Download medicines in development for neurological disorders

Neurological Disorders
A Report on Disorders of the Brain, Spinal Cord and Nerves
2013 REPORT
Medicines in Development
presented by america’s biopharmaceutical research companies
Biopharmaceutical Companies Researching and
Developing Nearly 450 Medicines for
Neurological Disorders
Medicines in Development
For Neurological Disorders
Application
Submitted
Phase III
Phase II
Phase I
82
on
’s
•28 medicines for epilepsy and seizures, which impact more than
3 million Americans.
•27 medicines for Parkinson’s disease,
which affects as many as 1 million
Americans.
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•Gene therapy to reverse the effects of
Parkinson’s disease.
•38 medicines for multiple sclerosis,
which afflicts an estimated 500,000
Americans.
28
Br
ain
•82 medicines for Alzheimer’s disease,
which afflicts more than 5 million
Americans.
•62 medicines for brain tumors—nearly
70,000 Americans are diagnosed
each year with a primary brain tumor.
38
zh
Other medicines in development target
amyotrophic lateral sclerosis (ALS),
brain injuries, Huntington’s disease,
spinal cord injury, cerebral palsy, and
stroke. Among the many promising new
medicines in development are:
•82 medicines for pain—100 million
U.S. adults experience chronic pain.
82
62
Al
Neurological disorders—such as epilepsy, multiple sclerosis and Parkinson’s
disease—inflict great pain and suffering
on patients and their families, and every
year costs the U.S. economy billions
of dollars. Biopharmaceutical research
companies are developing 444 new
medicines to prevent and treat neurological disorders. The medicines in
development are either in human clinical
trials or under review at the Food and
Drug Administration. They include:
•25 medicines for headache, including migraine, a condition that affects
more than 37 million people.
•A medicine that prompts the immune
system to protect neurons affected by
ALS.
•Gene therapy for the treatment of
Alzheimer’s disease.
The new medicines being developed by
biopharmaceutical research companies
reflect a growing understanding of the
underlying mechanisms of neurological
disorders, which fuels scientific progress
and pharmaceutical research. These medicines offer patients hope that more effective treatments may soon be available.
Contents
Key Issues.......................................2
Disease Facts...................................7
Medicines in Development................11
Glossary....................................... 49
Drug Development/
Approval Process........................ 51
Innovative Medicines in the Pipeline
orders such as Duchenne muscular dystrophy (DMD).
In DMD, DNA deletions cause mutations in important
genes that encode for dystrophin, a structural protein found in normal muscle. The loss of this protein
causes muscle fibers to disintegrate faster than they
can be regenerated. One medicine in development
targets restoration of dystrophin function and, as dystrophin expression increases, there have been demonstrated improvements in patients’ ability to walk.
Of the 444 medicines in development in the United States
listed in this report (see page 11), many present innovative
new ways to target diseases. Some of them include:
•Gene Therapy to Restore Neuronal Function in
Alzheimer’s—A gene therapy for the treatment
of Alzheimer’s disease in clinical trials is designed
to deliver nerve growth factor (NGF) to the brain.
NGF is a naturally occurring protein important for
neuron survival. The gene treatment is injected into
the brain region where neuron degeneration occurs
in Alzheimer’s disease. It is thought that the resulting sustained expression of NGF in the neurons can
restore their lost function, leading to memory and
cognition improvement.
•Gene Therapy as a Possible Approach for Parkinson’s
Disease—Inserting genes into cells can alter the impact the genes have on the proteins that are involved
in a particular disease. These genes might alter or
replace a mutated gene or produce a new therapeutic protein entirely. In Parkinson’s disease, there
are a number of treatments addressing the disease’s
symptoms, but none that replace the lost nerve cells
resulting from Parkinson’s or that would stop disease
progression. More than one gene therapy in clinical
trials uses an adeno-associated virus (AAV) as a vector
•Targeted RNAi Therapy Approach for Duchenne
Muscular Dystrophy—In clinical trials, RNAi therapies
have shown potential in treating neuromuscular dis-
Medicines in Development for Neurological Disorders By Disease and Phase
Some medicines are listed in more than one category
Alzheimer’s Disease
82
Amyotrophic Lateral Sclerosis (ALS)
8
Attention-Deficit/Hyperactivity Disorder
Phase III
19
Brain Injury
Phase II
8
Brain Tumors
28
Genetic Disorders
14
Headache
Huntington’s Disease
25
7
Multiple Sclerosis
Muscular Dystrophy
38
7
Pain
82
Parkinson’s Disease
Spasticity
Spinal Cord Injury
Stroke
Other
2
Phase I
62
Epilepsy
Application
Submitted
27
6
9
19
33
Medicines in Development
Neurological Disorders 2013
to deliver neurturin to restore cells damaged in
Parkinson’s patients and to protect them from further degeneration; these inactivated viruses present a safe way to get these important therapeutic
options to patients.
IMPORTANCE TREATMENT
OF
ALZHEIMER’S
5
•ALS: Fighting a Devastating Disease—ALS, or
Lou Gehrig’s disease, is a progressive neurodegenerative disease that causes the brain to lose
control over body movement, ultimately resulting
in paralysis and death. The one drug approved to
treat ALS can modestly slow progression of the
disease, but new treatments are needed. As our
scientific understanding of the disease has grown,
researchers are pursuing many new approaches
to halt or slow disease, including the use of the
patient’s own bone marrow stem-cells to create healthy neuron-like cells to replace diseased
neurons. Other trials are studying ways to prompt
the immune system to protect neurons affected
by ALS.
MILLION
AMERICANS
ARE LIVING WITH ALZHEIMER’S DISEASE
15
MILLION
AMERICANS
Alzheimer’s:
Effective Treatment is Needed
Alzheimer’s is the 6th leading cause of
death in the United States, and its impact
on patients and the health care system is
growing. Existing medicines are able to treat
the symptoms of the disease but cannot slow,
prevent, or reverse the progressive dementia.
Disease-modifying treatments that could
delay the onset of the disease could reduce
the cost of care of Alzheimer’s patients in
2050 by $447 billion. Researchers continue
to unravel the mysteries of the disease
and are studying many new treatments
in this area. Recent research has focused
on the plaques and tangles which form
in the brains of Alzheimer’s patients and
are thought to contribute to the death of
nerve cells. One medicine in development
has shown promise in reducing both brain
plaques and tangles. A gene therapy also in
clinical trials is being explored to restore lost
neuron function.
Medicines in Development
Neurological Disorders 2013
BY 2050, THE NUMBER OF PATIENTS COULD TRIPLE
WITHOUT EFFECTIVE TREATMENT
2050
2013
203
1.2
$
$
TRILLION
BILLION
ALZHEIMER’S COSTS SOCIETY $203 BILLION
AND COULD INCREASE TO $1.2 TRILLION BY 2050
5 50%
YEARS
DELAYED
ONSET
=
FEWER
VICTIMS
447
+
$
BILLION
SAVED
DELAYING DISEASE ONSET BY 5 YEARS COULD RESULT IN
50% FEWER PATIENTS/SAVE $447 BILLION BY 2050
3
Advancing Biomedical Science
Over the past decade, scientific advances and new technologies have dramatically changed how medicines are discovered. This new information is critical to the development of
new treatments for neurological disorders. Greater knowledge of how diseases work at the genetic and molecular level
has allowed researchers to pursue new targets for therapy
and better predict how certain biopharmaceuticals will affect
specific subpopulations of patients.
• B
ioinformatics—Bioinformatics use systems and
mathematical models to advance the scientific understanding of living systems. At its simplest level,
bioinformatics involves the creation and maintenance
of biological databases, including DNA sequences.
• Biomarkers—Every
disease leaves a signature of
molecular “biomarkers” in our body—genes that turn
on and off or proteins released into the bloodstream.
Biomarkers measured in blood and other samples
can tell us the state of our health and how we might
respond to treatment.
• Molecular
Targeting—The idea behind molecular
targeting is to design drugs that specifically attack the
molecular pathways that cause disease without disrupting the normal functions in our cells and tissues.
• Nanotechnology—You
can’t see it, but soon it will be
everywhere. Nanotechnology is the science of building microscopic devices at the molecular and atomic
levels. In medicine, nanotechnology may also be used
to help diagnose and treat diseases.
