II completed
www.irxtherapeutics.com
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
advanced head and neck cancer
(see also bladder, breast,
hematological, kidney, lung,
myeloma, stomach)
Phase I
www.merck.com
MGCD265
(MET/Axl/VEGFR kinase inhibitor)
Mirati Therapeutics
San Diego, CA
squamous cell carcinoma of the
head and neck
(see also lung)
Phase I
www.mirati.com
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
27
Medicines and Vaccines in Development for Cancers
Head/Neck Cancer
Product Name
Sponsor
Indication
Development Phase
Multikine®
leukocyte interleukin, injection
CEL-SCI
Vienna, VA
head and neck cancer (1st-line)
Phase III
www.cel-sci.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
head and neck cancer (2nd-line)
(see also brain, colorectal,
hematological, kidney, liver, lung,
lymphoma, skin, solid tumors)
Phase III
www.bms.com
nivolumab + Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
head and neck cancer (2nd-line)
(see also brain, colorectal,
hematological, kidney, lung, skin)
Phase III
www.bms.com
pelareorep
Oncolytics Biotech
Calgary, Canada
advanced head and neck cancer
(see also colorectal, lung, myeloma,
ovarian, pancreatic, skin, solid tumors,
other)
Phase III completed
www.oncolyticsbiotech.com
RG7597
(HER3/EGFR DAF mAb)
Genentech
South San Francisco, CA
head and neck cancer
(see also colorectal)
Phase II
www.gene.com
Sym004
(anti-EGFR mAb)
EMD Serono
Rockland, MA
squamous cell carcinoma of the
head and neck
(see also colorectal, solid tumors)
Phase II
www.emdserono.com
VTX-2337
(TLR8 agonist immunotherapy)
VentiRx Pharmaceuticals
Seattle, WA
head and neck cancer
(see also ovarian)
Phase II
www.ventirx.com
Hematological Malignancies
Product Name
Sponsor
Indication
Development Phase
ABT-199/RG7601
(Bcl-2 inhibitor)
AbbVie
North Chicago, IL
Genentech
South San Francisco, CA
hematological malignancies
(see also leukemia)
Phase I
www.abbvie.com
www.gene.com
ACE-536
(TGF-ß protein super family
inhibitor)
Acceleron Pharma
Cambridge, MA
Celgene
Summit, NJ
myelodysplastic syndromes
Phase II
www.acceleronpharma.com
www.celgene.com
ORPHAN DRUG
28
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Hematological Malignancies
Product Name
Sponsor
Indication
Development Phase
AG-120
(IDH1 inhibitor)
Agios Pharmaceuticals
Cambridge, MA
Celgene
Summit, NJ
hematological malignancies
(see also solid tumors)
Phase I
www.agios.com
www.celgene.com
AG-221
(IDH2 inhibitor)
Agios Pharmaceuticals
Cambridge, MA
Celgene
Summit, NJ
hematological malignancies
Phase I
www.agios.com
www.celgene.com
ALT-803
(IL-15 superagonist complex)
Altor BioScience
Miramar, FL
hematological malignancies
(see also bladder, myeloma, skin)
Phase I/II
www.altorbioscience.com
AMG 319
(PI3K-delta inhibitor)
Amgen
Thousand Oaks, CA
hematological malignancies
Phase I
www.amgen.com
AMG 900
(aurora kinase inhibitor)
Amgen
Thousand Oaks, CA
hematological malignancies
(see also solid tumors)
Phase I
www.amgen.com
anti-LAG3 antibody
Bristol-Myers Squibb
Princeton, NJ
hematological malignancies
Phase I
www.bms.com
AR-42
(pan-DAC inhibitor)
Arno Therapeutics
Flemington, NJ
hematological malignancies
(see also solid tumors)
Phase I
www.arnothera.com
AZD1208
(PIM kinase inhibitor)
AstraZeneca
Wilmington, DE
hematological malignancies
Phase I
www.astrazeneca.com
AZD9150
(STAT3 inhibitor)
AstraZeneca
Wilmington, DE
Isis Pharmaceuticals
Carlsbad, CA
hematological malignancies
Phase I
www.astrazeneca.com
www.isispharm.com
BGJ398
(FGF-R kinase inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
hematological malignancies
(see also brain, liver, solid tumors)
Phase II
www.novartis.com
birinapant
(apoptosis stimulator)
TetraLogic Pharmaceuticals
Malvern, PA
myelodysplastic syndromes
(see also colorectal, ovarian)
Phase II
www.tetralogicpharm.com
BP-100-1.01
(loposomal Grb 2)
Bio-Path Holdings
Houston, TX
myelodysplastic syndromes
(see also leukemia)
Phase I
www.biopathholdings.com
BPX-501
(T cell replacement therapy)
Bellicum Pharmaceuticals
Houston, TX
bone marrow transplantation in
hematological malignancies
Phase I/II
www.bellicum.com
CB-839
(glutaminase inhibitor)
Calithera Biosciences
South San Francisco, CA
hematological malignancies
(see also solid tumors)
Phase I
www.calithera.com
ORPHAN DRUG
Medicines in Development
Cancer 2014
29
Medicines and Vaccines in Development for Cancers
Hematological Malignancies
Product Name
Sponsor
Indication
Development Phase
CC-486
Celgene
Summit, NJ
myelodysplastic syndromes
(see also leukemia, solid tumors)
Phase III
www.celgene.com
CPI-0610
(BET inhibitor)
Constellation Pharmaceuticals
Cambridge, MA
myelodysplastic syndromes
(see also leukemia, lymphoma,
myeloma)
Phase I
www.constellationpharma.com
CPX-351
(cytarabine/daunorubicin)
Celator Pharmaceuticals
Ewing, NJ
myelodysplastic syndromes
(see also leukemia)
Phase II
www.celatorpharma.com
EP-100
(membrane disrupting peptide)
Esperance Pharmaceuticals
San Francisco, CA
hematological malignancies
(see also solid tumors)
Phase II
www.esperancepharma.com
FF-10501
(cell differentiation stimulant)
Boston Strategics
Boston, MA
FUJIFILM Pharmaceuticals USA
Boston, MA
hematological malignancies
Phase I/II
www.bostonstrategics.com
GDC-0853
Genentech
South San Francisco, CA
hematological malignancies
Phase I
www.gene.com
GS-9973
(Syk inhibitor)
Gilead Sciences
Foster City, CA
hematological malignancies
Phase II
www.gilead.com
HDM201
(tumor suppressor protein
p53 modulator)
Novartis Pharmaceuticals
East Hanover, NJ
hematological malignancies
(see also solid tumors)
Phase I
www.novartis.com
HSC835
(stem cell therapy)
Novartis Pharmaceuticals
East Hanover, NJ
hematological malignancies
Phase I/II
www.novartis.com
imetelstat
Geron
Menlo Park, CA
hematological malignancies
Phase II
www.geron.com
ixazomib
(proteasome inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
hematological malignancies
(see also lymphoma, myeloma, solid
tumors)
Phase I
www.millennium.com
KB004
(EphA3 kinase inhibitor)
KaloBios Pharmaceuticals
South San Francisco, CA
hematological malignancies
Phase II
www.kalobios.com
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
hematological malignancies
(see also bladder, breast, head/neck,
kidney, lung, myeloma, stomach)
Phase I
www.merck.com
KPT-330
(XPO1 inhibitor)
Karyopharm Therapeutics
Newton, MA
relapsed or refractory hematological
malignancies
(see also brain, leukemia, prostate,
solid tumors)
Phase I
www.karyopharm.com
30
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Hematological Malignancies
Product Name
Sponsor
Indication
Development Phase
LGH447
(proto oncogene protein c
pim-1 inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
myelodysplastic syndromes
(see also leukemia, myeloma)
Phase I
www.novartis.com
MEDI-551 + rituximab
(anti-CD19 mAb/anti-CD20 mAb)
MedImmune
Gaithersburg, MD
hematological malignancies
Phase I
www.medimmune.com
MLN0128
(TORC1/TORC2 inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
hematological malignancies
(see also breast, solid tumors)
Phase I
www.millennium.com
MLN4924
(Nedd8-activating enzyme
inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
hematological malignancies
(see also solid tumors)
Phase I
www.millennium.com
mocetinostat
(HDAC inhibitor)
Mirati Therapeutics
San Diego, CA
myelodysplastic syndromes
(see also bladder, lymphoma)
Phase II
www.mirati.com
Gilead Sciences
Foster City, CA
myelofibrosis
(see also pancreatic)
Phase III
www.gilead.com
MRX34
(liposome-encapsulated
miR-34 mimic)
Mirna Therapeutics
Austin, TX
hematological malignancies
(see also liver)
Phase I
www.mirnarx.com
NiCord®
cord blood stem cell therapy
Gamida Cell
Jerusalem, Israel
hematological malignancies
Phase III
www.gamide-cell.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
hematological malignancies
(see also brain, colorectal, head/neck,
kidney, liver, lung, lymphoma, skin,
solid tumors)
Phase I
www.bms.com
nivolumab + Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
hematological malignancies
(see also brain, colorectal, head/neck,
kidney, lung, skin)
Phase I
www.bms.com
OMP-52M51
(NOTCH-1 inhibitor)
OncoMed Pharmaceuticals
Redwood City, CA
hematological malignancies
(see also solid tumors)
Phase I
www.oncomed.com
oprozomib
(proteasome inhibitor)
Onyx Pharmaceuticals
South San Francisco, CA
hematological malignancies
(monotherapy)
(see also solid tumors)
----------------------------------------hematological malignancies
(combination therapy)
Phase II
www.onyx.com
ORPHAN DRUG
momelotinib
(JAK inhibitor)
ORPHAN DRUG
Medicines in Development
Cancer 2014
----------------------------------------Phase I
www.onyx.com
31
Medicines and Vaccines in Development for Cancers
Hematological Malignancies
Product Name
Sponsor
Indication
Development Phase
OXi4503
(vascular disrupting agent)
OxiGENE
South San Francisco, CA
myelodysplastic syndromes
(see also leukemia)
Phase I
www.oxigene.com
pracinostat
(HDAC inhibitor)
MEI Pharma
San Diego, CA
myelodysplastic syndromes (1st-line),
refractory myelodysplastic syndromes
(see also leukemia)
Phase II
www.meipharma.com
Promacta®
eltrombopag
GlaxoSmithKline
Research Triangle Park, NC
myelodysplastic syndromes
(see also leukemia)
Phase II
www.gsk.com
Revlimid®
lenalidomide
Celgene
Summit, NJ
myelodysplastic syndromes
(non deletion 5q)
(see also leukemia, lymphoma,
myeloma)
Phase III
www.celgene.com
RG7388
(dual Raf/MEK inhibitor)
Genentech
South San Francisco, CA
hematological malignancies
(see also solid tumors)
Phase I
www.gene.com
rigosertib
(PI3K/Plk1 inhibitor)
Onconova Therapeutics
Newtown, PA
high-risk and low-risk
myelodysplastic syndromes
Phase III
www.onconova.com
ORPHAN DRUG
Cyclacel Pharmaceuticals
Berkeley Heights, NJ
myelodysplastic syndromes
(see also leukemia, lung)
Phase II
www.cyclacel.com
SGI110
(DNMT inhibitor)
Astex Pharmaceuticals
Dublin, CA
myelodysplastic syndromes
(see also leukemia, liver, ovarian)
Phase II
www.astx.com
simtuzumab
Gilead Sciences
Foster City, CA
myelofibrosis
(see also colorectal)
Phase II
www.gilead.com
SL-401
(IL-3R inhibitor)
Stemline Therapeutics
New York, NY
blastic plasmacytoid dendritic cell
neoplasm
(see also leukemia)
Phase I/II
www.stemline.com
sotatercept
(activin inhibitor)
Acceleron Pharma
Cambridge, MA
Celgene
Summit, NJ
myelodysplastic syndromes
Phase II
www.acceleronpharma.com
www.celgene.com
SP-02
(darinaparsin)
Solasia Pharma
Tokyo, Japan
hematological malignancies
(see also solid tumors)
Phase I
www.solasia.co.jp
StemEx®
carlecortemcel-L
Gamida Cell-Teva Joint Venture
Jerusalem, Israel
hematological malignancies
(Fast Track)
Phase III
www.gamida-cell.com
Millennium Pharmaceuticals
Cambridge, MA
hematological malignancies
(see also solid tumors)
Phase I
www.millennium.com
ORPHAN DRUG
ORPHAN DRUG
sapacitabine
ORPHAN DRUG
ORPHAN DRUG
TAK-659
(SYK kinase inhibitor)
32
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Hematological Malignancies
Product Name
Sponsor
Indication
Development Phase
TGR-1202
(PI3-delta kinase inhibitor)
TG Therapeutics
New York, NY
hematological malignancies
Phase I
www.tgtherapeutics.com
TH-302
(hypoxia-activated prodrug)
EMD Serono
Rockland, MA
Threshold Pharmaceuticals
South San Francisco, CA
hematological malignancies
(see also lung, pancreatic, sarcoma,
skin, solid tumors)
Phase I
www.emdserono.com
www.thresholdpharm.com
TZ101
(human recombinant enzyme)
Targazyme
San Antonio, TX
cord blood stem cell transplantation
for hematological malignancies
Phase I
www.targazyme.com
ulocuplumab
Bristol-Myers Squibb
Princeton, NJ
hematological malignancies
Phase I
www.bms.com
urelumab
Bristol-Myers Squibb
Princeton, NJ
hematological malignancies
(see also solid tumors)
Phase I
www.bms.com
varlilumab
(CD27 antigen inhibitor)
Celldex Therapeutics
Hampton, NJ
hematological malignancies
(see also solid tumors)
Phase I
www.celldex.com
WT2725
(cancer immunotherapy)
Sunovion Pharmaceuticals
Marlborough, MA
hematological malignancies
(see also solid tumors)
Phase I
www.sunovion.com
Product Name
Sponsor
Indication
Development Phase
AGS-003
(personalized dendritic cell-based
vaccine)
Argos Therapeutics
Durham, NC
metastatic renal cell carcinoma
(combination therapy) (Fast Track)
----------------------------------------metastatic renal cell carcinoma
(monotherapy)
Phase III
www.argostherapeutics.com
----------------------------------------Phase II
www.argostherapeutics.com
AGS-16M8F/AGS-16C3F
(antibody drug conjugate)
Agensys
Santa Monica, CA
Astellas Pharma
Tokyo, Japan
kidney cancer
Phase I
www.agensys.com
www.astellas.com
AMG 172
(antibody drug conjugate)
Amgen
Thousand Oaks, CA
kidney cancer
Phase I
www.amgen.com
Archexin®
AKT1 inhibitor
Rexahn
Rockville, MD
metastatic kidney cancer
Phase I/II
www.rexahn.com
Kidney Cancer
ORPHAN DRUG
Medicines in Development
Cancer 2014
33
Medicines and Vaccines in Development for Cancers
Kidney Cancer
Product Name
Sponsor
Indication
Development Phase
BNC105
(vascular disrupting agent)
Bionomics
Thebarton, Australia
renal cell carcinoma
(see also ovarian)
Phase II
www.bionomics.com.au
Cometriq®
cabozantinib
Exelixis
South San Francisco, CA
metastatic renal cell carcinoma
(see also liver)
Phase III
www.exelixis.com
dalantercept
(ALK1 antagonist)
Acceleron Pharma
Cambridge, MA
renal cell carcinoma
(see also liver)
Phase II
www.acceleronpharma.com
HyperAcute™ Renal
renal cancer immunotherapy
vaccine
NewLink Genetics
Ames, IA
metastatic renal cancer
Phase I
www.newlinkgenetics.com
IMA901
(multiple tumor-associated
peptides)
immatics Biotechnologies
Tuebingen, Germany
renal cancer
Phase III
www.immatics.com
Inlyta®
axitinib
Pfizer
New York, NY
renal cell carcinoma (adjuvant)
(see also liver)
Phase III
www.pfizer.com
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
kidney cancer
(see also bladder, breast, head/
neck, hematological, lung, myeloma,
stomach)
Phase I/II
www.merck.com
LY2510924
(CXCR4 peptide antagonist)
Lilly
Indianapolis, IN
metastatic clear-cell renal cell
carcinoma
(see also lung)
Phase II
www.lilly.com
Nexavar®
sorafenib
Bayer HealthCare Pharmaceuticals
Whippany, NJ
renal cell carcinoma (adjuvant)
(see also breast)
Phase III
www.bayerpharma.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
renal cell carcinoma (2nd/3rd-line)
(see also brain, colorectal, head/neck,
hematological, liver, lung, lymphoma,
skin, solid tumors)
----------------------------------------renal cell carcinoma
Phase II/III
www.bms.com
renal cell carcinoma (1st-line)
(see also brain, colorectal, head/neck,
hematological, lung, skin)
----------------------------------------renal cell carcinoma
Phase III
www.bms.com
treatment refractory kidney cancer
(see also colorectal, liver)
Phase II
www.sillajen.com
nivolumab + Yervoy®
ipilimumab
pexastimogene devacirepvec
(genetically-engineered oncolytic
vaccinia virus)
34
Bristol-Myers Squibb
Princeton, NJ
SillaJen Biotherapeutics
San Francisco, CA
----------------------------------------Phase I
www.bms.com
----------------------------------------Phase I
www.bms.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Kidney Cancer
Product Name
Sponsor
Indication
Development Phase
RG7446
(anti-PD-L1 mAb)
Genentech
South San Francisco, CA
renal cell carcinoma
(combination therapy)
(see also bladder, lung, skin)
Phase II
www.gene.com
SGN-CD70A
Seattle Genetics
Bothell, WA
renal cell carcinoma
(see also leukemia)
Phase I
www.seattlegenetics.com
sonepcizumab
(anti-sphingosine-1-phosphate
mAb)
Lpath
San Diego, CA
renal cell carcinoma
Phase II
www.lpath.com
Stivarga®
regorafenib
Bayer HealthCare Pharmaceuticals
Whippany, NJ
renal cancer
(see also colorectal, liver, solid
tumors)
Phase II
www.bayerpharma.com
Sutent®
sunitinib
Pfizer
New York, NY
renal cell carcinoma (adjuvant)
Phase III
www.pfizer.com
TRC105
(ENG protein inhibitor)
TRACON Pharmaceuticals
San Diego, CA
renal cell carcinoma
(see also brain, sarcoma)
Phase I/II
www.traconpharma.com
volitinib
(MET tyrosine kinase inhibitor)
AstraZeneca
Wilmington, DE
papillary renal cell carcinoma
Phase II
www.astrazeneca.com
Votrient®
pazopanib
GlaxoSmithKline
Research Triangle Park, NC
renal cell cancer (adjuvant)
(see also ovarian)
----------------------------------------renal cell cancer (combination with
MK-3475)
Phase III
www.gsk.com
----------------------------------------Phase I
www.gsk.com
Product Name
Sponsor
Indication
Development Phase
Å6 peptide
Angstrom Pharmaceuticals
Solana Beach, CA
chronic lymphocytic leukemia (CLL),
small lymphocytic leukemia
Phase II
www.angstrominc.com
ABL 001
(Bcr-abl tyrosine kinase inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
leukemia
Phase I
www.novartis.com
ABT-199/RG7601
(Bcl-2 inhibitor)
AbbVie
North Chicago, IL
Genentech
South San Francisco, CA
CLL
(see also hematological)
Phase III
www.abbvie.com
www.gene.com
----------------------------------------Phase II
www.abbvie.com
www.gene.com
Leukemia
Medicines in Development
Cancer 2014
----------------------------------------acute myeloid leukemia (AML)
35
Medicines and Vaccines in Development for Cancers
Leukemia
Product Name
Sponsor
Indication
Development Phase
ACP-196
(Btk inhibitor)
Acerta Pharma
San Carlos, CA
CLL (combination therapy)
(see also lymphoma)
Phase I
www.acerta-pharma.com
ACP-319
(Btk inhibitor)
Acerta Pharma
San Carlos, CA
CLL (combination therapy)
Phase I
www.acerta-pharma.com
ADI-PEG-20
(PEG arginine deaminase)
Polaris Pharmaceuticals
San Diego, CA
AML
(see also breast, liver, lung,
lymphoma, skin)
Phase II
www.polarispharma.com
alvocidib
(angiogenesis inhibitor)
Tolero Pharmaceuticals
Lehi, UT
AML (1st-line)
Phase III
www.toleropharmaceuticals.com
----------------------------------------Phase II
www.toleropharmaceuticals.com
----------------------------------------relapsed/refractory AML
AMG 232
Amgen
Thousand Oaks, CA
AML
(see also solid tumors)
Phase I
www.amgen.com
Arzerra™
ofatumumab
GlaxoSmithKline
Research Triangle Park, NC
relapsed CLL
(see also lymphoma)
Phase III
www.gsk.com
ASP2215
Astellas Pharma US
Northbrook, IL
AML
Phase I/II
www.astellas.com
AZD6738
(ATR serine/threonine kinase
inhibitor)
AstraZeneca
Wilmington, DE
CLL
(see also head/neck)
Phase I
www.astrazeneca.com
bendamustine ready-to-dilute
liquid formulation
Eagle Pharmaceuticals
Woodcliff Lake, NJ
CLL
(see also lymphoma, solid tumors)
application submitted
www.eagleus.com
BI 811283
(aurora kinase B inhibitor)
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
AML
Phase II completed
www.boehringer-ingelheim.com
BI 836858
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
AML
Phase I
www.boehringer-ingelheim.com
BL-8040
(CXCR4 receptor antagonist)
BioLineRx
Jerusalem, Israel
AML (combination therapy)
Phase II
www.biolinerx.com
Amgen
Thousand Oaks, CA
Philadelphia-negative relapsed/
refractory ß-precursor ALL
(Breakthrough Therapy)
(see also lymphoma)
application submitted
www.amgen.com
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
blinatumomab
ORPHAN DRUG
36
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Leukemia