• Personalized
Medicine—The sequencing of the
human genome produced a “map” of the human
genes in DNA. This new genetic knowledge opens
up the possibility of developing “targeted” therapies
for people with specific gene sequences, and it can
help physicians choose the best treatments based on
individual genetic, lifestyle, and environmental factors.
4
Breakthrough Research in
Neurological Disorders
Early research discoveries often fuel the drug development
pathways that biopharmaceutical company researchers undertake. These discoveries help researchers target a specific
disease through certain mechanisms that may have been
unknown before. Some noteworthy recent scientific discoveries in the field of neurology include:
• R
esearchers at Mount Sinai Medical Center found
10 genes that account for half of the genetic risk for
Alzheimer’s.
• R
esearch at the National Institutes of Health found
that a protein linked with some early-onset Parkinson’s disease cases regulates how the body processes
dietary fats. The study suggests there could be a link
between the defective protein and early-onset Parkinson’s disease.
• S
cientists at the University of Chicago have uncovered
a previously unknown process of protein production
where a single gene can create two separate proteins
from the same messenger RNA simultaneously. They
believe this discovery will open the door for new
research into therapies for neurological disorders.
• R
esearch at the University of Missouri suggests that
two identical neurons can reach the same electrical
activity in different ways and could help doctors in
treating patients with epilepsy.
• T
wo separate research groups have identified a mutation on the TREM2 gene that may increase a person’s
chance of developing late-stage Alzheimer’s disease
by three to five times.
• W
hile studying at PennVet, a researcher discovered
the gene responsible for Alexander disease, a rare,
genetic neurological disorder where the white matter
in the brain is destroyed and Rosenthal fibers form
causing both mental and physical declines.
Medicines in Development
Neurological Disorders 2013
• A
t the University of Notre Dame, researchers have
created a prodrug (an inactive drug that is converted
into its active form once it’s in the body) of an inhibitor of the gelatinase enzyme. Gelatinase is associated
with certain neurological disorders, such as strokes,
aneurysms and traumatic brain injury.
• R
esearchers at the Manchester Institute of Biotechnology in the United Kingdom have identified an
enzyme in the brain that interacts with a compound
for Huntington’s disease to inhibit its activity. Animal
studies have shown that by switching off the enzyme’s
activity through drug-binding, the enzyme is effective in treating brain disorders such as Huntington’s,
Alzheimer’s, and Parkinson’s.
The BRAIN Initiative
Brain Research through Advancing Innovative
Neurotechnologies is a research effort launched
by the Obama Administration to map the human
brain and increase our understanding of how the
human mind works. Approximately $100 million
will be directed to government agencies, such as
the National Institutes of Health, beginning in FY
2014. The BRAIN Initiative aims to accelerate the
development and application of new technologies
to help researchers understand the interworking’s
of the brain, leading to new ways to treat and
maybe one day prevent or cure diseases, such as
Alzheimer’s, epilepsy and traumatic brain injury.
Public/Private Partnerships Critical
to Advancing Science
Collaboration among partners in the entire medical innovation
ecosystem is critical to help advance scientific understanding
of some of the most complex diseases facing patients. Federal
research institutions, academia, biopharmaceutical research
companies and patient communities all play an important role
in furthering research in the neurological field.
These evolving partnerships take many forms, for example:
• O
ne biopharmaceutical company recently formed a
research consortium to bring together several leading academic research centers to coordinate their
research and share results. Through this collaboration,
researchers hope to accelerate their understanding of
the disease and identify new approaches for targeting
and treating amyotrophic lateral sclerosis (ALS).
• T
he Alzheimer’s Disease Neuroimaging Initiative is
a collaborative effort between several federal agencies, non-profit organizations, and biopharmaceutical
industry members. The goal of the Initiative is to track
Alzheimer’s disease progression, establish quality standards, and validate biomarkers to be used in clinical
Medicines in Development
Neurological Disorders 2013
trials. Data collected are made available at no cost to
researchers when designing clinical trials and research
projects.
• A
nother biopharmaceutical company is collaborating
with a medical school’s neurodegenerative disease
research center to screen for Alzheimer’s drug candidates. The company is sharing their basic research
with the research center’s screening assays and
knowledge of the biology of the tau protein, which
is thought to play a significant role in Alzheimer’s
disease.
• T
he Coalition Against Major Diseases, a program of
the Critical Path Institute, is a consortium of biopharmaceutical research companies, academic institutions, regulatory agencies, patient advocacy groups,
research foundations, scientific associations and
consultants that work collaboratively through a precompetitive partnership to accelerate development of
therapeutics for neurodegenerative diseases.
5
New Neurological Medicines
Approved for Patients
The new medicines being developed today build on the medical
progress seen over the last decade or so. Below are examples of innovative medicines approved recently to treat some
neurological conditions.
Attention-Deficit/Hyperactivity Disorder (ADHD)
• Vyvanse® (lisdexamphetamine dimesylate) is the first
stimulant prodrug approved for the treatment of
ADHD. It is an oral biologic medicine that is therapeutically inactive until metabolized in the body. It is
intended to provide a safer, abuse-resistant and effective alternative to amphetamine-based therapies for
ADHD.
Alzheimer’s Disease
• N
amenda® (memantine) is the first treatment approved for moderate to severe Alzheimer’s disease.
It was also the first in a new class of medicines called
NMDA receptor antagonists to be approved for
the disease. Namenda interferes with the effects of
excess glutamate release—glutamate plays a key role
in the neural pathways associated with learning and
memory.
6
Multiple Sclerosis (MS)
• T
ecfidera™ (dimethyl fumarate; BG-12) is a first-line
oral treatment for relapsing forms of MS. It is believed to treat the disease in a new way by stimulating
the Nrf2 transcriptional pathway that provides a way
for cells in the body to defend against inflammation and neuronal death that is induced by oxidative
stress. In clinical trials it was shown to reduce relapses
and the development of brain lesions, as well as slow
disability progression.
• G
ilenya® (fingolimod) is a first-in-class oral medicine
also approved to treat relapsing forms of MS. With
a different mechanism of action, it binds to the S1PR
lipid and traps certain white blood cells in the lymph
nodes, thereby reducing the immune system’s attack
on the central nervous system (CNS). By preventing
the blood cells from reaching the CNS where they
can damage the covering around the nerve fibers, this
medicine can reduce damage to the nerve cells.
Seizures
• P
otiga® (ezogabine) is a first-in-class potassium channel blocker approved for the adjunctive treatment of
partial-onset seizures in adults. While the exact mechanism of action is unknown, it is believed to work by
opening the potassium channels, which are thought to
stabilize the neurons and reduce brain excitability.
Medicines in Development
Neurological Disorders 2013
Selected Facts about Neurological Disorders
Alzheimer’s Disease and Other Dementias1
•An estimated 5.4 million Americans have Alzheimer’s disease (AD). Today, someone in America develops AD every 68 seconds. By 2050, there is expected to be one new case of AD every 33 seconds, or nearly a million new cases per year, and
AD prevalence is projected to be 11 million to 16 million.
•AD is the sixth leading cause of death in the United States and the fifth leading cause of death in Americans age 65 and older.
Amyotrophic Lateral Sclerosis2
•An estimated 30,000 Americans may have amyotrophic lateral sclerosis (ALS) at any given time, and some 5,600 people in
the United States are diagnosed with ALS each year. It is estimated that ALS is responsible for nearly 2 deaths per 100,000
population annually.
Brain Injury3
•According to the U.S. Centers for Disease Control (CDC) Injury Prevention Center, the leading causes of traumatic brain injury (TBI) are: falls, 35.2 percent; unknown/other, 21 percent; motor vehicle, 17.3 percent; head strikes, 16.5 percent; assault,
10 percent.
•Traumatic brain injury is the leading cause of disability and death in children and adolescents in the United States. According
to the CDC, the two age groups at greatest risk for TBI are ages 0-4 and 15-19. Among children ages 0 to 14 years, each
year TBI results in an estimated: 2,685 deaths; 37,000 hospitalizations; and 435,000 emergency department visits.