Product Name
Sponsor
Indication
Development Phase
Bosulif®
bosutinib
Avillion
London, United Kingdom
Pfizer
New York, NY
Philadelphia-chromosome positive
chronic myeloid leukemia (CML)
(1st-line)
Phase III
www.avillionllp.com
www.pfizer.com
BP-100-1.01
(liposomal Grb 2)
Bio-Path Holdings
Houston, TX
ALL, AML, CML
(see also hematological)
Phase I
www.biopathholdings.com
CC-292
(Btk inhibitor)
Celgene
Summit, NJ
CLL
(see also lymphoma)
Phase I
www.celgene.com
CC-486
Celgene
Summit, NJ
post-induction AML maintenance
(see also hematological, solid tumors)
Phase III
www.celgene.com
cerdulatinib
(dual Syk-JAK inhibitor)
Portola Pharmaceuticals
South San Francisco, CA
CLL
(see also lymphoma)
Phase I/II
www.portola.com
CNDO-109
(natural killer cell stimulant)
Coronado Biosciences
Burlington, MA
AML
Phase I/II
www.coronadobiosciences.com
CPI-0610
(BET inhibitor)
Constellation Pharmaceuticals
Cambridge, MA
acute leukemia
(see also hematological, lymphoma,
myeloma)
Phase I
www.constellationpharma.com
CPX-351
(cytarabine/daunorubicin)
Celator Pharmaceuticals
Ewing, NJ
ALL, AML
(see also hematological)
Phase II
www.celatorpharma.com
CSL-362
(anti-CD123 mAb)
CSL
Victoria, Australia
Janssen Research & Development
Raritan, NJ
AML
Phase I
www.csl.com.au
www.janssenrnd.com
CTL019
(chimeric antigen receptor T-cell
therapy)
Novartis Pharmaceuticals
East Hanover, NJ
ALL
(Breakthrough Therapy)
Phase II
www.novartis.com
CWP232291
JW Pharmaceutical
Seoul, Korea
AML
Phase I
DFP-10917
(cell cycle inhibitor)
Delta-Fly Pharma
Tokushima, Japan
ALL, AML
Phase I/II
EPZ-5676
(DOT1L inhibitor)
Celgene
Summit, NJ
Epizyme
Cambridge, MA
ALL and AML with altered MLL gene
Phase I
www.celgene.com
www.epizyme.com
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
37
Medicines and Vaccines in Development for Cancers
Leukemia
Product Name
Sponsor
Indication
Development Phase
Erivedge®
vismodegib
Genentech
South San Francisco, CA
AML
(see also skin)
Phase II
www.gene.com
Erwinaze®
asparaginase erwinia
chrysanthemi intravenous
Jazz Pharmaceuticals
Palo Alto, CA
ALL
application submitted
www.jazzpharma.com
----------------------------------------Phase II
www.jazzpharma.com
----------------------------------------ALL in patients who have developed a
hypersensitivity to 1st-line
asparaginase therapy
ORPHAN DRUG
ERYTECH Pharmaceuticals
Lyon, France
ALL (adults)
Phase I
www.erytech.com
EZN-2285
(calaspargase pegol)
Sigma-Tau Pharmaceuticals
Gaithersburg, MD
ALL
Phase III completed
www.sigmatau.com
FPI-01
(multi-peptide
immunotherapeutic)
Formula Pharmaceuticals
Berwyn, PA
ALL, AML
(see also myeloma)
Phase II
www.formulapharma.com
ganetespib
(Hsp90 inhibitor)
Synta Pharmaceuticals
Lexington, MA
AML
(see also breast, lung, ovarian)
Phase II
www.syntapharma.com
GMI-1271
(E-selectin antagonist)
GlycoMimetics
Gaithersburg, MD
AML
Phase I
www.glycomimetics.com
HSV-Tk
(thymidine kinase cell therapy)
MolMed
Milan, Italy
high risk acute leukemia
Phase III
www.molmed.com
Iclusig®
ponatinib
ARIAD Pharmaceuticals
Cambridge, MA
AML
(see also stomach)
Phase II
www.ariad.com
IGN523
(antibody drug conjugate)
Igenica
Burlingame, CA
AML
Phase I
www.igenica.com
Imbruvica®
ibrutinib
Janssen Research & Development
Raritan, NJ
relapsed/refractory CLL
(see also lymphoma)
----------------------------------------relapsed/refractory CLL (combination
therapy), treatment naïve CLL
application submitted
www.janssenrnd.com
----------------------------------------Phase III
www.janssenrnd.com
IMMU-114
(humanized anti-HLA-DR)
Immunomedics
Morris Plains, NJ
CLL
(see also lymphoma)
Phase I
www.immunomedics.com
Imprime PGG®
intravenous immunostimulant
Biothera
Eagan, MN
high-risk CLL (1st-line)
(see also colorectal, lung, lymphoma,
pancreatic)
Phase II
www.biothera.com
ERY-ASP
(asparagine modulator)
ORPHAN DRUG
ORPHAN DRUG
38
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Leukemia
Product Name
Sponsor
Indication
Development Phase
inotuzumab ozogamicin
(CD22-targeted cytotoxic agent)
Pfizer
New York, NY
ALL
Phase III
www.pfizer.com
Iomab™ B
BC8-I-131 construct
Actinium Pharmaceuticals
New York, NY
hematopoietic stem cell
transplantation in ALL
Phase II
www.actiniumpharma.com
IPI-145
(PI3K inhibitor)
Infinity Pharmaceuticals
Cambridge, MD
CLL
(see also lymphoma)
----------------------------------------high-risk CLL (treatment naïve)
Phase III
www.infi.com
----------------------------------------Phase I
www.infi.com
JCAR014
(T lymphocyte immunotherapy)
Juno Therapeutics
Seattle, WA
acute ALL, refractory CLL
(see also lymphoma)
Phase I/II
www.junotherapeutics.com
JCAR015
(T lymphocyte immunotherapy)
Juno Therapeutics
Seattle, WA
B-cell ALL
Phase I
www.junotherapeutics.com
JCAR017
(T lymphocyte immunotherapy)
Juno Therapeutics
Seattle, WA
leukemia
Phase I/II
www.junotherapeutics.com
KPT-330
(XPO1 inhibitor)
Karyopharm Therapeutics
Newton, MA
elderly AML (2nd-line)
(see also brain, hematological,
prostate, solid tumors)
Phase II
www.karyopharm.com
KX01
(non-ATP competitive Sre kinase
inhibitor)
Kinex Pharmaceuticals
Buffalo, NY
AML
Phase I completed
www.kinexpharma.com
LGH447
(proto oncogene protein c
pim-1 inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
AML
(see also hematological, myeloma)
Phase I
www.novartis.com
lintuzumab Ac-226
Actinium Pharmaceuticals
New York, NY
AML (1st-line)
Phase I/II
www.actiniumpharma.com
Marqibo®
vinCRIStine sulfate LIPOSOME
injection
Spectrum Pharmaceuticals
Henderson, NV
elderly Ph-negative ALL (1st-line)
(Fast Track)
(see also lymphoma, other)
Phase III
www.sppirx.com
MEDI-551
(anti-CD19 mAb)
MedImmune
Gaithersburg, MD
CLL
(see also lymphoma)
Phase II
www.medimmune.com
MGD006
(CD123/CD3 bispecific antibody)
Macrogenics
Rockville, MD
AML
Phase I
www.macrogenics.com
milatuzumab-doxorubicin
(antibody drug conjugate)
Immunomedics
Morris Plains, NJ
CLL
(see also lymphoma)
Phase I/II
www.immunomedics.com
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
39
Medicines and Vaccines in Development for Cancers
Leukemia
Product Name
Sponsor
Indication
Development Phase
mogamulizumab
(anti-CCR4 humanized antibody)
Kyowa Hakko Pharma
Princeton, NJ
adult T-cell leukemia
(see also lymphoma)
Phase II
www.kyowa-kirin.com
MorphoSys
Martinsried, Germany
ALL, CLL
(see also lymphoma)
Phase II
www.morphosys.com
moxetumomab pasudotox
(anti-CD22 recombinant
immunotoxin)
MedImmune
Gaithersburg, MD
hairy cell leukemia
Phase III
www.medimmune.com
----------------------------------------Phase I
www.medimmune.com
nivolumab + Sprycel®
dasatinib
Bristol-Myers Squibb
Princeton, NJ
CML
Phase I
www.bms.com
Oncoquest™-CLL
personalized cancer vaccine
XEME Biopharma
Monmouth Junction, NJ
CLL (1st-line)
Phase I
www.xemebiopharma.com
otlertuzumab
(CD37 protein inhibitor)
Emergent BioSolutions
Rockville, MD
CLL
Phase II
www.emergentbiosolutions.com
OxiGENE
South San Francisco, CA
refractory AML
(see also hematological)
Phase I
www.oxigene.com
PF-04449913
(SMO [smoothened] antagonist)
Pfizer
New York, NY
AML
Phase II
www.pfizer.com
PKC412
(signal transduction inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
AML
Phase III
www.novartis.com
PLX3397
(Fms/Kit/Flt3-ITD inhibitor)
Daiichi Sankyo
Parsippany, NJ
Plexxikon
Berkeley, CA
AML
(see also brain, skin)
Phase II
www.dsi.com
www.plexxikon.com
PLX8394
(BRAF inhibitor)
Daiichi Sankyo
Parsippany, NJ
Plexxikon
Berkeley, CA
hairy cell leukemia
(see also solid tumors)
Phase I
www.dsi.com
www.plexxikon.com
PM01183
(marine-derived alkylating agent)
Pharma Mar
Madrid, Spain
acute leukemia
(see also breast)
Phase II
www.pharmamar.com
pracinostat
(HDAC inhibitor)
MEI Pharma
San Diego, CA
AML (1st-line)
(see also hematological)
Phase II
www.meipharma.com
ORPHAN DRUG
MOR208
(CD19 antigen inhibitor)
ORPHAN DRUG
----------------------------------------pediatric ALL
ORPHAN DRUG
OXi4503
(vascular disrupting agent)
ORPHAN DRUG
ORPHAN DRUG
40
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Leukemia
Product Name
Sponsor
Indication
Development Phase
PRI-724
(CBP/β-catenin inhibitor)
PRISM Pharma
Tokyo, Japan
AML, CML
(see also pancreatic, solid tumors)
Phase I/II
www.prismbiolab.com
Promacta®
eltrombopag
GlaxoSmithKline
Research Triangle Park, NC
AML
(see also hematological)
Phase II
www.gsk.com
Qinprezzo™
vosaroxin
Sunesis Pharmaceuticals
South San Francisco, CA
relapsed/refractory AML
(Fast Track)
Phase III
www.sunesis.com
quizartinib
(FLT3 inhibitor)
Ambit Biosciences
San Diego, CA
relapsed refractory AML
(Fast Track)
Phase III
www.ambitbio.com
Revlimid®
lenalidomide
Celgene
Summit, NJ
CLL maintenance (2nd-line)
(see also hematological, lymphoma,
myeloma)
Phase III
www.celgene.com
RG7775
(MDM2 IN prodrug)
Genentech
South San Francisco, CA
AML
Phase I
www.gene.com
sapacitabine
ORPHAN DRUG
Cyclacel Pharmaceuticals
Berkeley Heights, NJ
AML
(see also hematological, lung)
Phase III
www.cyclacel.com
SG2000
(DNA cross linking agent)
Spirogen
London, United Kingdom
AML, CLL
Phase I/II
SGI110
(DNMT inhibitor)
Astex Pharmaceuticals
Dublin, CA
AML
(see also hematological, liver, ovarian)
Phase II
www.astx.com
SGN-CD19A
(antibody drug conjugate)
Seattle Genetics
Bothell, WA
AML
(see also lymphoma)
Phase I
www.seattlegenetics.com
SGN-CD33A
(antibody drug conjugate)
Seattle Genetics
Bothell, WA
AML
Phase I
www.seattlegenetics.com
SL-401
(IL-3R inhibitor)
Stemline Therapeutics
New York, NY
advanced AML
(see also hematological)
Phase II
www.stemline.com
Tasigna®
nilotinib
Novartis Pharmaceuticals
East Hanover, NJ
CML treatment free remission
Phase III
www.novartis.com
tetradecanoylphorbol acetate
Rich Pharmaceuticals
Beverly Hills, CA
AML
Phase II
www.richpharmaceuticals.com
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
41
Medicines and Vaccines in Development for Cancers
Leukemia
Product Name
Sponsor
Indication
Development Phase
TG02
(CDK inhibitor)
Tragara Pharmaceuticals
Carlsbad, CA
refractory AML, relapsed CLL
(see also myeloma)
Phase I
www.tragarapharma.com
TG-1101
(anti-CD20 mAb)
TG Therapeutics
New York, NY
CLL
(see also lymphoma)
Phase I
www.tgtherapeutics.com
volasertib
(PLK-1 antagonist)
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
AML
(Breakthrough Therapy)
Phase III
www.boehringer-ingelheim.com
Gilead Sciences
Foster City, CA
CLL (1st-line)
(see also lymphoma)
Phase III
www.gilead.com
Product Name
Sponsor
Indication
Development Phase
ADI-PEG 20
(PEG arginine deiminase)
Polaris Pharmaceuticals
San Diego, CA
hepatocellular carcinoma
(see also breast, leukemia, lung,
lymphoma, skin)
Phase III
www.polarispharma.com
BA-003
(doxorubicin nanoparticles)
Onexo
Paris, France
hepatocellular carcinoma (2nd-line)
(Fast Track)
Phase III
www.onexo.com
bavituximab
Peregrine Pharmaceuticals
Tustin, CA
liver cancer
(see also breast, lung, skin, other)
Phase II
www.peregrineinc.com
BGJ398
(FGF-R kinase inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
advanced cholangiocarcinoma
(see also brain, hematological, solid
tumors)
Phase II
www.novartis.com
CBL0102
(SSRP1 protein inhibitor)
Cleveland BioLabs
Buffalo, NY
Incuron
Moscow, Russia
hepatocellular carcinoma
Phase I/II
www.cbiolabs.com
www.incuron.ru
CF102
(A3 adenosine receptor agonist)
Can-Fite BioPharma
Petach Tikva, Israel
hepatocellular carcinoma
Phase II
www.canfite.com
Cometriq®
cabozantinib
Exelixis
South San Francisco, CA
late-stage hepatocellular carcinoma
(see also kidney)
Phase III
www.exelixis.com
ORPHAN DRUG
Zydelig®
idelalisib
Liver Cancer
ORPHAN DRUG
ORPHAN DRUG
42
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Liver Cancer
Product Name
Sponsor
Indication
Development Phase
CX-4945
(casein kinase II inhibitor)
Senhwa Biosciences
San Diego, CA
cholangiocarcinoma
Phase II
www.senhwabiosciences.com
Cyramza®
ramucirumab
Lilly
Indianapolis, IN
hepatocellular carcinoma
(combination therapy)
(see also bladder, breast, colorectal,
lung, ovarian, solid tumors, stomach)
Phase III
www.lilly.com
dalantercept
(ALK1 antagonist)
Acceleron Pharma
Cambridge, MA
hepatocellular carcinoma
(see also kidney)
Phase II
www.acceleronpharma.com
DCR-MYC
(c-myc inhibitor)
Dicerna Pharmaceuticals
Watertown, MA
hepatocellular carcinoma
Phase I
www.dicerna.com
doxorubicin nanoparticles
BioAlliance Pharma
Paris, France
liver cancer (2nd-line)
ORPHAN DRUG
Phase III
www.bioalliancepharma.com
E7080
(multi-targeted kinase inhibitor)
Eisai
Woodcliff Lake, NJ
hepatocellular carcinoma
(see also brain, lung, skin, other)
Phase III
www.eisai.com
G-202
(calcium-transporting ATPase
inhibitor)
GenSpera
San Antonio, TX
liver cancer
(see also brain)
Phase II
www.genspera.com
galunisertib
(TGF-beta1 inhibitor)
Lilly
Indianapolis, IN
liver cancer
(see also brain, pancreatic)
Phase II
www.lilly.com
GC33/RG7686
(anti-GPC3 antibody)
Chugai Pharmaceutical
Tokyo, Japan
Roche
New York, NY
metastatic liver cancer
Phase II
www.chugai.com
www.roche.com
INC280
(c-Met inhibitor)
Incyte
Wilmington, DE
Novartis Pharmaceuticals
East Hanover, NJ
advanced liver cancer with c-Met
dysregulation
Phase II
www.incyte.com
www.novartis.com
Inlyta®
axitinib
Pfizer
New York, NY
liver cancer
(see also kidney)
Phase II
www.pfizer.com
ipafricept
(Wnt protein inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
OncoMed Pharmaceuticals
Redwood City, CA
hepatocellular cancer
(see also ovarian, pancreatic)
Phase I
www.bayerpharma.com
www.oncomed.com
KD018
(Chinese botanical extract)
Kadmon Pharmaceuticals
Warrendale, PA
liver cancer
(see also colorectal)
Phase I/II
www.kadmon.com
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
43
Medicines and Vaccines in Development for Cancers
Liver Cancer
Product Name
Sponsor
Indication
Development Phase
melphalan drug delivery system
(drug-device combination)
Delcath Systems
New York, NY
secondary liver cancer arising from
metastatic ocular melanoma
(Fast Track)
application submitted
www.delcath.com
GenSpera
San Antonio, TX
liver cancer
(see also brain, prostate)
Phase II
www.genspera.com
MRX34
(liposome-encapsulated miR-34
mimic)
Mirna Therapeutics
Austin, TX
unresectable primary liver cancer,
liver metastatases from other solid
tumors
(see also hematological)
Phase I
www.mirnarx.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
hepatocellular carcinoma
(see also brain, colorectal, head/
neck, hematological, kidney, lung,
lymphoma, skin, solid tumors)
Phase I
www.bms.com
PDT with Photofrin®
porfimer sodium
Pinnacle Biologics
Bannockburn, IL
unresectable advanced perihilar
cholangiocarcinoma Bismuth type
III/IV
(see also lung)
Phase III
www.pinnaclebiologics.com
StillaJen Biotherapeutics
San Francisco, CA
advanced primary liver cancer
(see also colorectal, kidney)
Phase II
www.sillajen.com
PF-03446962
(ALK1 inhibitor)
Pfizer
New York, NY
hepatocellular carcinoma (2nd-line)
Phase II
www.pfizer.com
PV-10
(rose bengal disodium)
Provectus Biopharmaceuticals
Knoxville, TN
liver metastases
(see also skin)
Phase I
www.pvct.com
refametinib
(MEK inihibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
RAS mutant hepatocellular carcinoma
(see also solid tumors)
Phase II
www.bayerpharma.com
SGI110
(DNMT inhibitor)
Astex Pharmaceuticals
Dublin, CA
liver cancer
(see also hematological, leukemia,
ovarian)
Phase II
www.astx.com
SM04755
(Wnt inhibitor)
Samumed
San Diego, CA
advanced liver cancer
(see also colorectal, pancreatic,
stomach)
Phase I
www.samumed.com
SPI-1620
(endothelin B receptor agonist)
Spectrum Pharmaceuticals
Henderson, NV
biliary cancer
(see also lung)
Phase II
www.sppirx.com
ORPHAN DRUG
mipsagargin
(ATPase inhibitor)
ORPHAN DRUG
ORPHAN DRUG
pexastimogene devacirepvec
(genetically-engineered oncolytic
vaccinia virus)
ORPHAN DRUG
ORPHAN DRUG
44
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Liver Cancer
Product Name
Sponsor
Indication
Development Phase
Stivarga®
regorafenib
Bayer HealthCare Pharmaceuticals
Whippany, NJ
hepatocellular carcinoma (2nd-line)
(see also colorectal, kidney, solid
tumors)
Phase III
www.bayerpharma.com
ThermoDox®
doxorubicin liposomal
Celsion
Lawrenceville, NJ
primary liver cancer
(Fast Track)
(see also breast, colorectal)
----------------------------------------liver metastases
Phase III
www.celsion.com
ORPHAN DRUG
----------------------------------------Phase III
www.celsion.com
tivantinib
(c-Met inhibitor)
ArQule
Woburn, MA
Daiichi Sankyo
Parsippany, NJ
hepatocellular carcinoma
Phase III
www.arqule.com
www.dsi.com
TKM-PLK1
(RNA interference)
Tekmira Pharmaceuticals
Burnaby, Canada
hepatocellular carcinoma
(see also stomach, other)
Phase II
www.tekmira.com
VT-122
(cancer immunotherapy)
Vicus Therapeutics
Morristown, NJ
advanced liver cancer
(see also prostate)
Phase II
www.vicustherapeutics.com
Product Name
Sponsor
Indication
Development Phase
abemaciclib
(CDK 4/6 inhibitor)
Lilly
Indianapolis, IN
non-small-cell lung cancer (NSCLC)
(see also breast, lymphoma)
Phase I
www.lilly.com
ABT-RTA-408
AbbVie
North Chicago, IL
Reata Pharmaceuticals
Irving, TX
metastatic NSCLC
(see also skin)
Phase I
www.abbvie.com
www.reatapharma.com
ADI-PEG 20
(PEG arginine deiminase)
Polaris Pharmaceuticals
San Diego, CA
small-cell lung cancer (SCLC)
(see also breast, leukemia, liver,
lymphoma, skin)
----------------------------------------mesothelioma, NSCLC
Phase II
www.polarispharma.com
neuroendocrine lung tumors
(see also breast, lymphoma, stomach)
Phase III
www.novartis.com
Lung Cancer
Afinitor®
everolimus
Medicines in Development
Novartis Pharmaceuticals
East Hanover, NJ
Cancer 2014
----------------------------------------Phase I
www.polarispharma.com
45
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
aldoxorubicin
(delivery-enhanced doxorubicin)
CytRx
Los Angeles, CA
SCLC
(see also brain, sarcoma)
Phase II
www.cytrx.com
alectinib
(ALK inhibitor)
Chugai Pharma USA
Berkeley Heights, NJ
Roche
Basel, Swizterland
NSCLC
(Breakthrough Therapy)
Phase II
www.chugai-pharm.com
www.roche.com
alisertib
(auroura A kinase inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
SCLC
(see also lymphoma, ovarian)
Phase II
www.millennium.com
antroquinonol
Golden Biotechnology
New Taipei City, Taiwan
NSCLC
Phase II
www.goldenbiotech.com.tw
AP26113
(ALK inhibitor)
ARIAD Pharmaceuticals
Cambridge, MA
NSCLC
Phase II
www.ariad.com
apatorsen
(Hsp27 inhibitor)
OncoGenex Pharmaceuticals
Bothell, WA
non-squamous NSCLC, squamous
NSCLC
(see also bladder, pancreatic,
prostate)
Phase II
www.oncogenex.com
ASP8273
Astellas Pharma US
Northbrook, IL
NSCLC
Phase I/II
www.astellas.com
AT13387
(HSP90 inhibitor)
Astex Pharmaceuticals
Dublin, CA
NSCLC
(see also skin)
Phase II
www.astx.com
Avastin®
bevacizumab
Genentech
South San Francisco, CA
NSCLC (adjuvant)
(see also brain, ovarian, other)
Phase III
www.gene.com
AZD9291
(EGFR tyrosine kinase inhibitor)
AstraZeneca
Wilmington, DE
advanced T790M mutation-positive
NSCLC
(Breakthrough Therapy)
Phase II/III
www.astrazeneca.com
bavituximab
Peregrine Pharmaceuticals
Tustin, CA
NSCLC (2nd-line)
(see also breast, liver, skin, other)
----------------------------------------NSCLC (1st-line)
Phase III
www.peregrineinc.com
----------------------------------------Phase I
www.peregrineinc.com
BBI608
(cancer stemness kinase inhibitor)
Boston Biomedical
Cambridge, MA
NSCLC
(see also breast, colorectal, ovarian,
skin, stomach)
Phase I/II
www.bostonbiomedical.com
belinostat
Spectrum Pharmaceuticals
Henderson, NV
NSCLC
Phase I/II
www.sppirx.com
46
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
bevacizumab biosimilar
Actavis
Parsippany, NJ
Amgen
Thousand Oaks, CA
NSCLC
Phase III
www.actavis.com
www.amgen.com
BIND-014
(PSMA-targeted docetaxel)
BIND Therapeutics
Cambridge, MA
NSCLC
(see also prostate)
Phase II
www.bindtherapeutics.com
CRS-207
(cancer immunotherapy)
Aduro BioTech
Berkeley, CA
mesothelioma (combination therapy)
(see also pancreatic)
Phase I
ww.adurobiotech.com
custirsen (OGX-111)
(antisense oligonucleotide)
OncoGenex Pharmaceuticals
Bothell, WA
Teva Pharmaceutical
Frazier, PA
NSCLC (Fast Track)
(see also prostate)
Phase III
www.oncogenex.com
www.tevapharm.com
Cynviloq™
paclitaxel polymeric micelle for
injection
Sorrento Therapeutics
San Diego, CA
NSCLC
(see also bladder, breast, ovarian,
pancreatic)
Phase III
www.sorrentotherapeutics.com
Cyramza®
ramucirumab
Lilly
Indianapolis, IN
NSCLC (combination therapy)
(see also bladder, breast, colorectal,
liver, ovarian, solid tumors, stomach)
Phase III
www.lilly.com
CYT-6091
(nanomedicine)
CytImmune Sciences
Rockville, MD
NSCLC (2nd-line)
Phase II
www.cytimmune.com
dacomitinib
(pan-HER inhibitor)
Pfizer
New York, NY
EGFR-mutant NSCLC (1st-line)
(see also solid tumors)
Phase III
www.pfizer.com
DKN-01
(DKK1 protein inhibitor)
HealthCare Pharmaceuticals
Cambridge, MA
NSCLC
(see also myeloma, other)
Phase I
www.healthcarepharmaceuticals.com
DPV-001
(toll-like receptor agonist)
UbiVac
Portland, OR
NSCLC
Phase II
www.ubivac.com
E7080
(multi-targeted kinase inhibitor)
Eisai
Woodcliff Lake, NJ
NSCLC (3rd-line monotherapy), NSCLC
(RET translocations)
(see also brain, liver, skin, other)
Phase II
www.eisai.com
EGF816
(EGFR TK inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
NSCLC
Phase I/II
www.novartis.com
ficlatuzumab
(HGF/c-MET inhibitor)
AVEO Oncology
Cambridge, MA
NSCLC
Phase II
www.aveooncology.com
foretinib
(c-MET kinase inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
NSCLC
Phase II
www.gsk.com
Medicines in Development
Cancer 2014
47
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
ganetespib
(Hsp90 inhibitor)
Synta Pharmaceuticals
Lexington, MA
NSCLC
(Fast Track)
(see also breast, leukemia, ovarian)
Phase III
www.syntapharma.com
GI-4000
(natural killer cell stimulant)
GlobeImmune
Louisville, CO
NSCLC
(see also pancreatic)
Phase II
www.globeimmune.com
GL-ONC1
(oncolytic virus immunomodulator)
Genelux
San Diego, CA
lung, malignant mesothelioma
(see also head/neck, solid tumors,
other)
Phase I
www.genelux.com
GSK2879552
(LSD1/CoREST inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
relapsed/refractory SCLC
Phase I
www.gsk.com
Halaven®
eribulin
Eisai
Woodcliff Lake, NJ
NSCLC
(see also breast, sarcoma)
Phase III
www.eisai.com
Imprime PGG®
intravenous immunostimulant
Biothera
Eagan, MN
metastatic NSCLC (1st-line)
(see also colorectal, leukemia,
lymphoma, pancreatic)
Phase II
www.biothera.com
INCB24360
(IDO1 inhibitor)
Incyte
Wilmington, DE
NSCLC
(see also ovarian, skin, other)
Phase I/II
www.incyte.com
INCB39110
(JAK1 inhibitor)
Incyte
Wilmington, DE
NSCLC
(see also solid tumors)
Phase I/II
www.incyte.com
ISIS-EIF4ERX
(antisense oligonucleotide)
Isis Pharmaceuticals
Carlsbad, CA
NSCLC
(see also prostate)
Phase II
www.isispharm.com
Jakafi®
ruxolitinib
Incyte
Wilmington, DE
NSCLC
(see also breast, colorectal,
pancreatic, other)
Phase I
www.incyte.com
KD019
(TKI inhibitor)
Kadmon Pharmaceuticals
Warrendale, PA
NSCLC
(see also breast)
----------------------------------------Src-positive NSCLC
Phase II
www.kadmon.com
----------------------------------------Phase I
www.kadmon.com
KD032
(Ras antagonist)
Kadmon Pharmaceuticals
Warrendale, PA
NSCLC
(see also colorectal, pancreatic)
Phase II
www.kadmon.com
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
NSCLC
(see also bladder, breast, head/neck,
hematological, kidney, myeloma,
stomach)
Phase III
www.merck.com
Kyprolis®
carfilzomab
Onyx Pharmaceuticals
South San Francisco, CA
SCLC
(see also myeloma)
Phase II
www.onyx.com
48
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
lifastuzumab vedotin
(anti-NaPi2b antibody drug
conjugate)
(RG7599)
Genentech
South San Francisco, CA
NSCLC
(see also ovarian)
Phase I
www.gene.com
lucitanib
(FGFR/VEGFR/PDGFR inhibitor)
Clovis Oncology
Boulder, CO
squamous NSCLC
(see also breast)
Phase II
www.clovisoncology.com
lung cancer DNA vaccine
(IC 1001)
Immune Cell Therapy
Chicago, IL
NSCLC
Phase I
www.immcellther.com
LY2510924
(CXCR4 peptide antagonist)
Lilly
Indianapolis, IN
SCLC
(see also kidney)
Phase II
www.lilly.com
LY2875358
(c-Met mAb)
Lilly
Indianapolis, IN
NSCLC
(see also stomach)
Phase II
www.lilly.com
LY2940680
(hedgehog/SMO antagonist)
Lilly
Indianapolis, IN
SCLC
Phase I/II
www.lilly.com
MAGE-A3 immunotherapeutic
(recombinant antigen-specific
cancer immunotherapeutic)
GlaxoSmithKline
Research Triangle Park, NC
NSCLC
Phase III
www.gsk.com
ME-344
(mitochondrial inhibitor)
MEI Pharma
San Diego, CA
SCLC
(see also ovarian)
Phase I
www.meipharma.com
MEDI4736
(anti-PD-L1 mAb)
MedImmune
Gaithersburg, MD
stage III NSCLC, NSCLC (3rd-line)
Phase III
www.medimmune.com
MEDI4736 + Iressa® gefitinib
(anti-PD-L1 mAb/EGFR inhibitor)
MedImmune
Gaithersburg, MD
NSCLC
Phase I
www.medimmune.com
MGCD265
(MET/Axl/VEGFR kinase inhibitor)
Mirati Therapeutics
San Diego, CA
NSCLC
(see also head/neck)
Phase I
www.mirati.com
MM-121
(ErbB-3 receptor antagonist)
Merrimack Pharmaceuticals
Cambridge, MA
NSCLC
(see also breast, ovarian)
Phase II
www.merrimackpharma.com
MORAb-003
(IgG1 mAb)
Eisai
Woodcliff Lake, NJ
Morphotek
Exton, PA
NSCLC
(see also ovarian)
Phase II
www.eisai.com
MORAb-009
(megakaryocyte potentiating
factor inhibitor)
Eisai
Woodcliff Lake, NJ
Morphotek
Exton, PA
mesothelioma
Phase II
www.eisai.com
----------------------------------------Phase I
www.eisai.com
ORPHAN DRUG
Medicines in Development
Cancer 2014
----------------------------------------solid tumors
49
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
NC-6004
(nanoparticle cisplatin)
NanoCarrier
Tokyo, Japan
NSCLC
Phase I/II
www.nanocarrier.co.jp
necitumumab
(EGFR inhibitor)
Lilly
Indianapolis, IN
squamous NSCLC
Phase III
www.lilly.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
NSCLC (1st-line),
squamous NSCLC (2nd-line),
non-squamous NSCLC (2nd-line)
(see also brain, colorectal,
head/neck, hematological, kidney,
liver, lymphoma, skin, solid tumors)
----------------------------------------squamous NSCLC (3rd-line)
Phase III
www.bms.com
----------------------------------------NSCLC, SCLC
----------------------------------------Phase II
www.bms.co
----------------------------------------Phase II
www.bms.com
nivolumab + Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
NSCLC, SCLC
(see also brain, colorectal, head/neck,
hematological, kidney, skin)
Phase I
www.bms.com
patritumab
(anti-HER3 antibody)
Daiichi Sankyo
Parsippany, NJ
NSCLC
(see also breast)
Phase II
www.dsi.com
PDT with Photofrin®
porfimer sodium
Pinnacle Biologics
Bannockburn, IL
epithelioid malignant pleural
mesothelioma
(see also liver)
Phase III
www.pinnaclebiologics.com
pelareorep
Oncolytics Biotech
Calgary, Canada
NSCLC, squamous cell carcinoma of
the lung
(see also colorectal, head/neck,
myeloma, ovarian, pancreatic, skin,
solid tumors, other)
Phase II
www.oncolyticsbiotech.com
pictrelisib
(PI3K inhibitor)
Genentech
South San Francisco, CA
metastatic NSCLC
(see also breast)
Phase II
www.gene.com
PRAME immunotherapeutic
(recombinant antigen-specific
cancer immunotherapeutic)
GlaxoSmithKline
Research Triangle Park, NC
resectable NSCLC
Phase II
www.gsk.com
PT 107
(allogeneic B7.1/HLA-A1
transfected tumor cell vaccine)
Pique Therapeutics
Durham, NC
NSCLC
Phase II
www.piquetherapeutics.com
ORPHAN DRUG
50
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
RG7446
(anti-PD-L1 mAb)
Genentech
South San Francisco, CA
NSCLC (2nd-line)
(see also bladder, kidney, skin)
Phase III
www.gene.com
----------------------------------------NSCLC (3rd-line)
----------------------------------------Phase II
www.gene.com
----------------------------------------Phase I
www.gene.com
----------------------------------------EGFR-positive NSCLC
rociletinib (CO-1686)
(EGFR inhibitor)
Clovis Oncology
Boulder, CO
EGFR-mutated NSCLC
(Breakthrough Therapy)
Phase II
www.clovisoncology.com
roniciclib
(CDK inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
SCLC
Phase II
www.bayerpharma.com
sapacitabine
ORPHAN DRUG
Cyclacel Pharmaceuticals
Berkeley Heights, NJ
NSCLC
(see also hematological, leukemia)
Phase II
www.cyclacel.com
SC16LD6.5
(antibody drug conjugate)
Stem CentRx
South San Francisco, CA
SCLC
Phase I/II
www.stemcentrx.com
seliciclib
(CDK inhibitor)
Cyclacel Pharmaceuticals
Berkeley Heights, NJ
NSCLC
(see also other)
Phase II
www.cyclacel.com
selumetinib
(MEK inhibitor)
Array BioPharma
Boulder, CO
AstraZeneca
Wilmington, DE
KRAS-positive NSCLC (2nd-line)
(see also other)
Phase III
www.arraybiopharma.com
www.astrazeneca.com
----------------------------------------Phase II
www.arraybiopharma.com
www.astrazeneca.com
SPI-1620
(endothelin B receptor agonist)
Spectrum Pharmaceuticals
Henderson, NV
NSCLC
(see also liver)
Phase II
www.sppirx.com
Tafinlar®
dabrafenib
GlaxoSmithKline
Research Triangle Park, NC
NSCLC
(Breakthrough Therapy)
Phase II
www.gsk.com
tarextumab
(anti-NOTCH 2/3)
OncoMed Pharmaceuticals
Redwood City, CA
SCLC
(see also pancreatic)
Phase I/II
www.oncomed.com
Tavocept™
BioNumerik Pharmaceuticals
San Antonio, TX
lung cancer
Phase III
www.bionumerik.com
tergenpumatucel-L
NewLink Genetics
Ames, IA
advanced or metastatic NSCLC
Phase II/III
www.newlinkgenetics.com
Medicines in Development
Cancer 2014
----------------------------------------KRAS-negative NSCLC (2nd-line)
51
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
TG4010
(MVA-MUC1-IL2 cancer vaccine)
Transgene
Cambridge, MA
advanced NSCLC
Phase II/III
www.transgene.fr
TH-302
(hypoxia-activated prodrug)
EMD Serono
Rockland, MA
Threshold Pharmaceuticals
South San Francisco, CA
NSCLC
(see also hematological, pancreatic,
skin, sarcoma, solid tumors)
Phase II
www.emdserono.com
www.thresholdpharm.com
topotecan liposomal
Spectrum Pharmaceuticals
Henderson, NV
lung cancer
(see also breast)
Phase I
www.sppirx.com
tremelimumab
(anti-CTLA-4 mAb)
MedImmune
Gaithersburg, MD
mesothelioma
Phase III
www.medimmune.com
tremelimumab
(anti-CTLA-4 mAb)/Iressa®
gefitinib combination
AstraZeneca
Wilmington, DE
MedImmune
Gaithersburg, MD
NSCLC
Phase I
www.astrazeneca.com
www.medimmune.com
TSR-011
(ALK/TRK inhibitor)
TESARO
Waltham, MA
NSCLC
Phase I/II
www.tesarobio.com
TUSC2
(gene therapy)
Genprex
Austin, TX
NSCLC
Phase I/II
www.genprex.com
Vargatef™
nintedanib
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
NSCLC
(see also ovarian)
----------------------------------------mesothelioma
Phase III
www.boehringer-ingelheim.com
----------------------------------------Phase II
www.boehringer-ingelheim.com
veliparib
AbbVie
North Chicago, IL
NSCLC
(see also brain, breast)
Phase III
www.abbvie.com
viagenpumatucel-L
(cancer immunotherapy)
Heat Biologics
Durham, NC
NSCLC
Phase II
www.heatbio.com
vinorelbine liposomal
Spectrum Pharmaceuticals
Henderson, NV
SCLC
(see also ovarian)
Phase I
www.sppirx.com
vintafolide
(folate-DAVLBH)
Endocyte
West Lafayette, IN
NSCLC
(see also solid tumors)
Phase II
www.endocyte.com
VS-6063
(FAK inhibitor)
Verastem
Cambridge, MA
KRAS-mutant NSCLC, mesothelioma
(see also ovarian)
Phase II
www.verastem.com
Pfizer
New York, NY
ALK-positive NSCLC (1st-line)
(see also solid tumors)
Phase III
www.pfizer.com
ORPHAN DRUG
Xalkori®
crizotinib
ORPHAN DRUG
52
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Lung Cancer
Product Name
Sponsor
Indication
Development Phase
Xgeva®
denosumab
Amgen
Thousand Oaks, CA
metastatic NSCLC (1st-line)
(see also breast)
Phase II
www.amgen.com
Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
SCLC (1st-line), squamous NSCLC
(1st-line)
(see also ovarian, prostate, skin,
stomach)
Phase III
www.bms.com
Zykadia™
ceritinib
Novartis Pharmaceuticals
East Hanover, NJ
ALK-positive advanced NSCLC
(1st-line, treatment naïve)
Phase II
www.novartis.com
Product Name
Sponsor
Indication
Development Phase
abemaciclib
(CDK 4/6 inhibitor)
Lilly
Indianapolis, IN
mantle cell lymphoma
(see also breast, lung)
Phase II
www.lilly.com
abexinostat
(HDAC inhibitor)
Pharmacyclics
Sunnyvale, CA
follicular lymphoma,
mantle cell lymphoma
Phase II
www.pharmacyclics.com
ACP-196
(Btk inhibitor)
Acerta Pharma
San Carlos, CA
diffuse large B-cell lymphoma
(DLBCL),
Waldenström’s macroglobulinemia
(see also leukemia)
Phase I
www.acerta-pharma.com
Adcetris®
brentuximab vedotin
Seattle Genetics
Bothell, WA
post-transplant Hodgkin lymphoma
prevention, relapsed
CD30-positive cutaneous lymphoma,
Hodgkin lymphoma (1st-line),
CD30-positive mature
T-cell lymphoma (1st-line)
----------------------------------------relapsed/refractory CD30-positive
non-Hodgkin lymphoma (NHL),
Hodgkin lymphoma in the elderly
(1st-line), DLBCL (1st-line)
----------------------------------------Hodgkin lymphoma (2nd-line)
Phase III
www.seattlegenetics.com
NHL
(see also breast, leukemia, liver, lung,
skin)
Phase II
www.polarispharma.com
Lymphoma
ORPHAN DRUG
ADI-PEG 20
(PEG arginine deiminase)
Medicines in Development
Polaris Pharmaceuticals
San Diego, CA
Cancer 2014
----------------------------------------Phase II
www.seattlegenetics.com
----------------------------------------Phase I
www.seattlegenetics.com
53
Medicines and Vaccines in Development for Cancers
Lymphoma
Product Name
Sponsor
Indication
Development Phase
AEB071
(PKC inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
CD79-mutant or ABC-subtype DLBCL
(combination therapy)
(see also other)
Phase I/II
www.novartis.com
Afinitor®
everolimus
Novartis Pharmaceuticals
East Hanover, NJ
DLBCL
(see also breast, lung, stomach)
Phase III
www.novartis.com
AFM 13
(CD30 antigen modulator)
Affimed Therapeutics
Heidelberg, Germany
Hodgkin lymphoma
Phase II
www.affimed.com
alisertib
(auroura A kinase inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
relapsed or refractory peripheral
T-cell lymphoma
(see also lung, ovarian)
Phase III
www.millennium.com
AR-12
(PI3K/Akt inhibitor)
Arno Therapeutics
Flemington, NJ
lymphoma
(see also solid tumors)
Phase I
www.arnothera.com
Arzerra™
ofatumumab
GlaxoSmithKline
Research Triangle Park, NC
relapsed DLBCL, refractory and
relapsed follicular lymphoma
(see also leukemia)
Phase III
www.gsk.com
bendamustine ready-to-dilute
liquid formulation
Eagle Pharmaceuticals
Woodcliff Lake, NJ
NHL
(see also leukemia, solid tumors)
application submitted
www.eagleus.com
BI 836826
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
NHL
Phase I
www.boehringer-ingelheim.com
BiovaxID®
dasiprotimut T
(personalized cancer vaccine)
Biovest International
Minneapolis, MN
follicular NHL (Fast Track),
mantle cell lymphoma
Phase III
www.bioinvest.com
ORPHAN DRUG
Amgen
Thousand Oaks, CA
DLBCL
(see also leukemia)
Phase II
www.amgen.com
CC-122
(pleiotropic pathway modulator)
Celgene
Summit, NJ
DLBCL
(see also solid tumors)
Phase I
www.celgene.com
CC-292
(Btk inhibitor)
Celgene
Summit, NJ
lymphoma
(see also leukemia)
Phase I
www.celgene.com
cerdulatinib
(dual Syk-JAK inhibitor)
Portola Pharmaceuticals
South San Francisco, CA
NHL
(see also leukemia)
Phase I/II
www.portola.com
CMD-003
(cell therapy)
Cell Medica
London, United Kingdom
Epstein-Barr virus lymphoma
Phase II
www.cellmedica.co.uk
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
blinatumomab
54
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Lymphoma
Product Name
Sponsor
Indication
Development Phase
copanlisib
(PI3K inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
NHL
Phase II
www.bayerpharma.com
CPI-0610
(BET inhibitor)
Constellation Pharmaceuticals
Cambridge, MA
lymphoma
(see also hematological, leukemia,
myeloma)
Phase I
www.constellationpharma.com
CUDC-427
(IAP inhibitor)
Curis
Lexington, MA
late-stage lymphoma
(see also solid tumors)
Phase I
www.curis.com
CUDC-907
(HDAC, PI3K inhibitor)
Curis
Lexington, MA
relapsed/refractory lymphoma
(see also myeloma)
Phase I
www.curis.com
Debio 1143
(IAP inhibitor)
Debiopharma
Lausanne, Switzerland
(see also solid tumors)
Phase I
www.debiopharm.com
DI-Leu16-IL2
(CD20 antigen inhibitor)
ALOPEXX Oncology
Concord, MA
NHL
Phase I/II
www.alopexx.com
DS-3032
(MDM2 inhibitor)
Daiichi Sankyo
Parsippany, NJ
(see also solid tumors)
Phase I
www.dsi.com
DS-3078
(mTOR inhibitor)
Daiichi Sankyo
Parsippany, NJ
(see also solid tumors)
Phase I
www.dsi.com
DS-8273
(anti-DR5 antibody)
Daiichi Sankyo
Parsippany, NJ
late-stage lymphoma
(see also solid tumors)
Phase I
www.dsi.com
EPZ-6438
(EZH2 inhibitor)
Epizyme
Cambridge, MA
NHL
(see also solid tumors)
Phase I/II
www.epizyme.com
fenretinide intravenous
CerRx
Lubbock, TX
adult lymphoma
(see also solid tumors)
Phase II
www.cerrx.com
Folotyn®
pralatrexate injection
Spectrum Pharmaceuticals
Henderson, NV
peripheral T-cell lymphoma (1st-line)
Phase III
www.sppirx.com
Gazyva®
obinutuzumab
Genentech
South San Francisco, CA
DLBCL, indolent NHL (1st-line),
refractory indolent NHL
Phase III
www.gene.com
GDC-0575
(ChK1 inhibitor)
Genentech
South San Francisco, CA
lymphoma
(see also solid tumors)
Phase I
www.gene.com
Medicines in Development
Cancer 2014
55
Medicines and Vaccines in Development for Cancers
Lymphoma
Product Name
Sponsor
Indication
Development Phase
Imbruvica®
ibrutinib
Janssen Research & Development
Raritan, NJ
relapsed/refractory mantle cell
lymphoma, treatment naïve mantle
cell lymphoma (combination
therapy), relapsed/refractory indolent
NHL, non-germinal center B-cell
subtype of DLBCL, Waldenström’s
macroglobulinemia
(Breakthrough Therapy)
Phase III
www.janssenrnd.com
IMGN529
(CD37 protein inhibitor)
ImmunoGen
Waltham, MA
NHL
Phase I
www.immunogen.com
IMMU-114
(humanized anti-HLA-DR)
Immunomedics
Morris Plains, NJ
NHL
(see also leukemia)
Phase I
www.immunomedics.com
IMO-8400
(TLR antagonist)
Idera Pharmaceuticals
Cambridge, MA
DLBCL, Waldenström’s
macroglobulinemia
Phase I/II
www.iderapharma.com
Imprime PGG®
intravenous immunostimulant
Biothera
Eagan, MN
advanced indolent NHL
(see also colorectal, leukemia, lung,
pancreatic)
Phase II
www.biothera.com
INCB39110/INCB40093
(JAK1 inhibitor/PI3K delta
inhibitor)
Incyte
Wilmington, DE
B-lymphoid malignancies
Phase I
www.incyte.com
INCB40093
(PI3K delta inhibitor)
Incyte
Wilmington, DE
B-lymphoid malignancies
Phase I
www.incyte.com
interleukin-12 gene therapy
OncoSec Medical
San Diego, CA
cutaneous T-cell lymphoma (CTCL),
Merkel cell carcinoma