•
Approximately 1,300 U.S. children experience severe or fatal brain trauma from child abuse every year.
Brain Tumors4
•An estimated 69,720 new cases of primary brain tumors (those that begin and tend to stay in the brain) are expected to be
diagnosed in 2013, including both malignant (24,620) and non-malignant (45,100) brain tumors.
•In 2010, more than 688,096 people in the United States were living with the diagnosis of a primary brain or central nervous system tumor—more than 138,054 were malignant tumors and more than 550,042 were non-malignant.
•Gliomas, a broad term which includes all tumors arising from the gluey or supportive tissue of the brain, represent 30 percent
of all brain tumors and 80 percent of all malignant tumors. Glioblastomas represent 17 percent of all primary brain tumors
and 54 percent of all gliomas.
Medicines in Development
Neurological Disorders 2013
7
Selected Facts about Neurological Disorders
Epilepsy5
•Epilepsy affects nearly 3 million Americans. Epilepsy is the fourth most common neurological disorder in the United States
after migraine, stroke, and Alzheimer’s disease. And it costs society $17.6 billion in direct and indirect costs.
•Epilepsy strikes most often among the very young and the very old, although anyone can develop the disorder at any age.
In this country, it affects more than 300,000 children under the age of 15—more than 90,000 of whom have seizures that
cannot be adequately treated.
•The number of epilepsy cases in the elderly is climbing as the baby boom generation reaches retirement age. More than
300,000 adults age 65 and above have the condition.
Genetic Disorders6
•More than 6,000 known genetic disorders account for a significant portion of human disease and conditions and can present
themselves in several different ways, such as Down syndrome, spina bifida, and sickle cell anemia.
•Up to 4 percent of the approximately 4 millon babies born each year have a genetic disease or major birth defect. More than
20 percent of infant deaths are caused by birth defects or genetic conditions (e.g., congenital heart defects, abnormalities of the
nervous system, or chromosomal abnormalities).
•Approximately 10 percent of all adults and 30 percent of children in hospitals are there due to genetically-related problems.
Headache
•Headaches are the most prevalent neurological disorders and among the most frequent symptoms seen in general
practice—50 percent of the general population have headaches during any given year, and more than 90 percent report a
lifetime history of headache.7
•Chronic headache (a headache that occurs 15 or more days per month) affects 3 percent of the general population, and
those people are the most severely disabled.7
•More than 37 million Americans suffer from migraine, with women being affected three times more often than men. This
vascular headache is most commonly experienced between the ages of 15 and 55, and 70 percent to 80 percent of sufferers have a family history of migraine.8
•The financial cost of headache arises partly from direct treatment costs, but much more from loss of work time and productivity. The annual U.S. direct medical costs attributable to migraine were estimated at $1 billion in 1999.7
Huntington’s Disease9
•More than 15,000 Americans have Huntington’s disease (HD). At least 150,000 others have a 50 percent risk of developing the disease, and thousands more of their relatives live with the possibility that they, too, might develop HD.
8
Medicines in Development
Neurological Disorders 2013
Selected Facts about Neurological Disorders
Muscular Dystrophy10
•The incidence rates of muscular dystrophies (MD) vary depending on the specific type. Duchenne MD is the most common MD and is sex-linked, with an inheritance pattern of 1 case per 3,500 live male births. Becker MD is the second most
common form, with an incidence of 1 case per 30,000 live male births. Other types of MD are rare. For example, limb-girdle
dystrophy occurs in only 1.3 percent of patients with MDs.
Multiple Sclerosis11
•Some 350,000 to 500,000 patients suffer from multiple sclerosis (MS) in the United States. Most cases are diagnosed
between 20 and 50 years.
Pain12
•In 2011, at least 100 million adult Americans had common chronic pain conditions.
•Recent Centers for Disease Control and Prevention (CDC) and National Center for Health Statistics (NCHS) data suggest substantial rates of pain from various causes and that most people in chronic pain have multiple sites of pain. For U.S. adults reporting pain, causes include: severe headache or migraine (16.1 percent), low back pain (28.1 percent), neck pain (15.1 percent), knee
pain (19.5 percent), shoulder pain (9.0 percent), finger pain (7.6 percent), and hip pain (7.1 percent).
•Pain is a significant public health problem that costs society at least $560-$635 billion annually (an amount equal to about
$2,000.00 for everyone living in the United States).
Parkinson’s Disease
•In the United States, 50,000-60,000 new cases of Parkinson’s disease (PD) are diagnosed each year, adding to the 1 million
people who currently have PD. The U.S. Centers for Disease Control and Prevention rated complications from Parkinson’s
disease as the 14th leading cause of death in the United States.13
•
Men are one and a half times more likely to have Parkinson’s than women.14
•The combined direct and indirect costs of Parkinson’s, including treatment, Social Security payments and lost income from
inability to work, is estimated to be nearly $25 billion per year in the United States. Medication costs for an individual person
with PD average $2,500 a year, and therapeutic surgery can cost up to $100,000 dollars per patient.14
Spasticity15
•Spasticity is a common finding in multiple sclerosis (MS), stroke, traumatic brain injury (TBI), cerebral palsy (CP), and spinal
cord injury (SCI). Within those patient populations, spasticity occurs at a variable rate. Studies have shown that spasticity
affects between 37 percent and 78 percent of people with MS, 40 percent of those with SCI, approximately 35 percent of
those with stroke, more than 90 percent with CP, and approximately 50 percent of patients with TBI.
Medicines in Development
Neurological Disorders 2013
9
Selected Facts about Neurological Disorders
Spinal Cord Injury16
•The estimated annual incidence of spinal cord injury (SCI), not including those who die at an accident scene, is approximately 12,000 new cases each year. The estimated number of people living today with SCI ranges from 238,000 to 332,000.
•The costs for those living with SCI vary greatly according to injury severity. For example, the lifetime direct medical costs are
more than $4.6 million for someone with high quadriplegia injured at age 25, compared with the more than $1.5 million it
will cost someone injured at the same age who has incomplete motor functions.
Stroke17
•Stroke is the nation’s fourth leading killer and a leading cause of long-term disability. Each year, about 795,000 people suffer a stroke. On average, someone in the United States has a stroke every 40 seconds, and every 4 minutes someone dies
from one.
•Deaths from ischemic stroke, the most common type, are predicted to nearly double between 2000 and 2032. Conservative estimates forecast that ischemic stroke alone will cost the United States $2.2 trillion from 2005 to 2050.
•The direct and indirect costs of stroke in the United States for 2009 were $38.6 billion, with an average per person expenditure of $6,018.
Sources:
1. Alzheimer’s Association, www.alz.org
2. ALS Association, www.alsa.org
3. Brain Injury Association of America, www.biausa.org
4. American Brain Tumor Association, www.abta.org
5. Epilepsy Foundation, www.epilepsyfoundation.org
6.NetWellness from Case Western Reserve University, Ohio State University and University of Cincinnati, www.netwellness.org
7. International Association for the Study of Pain, www.iasp-pain.org
8. National Headache Foundation, www.headaches.org
9. National Institute of Neurological Disorders and Stroke, www.ninds.nih.gov
10. E-Medicine, www.emedicine.medscape.com
11. Multiple Sclerosis Foundation, www.msfocus.org
12. The American Academy of Pain Medicine, www.painmed.org
13. National Parkinson Foundation, www.parkinson.org
14. Parkinson Disease Foundation, www.pdf.org
15. Medscape, www.medscape.org
16. National Spinal Cord Injury Statistical Center, www.nscisc.uab.edu
17. American Heart Association, www.heart.org/advocacy
10
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Alzheimer’s Disease
Product Name
Sponsor
Indication
Development Phase*
AAB-003/PF-05236812
(beta-amyloid protein inhibitor
mAB)
Janssen Alzheimer Immunotherapy
South San Francisco, CA
Pfizer
New York, NY
Alzheimer’s disease
Phase I
www.janssenrnd.com
www.pfizer.com
ABT-126
(alpha-7-NNR antagonist)
AbbVie
North Chicago, IL
Alzheimer’s disease
Phase II
www.abbvie.com
ABT-288
(neurotransmitter receptor
modulator)
AbbVie
North Chicago, IL
Alzheimer’s disease
Phase II completed
www.abbvie.com
ABT-384
AbbVie
North Chicago, IL
Alzheimer’s disease
(see also other)
Phase II completed
www.abbvie.com
ABT-957
(calpain inhibitor)
AbbVie
North Chicago, IL
Alzheimer’s disease
Phase I
www.abbvie.com
AC-1204
(glucose stimulant)
Accera
Broomfield, CO
mild to moderate Alzheimer’s disease
Phase II/III
www.accerapharma.com
AD02 vaccine
(amyloid-beta protein inhibitor)
Affiris
Vienna, Austria
GlaxoSmithKline
Rsch. Triangle Park, NC
Alzheimer’s disease
Phase II
www.affiris.com
www.gsk.com
AD03 vaccine
(amyloid-beta protein inhibitor)
Affiris
Vienna, Austria
GlaxoSmithKline
Rsch. Triangle Park, NC
Alzheimer's disease
Phase I
www.affiris.com
www.gsk.com
APH-0703
(protein kinase C stimulant)
Aphios Corporation
Woburn, MA
Alzheimer’s disease
Phase I/II
www.aphios.com
ARC029
(nilvadipine)
Archer Pharmaceuticals
Sarasota, FL
Alzheimer’s disease
Phase I/II
www.archerpharma.com
ARC031
(soluble amyloid reducing/clearing
agent)
Archer Pharmaceuticals
Sarasota, FL
Alzheimer’s disease
Phase I
www.archerpharma.com
ASP0777
Astellas Pharma US
Northbrook, IL
Alzheimer’s disease
Phase I
www.astellas.com
*For more information about a specific medicine or company in the report, please use the website provided.