(see also skin)
Phase II
www.oncosec.com
IPI-145
(PI3K inhibitor)
Infinity Pharmaceuticals
Cambridge, MD
indolent NHL
(see also leukemia)
----------------------------------------mantle cell lymphoma
Phase II
www.infi.com
----------------------------------------Phase I
www.infi.com
Istodax®
romidepsin
Celgene
Summit, NJ
peripheral T-cell lymphoma (1st-line)
Phase III
www.celgene.com
ixazomib
(proteasome inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
relapsed/refractory follicular
lymphoma
(see also hematological, myeloma,
solid tumors)
Phase II
www.millennium.com
JCAR014
(T lymphocyte immunotherapy)
Juno Therapeutics
Seattle, WA
NHL
(see also leukemia)
Phase I/II
www.junotherapeutics.com
56
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Lymphoma
Product Name
Sponsor
Indication
Development Phase
JNJ-26481585
(HDAC inhibitor)
Janssen Research & Development
Raritan, NJ
CTCL
Phase II
www.janssenrnd.com
JNJ-40346527
(Fms inhibitor)
Janssen Research & Development
Raritan, NJ
Hodgkin lymphoma
Phase II completed
www.janssenrnd.com
JNJ-42756493
(FGFR inhibitor)
Janssen Research & Development
Raritan, NJ
late-stage lymphoma
(see also solid tumors)
Phase I
www.janssenrnd.com
KTE-C19 CAR
(chimeric antigen receptor)
Kite Pharma
Santa Monica, CA
National Cancer Institute
Bethesda, MD
DLBCL
Phase I/II
www.kitepharma.com
Marqibo®
vinCRIStine sulfate LIPOSOME
injection
Spectrum Pharmaceuticals
Henderson, NV
aggressive NHL (1st-line)
(see also leukemia, other)
Phase III
www.sppirx.com
MEDI-551
(anti-CD19 mAb)
MedImmune
Gaithersburg, MD
DLBCL
(see also leukemia)
Phase II
www.medimmune.com
milatuzumab-doxorubicin
(antibody drug conjugate)
Immunomedics
Morris Plains, NJ
NHL
(see also leukemia)
Phase I/II
www.immunomedics.com
mocetinostat
(HDAC inhibitor)
Mirati Therapeutics
San Diego, CA
DLBCL
(see also bladder, hematological)
Phase II
www.mirati.com
Kyowa Hakko Pharma
Princeton, NJ
CTCL
(see also leukemia)
----------------------------------------adult T-cell lymphoma
Phase III
www.kyowa-kirin.com
----------------------------------------Phase II
www.kyowa-kirin.com
MOR208
(CD19 antigen inhibitor)
MorphoSys
Martinsried, Germany
NHL
(see also leukemia)
Phase II
www.morphosys.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
Hodgkin lymphoma (Breakthrough
Therapy), NHL (1st-line), DLBCL
(see also brain, colorectal, head/neck,
hematological, kidney, liver, lung,
skin, solid tumors)
Phase II
www.bms.com
non-Hodgkin’s lymphoma vaccine
(personalized cancer vaccine)
XEME Biopharma
Monmouth Junction, NJ
non-Hodgkin’s follicular lymphoma
Phase II
www.xemebiopharma.com
ocaratuzumab
(AME-133v)
MENTRIK Biotech
Dallas, TX
lymphoma
Phase I compeleted
www.mentrik.com
ORPHAN DRUG
ORPHAN DRUG
mogamulizumab
(anti-CCR4 humanized antibody)
ORPHAN DRUG
Medicines in Development
Cancer 2014
57
Medicines and Vaccines in Development for Cancers
Lymphoma
Product Name
Sponsor
Indication
Development Phase
ONO-4059
(Btk inhibitor)
Ono Pharmaceuticals
Osaka, Japan
B cell lymphoma
Phase I
pinatuzumab vedotin
(anti-CD22 antibody drug
conjugate)
Genentech
South San Francisco, CA
DLBCL, NHL
Phase II
www.gene.com
Pixuvri®
pixantrone
CTI BioPharma
Seattle, WA
DLBCL
Phase III
www.ctibiopharma.com
PNT2258
(bcl-2 oncogene inhibitor)
ProNAi Therapeutics
Plymouth, MI
NHL
(see also solid tumors)
Phase II
www.pronai.com
polatuzumab vedotin
(anti-CD79b antibody drug
conjugate)
Genentech
South San Francisco, CA
DLBCL, NHL
Phase II
www.gene.com
Resimmune™
A-dmDT390-bisFv immunotoxin
Angimmune
Rockville, MD
CTCL
(see also skin)
Phase I/II
www.angimmune.com
Revlimid®
lenalidomide
Celgene
Summit, NJ
DLBCL (maintenance), follicular
lymphoma (1st-line), relapsed/
refractory indolent lymphoma
(see also hematological, leukemia,
myeloma)
----------------------------------------DLBCL-ABC subtype (1st-line)
Phase III
www.celgene.com
----------------------------------------Phase II
www.celgene.com
RRx-001
(free radical stimulant)
RadioRx
Mountain View, CA
late-stage lymphoma
(see also colorectal, solid tumors)
Phase I
www.radiorx.com
SAR3419
(maytansin-loaded anti-CD19
mAb)
Sanofi US
Bridgewater, NJ
B-cell refractory/relapsed
malignancies
Phase II
www.sanofi.com
SGN-CD19A
(antibody drug conjugate)
Seattle Genetics
Bothell, WA
NHL
(see also leukemia)
Phase I
www.seattlegenetics.com
SGN-CD70A
Seattle Genetics
Bothell, WA
NHL
(see also kidney)
Phase I
www.seattlegenetics.com
SHAPE
(HDAC inhibitor)
TetraLogic Pharmaceuticals
Malvern, PA
CTCL
Phase I
www.tetralogicpharma.com
Sevion Therapeutics
Bridgewater, NJ
DLBCL, mantle cell lymphoma
(see also myeloma)
Phase I/II
www.seviontherapeutics.com
ORPHAN DRUG
SNS-01
(nano-encapsulated gene therapy)
ORPHAN DRUG
58
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Lymphoma
Product Name
Sponsor
Indication
Development Phase
TG-1101
(anti-CD20 mAb)
TG Therapeutics
New York, NY
mantle cell lymphoma
(see also leukemia)
Phase I
www.tgtherapeutics.com
Teva Pharmaceutical
Frazier, PA
advanced indolent NHL, mantle cell
lymphoma
----------------------------------------relapsed or refractory mantle cell
lymphoma
(combination therapy)
Phase III
www.tevapharm.com
----------------------------------------Phase II
www.tevapharm.com
trifarotene
Galderma
Lausanne, Switzerland
CTCL
Phase I
www.galderma.com
Velcade®
bortezomib
Millennium Pharmaceuticals
Cambridge, MA
mantle cell lymphoma (1st-line)
Phase III
www.millennium.com
----------------------------------------Phase II
www.millennium.com
ORPHAN DRUG
Treanda®
bendamustine liquid
ORPHAN DRUG
----------------------------------------DLBCL
veltuzumab
(humanized anti-CD20 mAb)
Immunomedics
Morris Plains, NJ
NHL (combination therapy)
Phase II
www.immunomedics.com
VS-5584
(PI3K/mTOR inhibitor)
Verastem
Cambridge, MA
late-stage lymphoma
(see also solid tumors)
Phase I
www.verastem.com
Zydelig®
idelalisib
Gilead Sciences
Foster City, CA
indolent NHL
(see also leukemia)
Phase III
www.gilead.com
Product Name
Sponsor
Indication
Development Phase
afuresertib
(AKT protein kinase inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
multiple myeloma
(see also ovarian)
Phase I
www.gsk.com
ALT-803
(IL-15 superagonist complex)
Altor BioScience
Miramar, FL
multiple myeloma
(see also bladder, hematological, skin)
Phase I/II
www.altorbioscience.com
AT7519
(CDK inhibitor)
Astex Pharmaceuticals
Dublin, CA
multiple myeloma
Phase II
www.astx.com
Myeloma
Medicines in Development
Cancer 2014
59
Medicines and Vaccines in Development for Cancers
Myeloma
Product Name
Sponsor
Indication
Development Phase
BT062
(tubulin polymerisation inhibitor)
Biotest Pharmaceuticals
Dreieich, Germany
multiple myeloma
Phase I/II
www.biotest.de
CB-5083
(P97 ATPase inhibitor)
Cleave Biosciences
Burlingame, CA
multiple myeloma
Phase I
www.cleavebio.com
CPI-0610
(BET inhibitor)
Constellation Pharmaceuticals
Cambridge, MA
multiple myeloma
(see also hematological, leukemia,
lymphoma)
Phase I
www.constellationpharma.com
CUDC-907
(HDAC, PI3K inhibitor)
Curis
Lexington, MA
multiple myeloma
(see also lymphoma)
Phase I
www.curis.com
daratumumab
Janssen Research & Development
Raritan, NJ
relapsed/refractory multiple myeloma
(Breakthrough Therapy)
Phase III
www.janssenrnd.com
DKN-01
(DKK1 protein inhibitor)
HealthCare Pharmaceuticals
Cambridge, MA
multiple myeloma
(see also lung, other)
Phase I/II
ww.healthcarepharmaceuticals.com
elotuzumab
AbbVie
North Chicago, IL
Bristol-Myers Squibb
Princeton, NJ
multiple myeloma (1st-line)
(combination therapy), relapsed/
refractory multiple myeloma
(combination therapy)
(Breakthrough Therapy)
----------------------------------------multiple myeloma (2nd-line)
(combination therapy)
Phase III
www.abbvie.com
www.bms.com
----------------------------------------Phase II
www.abbvie.com
www.bms.com
filanesib
(KSP inhibitor)
Array BioPharma
Boulder, CO
multiple myeloma
Phase II
www.arraybiopharma.com
FPI-01
(multi-peptide
immunotherapeutic)
Formula Pharmaceuticals
Berwyn, PA
multiple myeloma
(see also leukemia)
Phase I
www.formulapharma.com
GSK2110183
(AKT protein kinase inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
multiple myeloma
Phase I
www.gsk.com
ixazomib
(proteasome inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
relapsed/refractory multiple myeloma
(see also hematological, lymphoma,
solid tumors)
Phase III
www.millennium.com
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
multiple myeloma
(see also bladder, breast, head/neck,
hematological, kidney, lung, stomach)
Phase I
www.merck.com
Kyprolis®
carfilzomib
Onyx Pharmaceuticals
South San Francisco, CA
multiple myeloma
(combination therapy)
(see also lung)
Phase III
www.onyx.com
ORPHAN DRUG
60
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Myeloma
Product Name
Sponsor
Indication
Development Phase
LGH447
(proto oncogene protein c
pim-1 inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
multiple myeloma
(see also hematological, leukemia)
Phase I/II
www.novartis.com
marizomib intravenous
Triphase Accelerator
San Diego, CA
multiple myeloma
ORPHAN DRUG
Phase II
www.triphaseco.com
melphalan intravenous
(Captisol-Enabled® melphalan)
Spectrum Pharmaceuticals
Henderson, NV
multiple myeloma
Phase II
www.sppirx.com
MOR202
(anti-CD38 antibody)
Celgene
Summit, NJ
MorphoSys
Martinsried, Germany
multiple myeloma
Phase I
www.celgene.com
www.morphosys.com
NY-ESO-1/LAGE-1-specific T-cells
Adaptimmune
Oxon, United Kingdom
advanced myeloma
(see also ovarian, sarcoma, skin)
Phase I/II
www.adaptimmune.com
panobinostat
(HDAC inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
multiple myeloma
application submitted
www.novartis.com
pelareorep
Oncolytics Biotech
Calgary, Canada
relapsed multiple myeloma
(see also colorectal, head/neck,
lung, ovarian, pancreatic, skin, solid
tumors, other)
Phase I
www.oncolyticsbiotech.com
plitidepsin
Pharmamar
Madrid, Spain
relapsed refractory multiple myeloma
ORPHAN DRUG
Phase III
www.pharmamar.com
PVX-410
(therapeutic cancer vaccine)
OncoPep
North Andover, MA
smoldering multiple myeloma
Phase I/II
www.oncopep.com
Revlimid®
lenalidomide
Celgene
Summit, NJ
multiple myeloma (1st-line)
(see also hematological, leukemia,
lymphoma)
----------------------------------------multiple myeloma (maintenance)
application submitted
www.celgene.com
ORPHAN DRUG
----------------------------------------Phase III
www.celgene.com
rocilinostat
(HDAC inhibitor)
Acetylon Pharmaceuticals
Boston, MA
multiple myeloma
Phase I/II
www.acetylon.com
SAR650984
(anti-CD38 naked mAb)
Sanofi US
Bridgewater, NJ
multiple myeloma
Phase II
www.sanofi.com
SNS-01
(nano-encapsulated gene therapy)
Sevion Therapeutics
Bridgewater, NJ
multiple myeloma
(see also lymphoma)
Phase I/II
www.seviontherapeutics.com
ORPHAN DRUG
Medicines in Development
Cancer 2014
61
Medicines and Vaccines in Development for Cancers
Myeloma
Product Name
Sponsor
Indication
Development Phase
Sylvant®
siltuximab
Janssen Research & Development
Raritan, NJ
smoldering multiple myeloma
Phase II
www.janssenrnd.com
TAS-120
(FGF/FGFR inhibitor)
Taiho Oncology
Princeton, NJ
multiple myeloma
(see also solid tumors)
Phase I
www.taihooncology.com
TG02
(CDK inhibitor)
Tragara Pharmaceuticals
Carlsbad, CA
multiple myeloma
(see also leukemia)
Phase I
www.tragarapharma.com
Zolinza®
vorinostat
Merck
Whitehouse Station, NJ
multiple myeloma (combination
therapy)
Phase III
www.merck.com
Product Name
Sponsor
Indication
Development Phase
afuresertib
(Akt protein kinase inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
ovarian cancer
(see also myeloma)
Phase II
www.gsk.com
alisertib
(auroura A kinase inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
ovarian cancer
(see also lung, lymphoma)
Phase II
www.millennium.com
Avastin®
bevacizumab
Genentech
South San Francisco, CA
platinum-resistant relapsed ovarian
cancer
(see also brain, lung, other)
----------------------------------------metastatic ovarian cancer (1st-line),
platinum-sensitive relapsed ovarian
cancer
application submitted
www.gene.com
Ovarian Cancer
ORPHAN DRUG
----------------------------------------Phase III
www.gene.com
AZD1775
(WEE-1 inhibitor)
AstraZeneca
Wilmington, DE
ovarian cancer
Phase II
www.astrazeneca.com
BBI608
(cancer stemness kinase inhibitor)
Boston Biomedical
Cambridge, MA
ovarian cancer
(see also breast, colorectal, lung, skin,
stomach)
Phase I/II
www.bostonbiomedical.com
birinapant
(apoptosis stimulator)
TetraLogic Pharmaceuticals
Malvern, PA
ovarian cancer
(combination therapy)
(see also colorectal, hematological)
Phase I/II
www.tetralogicpharm.com
BNC105
(vascular disrupting agent)
Bionomics
Thebarton, Australia
ovarian cancer
(see also kidney)
Phase I
www.bionomics.com.au
CVac™
cancer vaccine MUC-1
Prima BioMed
Sydney, Australia
ovarian cancer
(Fast Track)
Phase II
www.primabiomed.com.au
ORPHAN DRUG
62
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Ovarian Cancer
Product Name
Sponsor
Indication
Development Phase
Cynviloq™
paclitaxel polymeric micelle for
injection
Sorrento Therapeutics
San Diego, CA
ovarian cancer
(see also bladder, breast, lung,
pancreatic)
Phase II
www.sorrentotherapeutics.com
Cyramza®
ramucirumab
Lilly
Indianapolis, IN
ovarian cancer
(see also bladder, breast, colorectal,
liver, lung, solid tumors, stomach)
Phase II
www.lilly.com
demcizumab
(DLL4 inhibitor)
OncoMed Pharmaceuticals
Redwood City, CA
ovarian cancer
Phase I/II
www.oncomed.com
DPX-Survivac
(peptide cancer vaccine)
Immunovaccine
Halifax, Canada
ovarian cancer
Phase I/II
www.imvaccine.com
EGEN-001
(IL2-based immunotherapy)
EGEN
Huntsville, AL
ovarian cancer (monotherapy)
Phase II
www.egeninc.com
----------------------------------------Phase I
www.egeninc.com
----------------------------------------ovarian cancer (combination therapy)
ORPHAN DRUG
elesclomol
(HSP70 heat shock protein)
Synta Pharmaceuticals
Lexington, MA
recurrent, persistent ovarian cancer
Phase II
www.syntapharma.com
ENMD-2076
(aurora A/angiogenic kinase
inhibitor)
CASI Pharmaceuticals
Rockville, MD
ovarian clear cell carcinoma
(see also breast, sarcoma)
Phase II
www.casipharmaceuticals.com
Nektar Therapeutics
San Francisco, CA
platinum-resistant ovarian cancer
(see also breast, colorectal)
Phase II completed
www.nektar.com
FANG™ Vaccine
genetically modified autologous
tumor cell vaccine
Gradalis
Dallas, TX
high-risk stage IIIc ovarian cancer
(see also colorectal, skin, solid
tumors)
Phase II
www.gradalisinc.com
GALE-301
(folate binding protein
immunotherapy)
Galena Biopharma
Portland, OR
ovarian cancer
(see also other)
Phase II
www.galenabiopharma.com
ganetespib
(Hsp90 inhibitor)
Synta Pharmaceuticals
Lexington, MA
ovarian cancer
(see also breast, leukemia, lung)
Phase II
www.syntapharma.com
INCB24360
(IDO1 inhibitor)
Incyte
Wilmington, DE
ovarian cancer
(see also lung, skin, other)
Phase II
www.incyte.com
ORPHAN DRUG
etirinotecan pegol
(PEGylated irinotecan)
ORPHAN DRUG
Medicines in Development
Cancer 2014
63
Medicines and Vaccines in Development for Cancers
Ovarian Cancer
Product Name
Sponsor
Indication
Development Phase
ipafricept
(Wnt protein inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
OncoMed Pharmaceuticals
Redwood City, CA
ovarian cancer
(see also liver, pancreatic)
Phase I
www.bayerpharma.com
www.oncomed.com
Karenitecin®
cositecan
BioNumerik Pharmaceuticals
San Antonio, TX
advanced ovarian cancer
Phase III
www.bionumerik.com
lifastuzumab vedotin
(anti-NaPi2b antibody drug
conjugate)
(RG7599)
Genentech
South San Francisco, CA
platinum-resistant ovarian cancer
(see also lung)
----------------------------------------platinum-sensitive ovarian cancer
Phase II
www.gene.com
----------------------------------------Phase I
www.gene.com
LY2606368
(Chk1 inhibitor)
Lilly
Indianapolis, IN
BRCA 1/2 mutation ovarian cancer,
high grade ovarian cancer
(see also breast, solid tumors)
Phase II
www.lilly.com
ME-344
(mitochondrial inhibitor)
MEI Pharma
San Diego, CA
ovarian cancer
(see also lung)
Phase I
www.meipharma.com
MEK162
(MEK inhibitor)
Array BioPharma
Boulder, CO
Novartis Pharmaceuticals
East Hanover, NJ
low-grade serious ovarian cancer
(see also skin, solid tumors)
Phase III
www.arraybiopharma.com
www.novartis.com
MM-121
(ErbB-3 receptor antagonist)
Merrimack Pharmaceuticals
Cambridge, MA
ovarian cancer
(see also breast, lung)
Phase II
www.merrimackpharma.com
MORAb-003
(IgG1 mAb)
Eisai
Woodcliff Lake, NJ
Morphotek
Exton, PA
platinum-sensitive ovarian cancer
(see also lung)
Phase III
www.eisai.com
niraparib
(PARP inhibitor)
TESARO
Waltham, MA
ovarian cancer
(see also breast, sarcoma)
Phase III
www.tesarobio.com
NY-ESO-1/LAGE-1-specific T-cells
Adaptimmune
Oxon, United Kingdom
ovarian cancer
(see also myeloma, sarcoma, skin)
Phase I/II
www.adaptimmune.com
olaparib
(PARP inhibitor)
AstraZeneca
Wilmington, DE
BRCA-mutated platinum-sensitive
relapsed ovarian cancer
(see also breast, stomach)
----------------------------------------BRCA-mutation ovarian cancer
(1st-line)
application submitted
www.astrazeneca.com
ORPHAN DRUG
64
----------------------------------------Phase III
www.astrazeneca.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Ovarian Cancer
Product Name
Sponsor
Indication
Development Phase
oregovomab
Quest PharmaTech
Edmonton, Canada
ovarian cancer (1st-line)
Phase II
www.questpharmatech.com
AVAX Technologies
Philadelphia, PA
relapsed ovarian cancer
Phase I/II
www.avax-tech.com
pelareorep
Oncolytics Biotech
Calgary, Canada
persistant or recurrent ovarian cancer
(see also colorectal, head/neck, lung,
myeloma, pancreatic, skin, solid
tumors, other)
Phase II
www.oncolyticsbiotech.com
pimasertib/SAR245509
(MEK inihibitor/dual PI3K, mTOR
inhibitor)
EMD Serono
Rockland, MA
Sanofi US
Bridgewater, NJ
ovarian cancer
(see also solid tumors)
Phase II
www.emdserono.com
www.sanofi.com
RG7458
(MUC16 antibody drug conjugate)
Genentech
South San Francisco, CA
ovarian cancer
(see also pancreatic)
Phase I
www.gene.com
RG7882
(antibody drug conjugate)
Genentech
South San Francisco, CA
ovarian cancer
Phase I
www.gene.com
rucaparib
(PARP inhibitor)
Clovis Oncology
Boulder, CO
ovarian cancer
(see also pancreatic)
Phase III
www.clovisoncology.com
SGI110
(DNMT inhibitor)
Astex Pharmaceuticals
Dublin, CA
ovarian cancer
(see also leukemia, liver)
Phase II
www.astx.com
trebananib
(recombinant peptide-Fc fusion
protein)
Amgen
Thousand Oaks, CA
ovarian cancer
Phase III
www.amgen.com
Vargatef™
nintedanib
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
ovarian cancer
(see also lung)
Phase III
www.boehringer-ingelheim.com
VB-111
(vascular targeting system)
VBL Therapeutics
Or Yehuda, Israel
ovarian cancer
(see also brain, other)
Phase I/II
www.vblrx.com
vinorelbine liposomal
Spectrum Pharmaceuticals
Henderson, NV
ovarian cancer
(see also lung)
Phase I
www.sppirx.com
VS-6063
(FAK inhibitor)
Verastem
Cambridge, MA
ovarian (combination therapy)
(see also lung)
Phase II
www.verastem.com
ORPHAN DRUG
OVax®
autologous tumor cell-based
vaccine
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
65
Medicines and Vaccines in Development for Cancers
Ovarian Cancer
Product Name
Sponsor
Indication
Development Phase
VTX-2337
(TLR8 agonist immunotherapy)
VentiRx Pharmaceuticals
Seattle, WA
ovarian cancer
(see also head/neck)
Phase II
www.ventirx.com
Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
ovarian cancer
(see also lung, prostate, skin,
stomach)
Phase II
www.bms.com
Yondelis®
trabectedin
Janssen Research & Development
Raritan, NJ
relapsed ovarian cancer
(see also sarcoma)
Phase III
www.janssenrnd.com
Zybrestat®
fosbretabulin
OXiGENE
South San Francisco, CA
ovarian cancer
Phase II
www.oxigene.com
Product Name
Sponsor
Indication
Development Phase
177Lu-DOTATATE
(somatostatin receptor antagonist)
Advanced Accelerator Applications
New York, NY
metastatic gastro-entero-pancreatic
neuroendocrine tumors
Phase III
www.adacap.com
Celgene
Summit, NJ
pancreatic cancer (adjuvant)
(see also breast)
Phase III
www.celgene.com
NewLink Genetics
Ames, IA
pancreatic cancer (resected)
(Fast Track)
----------------------------------------pancreatic cancer (borderline
resectable of locally advanced
unresectable)
Phase III
www.newlinkgenetics.com
----------------------------------------Phase III
www.newlinkgenetics.com
anti-MUC1 AR20.5 mAb
(chemotherapy-enhanced
immunotherapy)
Quest PharmaTech
Edmonton, Canada
pancreatic cancer
Phase I/II
www.questpharmatech.com
apatorsen
(Hsp27 inhibitor)
OncoGenex Pharmaceuticals
Bothell, WA
metastatic pancreatic cancer
(see also bladder, lung, prostate)
Phase II
www.oncogenex.com
cancer gene therapy
(AdV-tK)
Advantagene
Auburndale, MA
pancreatic cancer
(see also brain, prostate)
Phase I
www.advantagene.com
ORPHAN DRUG
ORPHAN DRUG
Pancreatic Cancer
ORPHAN DRUG
Abraxane®
paclitaxel protein-bound particles
for injectable suspension
(albumin-bound)
ORPHAN DRUG
algenpantucel-L
(cancer immunotherapy vaccine)
ORPHAN DRUG
66
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Pancreatic Cancer
Product Name
Sponsor
Indication
Development Phase
CPI-613
Cornerstone Pharmaceuticals
Cranbury, NJ
pancreatic cancer
ORPHAN DRUG
Phase II
www.cornerstonepharma.com
CRS-207
(cancer immunotherapy)
Aduro BioTech
Berkeley, CA
pancreatic cancer in combination with
GVAX Pancreas
(Breakthrough Therapy)
(see also lung)
Phase II
www.adurobiotech.com
Cynviloq™
paclitaxel polymeric micelle
for injection
Sorrento Therapeutics
San Diego, CA
pancreatic cancer
(see also bladder, breast, lung,
ovarian)
Phase II
www.sorrentotherapeutics.com
ensituximab
(neoplasm antigen inhibitor)
Precision Biologics
Dallas, TX
advanced pancreatic cancer
(see also colorectal)
Phase II
www.precision-biologics.com
FG-3019
(connective tissue growth factor
inhibitor mAb)
Fibrogen
San Francisco, CA
advanced pancreatic cancer
Phase II
www.fibrogen.com
galunisertib
(TGF-beta1 inhibitor)
Lilly
Indianapolis, IN
pancreatic cancer
(see also brain, liver)
Phase I/II
www.lilly.com
GI-4000
(mutated Ras cancer vaccine)
GlobeImmune
Louisville, CO
resected pancreatic cancer
(see also lung)
Phase II
www.globeimmune.com
glufosfamide
ORPHAN DRUG
Eleison Pharmaceuticals
St. Petersburg, FL
metastatic pancreatic cancer
(2nd-line)
(Fast Track)
Phase III
www.eleison-pharma.com
Imprime PGG®
intravenous immunostimulant
Biothera
Eagan, MN
advanced pancreatic ductal cancer
(see also colorectal, leukemia, lung,
lymphoma)
Phase I
www.biothera.com
ipafricept
(Wnt protein inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
OncoMed Pharmaceuticals
Redwood City, CA
pancreatic cancer
(see also liver, ovarian)
Phase I
www.bayerpharma.com
www.oncomed.com
IRT-102
(methionine enkephalin)
TNI Biotech
Orlando, FL
pancreatic cancer
Phase II
www.tnibiotech.com
Jakafi®
ruxolitinib
Incyte
Wilmington, DE
pancreatic cancer
(see also breast, colorectal, lung,
other)
Phase II
www.incyte.com
KD032
(Ras antagonist)
Kadmon Pharmaceuticals
Warrendale, PA
pancreatic cancer
(see also colorectal, lung)
Phase II
www.kadmon.com
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
67
Medicines and Vaccines in Development for Cancers
Pancreatic Cancer
Product Name
Sponsor
Indication
Development Phase
masitinib
AB Science USA
Short Hills, NJ
metastatic pancreatic cancer
(see also stomach)
Phase III
www.ab-science.com
MLN0264
(GCC antibody drug conjugate)
Millennium Pharmaceuticals
Cambridge, MA
advanced pancreatic cancer
(see also stomach)
Phase II
www.millennium.com
MM-398
(irinotecan/nanotherapeutic)
Merrimack Pharmaceuticals
Cambridge, MA
pancreatic cancer (2nd-line)
(see also brain, colorectal)
Phase III
www.merrimackpharma.com
momelotinib
(JAK inhibitor)
Gilead Sciences
Foster City, CA
pancreatic cancer
(see also hematological)
Phase II
www.gilead.com
necuparanib
Momenta Pharmaceuticals
Cambridge, MA
pancreatic cancer
ORPHAN DRUG
Phase I/II
www.momentapharma.com
PCI-27483
(FVIIa inhibitor)
Pharmacyclics
Sunnyvale, CA
pancreatic cancer
Phase II
www.pharmacyclics.com
PEGPH20
(pegylated recombinant human
hyaluronidase)
Halozyme Therapeutics
San Diego, CA
metastatic pancreatic cancer
(Fast Track)
Phase II
www.halozyme.com
pelareorep
Oncolytics Biotech
Calgary, Canada
advanced pancreatic cancer
(see also colorectal, head/neck, lung,
myeloma, ovarian, skin, solid tumors,
other)
Phase II
www.oncolyticsbiotech.com
pimasertib
(MEK inhibitor)
EMD Serono
Rockland, MA
pancreatic cancer
(see also skin)
Phase II
www.emdserono.com
PLX7486
(TRK inhibitor)
Daiichi Sankyo
Parsippany, NJ
Plexxikon
Berkeley, CA
pancreatic cancer
(combination therapy)
(see also solid tumors)
Phase I
www.dsi.com
www.plexxikon.com
PRI-724
(CBP/ß-catenin inhibitor)
Prism Pharma
Tokyo, Japan
pancreatic cancer
(see also leukemia, solid tumors)
Phase I
www.prismbiolab.com
RG7458
(MUC16 antibody drug conjugate)
Genentech
South San Francisco, CA
pancreatic cancer
(see also ovarian)
Phase I
www.gene.com
RG7600
(mesothelin antibody drug
conjugate)
Genentech
South San Francisco, CA
unresectable pancreatic cancer
Phase I
www.gene.com
rucaparib
(PARP inhibitor)
Clovis Oncology
Boulder, CO
BRCA-mutated pancreatic cancer
(see also ovarian)
Phase II
www.clovisoncology.com
68
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Pancreatic Cancer
Product Name
Sponsor
Indication
Development Phase
SM04755
(Wnt inhibitor)
Samumed
San Diego, CA
advanced pancreatic cancer
(see also colorectal, liver, stomach)
Phase I
www.samumed.com
Sprycel®
dasatinib
Bristol-Myers Squibb
Princeton, NJ
Otsuka America Pharmaceutical
Rockville, MD
pancreatic cancer
(see also other)
Phase II
www.bms.com
www.otsuka.com
tarextumab
(anti-NOTCH 2/3)
OncoMed Pharmaceuticals
Redwood City, CA
pancreatic cancer
(see also lung)
Phase I/II
www.oncomed.com
TH-302
(hypoxia-activated prodrug)
EMD Serono
Rockland, MA
Threshold Pharmaceuticals
South San Francisco, CA
pancreatic cancer
(see also hematological, lung,
sarcoma, skin, solid tumors)
Phase III
www.emdserono.com
www.thresholdpharm.com
TL-118
Tiltan Pharma
Jerusalem, Israel
metastatic pancreatic cancer
(1st-line)
Phase II
www.tiltanpharma.com
Y-90 clivatuzumab tetraxetan
Immunomedics
Morris Plains, NJ
relapsed pancreatic cancer
(Fast Track)
----------------------------------------pancreatic cancer (1st-line)
(Fast Track)
Phase III
www.immunomedics.com
----------------------------------------Phase I/II
www.immunomedics.com
Product Name
Sponsor
Indication
Development Phase
abituzumab
(anti-integrin mAb)
EMD Serono
Rockland, MA
prostate cancer
(see also colorectal)
Phase II
www.emdserono.com
AE37
(immunotherapeutic)
Antigen Express
Worcester, MA
prostate cancer
(see also breast)
Phase II
www.antigenexpress.com
APC100
(androgen antagonist)
Adamis Pharmaceuticals
San Diego, CA
prostate cancer
Phase I/II
www.adamispharmaceuticals.com
apatorsen
(Hsp27 inhibitor)
OncoGenex Pharmaceuticals
Bothell, WA
prostate cancer
(see also bladder, lung, pancreatic)
Phase II
www.oncogenex.com
ATL101
(177 Lu-labeled anti-PSMA mAb)
ATLAB Pharma
Nantes, France
Weill Medical College
New York, NY
metastatic prostate cancer
Phase II
www.atlab-pharma.com
ORPHAN DRUG
ORPHAN DRUG
Prostate Cancer
Medicines in Development
Cancer 2014
69
Medicines and Vaccines in Development for Cancers
Prostate Cancer
Product Name
Sponsor
Indication
Development Phase
AZD5312
(androgen receptor inhibitor)
AstraZeneca
Wilmington, DE
Isis Pharmaceuticals
Carlsbad, CA
prostate cancer
Phase I
www.astrazeneca.com
www.isispharm.com
BIND-014
(PSMA-targeted docetaxel)
BIND Therapeutics
Cambridge, MA
prostate cancer
(see also lung)
Phase II
www.bindtherapeutics.com
BPX-201
(dendritic cell vaccine)
Bellicum Pharmaceuticals
Houston, TX
metastatic castration-resistant
prostate cancer
(combination therapy)
Phase I/II
www.bellicum.com
cancer gene therapy
(AdV-tK)
Advantagene
Auburndale, MA
newly diagnosed prostate cancer
(Fast Track)
(see also brain, pancreatic)
Phase III
www.advantagene.com
CFG920
(steroid 17-alpha-hydroxylase
inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
metastatic castration-resistant
prostate cancer
Phase I/II
www.novartis.com
custirsen (OGX-111)
(antisense oligonucleotide)
OncoGenex Pharmaceuticals
Bothell, WA
Teva Pharmaceutical
Frazier, PA
metastatic castration-resistant
prostate cancer (2nd-line)
(Fast Track)
(see also lung)
Phase III
www.oncogenex.com
www.tevapharm.com
DCVax®-Prostate
dendritic cell vaccine
Northwest Biotherapeutics
Bethesda, MD
late-stage hormone independent
prostate cancer
Phase II completed
www.nwbio.com
EC1169
(PSMA-tubulysin)
Endocyte
West Lafayette, IN
prostate cancer
Phase I
www.endocyte.com
galeterone
(androgen receptor antagonist)
Tokai Pharmaceuticals
Cambridge, MA
metastatic castration-resistant
prostate cancer
(Fast Track)
Phase II
www.tokaipharma.com
GTx-758
(ER alpha agonist)
GTx
Memphis, TN
secondary hormone therapy for
castration-resistant prostate cancer
Phase II
www.gtxinc.com
indoximod
(IDO pathway inhibitor)
NewLink Genetics
Ames, IA
metastatic castration-resistant
prostate cancer
(see also breast)
Phase II
www.newlinkgenetics.com
ipatasertib
(GDC-0068)
(Akt inhibitor)
Array BioPharma
Boulder, CO
Genentech
South San Francisco, CA
prostate cancer
(see also breast, stomach)
Phase II
www.arraybiopharma.com
www.gene.com
IRX4204
(retinoid X receptor agonist)
Io Therapeutics
Santa Ana, CA
prostate cancer
Phase II
www.io-therapeutics.com
70
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Prostate Cancer
Product Name
Sponsor
Indication
Development Phase
ISIS-EIF4ERX
(antisense oligonucleotide)
Isis Pharmaceuticals
Carlsbad, CA
castration-resistant prostate cancer
(see also lung)
Phase II
www.isispharm.com
Jevtana®
cabazitaxel
Sanofi US
Bridgewater, NJ
metastatic prostate cancer
Phase III
www.sanofi.com
JNJ-56021927/ARN-509
(androgen receptor antagonist)
Janssen Research & Development
Raritan, NJ
hormone refractory prostate cancer
Phase III
www.janssenrnd.com
KPT-330
(XPO1 inhibitor)
Karyopharm Therapeutics
Newton, MA
metastatic prostate cancer
(see also brain, hematological,
leukemia, solid tumors)
Phase II
www.karyopharm.com
LFA102
(anti-prolactin receptor mAb)
Novartis Pharmaceuticals
East Hanover, NJ
XOMA
Berkeley, CA
prolactin receptor-positive
castration-resistant prostate cancer
(see also breast)
Phase I
www.novartis.com
www.xoma.com
MER-104
(oral acyline)
Merrion Pharmaceuticals
Dublin, Ireland
prostate cancer
Phase I
www.merrionpharma.com
mipsagargin
(ATPase inhibitor)
GenSpera
San Antonio, TX
prostate cancer
(see also brain, liver)
Phase II
www.genspera.com
MVA-BN-PRO
(immunotherapy vaccine)
Bavarian Nordic
Mountain View, CA
hormone refractory prostate cancer
Phase I/II
www.bavarian-nordic.com
MVI-816
(PAP plasmid DNA)
Madison Vaccines
Madison, WI
prostate cancer
Phase II
www.madisonvaccinesinc.com
NX-1207
(apoptosis stimulant)
Nymox Pharmaceutical
Hasbrouck Heights, NJ
early-stage prostate cancer
Phase II completed
www.nymox.com
ODM-201
(androgen receptor inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Orion
Espoo, Finland
prostate cancer
Phase II
www.bayerpharma.com
www.orion.fi
ONC1-13B
(androgen receptor antagonist)
AllaChem
Hallandale Beach, FL
metastatic prostate cancer
Phase I/II
www.allachem.com
orteronel
(lyase inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
metastatic castration-resistant
prostate cancer
----------------------------------------non-metastatic prostate cancer
Phase III
www.millennium.com
----------------------------------------Phase II
www.millennium.com
ozarelix
(GnRH inhibitor)
Spectrum Pharmaceuticals
Henderson, NV
hormone dependent prostate cancer
Phase II
www.sppirx.com
Medicines in Development
Cancer 2014
71
Medicines and Vaccines in Development for Cancers
Prostate Cancer
Product Name
Sponsor
Indication
Development Phase
prostate cancer vaccine
(PSA/IL-2/GM-CSF vaccine)
OncBioMune
Baton Rouge, LA
prostate cancer
Phase I
www.oncbiomune.com
Prostvac®
targeted immunotherapy
Bavarian Nordic
Mountain View, CA
National Cancer Institute
Bethesda, MA
metastatic castration-resistant
prostate cancer
Phase III
www.bavarian-nordic.com
Provenge®
sipuleucel-T
Dendreon
Seattle, WA
recurrent early stage prostate cancer
Phase III
www.dendreon.com
----------------------------------------Phase II
www.dendreon.com
----------------------------------------recurrent hormone refractory,
metastatic prostate cancer
PSMA ADC therapeutic
(antibody drug conjugate)
Progenics Pharmaceuticals
Tarrytown, NY
prostate cancer
Phase II
www.progenics.com
RG7450
(anti-STEAP1 antibody drug
conjugate)
Genentech
South San Francisco, CA
prostate cancer
Phase I
www.gene.com
TAK-385
(GnRH angonist)
Millennium Pharmaceuticals
Cambridge, MA
prostate cancer
Phase II
www.millennium.com
tasquinimod
Active Biotech
Lund, Sweden
metastatic hormone refractory
prostate cancer
Phase III
www.activebiotech.com
TeloB-VAX
(telomerase vaccine)
Adamis Pharmaceuticals
San Diego, CA
prostate cancer
Phase I
www.adamispharmaceuticals.com
VT-122
(cancer immunotherapy)
Vicus Therapeutics
Morristown, NJ
pre-chemotherapy prostate cancer
(see also liver)
Phase II
www.vicustherapeutics.com
Xofigo®
radium-223 dichloride
Bayer HealthCare Pharmaceuticals
Whippany, NJ
bone metastases in
castration-resistant prostate cancer
(1st-line) (combination therapy)
----------------------------------------bone metastases in
castration-resistent prostate cancer
(combination therapy)
Phase III
www.bayerpharma.com
metastatic castration-resistant
prostate cancer (treatment naïve)
(see also breast)
----------------------------------------non-metastatic castration-resistant
prostate cancer
application submitted
www.astellas.com
www.medivation.com
----------------------------------------Phase III
www.astellas.com
www.medivation.com
Xtandi®
enzalutamide
(androgen receptor inhibitor)
72
Astellas Pharma US
Northbrook, IL
Medivation
San Francisco, CA
----------------------------------------Phase II
www.bayerpharma.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Prostate Cancer
Product Name
Sponsor
Indication
Development Phase
Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
prostate cancer (1st-line)
(see also lung, ovarian, skin, stomach)
Phase III
www.bms.com
zoptarelin doxorubicin
(type II DNA topoisomerase
inhibitor)
AEterna Zentaris
Quebec, Canada
castration-/taxane-resistant prostate
cancer
(see also other)
Phase II
www.aezsinc.com
Product Name
Sponsor
Indication
Development Phase
aldoxorubicin
(delivery-enhanced doxorubicin)
CytRx
Los Angeles, CA
soft tissue sarcoma (2nd-line)
(see also brain, lung)
----------------------------------------Kaposi’s sarcoma,
soft tissue sarcoma (1st-line)
Phase III
www.cytrx.com
----------------------------------------Phase II
www.cytrx.com
ENMD-2076
(aurora A/angiogenic
kinase inhibitor)
CASI Pharmaceuticals
Rockville, MD
soft tissue sarcoma
(see also breast, ovarian)
Phase II
www.casipharmaceuticals.com
Halaven®
eribulin
Eisai
Woodcliff Lake, NJ
soft tissue sarcoma
(see also breast, lung)
Phase III
www.eisai.com
inhaled lipid cisplatin
Eleison Pharmaceuticals
St. Petersburg, FL
prevention of pulmonary metastases
from osteosarcoma (pediatric)
Phase II
www.eleison-pharma.com
MORAb-004
(CD248 antigen inhibitor)
Eisai
Woodcliff Lake, NJ
soft tissue sarcoma
(see also colorectal, skin)
Phase II
www.eisai.com
niraparib
(PARP inhibitor)
TESARO
Waltham, MA
Ewing’s sarcoma
(see also breast, ovarian)
Phase I
www.tesarobio.com
NY-ESO-1/LAGE-1-specific T-cells
Adaptimmune
Oxon, United Kingdom
synovial sarcoma
(see also myeloma, ovarian, skin)
Phase I
www.adaptimmune.com
sarcoma vaccine
MabVax Therapeutics
San Diego, CA
metastatic sarcoma
Phase II
www.mabvax.com
TH-302
(hypoxia-activated prodrug)
EMD Serono
Rockland, MA
Threshold Pharmaceuticals
South San Francisco, CA
soft tissue sarcoma
(see also hematological, lung,
pancreatic, skin, solid tumors)
Phase III
www.emdserono.com
www.thresholdpharm.com
Sarcoma
ORPHAN DRUG
ORPHAN DRUG
ORPHAN DRUG
Medicines in Development
Cancer 2014
73
Medicines and Vaccines in Development for Cancers
Sarcoma
Product Name
Sponsor
Indication
Development Phase
TRC105
(ENG protein inhibitor)
TRACON Pharmaceuticals
San Diego, CA
advanced soft tissue sarcoma
(see also brain, kidney)
Phase I/II
www.traconpharma.com
Yondelis®
trabectedin
Janssen Research & Development
Raritan, NJ
soft tissue sarcoma
(see also ovarian)
Phase III
www.janssenrnd.com
Product Name
Sponsor
Indication
Development Phase
ABT-RTA-408
AbbVie
North Chicago, IL
Reata Pharmaceuticals
Irving, TX
relapsed/refractory melanoma
(see also lung)
Phase I
www.abbvie.com
www.reatapharma.com
ADI-PEG 20
(PEG arginine deiminase)
Polaris Pharmaceuticals
San Diego, CA
metastatic melanoma
(see also breast, leukemia, liver, lung,
lymphoma)
Phase I
www.polarispharma.com
Ad-RTS-IL-12
(interleukin-12 gene therapy)
ZIOPHARM Oncology
Boston, MA
malignant melanoma
(see also breast)
Phase II
www.ziopharm.com
AE-M
(immunotherapeutic)
Antigen Express
Worcester, MA
melanoma
Phase I
www.antigen-express.com
alpha-gal glycosphingolipid
(immunotherapy)
Agalimmune
Irvine, CA
melanoma
Phase I completed
www.agalimmune.com
ALT-803
(IL-15 superagonist complex)
Altor BioScience
Miramar, FL
metastatic melanoma
(see also bladder, hematological,
myeloma)
Phase I/II
www.altorbioscience.com
AT13387
(HSP90 inhibitor)
Astex Pharmaceuticals
Dublin, CA
melanoma
(see also lung)
Phase II
www.astx.com
bavituximab
Peregrine Pharmaceuticals
Tustin, CA
advanced melanoma
(see also breast, liver, lung, other)
Phase I
www.peregrineinc.com
BBI608
(cancer stemness kinase inhibitor)
Boston Biomedical
Cambridge, MA
melanoma
(see also breast, colorectal, lung,
ovarian, stomach)
Phase I/II
www.bostonbiomedical.com
Cavatak™
coxsackievirus A21
ORPHAN DRUG
Viralytics
Sydney, Australia
malignant melanoma
Phase II
www.viralytics.com
Skin Cancer
ORPHAN DRUG
74
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Skin Cancer
Product Name
Sponsor
Indication
Development Phase
CEP-32496
(BRAF kinase inhibitor)
Teva Pharmaceutical
Frazier, PA
advanced melanoma
(see also colorectal, solid tumors)
Phase II
www.tevapharm.com