Medicines in Development
Neurological Disorders 2013
11
Medicines in Development for Neurological Disorders
Alzheimer’s Disease
Product Name
Sponsor
Indication
Development Phase
AVN 101
(serotonin 5-HT6 receptor
antagonist)
Avineuro Pharmaceuticals
San Diego, CA
Alzheimer’s disease
Phase II
www.avineuro.com
AVN 322
(serotonin 6 receptor antagonist)
Avineuro Pharmaceuticals
San Diego, CA
Alzheimer’s disease
Phase I
www.avineuro.com
AVN 397
Avineuro Pharmaceuticals
San Diego, CA
Alzheimer’s disease
Phase II
www.avineuro.com
AVP-923
(dextromethorphan/quinidine
fixed-dose combination)
Avanir Pharmaceuticals
Aliso Viejo, CA
agitation in Alzheimer’s disease
(see also pain)
Phase II
www.avanir.com
AZD1446
(alpha4/beta2 neuronal nicotinic
receptor agonist)
AstraZeneca
Wilmington, DE
Targacept
Winston-Salem, NC
Alzheimer’s disease
Phase I
www.astrazeneca.com
www.targacept.com
AZD2184
(PET enhancer)
Navidea Pharmaceuticals
Dublin, OH
Alzheimer’s disease (diagnosis)
Phase I
www.navidea.com
AZD2995
(PET enhancer)
Navidea Pharmaceuticals
Dublin, OH
Alzheimer’s disease (diagnosis)
Phase I
www.navidea.com
AZD3293
(beta secretase)
Astex Pharmaceuticals
Dublin, CA
AstraZeneca
Wilmington, DE
Alzheimer’s disease
Phase I
www.astx.com
www.astrazeneca.com
AZD5213
(histamine-3 receptor antagonist)
AstraZeneca
Wilmington, DE
Alzheimer’s disease
Phase II
www.astrazeneca.com
BACE inhibitor
Janssen Pharmaceuticals
Titusville, NJ
Shionogi
Florham Park, NJ
Alzheimer’s disease
Phase I
www.janssenpharmaceuticalsinc.com
www.shionogi.com
BAN2401
(amyloid beta-protein inhibitor)
Eisai
Woodcliff Lake, NJ
early stage Alzheimer’s disease
Phase II
www.eisai.com
BIIB037
(human anti-amyloid beta mAb)
Biogen Idec
Weston, MA
Alzheimer’s disease
Phase I
www.biogenidec.com
bisnorcymserine
(BNC)
QR Pharma
Berwyn, PA
Alzheimer’s disease
Phase I
www.qrpharma.com
BMS-241027
(microtubule stabilizer)
Bristol-Myers Squibb
Princeton, NJ
Alzheimer’s disease
Phase I
www.bms.com
12
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Alzheimer’s Disease
Product Name
Sponsor
Indication
Development Phase
CAD106
(amyloid beta-protein inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
Alzheimer’s disease
Phase II
www.novartis.com
CERE-110
(AAV-NGF)
Ceregene
San Diego, CA
Alzheimer’s disease
Phase II
www.ceregene.com
crenezumab
(anti-amyloid-beta mAb)
Genentech
South San Francisco, CA
Alzheimer’s disease
Phase II
www.gene.com
davunetide intranasal
Allon Therapeutics
Vancouver, Canada
Alzheimer’s disease
(see also Parkinson’s, other)
Phase II
www.allontherapeutics.com
donepezil/memantine
extended release
(fixed-dose combination)
Adamas Pharmaceuticals
Emeryville, CA
Forest Laboratories
New York, NY
moderate to severe Alzheimer’s
disease
Phase II
www.adamaspharma.com
www.frx.com
DSP-8658
(PPAR alpha/gamma agonist)
Sunovion Pharmaceuticals
Marlborough, MA
Alzheimer’s disease
Phase I
www.sunovion.com
E2212
(amyloid precursor protein
secretase modulator)
Eisai
Woodcliff Lake, NJ
Alzheimer’s disease
Phase I completed
www.eisai.com
E2609
(BACE1 protein inhibitor)
Eisai
Woodcliff Lake, NJ
Alzheimer’s disease
Phase I
www.eisai.com
ELND005
Speranza Therapeutics
Dublin, Ireland
neuropsychiatric symptoms in
Alzheimer’s disease
(Fast Track)
Phase II
EVP-0962
(gamma secretase modulator)
EnVivo Pharmaceuticals
Watertown, MA
Alzheimer’s disease
Phase II
www.envivopharma.com
EVP-6124
(alpha7 nicotinic acetylcholine
receptor agonist)
EnVivo Pharmaceuticals
Watertown, MA
Alzheimer’s disease
Phase II
www.envivopharma.com
Exebryl-1®
amyloid-beta-protein/tau protein
inhibitor
ProteoTech
Kirkland, WA
Alzheimer’s disease
Phase I
www.proteotech.com
F-18 T808
(PET imaging)
Siemens Medical Solutions
Malvern, PA
Alzheimer’s disease (diagnosis)
Phase 0
www.usa.healthcare.siemens.com
F18-florbetaben
(molecular imaging agent)
IBA Molecular
Dulles, VA
Piramal Healthcare
Mumbai, India
Alzheimer’s disease (diagnosis)
application submitted
www.iba-molecular.com
www.piramal.com
Medicines in Development
Neurological Disorders 2013
13
Medicines in Development for Neurological Disorders
Alzheimer’s Disease
Product Name
Sponsor
Indication
Development Phase
F18-flutemetamol
(PET imaging agent)
GE Healthcare
Waukesha, WI
Alzheimer’s disease (diagnosis)
application submitted
www.gehealthcare.com
gantenerumab
(RG1450)
Roche
Nutley, NJ
early stage Alzheimer’s disease
Phase II/III
www.roche.com
GM6
(peptide therapeutic)
Genervon Biopharmaceuticals
Pasadena, CA
Alzheimer’s disease
(see also amyotrophic lateral sclerosis,
multiple sclerosis, Parkinson’s, spinal
cord injury, stroke)
Phase I
www.genervon.com
GSK2647544
(Lp-PLA2 inhibitor)
GlaxoSmithKline
Rsch. Triangle Park, NC
Alzheimer’s disease
Phase I
www.gsk.com
GSK2981710
(medium chain triglycerides)
GlaxoSmithKline
Rsch. Triangle Park, NC
Alzheimer’s disease
Phase I
www.gsk.com
HPP-854
(BACE1 inhibitor)
High Point Pharmaceuticals
High Point, NC
Alzheimer’s disease
Phase I
www.highpointpharma.com
JNJ-54861911
Janssen Research & Development
Raritan, NJ
Alzheimer’s disease
Phase I
www.janssenrnd.com
KU-046
(amyloid beta-protein modulator)
Kareus Therapeutics
La Chaux-de-Fonds, Switzerland
Alzheimer’s disease
Phase I
www.kareustherapeutics.com
LMTX
(TRx-0238)
TauRx Pharmaceuticals
Singapore
Alzheimer’s disease,
frontotemporal dementia
Phase III
www.taurx.com
Lu AE58054
(5-HT6 receptor antagonist)
Lundbeck
Deerfield, IL
Otsuka America Pharmaceutical
Rockville, MD
Alzheimer’s disease (cognition)
Phase II
www.lundbeck.com
www.otsuka.com
LY2886721
(beta secretase inhibitor)
Eli Lilly
Indianapolis, IN
Alzheimer’s disease
(slow disease progression)
Phase I
www.lilly.com
LY3002813
(biological)
Eli Lilly
Indianapolis, IN
Alzheimer’s disease
Phase I
www.lilly.com
Lym Pro®
neurotrophic factor companion
diagnostic
Amarantus BioSciences
Sunnyvale, CA
Alzheimer’s disease (diagnosis)
Phase II
www.amarantus.com
MK-8931
(BACE1 protein inhibitor)
Merck
Whitehouse Station, NJ
Alzheimer’s disease
Phase II/III
www.merck.com
14
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Alzheimer’s Disease