cobimetinib
(MEK inhibitor)
Genentech
South San Francisco, CA
metastatic melanoma
(see also solid tumors)
Phase III
www.gene.com
dorgenmeltucel-L
NewLink Genetics
Ames, IA
malignant melanoma
Phase II
www.newlinkgenetics.com
E7016
(PARP inhibitor)
Eisai
Woodcliff Lake, NJ
malignant melanoma
Phase II
www.eisai.com
E7080
(multi-targeted kinase inhibitor)
Eisai
Woodcliff Lake, NJ
melanoma
(see also brain, liver, lung, other)
Phase II
www.eisai.com
Erivedge®
vismodegib
Genentech
South San Francisco, CA
operable basal cell carcinoma
(see also leukemia)
Phase II
www.gene.com
FANG™ Vaccine
genetically modified autologous
tumor cell vaccine
Gradalis
Dallas, TX
advanced melanoma
(see also colorectal, ovarian,
solid tumors)
Phase II
www.gradalisinc.com
G100
(TLR4 agonist)
Immune Design
Seattle, WA
Merkel cell carcinoma
Phase I
www.immunedesign.com
glembatumumab vedotin
(antibody drug conjugate)
CellDex Therapeutics
Hampton, NJ
stage III or IV melanoma
(see also skin)
Phase II
www.celldex.com
GM-CT-01
(galectin inhibitor)
Galectin Therapeutics
Norcross, GA
melanoma
Phase I
www.galectintherapeutics.com
GR-MD-02
(galectin-3 inhibitor)
Galectin Therapeutics
Norcross, GA
melanoma
Phase I
www.galectintherapeutics.com
heat shock protein vaccine
ORPHAN DRUG
Agenus
Lexington, MA
melanoma
(see also brain)
Phase I
www.agenusbio.com
IMCgp100
(CD3 antigen inhibitor)
Immunocore
Oxon, United Kingdom
malignant melanoma
Phase I
www.immunocore.com
INCB24360
(IDO1 inhibitor)
Incyte
Wilmington, DE
metastatic melanoma
(see also lung, ovarian, other)
Phase II
www.incyte.com
interleukin-12 gene therapy
OncoSec Medical
San Diego, CA
malignant melanoma
(see also lymphoma)
Phase II
www.oncosec.com
LDE225
(SMO protein inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
advanced basal cell carcinoma
(see also brain, solid tumors)
Phase II
www.novartis.com
Medicines in Development
Cancer 2014
75
Medicines and Vaccines in Development for Cancers
Skin Cancer
Product Name
Sponsor
Indication
Development Phase
LGX818
(BRAF inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
BRAF-mutant melanoma
(see also colorectal, solid tumors)
Phase III
www.novartis.com
MAGE-A3 immunotherapeutic
(recombinant antigen-specific
cancer immunotherapeutic)
GlaxoSmithKline
Research Triangle Park, NC
melanoma
Phase III
www.gsk.com
MEDI4736 + dabrafenib
+ trametinib
(anti-PD-L1 mAb/BRAF inhibitor/
MEK inhibitor)
MedImmune
Gaithersburg, MD
melanoma
Phase I
www.medimmune.com
MEK162
(MEK inhibitor)
Array BioPharma
Boulder, CO
Novartis Pharmaceuticals
East Hanover, NJ
NRAS-mutant melanoma
(see also ovarian, solid tumors)
Phase III
www.arraybiopharma.com
www.novartis.com
MEK162 and LGX818
(MEK inhibitor/BRAF inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
BRAF-mutant melanoma
Phase III
www.novartis.com
Mekinist® trametinib +
Tafinlar® dabrafenib combination
GlaxoSmithKline
Research Triangle Park, NC
metastatic melanoma (adjuvant)
Phase III
www.gsk.com
NeoStem Oncology
New York, NY
malignant melanoma
(Fast Track)
Phase II
www.neostem.com
MORAb-004
(CD248 antigen inhibitor)
Eisai
Woodcliff Lake, NJ
melanoma
(see also colorectal, sarcoma)
Phase II
www.eisai.com
MSB0010445
(NHS-IL2 cancer immunotherapy)
EMD Serono
Rockland, MA
melanoma
Phase II
www.emdserono.com
MSB0010718C
(anti-PD-L1 mAb)
EMD Serono
Rockland, MA
Merkel cell carcinoma
(see also solid tumors)
Phase II
www.emdserono.com
MVax®
autologous tumor cell-based
vaccine
AVAX technologies
Philadelphia, PA
melanoma
Phase III
www.avax-tech.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
melanoma (1st-line),
melanoma, (2nd-3rd-line)
(see also brain, colorectal, head/neck,
hematological, kidney, liver, lung,
lymphoma, solid tumors)
----------------------------------------melanoma
Phase III
www.bms.com
ORPHAN DRUG
melapuldencel-T
(dendritic cell immunotherapy)
ORPHAN DRUG
76
----------------------------------------Phase I/II
www.bms.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Skin Cancer
Product Name
Sponsor
Indication
Development Phase
nivolumab + Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
melanoma (1st-line)
(see also brain, colorectal, head/neck,
hematological, kidney, lung)
----------------------------------------melanoma
Phase II/III
www.bms.com
----------------------------------------Phase I
www.bms.com
NY-ESO-1/LAGE-1-specific T-cells
Adaptimmune
Oxon, United Kingdom
melanoma
(see also myeloma, ovarian, sarcoma)
Phase I/II
www.adaptimmune.com
Ontak®
denileukin diftitox
Eisai
Woodcliff Lake, NJ
melanoma
Phase II
www.eisai.com
pelareorep
Oncolytics Biotech
Calgary, Canada
metastatic melanoma
(see also colorectal, head/neck, lung,
myeloma, ovarian, pancreatic, solid
tumors, other)
Phase II
www.oncolyticsbiotech.com
Picato®
ingenol mebutate
LEO Pharma
Parsippany, NJ
basal cell cancer
Phase II
www.leo-pharma.us
pimasertib
(MEK inhibitor)
EMD Serono
Rockland, MA
melanoma
(see also pancreatic)
Phase II
www.emdserono.com
PLX3397
(Fms/Kit/Flt3-ITD inhibitor)
Daiichi Sankyo
Parsippany, NJ
Plexxikon
Berkeley, CA
melanoma
(combination therapy)
(see also brain, leukemia)
----------------------------------------melanoma
Phase II
www.dsi.com
www.plexxikon.com
----------------------------------------Phase I
www.dsi.com
www.plexxikon.com
POL-103A
(immunotherapy vaccine)
Polynoma
San Diego, CA
late-stage malignant melanoma
Phase III
www.polynoma.com
Provectus Biopharmaceuticals
Knoxville, TN
malignant melanoma
(see also liver)
Phase II
www.pvct.com
Resimmune™
A-dmDT390-bisFv immunotoxin
Angimmune
Rockville, MD
metastatic melanoma
(see also lymphoma)
Phase I/II
www.angimmune.com
RG7446
(anti-PD-L1 mAb)
Genentech
South San Francisco, CA
malignant melanoma
(see also bladder, kidney, lung)
Phase I
www.gene.com
RG7636
(ETBR antibody drug conjugate)
Genentech
South San Francisco, CA
metastatic melanoma
Phase I
www.gene.com
ORPHAN DRUG
PV-10
(rose bengal sodium)
ORPHAN DRUG
Medicines in Development
Cancer 2014
77
Medicines and Vaccines in Development for Cancers
Skin Cancer
Product Name
Sponsor
Indication
Development Phase
talimogene laherparepvec
(cancer immunotherapy)
Amgen
Thousand Oaks, CA
regionally and distantly metastatic
melanoma
application submitted
www.amgen.com
TH-302
(hypoxia-activated prodrug)
EMD Serono
Rockland, MA
Threshold Pharmaceuticals
South San Francisco, CA
advanced melanoma
(see also hematological, lung,
pancreatic, sarcoma, solid tumors)
Phase II
www.emdserono.com
www.thresholdpharm.com
TRX518
(GITR receptor antagonist)
GITR
Cambridge, MA
advanced melanoma
(see also solid tumors)
Phase I
www.gitrrx.com
tumor infiltrating lymphocyte
(autologous cell therapy)
Lion Biotechnologies
Woodland Hills, CA
National Cancer Institute
Bethesda, MD
malignant melanoma (2nd-line)
Phase II
www.lbio.com
----------------------------------------Phase I
www.lbio.com
Bristol-Myers Squibb
Princeton, NJ
melanoma (adjuvant), metastatic
melanoma (dose optimization)
(see also lung, ovarian, prostate,
stomach)
----------------------------------------melanoma (adolescents)
Phase III
www.bms.com
BRAF-mutation-positive metastatic
melanoma (adjuvant)
(see also other)
----------------------------------------BRAF-mutation-positive metastatic
melanoma
Phase III
www.gene.com
Indication
Development Phase
Yervoy®
ipilimumab
Zelboraf®
vemurafenib
Genentech
South San Francisco, CA
----------------------------------------malignant melanoma (1st-line
combination therapy)
----------------------------------------Phase II
www.bms.com
----------------------------------------Phase I
www.gene.com
Solid Tumors
Product Name
Sponsor
ABBV-399
(antibody drug conjugate)
AbbVie
North Chicago, IL
ABC294640
(sphingosine kinase-2 inhibitor)
Apogee Biotechnology
Hummelstown, PA
advanced solid tumors
Phase I
www.apogee-biotech.com
ABT-414
(antibody drug conjugate)
AbbVie
North Chicago, IL
squamous cell solid tumors
(see also brain)
Phase II
www.abbvie.com
ABT-700
(c-Met inhibitor)
AbbVie
North Chicago, IL
advanced solid tumors
Phase I
www.abbvie.com
78
Phase I
www.abbvie.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
ABT-767
(PARP inhibitor)
AbbVie
North Chicago, IL
ABT-806
(epidermal growth factor receptor
[EGFR] mAB)
AbbVie
North Chicago, IL
advanced solid tumors
Phase I
www.abbvie.com
AG-120
(IDH1 inhibitor)
Agios Pharmaceuticals
Cambridge, MA
Celgene
Summit, NJ
(see also hematological)
Phase I
www.agios.com
www.celgene.com
AGS-22ME
(antibody drug conjugate)
Agensys
Santa Monica, CA
Seattle Genetics
Bothell, WA
Phase I
www.agensys.com
www.seattlegenetics.com
ALB-109564(a)
(tubulin inhibitor)
Bessor-Brightwaters Pharma
Framingham, MA
Phase I
www.bessor-brightwaterspharma.
com
ALT-836
(recombinant chimeric
anti-tissue factor antibody)
Altor BioScience
Miramar, FL
Phase I/II
www.altorbioscience.com
altiratinib
(MET/TIE2/VEGFR2
kinase inhibitor)
Deciphera Pharmaceuticals
Cambridge, MA
Phase I
www.deciphera.com
AM0010
(PEGylated recombinant
human IL-10)
ARMO BioSciences
Redwood City, CA
Phase I
www.armobio.com
AMG 208
(c-Met inhibitor)
Amgen
Thousand Oaks, CA
Phase I
www.amgen.com
AMG 232
Amgen
Thousand Oaks, CA
(see also leukemia)
Phase I
www.amgen.com
AMG 337
(c-Met inhibitor)
Amgen
Thousand Oaks, CA
advanced solid tumors
(see also stomach, other)
Phase I
www.amgen.com
AMG 780
(angiogenesis inhibitor)
Amgen
Thousand Oaks, CA
advanced solid tumors
Phase I
www.amgen.com
AMG 820
(c-fms antagonist)
Amgen
Thousand Oaks, CA
Medicines in Development
Cancer 2014
Indication
Development Phase
Phase I
www.abbvie.com
Phase I
www.amgen.com
79
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
AMG 900
(aurora kinase inhibitor)
Amgen
Thousand Oaks, CA
late-stage solid tumors
(see also hematological)
Phase I
www.amgen.com
anti-LAG3 antibody
Bristol-Myers Squibb
Princeton, NJ
(combination therapy)
(see also other)
Phase I
www.bms.com
APS001F
(recombinant anaerobic bacteria)
Anaeropharma Science
Tokyo, Japan
APTO-253
(KLA4 activator)
Aptose Biosciences
Toronto, Canada
late-stage solid tumors
Phase I
www.aptose.com
AR-12
(PI3K/Akt inhibitor)
Arno Therapeutics
Flemington, NJ
(see also lymphoma)
Phase I
www.arnothera.com
AR-42
(pan-DAC inhibitor)
Arno Therapeutics
Flemington, NJ
(see also hematological)
Phase I
www.arnothera.com
ARQ 087
(FGFR antagonist)
ArQule
Woburn, MA
Phase I
www.arqule.com
ARQ 092
(AKT inhibitor)
ArQule
Woburn, MA
Phase I
www.arqule.com
ARQ 761
(E2F1 transcription factor
stimulant)
ArQule
Woburn, MA
Phase I
www.arqule.com
ATI-1123
(docetaxel liposomal)
Azaya Therapeutics
San Antonio, TX
Phase I
www.azayatherapeutics.com
AT13148
(ROCK inhibitor)
Astex Pharmaceuticals
Dublin, CA
Phase I
www.astx.com
AUY922
(Hsp90 inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
Phase I
www.novartis.com
AZD2014
(mTOR serine/threonine
kinase inhibitor)
AstraZeneca
Wilmington, DE
Phase II
www.astrazeneca.com
AZD4547
(FGFR tyrosine kinase inhibitor)
AstraZeneca
Wilmington, DE
Phase II
www.astrazeneca.com
AZD8186
(PI3K beta inhibitor)
AstraZeneca
Wilmington, DE
Phase I
www.astrazeneca.com
BAX-69
(anti-MIF antibody)
Baxter Healthcare
Deerfield, IL
80
Phase I
www.anaeropharma.co.jp
metastatic solid tumors
Phase I
www.baxter.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
BAY949343
(mesothelin antibody drug
conjugate)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
advanced solid tumors
Phase I
www.bayerpharma.com
BAY1129980
(C4.4a antibody drug conjugate)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
solid tumors with LYPD3 protein
expression
Phase I
www.bayerpharma.com
BBI503
(cancer stemness kinase inhibitor)
Boston Biomedical
Cambridge, MA
bendamustine ready-to-dilute
liquid formulation
Eagle Pharmaceuticals
Woodcliff Lake, NJ
BGB-283
(BRAF inhibitor)
BeiGene
Beijing, China
EMD Serono
Rockland, MA
Phase I
www.beigene.com
www.emdserono.com
BGB-290
(tankyrase inhibitor)
BeiGene
Beijing, China
EMD Serono
Rockland, MA
Phase I
www.beigene.com
www.emdserono.com
BGJ398
(FGF-R kinase inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
BI 836845
(somatomedin inhibitor)
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
Phase I
www.boehringer-ingelheim.com
BI 853520
(PTK2 protein inhibitor)
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
Phase I
www.boehringer-ingelheim.com
BI 860585
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
Phase I
www.boehringer-ingelheim.com
BKM120
(PI3K inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
BLY719
(PI3K-alpha inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
Phase I
www.novartis.com
BPM 31510 intravenous
(ubidecarenone IV)
Berg Pharma
Framingham, MA
Phase I
www.berghealth.com
briciclib
(cyclin D1 modulator)
Onconova Therapeutics
Newtown, PA
BVD-CNV
(C. novyi-NT)
BioMed Valley Discoveries
Kansas City, MO
Medicines in Development
Cancer 2014
Phase I/II
www.bostonbiomedical.com
(see also leukemia, lymphoma)
advanced solid tumors
(see also brain, hematological, liver)
(see also breast)
metastatic solid tumors
Phase I
www.eagleus.com
Phase I
www.novartis.com
Phase I
www.novartis.com
Phase I
www.onconova.com
Phase I
www.biomed-valley.com
81
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
CB-839
(glutaminase inhibitor)
Calithera Biosciences
South San Francisco, CA
advanced solid tumors
(see also hematological)
Phase I
www.calithera.com
CBL0137
(curaxin)
Cleveland BioLabs
Buffalo, NY
advanced solid tumors
Phase I
www.cbiolabs.com
CC-115
(dual TORK/DNA PK inhibitor
modulator)
Celgene
Summit, NJ
CC-122
(pleiotropic pathway modulator)
Celgene
Summit, NJ
CC-223
(dual TORK inhibitor)
Celgene
Summit, NJ
CC-486
Celgene
Summit, NJ
(see also hematological)
Phase I
www.celgene.com
CDX-1401
(NY-ESO-1 cancer vaccine)
Celldex Therapeutics
Hampton, NJ
solid tumors expressing NY-ESO-1
protein
Phase I
www.celldex.com
CEP-32496
(BRAF kinase inhibitor)
Teva Pharmaceutical
Frazier, PA
advanced solid tumors
(see also colorectal, skin)
Phase I
www.tevapharm.com
CEP-37250/KHK2801
Kyowa Hakko Kirin Pharma
Princeton, NJ
Teva Pharmaceutical
Frazier, PA
advanced solid tumors
Phase I
www.kyowa-hakko-kirin.com
www.tevapharm.com
CEP-37440
(dual anaplastic lymphoma kinase
and PTK2 protein inhibitor)
Teva Pharmaceutical
Frazier, PA
late-stage solid tumors
Phase I
www.tevapharm.com
CGM097
(p53/MDM2-interaction inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
late-stage solid tumors
Phase I
www.novartis.com
CLR457
(PI3K inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
advanced solid tumors
Phase I/II
www.novartis.com
cobimetinib
(MEK inhibitor)
Genentech
South San Francisco, CA
(see also skin)
Phase I
www.gene.com
crolibulin
Immune Pharmaceuticals
Cambridge, MA
CUDC-427
(IAP inhibitor)
Curis
Lexington, MA
82
Phase I
www.celgene.com
(see also lymphoma)
Phase I
www.celgene.com
Phase I
www.celgene.com
Phase II
www.immunepharmaceuticals.com
late-stage solid tumors
(see also lymphoma)
Phase I
www.curis.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
Cyramza®
ramucirumab
Lilly
Indianapolis, IN
combination therapy
(see also bladder, breast, colorectal,
liver, lung, ovarian, stomach)
Phase II
www.lilly.com
dacomitinib
(pan-HER inhibitor)
Pfizer
New York, NY
(see also lung)
Phase I
www.pfizer.com
DCVax®-Direct
dendritic cell vaccine
Northwest Biotherapeutics
Bethesda, MD
inoperable solid tumors
Phase I/II
www.nwbio.com
Debio 1143
(IAP inhibitor)
Debiopharma
Lausanne, Switzerland
(see also lymphoma)
Phase I
www.debiopharm.com
Debio 1347
(FGFR inhibitor)
Debiopharm
Lausanne, Switzerland
late-stage solid tumors
Phase I
www.debiopharm.com
DFP-11207
(thymidylate synthase expression
inhibitor)
Delta-Fly Pharma
Tokushima, Japan
advanced solid tumors
Phase I
DLYE-5953A
Genentech
South San Francisco, CA
refractory solid tumors
Phase I
www.gene.com
DS-3032
(MDM2 inhibitor)
Daiichi Sankyo
Parsippany, NJ
(see also lymphoma)
Phase I
www.dsi.com
DS-3078
(mTOR inhibitor)
Daiichi Sankyo
Parsippany, NJ
(see also lymphoma)
Phase I
www.dsi.com
DS-7423
(PI3K/mTOR inhibitor)
Daiichi Sankyo
Parsippany, NJ
DS-8273
(anti-DR5 antibody)
Daiichi Sankyo
Parsippany, NJ
late-stage solid tumors
(see also lymphoma)
Phase I
www.dsi.com
E6201
(MEK-1/MEKK-1 kinase inhibitor)
Eisai
Woodcliff Lake, NJ
advanced solid tumors
Phase I
www.eisai.com
E7389
(liposomal eribulin)
Eisai
Woodcliff Lake, NJ
Phase I
www.eisai.com
EC1456
(folate-tubulysin)
Endocyte
West Lafayette, IN
Phase I
www.endocyte.com
enoticumab
Regeneron Pharmaceuticals
Tarrytown, NY
Medicines in Development
Cancer 2014
Phase I
www.dsi.com
advanced solid malignancies
Phase I
www.regeneron.com
83
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
entolimod
(cancer immunotherapy)
Cleveland BioLabs
Buffalo, NY
advanced solid tumors
Phase I
www.cbiolabs.com
EP-100
(membrane disrupting peptide)
Esperance Pharmaceuticals
San Francisco, CA
(see also hematological)
Phase II
www.esperancepharma.com
EPZ-6438
(EZH2 inhibitor)
Epizyme
Cambridge, MA
INI1-deficient solid tumors
(see also lymphoma)
Phase I
www.epizyme.com
FANG™ Vaccine
genetically modified autologous
tumor cell vaccine
Gradalis
Dallas, TX
advanced solid tumors
(see also colorectal, ovarian, skin)
Phase I
www.gradalisinc.com
fenretinide intravenous
CerRx
Lubbock, TX
adult solid tumors, pediatric mixed
tumors
(see also lymphoma)
Phase II
www.cerrx.com
G305
(GLA-SE+NY-ESO-1 protein)
Immune Design
Seattle, WA
GDC-0575
(ChK1 inhibitor)
Genentech
South San Francisco, CA
GDC-0994
(ERK inhibitor)
Genentech
South San Francisco, CA
Phase I
www.gene.com
GI-6301
(brachyury peptide vaccine)
GlobeImmune
Lousiville, CO
Phase I completed
www.globeimmune.com
GL-ONC1
(oncolytic virus immunomodulator)
Genelux
San Diego, CA
GO-203-2c
(MUC1 inhibitor)
Genus Oncology
Vernon Hills, IL
Phase I
www.genusoncology.com
GS-5745
(MMP9 mAb inhibitor)
Gilead Sciences
Foster City, CA
Phase I
www.gilead.com
HDM201
(tumor suppressor protein
p53 modulator)
Novartis Pharmaceuticals
East Hanover, NJ
(see also hematological)
Phase I
www.novartis.com
I-131-CLR1404
(molecular radiotherapeutic)
Cellectar Biosciences
Madison, WI
advanced solid tumors
Phase I completed
www.cellectar.com
IMGN289
(EGFR kinase inhibitor)
ImmunoGen
Waltham, MA
EGFR-positive solid tumors
Phase I
www.immunogen.com
84
Phase I
www.immunedesign.com
(see also lymphoma)
Phase I
www.gene.com
(see also head/neck, lung, other)
Phase II
www.genelux.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
IMGN853
(folate receptor 1 inhibitor)
ImmunoGen
Waltham, MA
folate receptor alpha-positive solid
tumors
Phase I
www.immunogen.com
IMMU-132
(antibody drug conjugate)
Immunomedics
Morris Plains, NJ
metastatic solid tumors
Phase II
www.immunomedics.com
INC280
(c-Met inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
late-stage solid tumors
Phase II
www.novartis.com
INCB39110
(JAK1 inhibitor)
Incyte
Wilmington, DE
advanced solid tumors
(see also lung)
Phase I
www.incyte.com
IT-139
(GRP78 protein inhibitor)
Intezyne
Tampa, FL
ixazomib
(proteasome inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