Product Name
Sponsor
Indication
Development Phase
MSDC-0160
(mTOT modulator insulin sensitizer)
Metabolic Solutions Development
Company
Kalamazoo, MI
Alzheimer’s disease
Phase II
www.msdrx.com
NAV4694
(fluorine-18 labeled precision
radiopharmaceutical)
Navidea Biopharmaceuticals
Dublin, OH
Alzheimer’s disease (diagnosis)
Phase II
www.navidea.com
NAV5001
(123I-labelled imaging agent)
Navidea Biopharmaceuticals
Dublin, OH
dementia with Lewy bodies
(diagnosis)
(see also Parkinson’s)
Phase II
www.navidea.com
NIC5-15
(amyloid precursor protein
secretase inhibitor)
Humanetics
Minneapolis, MN
Alzheimer’s disease
Phase II
www.humaneticscorp.com
PF-05212377
(SAM-760)
Pfizer
New York, NY
Alzheimer’s disease
Phase II
www.pfizer.com
pioglitazone companion diagnostic
Takeda Pharmaceuticals U.S.A.
Deerfield, IL
Zinfandel Pharmaceuticals
Chapel Hill, NC
Alzheimer’s disease (diagnosis)
Phase I
www.takeda.com
Posiphen®
R-phenserine
QR Pharma
Berwyn, PA
Alzheimer’s disease,
mild cognitive impairment
(see also Parkinson’s)
Phase II
www.qrpharma.com
RG1577
(MAO-B inhibitor)
Roche
Nutley, NJ
Alzheimer’s disease
Phase II
www.roche.com
RG7129
(BACE1 protein inhibitor)
Roche
Nutley, NJ
Alzheimer’s disease
Phase I
www.roche.com
rilapladib
(Lp-PLA2 inhibitor)
GlaxoSmithKline
Rsch. Triangle Park, NC
Alzheimer’s disease
Phase II
www.gsk.com
RVX-208
(BET protein inhibitor)
Resverlogix
Calgary, Canada
Alzheimer’s disease
Phase I
www.resverlogix.com
SAR110894
(H3 antagonist)
Sanofi US
Bridgewater, NJ
Alzheimer’s disease
Phase II
www.sanofi.com
SAR228810
(anti-protofibrillar AB mAb)
Sanofi US
Bridgewater, NJ
Alzheimer’s disease
Phase I
www.sanofi.com
SB742457
(5HT6 antagonist)
GlaxoSmithKline
Rsch. Triangle Park, NC
Alzheimer’s disease
Phase II
www.gsk.com
Medicines in Development
Neurological Disorders 2013
15
Medicines in Development for Neurological Disorders
Alzheimer’s Disease
Product Name
Sponsor
Indication
Development Phase
sGC 1061
(nomethiazole)
sGC Pharma
Wellesley, MA
Alzheimer’s disease
Phase I
www.sgcpharma.com
solanezumab
(amyloid-beta protein inhibitor)
Eli Lilly
Indianapolis, IN
mild Alzheimer’s disease
Phase III
www.lilly.com
ST101
Sonexa Therapeutics
San Diego, CA
Alzheimer’s disease
Phase II
www.sonexa.com
T3D-959
(dual PPAR agonist)
T3D Therapeutics
Rsch. Triangle Park, NC
Alzheimer’s disease
Phase I completed
www.t3dtherapeutics.com
T-817MA
Toyama Chemical
Tokyo, Japan
Alzheimer’s disease
Phase II
www.toyama-chemical.co.jp
TC-1734
(ispronicline)
Targacept
Winston-Salem, NC
Alzheimer’s disease
Phase II
www.targacept.com
TC-5619
(alpha7nAChR)
Targacept
Winston-Salem, NC
Alzheimer’s disease
Phase I
www.targacept.com
TD-8954
(5-HT4 agonist)
Targacept
Winston-Salem, NC
Alzheimer’s disease
Phase I
www.targacept.com
TTP488
Transtech Pharma
High Point, NC
Alzheimer’s disease
(Fast Track)
Phase II
www.ttpharma.com
TTP4000
Transtech Pharma
High Point, NC
Alzheimer’s disease
Phase I
www.ttpharma.com
V950 vaccine
Merck
Whitehouse Station, NJ
Alzheimer’s disease
Phase I
www.merck.com
vanutide cridificar
(ACC-001/PF-05236806)
Janssen Alzheimer Immunotherapy
South San Francisco, CA
Pfizer
New York, NY
Alzheimer’s disease
Phase II
www.janimm.com
www.pfizer.com
VI-1121
VIVUS
Mountain View, CA
Alzheimer’s disease
Phase II
www.vivus.com
XEL 001HP
(transdermal patch)
Xel Pharmaceuticals
Draper, UT
Alzheimer’s disease
Phase I
www.xelpharmaceuticals.com
16
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Amyotrophic Lateral Sclerosis
Product Name
Sponsor
Indication
Development Phase
arimoclomol
(Orphan Drug)
ALS Association
Washington, DC
Orphazyme
Copenhagen, Denmark
University of Miami
Miami, FL
amyotrophic lateral sclerosis (ALS)
(Fast Track)
Phase II/III
www.orphazyme.com
GM6
(peptide therapeutic)
Genervon Biopharmaceuticals
Pasadena, CA
ALS (Fast Track)
(see also Alzheimer’s, multiple
sclerosis, Parkinson’s, spinal cord
injury, stroke)
Phase II
www.genervon.com
NP001
(macrophage regulator)
Neuraltus Pharmaceuticals
Palo Alto, CA
ALS
Phase II
www.neuraltus.com
NSI-566
(stem cell therapy)
(Orphan Drug)
Neuralstem
Rockville, MD
ALS
Phase II
www.neuralstem.com
NurOwn™
GDNF-producing adult
stem cell therapy
(Orphan Drug)
BrainStorm Cell Therapeutics
New York, NY
ALS
Phase II
www.brainstorm-cell.com
ozanezumab
(NOGO-A mAb)
GlaxoSmithKline
Rsch. Triangle Park, NC
ALS
Phase II
www.gsk.com
stem cell therapy
TCA Cellular Therapy
Covington, LA
ALS
(see also spinal cord injury)
Phase I
www.tcacellulartherapy.com
tirasemtiv
(Orphan Drug)
Cytokinetics
South San Francisco, CA
ALS
(Fast Track)
Phase II
www.cytokinetics.com
Attention-Deficit/Hyperactivity Disorder
Product Name
Sponsor
Indication
Development Phase
AR08
(adrenergic receptor agonist)
Arbor Pharmaceuticals
Atlanta, GA
attention-deficit/hyperactivity
disorder (ADHD)
Phase II
www.arborpharma.com
COL-171
(sustained release)
Collegium Pharmaceuticals
Cumberland, RI
ADHD
in clinical trials
www.collegiumpharma.com
Medicines in Development
Neurological Disorders 2013
17
Medicines in Development for Neurological Disorders
Attention-Deficit/Hyperactivity Disorder
Product Name
Sponsor
Indication
Development Phase
d-amphetamine transdermal
(ATS)
Noven Pharmaceuticals
Miami, FL
ADHD
Phase II
www.noven.com
EB-1020
(triple reuptake inhibitor)
Neurovance (Euthymics)
Cambridge, MA
ADHD
Phase I
www.euthymics.com
edivoxetine
(norepinephrine reuptake
inhibitor)
Eli Lilly
Indianapolis, IN
ADHD (pediatric)
Phase II/III
www.lilly.com
eltoprazine
(serotonin 1A/1B partial agonist)
PsychoGenics
Tarrytown, NY
ADHD (adults)
Phase II
www.psychogenics.com
HDL-100
(amphetamine modified release)
Highland Therapeutics
Toronto, Canada
ADHD
Phase I
www.highlandtherapeutics.com
HDL-200
(methylphenidate
modified-release)
Highland Therapeutics
Toronto, Canada
ADHD
Phase I
www.highlandtherapeutics.com
KP106
(d-amphetamine prodrug)
KemPharm
North Liberty, IA
ADHD
Phase I completed
www.kempharm.com
KRL-401
Krele Pharmaceuticals
(TONIX Pharmaceuticals)
New York, NY
ADHD
in clinical trials
www.krele.com