(see also hematological, lymphoma,
myeloma)
Phase I
www.millennium.com
JNJ-42756493
(FGFR inhibitor)
Janssen Research & Development
Raritan, NJ
late-stage solid tumors
(see also lymphoma)
Phase I
www.janssenrnd.com
KPT-330
(XPO1 inhibitor)
Karyopharm Therapeutics
Newton, MA
advanced solid tumors
(see also brain, leukemia, prostate,
other)
Phase I
www.karyopharm.com
KTN3379
(ErbB3 kinase inhibitor)
Kolltan Pharmaceuticals
New Haven, CT
late-stage solid tumors
Phase I
www.kolltan.com
LB-100
(protein phosphatase 2A inhibitor)
Lixte Biotechnology
East Setauket, NY
LDE225
(SMO protein inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
(see also brain, skin)
Phase II
www.novartis.com
LEE011
(CDK4/6 inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
(see also breast)
Phase I
www.novartis.com
LGX818
(BRAF inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
BRAF-mutant tumors
(see also colorectal, skin)
Phase II
www.novartis.com
LJM716
(ERBB-3 receptor antagonist)
Novartis Pharmaceuticals
East Hanover, NJ
lirilumab
(anti-KIR mAb)
Bristol-Myers Squibb
Princeton, NJ
Medicines in Development
Cancer 2014
Phase I
www.intezyne.com
Phase I
www.lixte.com
Phase I
www.novartis.com
solid tumors (with nivolumab),
solid tumors (with Yervoy® )
Phase I
www.bms.com
85
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
LOXO-101
(PanTrk inhibitor)
Array BioPharma
Boulder, CO
Loxo Oncology
Stamford, CT
Phase I
www.arraybiopharma.com
www.loxooncology.com
LV305
(NY-ESO-1 Zvex)
Immune Design
Seattle, WA
Phase I
www.immunedesign.com
LY2606368
(Chk1 inhibitor)
Lilly
Indianapolis, IN
advanced solid tumors
(see also breast, ovarian)
Phase I
www.lilly.com
margetuximab
(anti-HER2 mAb)
MacroGenics
Rockville, MD
(see also breast, other)
Phase I
www.macrogenics.com
ME-143
(NADH oxidase inhibitor)
MEI Pharma
San Diego, CA
refractory solid tumors
Phase I
www.meipharma.com
MEDI-565
(anti-CEA BITE mAb)
MedImmune
Gaithersburg, MD
Phase I
www.medimmune.com
MEDI0639
(anti-DLL-4 mAb)
MedImmune
Gaithersburg, MD
Phase I
www.medimmune.com
MEDI0680
(anti-PD-1 mAb)
MedImmune
Gaithersburg, MD
Phase I
www.medimmune.com
MEDI3617
(anti-ANG-2 mAb)
MedImmune
Gaithersburg, MD
Phase I
www.medimmune.com
MEDI4736 + MEDI0680
(anti-PD-L1 mAb/anti-PD-1 mAb)
MedImmune
Gaithersburg, MD
Phase I
www.medimmune.com
MEDI4736 + tremelimumab
(anti-PD-L1 mAb/anti-CTLA-4
mAB)
MedImmune
Gaithersburg, MD
Phase I
www.medimmune.com
MEDI6469
(murine anti-OX40 mAb)
AgonOx
Portland, OR
MedImmune
Gaithersburg, MD
Phase I
www.agonox.com
www.medimmune.com
MEK162
(MEK inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
MGA271
(CD276 protein inhibitor)
Macogenics
Rockville, MD
MGCD516
(multi kinase inhibitor)
Mirati Therapeutics
San Diego, CA
86
Indication
(see also ovarian, skin)
Development Phase
Phase I/II
www.novartis.com
Phase I
www.macrogenics.com
late-stage solid tumors
Phase I
www.mirati.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
MGN 1703
(TLR9 agonist)
Mologen
Berlin, Germany
Phase I completed
www.mologen.com
MINT1526A
(integrin-alpha-5/beta-1 inhibitor)
Genentech
South San Francisco, CA
Phase I
www.gene.com
MK-2206
(AKT inhibitor)
Merck
Whitehouse Station, NJ
Phase II
www.merck.com
MK-4166
(TNFRSF18 protein stimulant)
Merck
Whitehouse Station, NJ
Phase I
www.merck.com
MK-8033
(c-Met/RON inhibitor)
Merck
Whitehouse Station, NJ
Phase I completed
www.merck.com
MK-8242
(MDM-2 inhibitor)
Merck
Whitehouse Station, NJ
Phase I
www.merck.com
MLN0128
(TORC1/TORC2 inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
MLN1117
(PI3K alpha inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
Phase I
www.millennium.com
MLN2480
(Pan-Raf kinase inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
Phase I
www.millennium.com
MLN4924
(Nedd8-activating enzyme
inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
MLN7243
(UAE inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
MM-151
(EGFR oligoclonal antibody)
Merrimack Pharmaceuticals
Cambridge, MA
MNK-010
Mallinckrodt Pharmaceuticals
St. Louis, MO
MSB0010718C
(anti-PD-L1 mAb)
EMD Serono
Rockland, MA
MSC2156119
(c-Met kinase inhibitor)
EMD Serono
Rockland, MA
Phase I
www.emdserono.com
MSC2363318A
(p70S6K/Akt inhibitor)
EMD Serono
Rockland, MA
Phase I
www.emdserono.com
Medicines in Development
Cancer 2014
Indication
(see also breast, hematological)
(see also hematological)
Development Phase
Phase I
www.millennium.com
Phase I
www.millennium.com
Phase I
www.millennium.com
refractory advanced solid tumors
Phase I
www.merrimackpharma.com
Phase I
www.mallinckrodt.com
(see also skin)
Phase I
www.emdserono.com
87
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
MUC-1 dendritic cancer vaccine
MicroVAX
Manassas, VA
Phase I
MVA-BN Brachyury
(immunotherapy vaccine)
Bavarian-Nordic
Mountain View, CA
Phase I
www.bavarian-nordic.com
NC-4016
(DACH platin micelle)
NanoCarrier
Chiba, Japan
Phase I
www.nanocarrier.co.jp
NHS-IL12
(cancer immunotherapy)
EMD Serono
Rockland, MA
Phase I
www.emdserono.com
nivolumab
(PD-1 inhibitor)
Bristol-Myers Squibb
Princeton, NJ
NLG919
(immune checkpoint inhibitor)
NewLink Genetics
Ames, IA
OMP-52M51
(NOTCH-1 inhibitor)
OncoMed Pharmaceuticals
Redwood City, CA
ONT-10
(cancer immunotherapy)
Oncothyreon
Seattle, WA
Phase I
www.oncothyreon.com
OPB-111077
Otsuka Pharmaceutical
Rockville, MD
Phase I
www.otsuka.com
oprozomib
(proteasome inhibitor)
Onyx Pharmaceuticals
South San Francisco, CA
oraxol
(oral paclitaxel)
Kinex Pharmaceuticals
Buffalo, NY
Phase I
www.kinexpharma.com
OTS167
(MELK protein inhibitor)
OncoTherapy Science
Kanagawa Prefecture, Japan
Phase I
www.oncotherapy.co.jp
PB272
(neratinib)
Puma Biotechnology
Los Angeles, CA
HER2-mutated solid tumors
(see also breast)
Phase II
www.pumabiotechnology.com
PEG-SN38
(angiogenesis inhibitor)
Belrose Pharma
Princeton, NJ
(see also brain, breast)
Phase I
www.belrosepharma.com
pelareorep
Oncolytics Biotech
Calgary, Canada
relapsed or refractory solid tumors
(pediatric)
(see also colorectal, head/neck, lung,
myeloma, ovarian, pancreatic, skin,
other)
Phase I
www.oncolyticsbiotech.com
88
Indication
Development Phase
solid tumors (signal detection)
(see also brain, colorectal, head/neck,
hematological, kidney, liver, lung,
lymphoma, skin)
Phase I
www.bms.com
Phase I
www.newlinkgenetics.com
(see also hematological)
Phase I
www.oncomed.com
(see also hematological)
Phase I
www.onyx.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
PFK-158
(PFKFB3 inhibitor)
Advanced Cancer Therapeutics
Louisville, KY
Phase I
www.advancedcancertherapeutics.
com
pimasertib/SAR405838
(MEK inhibitor/hDM2 inhibitor)
EMD Serono
Rockland, MA
Sanofi US
Bridgewater, NJ
Phase I
www.emdserono.com
www.sanofi.com
pimasertib/SAR245409
(MEK inhibitor/dual PI3K, mTOR
inhibitor)
EMD Serono
Rockland, MA
Sanofi US
Bridgewater, NJ
(see also ovarian)
Phase I
www.emdserono.com
www.sanofi.com
PLX7486
(TRK inhibitor)
Daiichi Sankyo
Parsippany, NJ
Plexxikon
Berkeley, CA
TRK-driven tumors
(see also pancreatic)
Phase I
www.dsi.com
www.plexxikon.com
PLX8394
(BRAF inhibitor)
Daiichi Sankyo
Parsippany, NJ
Plexxikon
Berkeley, CA
(see also leukemia)
Phase I
www.dsi.com
www.plexxikon.com
PM060184
(tubulin polymerisation inhibitor)
Pharmamar
Madrid, Spain
PNT2258
(bcl-2 oncogene inhibitor)
ProNAi Therapeutics
Plymouth, MI
(see also lymphoma)
Phase II
www.pronai.com
PRI-724
(CBP/β-catenin inhibitor)
PRISM Pharma
Tokyo, Japan
(see also leukemia, pancreatic)
Phase I
www.prismbiolab.com
PT-112
(apoptosis stimulant)
Phosplatin Therapeutics
New York, NY
late-stage solid tumors
Phase I
www.phosplatin.com
purine nucleoside phosphorylase
gene therapy
PNP Therapeutics
Birmingham, AL
Phase I
www.pnptherapeutics.com
QBI-139
(RNA inhibitor)
Quintessence Biosciences
Madison, WI
Phase I
www.quintbio.com
rebastinib
(TRK, BCR-ABL, TIE2 inhibitor)
Deciphera Pharmaceuticals
Cambridge, MA
Phase I/II
www.deciphera.com
Medicines in Development
Cancer 2014
Indication
Development Phase
Phase I
www.pharmamar.com
89
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
refametinib
(MEK inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
advanced metastatic solid tumors
Phase I/II
www.bayerpharma.com
RG7304
(dual Raf/MEK inhibitor)
Genentech
South San Francisco, CA
RG7388
(dual Raf/MEK inhibitor)
Genentech
South San Francisco, CA
RG7841
(antibody drug conjugate)
Genentech
South San Francisco, CA
RRx-001
(free radical stimulant)
RadioRx
Mountain View, CA
late-stage solid tumors
(see also colorectal, lymphoma)
Phase I
www.radiorx.com
RX-3117
(cancer specific cytotoxic agent)
Rexahn
Rockville, MD
late-stage solid tumors
Phase I
www.rexahn.com
RX-5902
(p68 RNA helicase)
Rexahn
Rockville, MD
late-stage solid tumors
Phase I
www.rexahn.com
RXDX 101
(selective tyrosine kinase inhibitor)
Ignyta
San Diego, CA
Phase I/II
www.ignyta.com
SAR125844
(c-Met kinase inhibitor)
Sanofi US
Bridgewater, NJ
Phase I
www.sanofi.com
SAR245408
(PI3K inhibitor)
Sanofi US
Bridgewater, NJ
Phase I
www.sanofi.com
SAR260301
(PI3K-beta inhibitor)
Sanofi US
Bridgewater, NJ
SAR307746
(anti-ANG2 mAb)
Regeneron Pharmaceuticals
Tarrytown, NY
Sanofi US
Bridgewater, NJ
Phase I
www.regeneron.com
www.sanofi.com
SAR405838
(HDM2/p53 antagonist)
Ascenta Therapeutics
Malvern, PA
Sanofi US
Bridgewater, NJ
Phase I
www.ascenta.com
www.sanofi.com
SAR566658
(maytansin-loaded anti-CA6 mAb)
Sanofi US
Bridgewater, NJ
Phase I
ww.sanofi.com
90
Phase I
www.gene.com
(see also hematological)
Phase I
www.gene.com
Phase I
www.gene.com
PTEN-deficient tumors
Phase I
www.sanofi.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
sEphB4-HSA
(recombinant albumin fusion
protein)
VasGene Therapeutics
Los Angeles, CA
Phase I
SF1126
(pan-PI3K inhibitor)
SignalRx Pharmaceuticals
San Diego, CA
Phase I completed
www.signalrx.com
SP-02
(darinaparsin)
Solasia Pharma
Tokyo, Japan
(see also hematological)
Phase I
www.solasia.co.jp
Stivarga®
regorafenib
Bayer HealthCare Pharmaceuticals
Whippany, NJ
advanced solid tumors
(see also colorectal, kidney, liver)
Phase I/II
www.bayerpharma.com
Sym004
(anti-EGFR mAb)
EMD Serono
Rockland, MA
(see also colorectal, head/neck)
Phase I
www.emdserono.com
TAK-659
(SYK kinase inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
(see also hematological)
Phase I
www.millennium.com
TAK-733
(MEK inhibitor)
Millennium Pharmaceuticals
Cambridge, MA
Phase I
www.millennium.com
TAS-114
(dUTPase/DPD inhibitor)
Taiho Oncology
Princeton, NJ
Phase I
www.taihooncology.com
TAS-119
(aurora kinase A inhibitor)
Taiho Oncology
Princeton, NJ
late-stage solid tumors
Phase I
www.taihooncology.com
TAS-120
(FGF/FGFR inhibitor)
Taiho Oncology
Princeton, NJ
advanced solid tumors
(see also myeloma)
Phase I
www.taihooncology.com
taselisib
(PI3K inhibitor)
Genentech
South San Francisco, CA
(see also breast)
Phase I
www.gene.com
TCN-P
(triciribine)
Cahaba Pharmaceuticals
Princeton, NJ
TEN-010
(BET inhibitor)
Tensha Therapeutics
Cambridge, MA
TEW-7197
(TGF-beta type I receptor
antagonist)
MedPacto
Suwon, South Korea
TH-302
(hypoxia-activated prodrug)
EMD Serono
Rockland, MA
Threshold Pharmaceuticals
South San Francisco, CA
Medicines in Development
Cancer 2014
Indication
Development Phase
Phase II
www.cahabapharma.com
advanced solid tumors
Phase I
www.tenshatherapeutics.com
Phase I
www.medpacto.com
(see also hematological, lung,
pancreatic, sarcoma, skin)
Phase I
www.emdserono.com
www.thresholdpharm.com
91
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
thioureidobutyronitrile
(tumor suppressor protein p53
stimulant)
Cellceutix
Beverly, MA
Phase I
www.cellceutix.com
TKI258
(multi-targeted receptor tyrosine
kinase inhibitor)
Novartis Phamaceuticals
East Hanover, NJ
Phase II
www.novartis.com
TRX518
(GITR receptor antagonist)
GITR
Cambridge, MA
advanced solid tumors
(see also skin)
Phase I
www.gitrrx.com
TVB-2640
(FASN inhibitor)
3-V Biosciences
Menlo Park, CA
advanced solid tumors
Phase I
www.3vbio.com
U3-1565
(anti-HB-EGF antibody)
Daiichi Sankyo
Parsippany, NJ
urelumab
Bristol-Myers Squibb
Princeton, NJ
(see also hematological)
Phase I
www.bms.com
vanticitumab
(anti-Fzd7 antibody)
OncoMed Pharmaceuticals
Redwood City, CA
advanced solid tumors
Phase I
www.oncomed.com
varlilumab
(CD27 inhibitor)
Celldex Therapeutics
Hampton, NJ
(see also hematological)
Phase I
www.celldex.com
VGX 100
(VEGF-C antibody)
Ceres Oncology
Victoria, Australia
vintafolide
(folate-DAVLBH)
Endocyte
West Lafayette, IN
advanced solid tumors
(combination therapy)
(see also lung)
Phase I
www.endocyte.com
VS-4718
(FAK inhibitor)
Verastem
Cambridge, MA
late-stage solid tumors
Phase I
www.verastem.com
VS-5584
(PI3K/mTOR inhibitor)
Verastem
Cambridge, MA
late-stage solid tumors
(see also lymphoma)
Phase I
www.verastem.com
VX15
(CD100 antigen inhibitor)
Vaccinex
Rochester, NY
Phase I
www.vaccinex.com
VX-970
(ATR protein inhibitor)
Vertex Pharmaceuticals
Boston, MA
Phase I
www.vrtx.com
WT2725
(cancer immunotherapy)
Sunovion Pharmaceuticals
Marlborough, MA
92
Indication
Development Phase
Phase I
www.dsi.com
Phase I
www.ceresoncology.com
(see also hematological)
Phase I
www.sunovion.com
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Solid Tumors
Product Name
Sponsor
Indication
Development Phase
X396
(ALK inhibitor)
Xcovery
West Palm Beach, FL
advanced solid tumors
Phase I
www.xcovery.com
X82
(VEGFR/PDGFR antagonist)
Tyrogenex
West Palm Beach, FL
Xalkori®
crizotinib
Pfizer
New York, NY
(see also lung)
Phase I
www.pfizer.com
Product Name
Sponsor
Indication
Development Phase
Afinitor®
everolimus
Novartis Pharmaceuticals
East Hanover, NJ
non-functioning gastrointestinal
neuroendocrine tumors
(see also breast, lymphoma, stomach)
Phase III
www.novartis.com
AMG 337
(c-Met inhibitor)
Amgen
Thousand Oaks, CA
gastric cancer
(see also solid tumors, other)
Phase II
www.amgen.com
BBI608
(cancer stem cell inhibitor)
Boston Biomedical
Cambridge, MA
gastric cancer
(see also breast, colorectal, lung,
ovarian, skin, other)
----------------------------------------gastrointestinal cancers
(combination therapy)
Phase III
www.bostonbiomedical.com
Phase I
www.xcovery.com/tyrogenex-inc
Stomach Cancer
----------------------------------------Phase I
www.bostonbiomedical.com
crenolanib
(PDGFR inhibitor)
AROG Pharmaceuticals
Dallas, TX
GIST
(see also brain)
Phase II
www.arogpharma.com
Cyramza®
ramucirumab
Lilly
Indianapolis, IN
gastric cancer (2nd-line
combination therapy)
(see also bladder, breast, colorectal,
liver, lung, ovarian, solid tumors)
application submitted
www.lilly.com
Iclusig®
ponatinib
ARIAD Pharmaceuticals
Cambridge, MA
GIST (2nd-line)
(see also leukemia)
Phase II
www.ariad.com
ipatasertib
(GDC-0068)
(Akt inhibitor)
Array BioPharma
Boulder, CO
Genentech
South San Francisco, CA
gastric cancer
(see also breast, prostate)
Phase II
www.arraybiopharma.com
www.gene.com
ORPHAN DRUG
Medicines in Development
Cancer 2014
93
Medicines and Vaccines in Development for Cancers
Stomach Cancer
Product Name
Sponsor
Indication
Development Phase
Kadcyla®
ado-trastuzumab emtansine
Genentech
South San Francisco, CA
advanced HER2-positive gastric cancer
(see also breast)
Phase III
www.gene.com
Keytruda®
pembrolizumab
Merck
Whitehouse Station, NJ
gastric cancer
(see also bladder, breast,
head/neck, hematological, kidney,
lung, myeloma)
Phase I
www.merck.com
LY2875358
(c-Met mAb)
Lilly
Indianapolis, IN
advanced gastric cancer
(see also lung)
Phase II
www.lilly.com
masitinib
ORPHAN DRUG
AB Science USA
Short Hills, NJ
late-stage GIST (1st-line)
(see also pancreatic)
Phase III
www.ab-science.com
Minnelide™ 001
HSP70 inhibitor
Minneamrita Therapeutics
Moline, IL
advanced gastrointestinal tumors
Phase I
MLN0264
(GCC antibody drug conjugate)
Millennium Pharmaceuticals
Cambridge, MA
advanced gastrointestinal cancer
(see also pancreatic)
Phase II
www.millennium.com
MM-111
(ErbB3/ErbB2 receptor antagonist)
Merrimack Pharmaceuticals
Cambridge, MA
gastric cancer (2nd-line)
Phase II
www.merrimackpharma.com
NeuVax®
nelipepimut-S
Galena Biopharma
Portland, OR
gastric cancer
(see also breast, kidney, liver, lung,
lymphoma, skin, other)
Phase I completed
www.galenabiopharma.com
olaparib
(PARP inhibitor)
AstraZeneca
Wilmington, DE
gastric cancer (2nd-line)
(see also breast, stomach)
Phase III
www.astrazeneca.com
Perjeta®
pertuzumab
Roche
Nutley, NJ
advanced HER2-positive gastric cancer
(see also breast)
Phase III
www.roche.com
rilotumumab
(hepatocyte growth factor/
scatter factor inhibitor)
Amgen
Thousand Oaks, CA
MET-positive gastric cancer
Phase III
www.amgen.com
S-1
(gimeracil/oteracil/tegafur
fixed-dose combination)
Taiho Oncology
Princeton, NJ
gastric cancer
Phase III
www.taihooncology.com
SM04755
(Wnt inhibitor)
Samumed
San Diego, CA
advanced gastric cancer
(see also colorectal, liver, pancreatic)
Phase I
www.samumed.com
Somatuline® Depot
lanreotide auto-gel formulation
Ipsen Biopharmaceuticals
Basking Ridge, NJ
gastroenteropancreatic
neuroendocrine tumors
application submitted
www.ipsenus.com
ORPHAN DRUG
94
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Stomach Cancer
Product Name
Sponsor
Indication
Development Phase
telatinib
(VEGFR inhibitor)
Eddingpharm
Westlake Village, CA
gastric cancer
Phase II completed
www.eddingpharm.com
TKM-PLK1
(RNA interference)
Tekmira Pharmaceuticals
Burnaby, Canada
gastrointestinal neuroendocrine
tumors
(see also liver, other)
Phase II
www.tekmira.com
Yervoy®
ipilimumab
Bristol-Myers Squibb
Princeton, NJ
gastric cancer
(see also lung, ovarian, prostate, skin)
Phase II
www.bms.com
Product Name
Sponsor
Indication
Development Phase
Adcetris®
brentuximab vedotin
Seattle Genetics
Bothell, WA
CD30-positive non-lymphoma
malignancies
(see also lymphoma)
Phase II
www.seattlegenetics.com
ADXS-HPV
(live, attenuated Listeria
monocytogenes (Lm)-based
immunotherapy vaccine)
Advaxis
Princeton, NJ
cervical cancer
(see also head/neck, other)
----------------------------------------anal cancer
Phase II
www.advaxis.com
----------------------------------------Phase I/II
www.advaxis.com
AEB701
(PKC inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
uveal melanoma (combination
therapy)
(see also lymphoma)
Phase I/II
www.novartis.com
AMG 337
(c-Met inhibitor)
Amgen
Thousand Oaks, CA
gastroesophageal junction cancer,
esophageal cancer
(see also stomach, solid tumors)
Phase II
www.amgen.com
ATR-101
(sterol O-acyltransferase inhibitor)
Atterocor
Ann Arbor, MI
adrenocortical carcinoma
Phase I
www.atterocor.com
Genentech
South San Francisco, CA
Roche
Nutley, NJ
recurrent cervical cancer
(see also brain, lung, ovarian)
application submitted
www.gene.com
www.roche.com
----------------------------------------Phase III
www.gene.com
www.roche.com
ORPHAN DRUG
Other Cancers
ORPHAN DRUG
ORPHAN DRUG
Avastin®
bevacizumab
Medicines in Development
Cancer 2014
----------------------------------------metastatic or locally advanced
high-risk neuroendocrine tumor
95
Medicines and Vaccines in Development for Cancers
Other Cancers
Product Name
Sponsor
Indication
Development Phase
bavituximab