methylphenidate
extended release
Purdue Pharma
Stamford, CT
Rhodes Pharma
Coventry, RI
ADHD
Phase III
www.purduepharma.com
www.rhodespharma.com
NT0202
(amphetamine controlled release)
Neos Therapeutics
Grand Prairie, TX
ADHD
application submitted
www.neostx.com
NWP09
(methylphenidate
extended-release chewable
tablets)
Pfizer
New York, NY
ADHD
Phase III completed
www.pfizer.com
OPC-34712
(brexpiprazole)
Lundbeck
Deerfield, IL
Otsuka America Pharmaceutical
Rockville, MD
ADHD (adults)
Phase II
www.lundbeck.com
www.otsuka.com
18
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Attention-Deficit/Hyperactivity Disorder
Product Name
Sponsor
Indication
Development Phase
ORADUR®-ADHD
(sustained-release oral gel)
DURECT
Cupertino, CA
ADHD
Phase I
www.durect.com
SEP-225289
(triple reuptake inhibitor)
Sunovion Pharmaceuticals
Marlborough, MA
ADHD
Phase II
www.sunovion.com
SPN-810
(molindone)
Supernus Pharmaceuticals
Rockville, MD
impulsive aggression in ADHD
(pediatric)
Phase II completed
www.supernus.com
SPN-812
(norepinephrine uptake inhibitor)
Supernus Pharmaceuticals
Rockville, MD
ADHD
Phase II completed
www.supernus.com
TD-9855
(monoamine reuptake inhibitor)
Theravance
South San Francisco, CA
ADHD
Phase II
www.theravance.com
Product Name
Sponsor
Indication
Development Phase
BHR-100
(intravenous progesterone
infusion)
(Orphan Drug)
BHR Pharma
Herndon, VA
traumatic brain injury
(Fast Track)
Phase III
www.bhr-pharma.com
ciclosporin intravenous
(Orphan Drug)
Maas Biolab
Albuquerque, NM
NeuroVive Pharmaceutical
Lund, Sweden
traumatic brain injury
Phase I
www.maasbiolab.com
www.neurovive.com
methamphetamine intravenous
Sinapis Pharma
Jacksonville, FL
traumatic brain injury
(see also stroke)
Phase I completed
www.sinapispharma.com
NNZ-2566
(cytokine inhibitors/neuropeptide
receptor modulator)
Neuren Pharmaceuticals
Bethesda, MD
traumatic brain injury
(Fast Track)
(see also other)
Phase II
www.neurenpharma.com
NTx-428®
cell differentiation, nerve growth
factor and stem cell stimulant
Stem Cell Therapeutics
Toronto, Canada
traumatic brain injury
Phase II
www.stemcellthera.com
Oxycyte®
perfluorocarbon oxygen carrier
Oxygen Biotherapeutics
Morrisville, NC
traumatic brain injury
Phase II
www.oxybiomed.com
Brain Injury
Medicines in Development
Neurological Disorders 2013
19
Medicines in Development for Neurological Disorders
Brain Injury
Product Name
Sponsor
Indication
Development Phase
RP-1127
(glibenclamide)
Remedy Pharmaceuticals
New York, NY
moderate to severe traumatic brain
injury
(see also stroke)
Phase II
www.remedypharmaceuticals.com
SAR127963
(P75 receptor antagonist)
Sanofi US
Bridgewater, NJ
traumatic brain injury
Phase I
www.sanofi.com
Product Name
Sponsor
Indication
Development Phase
8H9 mAb
United Therapeutics
Silver Spring, MD
metastatic brain cancer
Phase I
www.unither.com
ABT-414
(EGFR antagonist)
AbbVie
North Chicago, IL
glioblastoma
Phase I
www.abbvie.com
AEE788
(multiple tyrosine kinase inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
glioblastoma
Phase I/II
www.novartis.com
afatinib
(ErbB tyrosine kinase inhibitor)
Boehringer Ingelheim Pharmaceuticals
Ridgefield, CT
glioblastoma
Phase I/II
www.boehringer-ingelheim.com
AMG 595
(anti-EGFRvIII antibody-drug
conjugate)
Amgen
Thousand Oaks, CA
anaplastic astrocytoma,
glioblastoma
Phase I
www.amgen.com
ANG1005
(paclitaxel prodrug)
AngioChem
Montreal, Canada
glioma
Phase I
www.angiochem.com
ARC 100
(tubulin polymerisation inhibitor)
Archer Biosciences
New York, NY
recurrent glioblastoma
Phase II
www.archerbiosciences.com
----------------------------------------Phase I/II
www.archerbiosciences.com
Brain Tumors
-------------------------------------medulloblastoma,
recurrent neuroblastoma
AT-101
(Bcl-2 inhibitor)
Ascenta Therapeutics
Malvern, PA
glioblastoma
Phase II
www.ascenta.com
Avastin®
bevacizumab
Genentech
South San Francisco, CA
first-line glioblastoma
Phase III
www.gene.com
Azedra™
iobenguane I-131
(Orphan Drug)
Progenics Pharmaceuticals
Tarrytown, NY
neuroblastoma (pediatric)
(Fast Track)
Phase II
www.progenics.com
20
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Brain Tumors
Product Name
Sponsor
Indication
Development Phase
BKM120
(PI3K inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
glioblastoma
Phase I
www.novartis.com
ch14.18 mAb
(Orphan Drug)
United Therapeutics
Silver Spring, MD
neuroblastoma
Phase III
www.unither.com
cilengitide
(Orphan Drug)
EMD Serono
Rockland, MA
glioblastoma
Phase III
www.emdserono.com
cilengitide companion diagnostic
EMD Serono
Rockland, MA
MDxHealth
Irvine, CA
glioblastoma (diagnosis)
Phase III
www.emdserono.com
www.mdxhealth.com
cintredekin besudotox
(IL13-PE38QQR)
(Orphan Drug)
INSYS Therapeutics
Chandler, AZ
National Institute of Neurological
Disorders and Stroke
Bethesda, MD
glioma
(Fast Track)
Phase I
www.insysrx.com
CLR 1404 I-124
(PET imaging)
Novelos Therapeutics
Madison, WI
brain tumor (diagnosis)
Phase I/II
www.novelos.com
Cotara®
(iodine-131 radiolabeled TNT mAb)
(Orphan Drug)
Peregrine Pharmaceuticals
Tustin, CA
recurrent glioblastoma
(Fast Track)
Phase II
www.peregrineinc.com
crenolanib
(receptor tyrosine kinase inhibitor)
(Orphan Drug)
AROG Pharmaceuticals
Dallas, TX
glioma (pediatric)
Phase I
www.arogpharma.com
dacomitinib
(PF-00299804)
Pfizer
New York, NY
glioblastoma
Phase II
www.pfizer.com
DCVax®-Brain
dendritic cell vaccine
(Orphan Drug)
Northwest Biotherapeutics
Bethesda, MD
glioblastoma
Phase III
www.nwbio.com
DNX-2401
(cell death stimulant)
DNAtrix
Houston, TX
glioblastoma
Phase I
www.dnatrix.com
E7080
(lenvatinib)
Eisai
Woodcliff Lake, NJ
glioma
Phase II
www.eisai.com
Medicines in Development
Neurological Disorders 2013
21
Medicines in Development for Neurological Disorders
Brain Tumors
Product Name
Sponsor
Indication
Development Phase
eflornithine
(CPP-1X)
Cancer Prevention Pharmaceuticals
Tucson, AZ
neuroblastoma
Phase II
www.canprevent.com
ETS2101
(synthetic terpene-based
cannabinoid)
e-Therapeutics
Oxfordshire, United Kingdom
University of California
San Diego, CA
glioma
Phase I
www.etherapeutics.co.uk
F18-ML-10
(molecular imaging agent)
IBA Molecular
Dulles, VA
Aposense
Petach-Tikva, Israel
brain tumors (diagnosis)
Phase II
www.iba-molecular.com
www.aposense.com
firtecan pegol
(Orphan Drug)
Belrose Pharma
Princeton, NJ
neuroblastoma
Phase I/II
www.belrosepharma.com
galunisertib
(LY2157299)
Eli Lilly
Indianapolis, IN
glioblastoma
Phase II
www.lilly.com
----------------------------------------Phase I/II
www.lilly.com
-------------------------------------newly diagnosed glioma
GDC-0084/RG7666
(PI3K inhibitor)
Genentech
South San Francisco, CA
glioma
Phase I
www.gene.com
GliAtak™
cancer gene therapy
(Orphan Drug)
Advantagene
Auburndale, MA
malignant glioma
Phase II
www.advantagene.com
Glionix™
belagenpumatucel-L
subcutaneous
NovaRx
San Diego, CA
glioma
Phase I
www.novarx.com
Hiltonol®
poly-ICLC
(Orphan Drug)
Oncovir
Washington, DC
glioblastoma
Phase II
www.oncovir.com
ICT-107
(autologous dendritic cell-based
vaccine)
(Orphan Drug)
ImmunoCellular Therapeutics
Woodland Hills, CA
glioblastoma
Phase II
www.imuc.com
IMA950
(tumor-associated peptide vaccine)
Immatics Biotechnologies
Tuebingen, Germany
glioblastoma
Phase I
www.immatics.com
LDE225
(SMO protein inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
medulloblastoma
Phase III
www.novartis.com
22
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Brain Tumors
Product Name
Sponsor
Indication
Development Phase
LEE011
(cyclin-dependent kinase-4/6
inhibitor)
Novartis Pharmaceuticals
East Hanover, NJ
neuroblastoma
Phase I
www.novartis.com
macitentan
Actelion Pharmaceuticals
South San Francisco, CA
recurrent glioblastoma
Phase I
www.actelion.us
mibefradil
Tau Therapeutics
Charlottesville, VA
recurrent glioma
Phase I
www.tautherapeutics.com
NEO100
(perillyl alcohol intranasal)
(Orphan Drug)
NEONC Technologies
Woodland Hills, CA
glioblastoma
in clinical trials
www.neonctech.com
neuroblastoma vaccine
MabVax
San Diego, CA
neuroblastoma
Phase I
www.mabvax.com
onartuzumab
(anti-c-Met mAb)
Genentech
South San Francisco, CA
recurrent glioblastoma
Phase II
www.gene.com
Opaxio®
paclitaxel poliglumex
(Orphan Drug)
Cell Therapeutics
Seattle, WA
glioblastoma
Phase II
www.celltherapeutics.com
palbociclib
(PD-0332991/CDK4-6 kinase
inhibitor)
Pfizer
New York, NY
Rb-positive glioblastoma
Phase II
www.pfizer.com
perifosine
AEterna Zentaris
Basking Ridge, NJ
glioma
Phase II
www.aezsinc.com
PLX3397
Plexxikon
Berkeley, CA
glioblastoma
Phase II
www.plexxikon.com
PX-866
(PI-3 kinase inhibitor)
Oncothyreon
Seattle, WA
glioblastoma
Phase II
www.oncothyreon.com
Reolysin®
pelareorep
Oncolytics Biotech
Calgary, Canada
glioma
Phase I/II completed
www.oncolyticsbiotech.com
rindopepimut
(EGFR varient III vaccine)
(Orphan Drug)
Celldex Therapeutics
Needham, MA
first-line glioblastoma
(Fast Track)
-------------------------------------recurrent glioblastoma
(Fast Track)
Phase III
www.celldextherapeutics.com
----------------------------------------Phase II
www.celldextheraputics.com
SB-313
(T-cell receptor gene stimulant)
Sangamo Biosciences
Richmond, CA
City of Hope National Medical Center
Duarte, CA
glioblastoma
Phase I
www.sangamo.com
Medicines in Development
Neurological Disorders 2013
23
Medicines in Development for Neurological Disorders
Brain Tumors
Product Name
Sponsor
Indication
Development Phase
SL-701
(dendritic cell vaccine)
Stemline Therapeutics
New York, NY
glioma
Phase I/II
www.stemline.com
Tarceva®
erlotinib
Astellas Pharma US
Northbrook, IL
recurrent ependymoma (pediatric)
Phase III
www.astellas.com
terameprocol
(intravenous)
Erimos Pharmaceuticals
Raleigh, NC
glioma
Phase I/II
www.erimos.com
Toca 511/Toca FC
(fluorouracil prodrug gene therapy)
Tocagen
San Diego, CA
recurrent glioblastoma
Phase I/II
www.tocagen.com
----------------------------------------Phase I
www.tocagen.com
-------------------------------------late-stage glioma
TRC105
(ENG protein inhibitor)
Tracon Pharmaceuticals
San Diego, CA
glioblastoma
Phase II
www.traconpharma.com
TSC
(trans-sodium crocetinate)
Diffusion Pharmaceuticals
Charlottesville, VA
glioblastoma
Phase I/II
www.diffusionpharma.com
TVI-Brain-1
(cancer vaccine)
TVAX Biomedical
Lenexa, KS
glioma
Phase II
www.tvaxbiomedical.com
Tykerb®
lapatinib
GlaxoSmithKline
Rsch. Triangle Park, NC
Jonsson Comprehensive Cancer Center
Los Angeles, CA
glioblastoma
Phase II
www.gsk.com
VAL-083
(Orphan Drug)
Del Mar Pharmaceuticals
Vancouver, Canada
glioblastoma
Phase I/II
www.delmarpharma.com
VB-111
(gene therapy)
VBL Therapeutics
Or Yehuda, Israel
glioblastoma
Phase I/II
www.vblrx.com
veliparib
AbbVie
North Chicago, IL
brain metastases
Phase II
www.abbvie.com
vemurafenib
Roche
Nutley, NJ
brain metastases
Phase II
www.roche.com
vitespen
(G-100 prophage)
Agenus
Lexington, MA
glioma
Phase II
www.agenusbio.com
Xeloda®
capecitabine
Roche
Nutley, NJ
glioma (pediatric)
Phase II
www.roche.com
24
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Epilepsy
Product Name
Sponsor
Indication
Development Phase
Banzel®
rufinamide
(Orphan Drug)
Eisai
Woodcliff Lake, NJ
Lennox-Gastaut syndrome (pediatric)
Phase III
www.eisai.com
BGG492
(selurampanel)
Novartis Pharmaceuticals
East Hanover, NJ
partial seizures
(see also headache)
Phase II completed
www.novartis.com
brivaracetam
UCB
Smyrna, GA
epilepsy
Phase III
www.ucb.com
CPP -15
(GABA-aminotransferase inhibitor)
(Orphan Drug)
Catalyst Pharmaceutical Partners
Coral Gables, FL
complex partial seizures,
infantile spasms
Phase I
www.catalystpharma.com
diazepam intranasal spray
(Orphan Drug)
Acorda Therapeutics
Ardsley, NY
acute repetitive seizures
Phase III
www.acorda.com
DSP-0565
(sodium/calcium channel blocker)
Sunovion Pharmaceuticals
Marlborough, MA
epilepsy
Phase I
www.sunovion.com
ezogabine extended release
GlaxoSmithKline
Rsch. Triangle Park, NC
Valeant Pharmaceuticals North America
Bridgewater, NJ
partial onset seizures
Phase III
www.gsk.com
www.valeant.com
Fycompa®
perampanel
Eisai
Woodcliff Lake, NJ
generalized seizures
Phase III
www.eisai.com
----------------------------------------Phase II