Peregrine Pharmaceuticals
Tustin, CA
rectal cancer
(see also breast, liver, lung, skin)
Phase I
www.peregrineinc.com
BBI608
(cancer stem cell inhibitor)
Boston Biomedical
Cambridge, MA
gastroesophageal junction cancer
(see also breast, colorectal, lung,
ovarian, skin, stomach)
Phase III
www.bostonbiomedical.com
DN24-02
(active cellular immunotherapy)
Dendreon
Seattle, WA
HER2-positive urothelial carcinoma
Phase II
www.dendreon.com
DKN-01
(DKK1 protein inhibitor)
HealthCare Pharmaceuticals
Cambridge, MA
esophageal cancer, gastroesophageal
junction cancer, gastroesophageal
cancer
(see also lung, myeloma)
Phase I
www.healthcarepharmaceuticals.com
E7080
(multi-targeted kinase inhibitor)
Eisai
Woodcliff Lake, NJ
thyroid cancer
(see also brain, liver, lung, skin)
----------------------------------------endometrial cancer
Phase III completed
www.eisai.com
----------------------------------------Phase II
www.eisai.com
GALE-301
(folate binding protein
immunotherapy)
Galena Biopharma
Portland, OR
endometrial cancer
(see also ovarian)
Phase II
www.galenabiopharma.com
GI-6207
(natural killer cell stimulant)
GlobeImmune
Louisville, CO
medullary thyroid cancer
Phase II
www.globeimmune.com
GL-ONC1
(oncolytic virus immunomodulator)
Genelux
San Diego, CA
peritoneal carcinomatosis
(see also head/neck, lung, solid
tumors)
Phase I/II
www.genelux.com
GSK525762
(bromodomain inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
NUT gene midline carcinoma
Phase I
www.gsk.com
INCB24360
(IDO1 inhibitor)
Incyte
Wilmington, DE
fallopian tube cancer,
peritoneal cancer
(see also lung, ovarian, skin)
Phase II
www.incyte.com
INO-3106
(therapeutic vaccine)
Inovio
Plymouth Meeting, PA
HPV-6-associated aerodigestive
malignancies
Phase I
www.inovio.com
INO-3112
(cancer therapeutic vaccine)
Inovio Pharmaceuticals
Plymouth Meeting, PA
cervical cancer caused by HPV
types 16/18
(see also head/neck)
Phase I
www.inovio.com
96
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Other Cancers
Product Name
Sponsor
Indication
Development Phase
Jakafi®
ruxolitinib
Incyte
Wilmington, DE
polycythemia vera
(see also breast, colorectal, lung,
pancreatic)
----------------------------------------advanced malignancies
Phase III
www.incyte.com
----------------------------------------Phase I
www.incyte.com
margetuximab
(anti-HER2 mAb)
MacroGenics
Rockville, MD
gastroesophageal cancer
(see also breast, solid tumors)
Phase II
www.macrogenics.com
Marqibo®
vinCRIStine sulfate LIPOSOME
injection
Spectrum Pharmaceuticals
Henderson, NV
pediatric cancers
(see also leukemia, lymphoma)
Phase I/II
www.sppirx.com
milciclib
(CDK inhibitor)
Nerviano Medical Sciences
Nerviano, Italy
malignant thymoma
Phase II
www.nervianoms.com
MORAb-066
(tissue factor antigen inhibitor)
Eisai
Woodcliff Lake, NJ
advanced TF-expressing malignancies
Phase I
www.eisai.com
NTO-1151
(ribonuclease inhibitor)
Nanotherapeutics
Alachua, FL
National Cancer Institute
Bethesda, MD
cervical cancer, vaginal cancer
Phase II
www.nanotherapeutics.com
pelareorep
Oncolytics Biotech
Calgary, Canada
fallopian tube cancer, primary
peritoneal cancer
(see also colorectal, head/neck, lung,
myeloma, ovarian, pancreatic, skin,
solid tumors)
Phase II
www.oncolyticsbiotech.com
seliciclib
(CDK inhibitor)
Cyclacel Pharmaceuticals
Berkeley Heights, NJ
nasopharyngeal cancer
(see also lung)
Phase II
www.cyclacel.com
selumetinib
(MEK inhibitor)
Array BioPharma
Boulder, CO
AstraZeneca
Wilmington, DE
differentiated thyroid cancer,
uveal melanoma
(see also lung)
Phase III
www.arraybiopharma.com
www.astrazeneca.com
SOR-C13
(TRPV6 protein inhibitor)
Soricimed Biopharma
Sackville, Canada
cancers that overexpress the TRPV6
calcium channel
Phase I
www.soricimed.com
Sprycel®
dasatinib
Bristol-Myers Squibb
Princeton, NJ
Otsuka America Pharmaceutical
Rockville, MD
pediatric cancer
(see also pancreatic)
Phase II
www.bms.com
www.otsuka.com
Medicines in Development
Cancer 2014
97
Medicines and Vaccines in Development for Cancers
Other Cancers
Product Name
Sponsor
Indication
Development Phase
telotristat etiprate
(TPH1 inhibitor)
Lexicon Pharmaceuticals
The Woodlands, TX
malignant carcinoid syndrome
(Fast Track)
Phase III
www.lexicon-genetics.com
tirapazamine
(type II DNA topoisomerase
inhibitor)
SRI International
Menlo Park, CA
cervical cancer
Phase III
www.sri.com
TKM-PLK1
(RNA interference)
Tekmira Pharmaceuticals
Nurnaby, Canada
adrenocortical carcinoma
(see also liver, stomach)
Phase II
www.tekmira.com
V503
(human papillomavirus vaccine
9-valent)
Merck
Whitehouse Station, NJ
prevention of HPV-related cancers
application submitted
www.merck.com
VB-111
(vascular targeting system)
VBL Therapeutics
Or Yehuda, Israel
thyroid cancer
(see also brain, ovarian)
Phase II
www.vblrx.com
Zelboraf®
vemurafenib
Genentech
South San Francisco, CA
BRAF-mutation-positive papillary
thyroid cancer
(see also skin)
Phase II
www.gene.com
zoptarelin doxorubicin
(type II DNA topoisomerase
inhibitor)
AEterna Zentaris
Quebec, Canada
endometrial cancer (2nd-line)
(see also prostate)
Phase III
www.aezsinc.com
Product Name
Sponsor
Indication
Development Phase
AGS-67E
(antibody drug conjugate)
Agensys
Santa Monica, CA
Astellas Pharma
Tokyo, Japan
Phase I
www.agensys.com
AMG 110
(recombinant bi-specific antibody)
Amgen
Thousand Oaks, CA
Phase I
www.amgen.com
BAY1082439
(PI3K inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Phase I
www.bayerpharma.com
BAY1125976
(allosteric AKT1/2 inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Phase I
www.bayerpharma.com
ORPHAN DRUG
Unspecified Cancer
98
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Unspecified Cancer
Product Name
Sponsor
BAY1143572
(PTEFb inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Phase I
www.bayerpharma.com
BAY1161909
(MPS1 inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Phase I
www.bayerpharma.com
BAY1163877
(Pan-FGFR inhibitor)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Phase I
www.bayerpharma.com
BAY1179470
(FGFR2 antibody)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Phase I
www.bayerpharma.com
BAY2010112
(PSMA BiTE antibody)
Bayer HealthCare Pharmaceuticals
Whippany, NJ
Phase I
www.bayerpharma.com
BVD-523
(ERK 1/2 kinase inhibitor)
BioMed Valley Discoveries
Kansas City, MO
Phase I/II
www.biomed-valley.com
CHML
(cytotropic heterogeneous
molecular lipids)
Glory Pharmaceuticals
Vienna, VA
Phase I
www.anticancerdrug.com
CSF-1R mAb
Lilly
Indianapolis, IN
Phase I
www.lilly.com
G1T28-1 (IV)
(CDK 4/6 inhibitor)
G1 Therapeutics
Research Triangle Park, NC
Phase I
www.g1therapeutics.com
GCS-100
(galectin-3 protein inhibitor)
La Jolla Pharmaceutical
San Diego, CA
Phase I completed
www.lajollapharmaceutical.com
GPX-150
Gem Pharmaceuticals
Birmingham, AL
Phase I
www.gempharmaceuticals.com
GSK2256098
(focal adhesion kinase inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
Phase I
www.gsk.com
GSK2636771
(PI3K inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
Phase I
www.gsk.com
GSK2816126
(EZH2 enzyme inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
Phase I
www.gsk.com
GSK2849330
(ErbB3 mAb)
GlaxoSmithKline
Research Triangle Park, NC
Phase I
www.gsk.com
GSK3052230
(FGF ligand trap)
GlaxoSmithKline
Research Triangle Park, NC
Phase I
www.gsk.com
Medicines in Development
Cancer 2014
Indication
Development Phase
99
Medicines and Vaccines in Development for Cancers
Unspecified Cancer
Product Name
Sponsor
HBI-8000
(HDAC inhibitor)
Huya Bioscience
San Diego, CA
Phase I
www.huyabio.com
high ADCC anticancer mAb
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
Xencor
Monrovia, CA
Phase I
www.boehringer-ingelheim.com
www.xencor.com
IGF-methotrexate conjugate
IGF Oncology
Birchwood, MN
Phase I
www.igfoncology.com
IP-112
InnoPharma
Piscataway, NJ
application submitted
www.innopharmainc.com
LGK974
(WNT signaling pathway inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
Phase I
www.novartis.com
LY2228820
(p38 MAP kinase inhibitor)
Lilly
Indianapolis, IN
Phase II
www.lilly.com
LY2874455
(FGF receptor inhibitor)
Lilly
Indianapolis, IN
Phase II
www.lilly.com
LY2780301
(p70S6/AKT dual inhibitor)
Lilly
Indianapolis, IN
Phase I
www.lilly.com
LY2801653
(c-Met inhibitor)
Lilly
Indianapolis, IN
Phase II
www.lilly.com
LY3009120
(pan-Raf inhibitor)
Lilly
Indianapolis, IN
Phase I
www.lilly.com
LY3023414
(PI3K/mTOR dual inhibitor)
Lilly
Indianapolis, IN
Phase I
www.lilly.com
LY3039478
(NOTCH inhibitor)
Lilly
Indianapolis, In
Phase I
www.lilly.com
MM-141
(IGF-1R/ErbB3 bispecific
tetravalent antibody)
Merrimack Pharmaceuticals
Cambridge, MA
Phase I
www.merrimackpharma.com
notch inhibitor
Bristol-Myers Squibb
Princeton, NJ
Phase I
www.bms.com
olaratumab
(PDGF-alpha receptor antagonist)
Lilly
Indianapolis, IN
Phase II
ww.lilly.com
100
Indication
Development Phase
Medicines in Development
Cancer 2014
Medicines and Vaccines in Development for Cancers
Unspecified Cancer
Product Name
Sponsor
OMN54
(signal transduction pathway
inhibitor)
Omnitura Therapeutics
Redwood Shores, CA
Phase I
www.omnitura.com
OPB-51602
(molecular targeting drug)
Otsuka Pharmaceutical
Rockville, MD
Phase I
www.otsuka.com
PBI-05204
(triple kinase inhibitor)
Phoenix Biotechnology
San Antonio, TX
Phase I
www.phoenixbiotechnology.com
PD-0325901
(MEK1/2 inhibitor)
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-03084014
(gamma secretase inhibitor)
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-05082566
(CD137 antigen agonist)
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-06263507
(auristatin-based antibody drug
conjugate)
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-06439535
(bevacizumab biosimilar)
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-06463922
(dual ALK/ROS1 inhibitor)
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-06664178
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-06647263
(immunoconjugate)
Pfizer
New York, NY
Phase I
www.pfizer.com
PF-06650808
Pfizer
New York, NY
Phase I
www.pfizer.com
REGN1400
(ERBB-3 receptor modulator)
Regeneron Pharmaceuticals
Tarrytown, NY
Phase I
www.regeneron.com
rintatolimod
Hemispherx Pharma
Philadelphia, PA
SNX-5422
(HSP90 heat-shock protein
inhibitor)
Esanex
Indianapolis, IN
Phase I
TGF-BR2 mAb
Lilly
Indianapolis, IN
Phase I
www.lilly.com
Medicines in Development
Cancer 2014
Indication
adjuvant therapy
Development Phase
Phase I/II
www.hemispherx.net
101
Medicines and Vaccines in Development for Cancers
Unspecified Cancer
Product Name
Sponsor
Indication
Development Phase
trametinib + GSK2141795
(MEK1/2 inhibitor + AKT protein
kinase inhibitor)
GlaxoSmithKline
Research Triangle Park, NC
Phase I
www.gsk.com
VEGFR-3 mAb
Lilly
Indianapolis, IN
Phase I
www.lilly.com
Definitions
Application Submitted—An application for marketing has been submitted by the company to the U.S. Food and Drug Administration (FDA).
Breakthrough Therapy—Upon request by a sponsor, the FDA can grant this designation to expedite the development and review of a drug or biologic intended, alone or in
combination with one or more other drugs, to treat a serious or life threatening disease or condition and preliminary clinical evidence indicates that it may demonstrate substantial improvement over existing therapies on one or more clinically-significant endpoints, such as substantial treatment effects observed early in clinical development. If a drug or
biologic is designated as a breakthrough therapy, the FDA will expedite the development and review. With this designation, all Fast Track features convey to the medicine.
Fast Track—Upon request by a sponsor, the FDA can grant this designation to facilitate the development and expedite the review of a drug or biologic to treat a serious
condition and fill an unmet medical need. When considering a biopharmaceutical company’s request for Fast Track designation for an investigational drug or biologic, the
FDA evaluates whether it will affect factors such as survival, day-to-day functioning, or the likelihood that the disease, if left untreated, will progress from a less severe
condition to a more serious one, and whether a condition can be adequately addressed by available therapy. With Fast Track designation, early and frequent
communication between the FDA and the biopharmaceutical company is encouraged throughout the entire drug development and review process to help to quickly resolve
any questions or issues that arise, potentially leading to an earlier approval and access by patients.
Orphan Designation—Upon request by a sponsor, the FDA can grant special status (“orphan status”) to a drug or biologic to treat a rare disease or condition. In order
to receive an orphan designation, a qualifying drug or biologic must be intended for the treatment, diagnosis, or prevention of a rare disease or condition that affects
fewer than 200,000 people in the United States, or that affects more than 200,000 persons but is not expected to recover the costs of its development and marketing.
Phase I—Researchers test the investigational drug or biologic in a small group of people, usually between 20 and 100 healthy adult volunteers, to evaluate its initial
safety and tolerability profile, determine a safe dosage range, and identify potential side effects.
Phase II—The investigational drug or biologic is given to volunteer patients, usually between 100 and 500, to determine whether it is effective, identify an optimal dose,
and to further evaluate its short-term safety.
Phase III—The investigational drug or biologic is given to a larger, more diverse patient population, often involving between 1,000 and 5,000 patients (but sometimes many more),
to generate statistically significant evidence to confirm its safety and effectiveness. Phase III studies are the longest studies and usually take place in multiple sites around the world.
The content of this report has been obtained through public, government and industry sources, and the Adis “R&D Insight”
database based on the latest information. Report current as of September 26, 2014. The medicines in this report include
medicines being developed by U.S.-based companies conducting trials in the United States and abroad, PhRMA-member
companies conducting trials in the United States and abroad, and foreign companies conducting clinical trials in the United
States. The information in this report may not be comprehensive. For more specific information about a particular product,
contact the individual company directly or go to www.clinicaltrials.gov. The entire series of Medicines in Development is
available on PhRMA’s website.
A publication of PhRMA’s Communications & Public Affairs Department (202) 835-3460
www.phrma.org | www.innovation.org | www.pparx.org
Provided as a public service by PhRMA. Founded in 1958 as the Pharmaceutical Manufacturers Association.
Copyright © 2014 by the Pharmaceutical Research and Manufacturers of America. Permission to reprint is awarded if proper
credit is given.
Pharmaceutical Research and Manufacturers of America • 950 F Street, NW, Suite 300, Washington, DC 20004
102
Medicines in Development
Cancer 2014
The Drug Discovery, Development and Approval Process
Developing a new medicine takes an average of 10-15 years.
Of the tens of thousands of compounds screened, only one is approved.
The Drug Development and Approval Process
The U.S. system of new drug approvals is
perhaps the most rigorous in the world.
It takes 10-15 years, on average, for an
investigational drug to travel from the lab to U.S.
patients, according to the Tufts Center for the
Study of Drug Development. Tens of thousands
of compounds may be screened early in
development, but only one ultimately receives
approval. Even medicines that reach clinical trials
have only a 16 percent chance of being approved.
On average, it costs a company $1.2 billion,
including the cost of failures, to get one new
medicine from the laboratory to U.S. patients,
according to a 2007 study by the Tufts Center
for the Study of Drug Development. More recent
studies estimate the cost to be even higher.
Once a new compound has been identified in
the laboratory, medicines are usually developed
as follows:
Preclinical Testing—A pharmaceutical company
conducts laboratory and animal studies to show
biological activity of the compound against the
targeted disease, and the compound is evaluated
for safety.
Investigational New Drug Application (IND)—
After completing preclinical testing, a company
Medicines in Development
Cancer 2014
files an IND with the U.S. Food and Drug
Administration (FDA) to begin to test the drug
in people. The IND shows results of previous
experiments; how, where and by whom the new
studies will be conducted; the chemical structure
of the compound; how it is thought to work in
the body; any toxic effects found in the animal
studies; and how the compound is manufactured.
All clinical trials must be reviewed and approved
by the Institutional Review Board (IRB) where
the trials will be conducted. Progress reports on
clinical trials must be submitted at least annually
to FDA and the IRB.
Clinical Trials, Phase I—Researchers test the
drug in a small group of people, usually between
20 and 100 healthy adult volunteers, to evaluate
its initial safety and tolerability profile, determine
a safe dosage range, and identify potential side
effects.
Clinical Trials, Phase II—The drug is given
to volunteer patients, usually between 100 and
500, to see if it is effective, identify an optimal
dose, and to further evaluate its short-term
safety.
Clinical Trials, Phase III—The drug is given to a
larger, more diverse patient population, often
involving between 1,000 and 5,000 patients
(but sometimes many more), to generate
statistically significant evidence to confirm its
safety and effectiveness. They are the longest
studies, and usually take place in multiple sites
around the world.
New Drug Application (NDA)/Biologic License
Application (BLA)—Following the completion
of all three phases of clinical trials, a company
analyzes all of the data and files an NDA or BLA
with FDA if the data successfully demonstrate
both safety and effectiveness. The applications
contain all of the scientific information that the
company has gathered. Applications typically
run 100,000 pages or more.
Approval—Once FDA approves an NDA or
BLA, the new medicine becomes available
for physicians to prescribe. A company must
continue to submit periodic reports to FDA,
including any cases of adverse reactions and
appropriate quality-control records. For some
medicines, FDA requires additional trials
(Phase IV) to evaluate long-term effects.
Discovering and developing safe and effective
new medicines is a long, difficult, and expensive
process. PhRMA member companies invested
an estimated $51.1 billion in research and
development in 2013.
103
ull
olor
l ack
white
Pharmaceutical Research and Manufacturers of America
950 F Street, NW, Washington, DC 20004
www.phrma.org