www.eisai.com
-------------------------------------partial-onset seizures (pediatric)
ganaxolone
(Orphan Drug)
Marinus Pharmaceuticals
New Haven, CT
infantile spasms (infants),
partial-onset seizures (adults)
Phase II
www.marinuspharma.com
INS-001
(huperzine A)
Insero Health
Miami, FL
epilepsy
Phase I
www.insero.com
I.V. carbamazepine
Lundbeck
Deerfield, IL
epilepsy
Phase III
www.lundbeck.com
JZP8
(clonazepam intranasal)
Jazz Pharmaceuticals
Palo Alto, CA
recurrent acute repetitive seizures
Phase II
www.jazzpharma.com
Lyrica®
pregabalin
Pfizer
New York, NY
generalized tonic clonic seizures
(see also pain)
Phase III
www.pfizer.com
midazolam intranasal
(USL261)
Upsher-Smith Laboratories
Maple Grove, MN
acute repetitive seizures
(Fast Track)
Phase III
www.upsher-smith.com
Medicines in Development
Neurological Disorders 2013
25
Medicines in Development for Neurological Disorders
Epilepsy
Product Name
Sponsor
Indication
Development Phase
naluzotan
(serotonin 1A receptor agonist/
sigma-1 receptor antagonist)
Proximagen
London, United Kingdom
epilepsy
Phase II
www.proximagen.com
NRL-1
(diazepam intranasal)
Biotie Therapies
Turku, Finland
Neuralis
San Diego, CA
epilepsy
Phase I
www.biotie.com
PF-04895162
Pfizer
New York, NY
epilepsy
Phase I completed
www.pfizer.com
Potiga™
ezogabine
GlaxoSmithKline
Rsch. Triangle Park, NC
Valeant Pharmaceuticals North America
Bridgewater, NJ
Lennox-Gastaut syndrome,
partial onset seizures
(patients age 12 and older)
Phase III
www.gsk.com
www.valeant.com
pregabalin controlled release
Pfizer
New York, NY
epilepsy (adjunctive treatment)
(see also pain)
Phase III
www.pfizer.com
Stedesa®
eslicarbazepine acetate
Sunovion Pharmaceuticals
Marlborough, MA
partial seizures in adults
(adjunctive therapy)
-------------------------------------partial seizures (monotherapy)
application submitted
www.sunovion.com
----------------------------------------Phase III
www.sunovion.com
tonabersat
(USL260)
Upsher-Smith Laboratories
Maple Grove, MN
epilepsy
Phase I
www.upsher-smith.com
topiramate extended release
(USL255)
Upsher-Smith Laboratories
Maple Grove, MN
partial-onset seizures
application submitted
www.upsher-smith.com
topiramate intravenous
(Captisol®-enabled)
CyDex Pharmaceuticals
(Ligand Pharmaceuticals)
Lenexa, KS
epilepsy
Phase I
www.ligand.com
Trokendi XR™
topiramate controlled release
Supernus Pharmaceuticals
Rockville, MD
epilepsy
application submitted
www.supernus.com
Vanquix® Auto-Injector
diazepam injection
Pfizer
New York, NY
acute repetitive seizures
(intermittent therapy)
Phase III
www.pfizer.com
Vimpat®
lacosamide
UCB
Smyrna, GA
epilepsy (monotherapy)
Phase III
www.ucb.com
----------------------------------------Phase II
www.ucb.com
-------------------------------------epilepsy in patients 2-17 years of age
(adjunctive therapy), primary generalized tonic clonic seizures (adjunctive
therapy)
26
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Epilepsy
Product Name
Sponsor
Indication
Development Phase
VX-765
(caspase 1 inhibitor)
Vertex Pharmaceuticals
Cambridge, MA
treatment-resistant partial epilepsy
Phase II
www.vrtx.com
YKP-3089
SK biopharmaceuticals
Fair Lawn, NJ
epilepsy
(see also pain)
Phase II
www.skbp.com
Product Name
Sponsor
Indication
Development Phase
Afinitor®
everlimus
Novartis Pharmaceuticals
East Hanover, NJ
seizures associated with tuberous
sclerosis complex (TSC)
Phase III
www.novartis.com
deferiprone
ApoPharma
Toronto, Canada
pantothenate kinase-associated
neurodegeneration
Phase III
www.apotex.com
ecopipam
(D1 dopamine receptor antagonist)
(Orphan Drug)
Psyadon Pharmaceuticals
Germantown, MD
Lesch-Nyhan syndrome
(see also other)
Phase III
www.psyadonrx.com
Gammagard Liquid™
immune globulin
Baxter Healthcare
Deerfield, IL
Friedreich’s ataxia
Phase II
www.baxter.com
HuCNS-SC®
adult neural stem cell therapy
StemCells
Newark, CA
Pelizaeus-Merzbacher disease
(see also spinal cord injury)
Phase I
www.stemcellsinc.com
ISIS-SMNRx
(antisense oligonucleotide)
(Orphan Drug)
Biogen Idec
Weston, MA
Isis Pharmaceuticals
Carlsbad, CA
spinal muscular atrophy
(Fast Track)
Phase II
www.biogenidec.com
www.isispharm.com
ISIS-TTRRx
(antisense RNA modulator)
(Orphan Drug)
Isis Pharmaceuticals
Carlsbad, CA
familial amyloid polyneuropathy
(Fast Track)
Phase II/III
www.isispharm.com
Lenti-D™
adrenoleukodystrophy
gene therapy
(Orphan Drug)
Bluebird Bio
Cambridge, MA
childhood cerebral
adrenoleukodystrophy
Phase I/II
www.bluebirdbio.com
PF-06687859
(DcpS protein inhibitor)
(Orphan Drug)
Pfizer
New York, NY
spinal muscular atrophy
Phase I
www.pfizer.com
RG2833
(HDAC inhibitor)
(Orphan Drug)
Repligen
Waltham, MA
Friedreich’s ataxia
Phase I completed
www.repligen.com
Genetic Disorders
Medicines in Development
Neurological Disorders 2013
27
Medicines in Development for Neurological Disorders
Genetic Disorders
Product Name
Sponsor
Indication
Development Phase
tafamidis meglumine
(transthyretin dissociation
inhibitor)
(Orphan Drug)
Pfizer
New York, NY
transthyretin familial amyloid polyneuropathy
(Fast Track)
application submitted
www.pfizer.com
vatiquinone
(EPI-743)
(Orphan Drug)
Edison Pharmaceuticals
Mountain View, CA
mitochondrial myopathies
Phase II/III
www.edisonpharma.com
----------------------------------------Phase II
www.edisonpharma.com
VP20629
ViroPharma
Exton, PA
Friedreich’s ataxia
Phase I
www.viropharma.com
UX001 sialic acid
(sialic acid extended release)
Ultragenyx Pharmaceuticals
Novato, CA
hereditary inclusion body myositis
Phase II
www.ultragenyx.com
Product Name
Sponsor
Indication
Development Phase
ALD403
(CGRP inhibitor mAb)
Alder Biopharmaceuticals
Bothell, WA
prevention of migraine
(subcutaneous)
-------------------------------------prevention of migraine
(intravenous)
Phase II
www.alderbio.com
----------------------------------------Phase I
www.alderbio.com
AMG 334
(CGRP receptor antagonist mAb)
Amgen
Thousand Oaks, CA
migraine
Phase I
www.amgen.com
Amrix®
cyclobenzaprine extended release
Cephalon (Teva)
Frazer, PA
migraine
Phase III
www.tevapharm.com
AP-1531
(EPH receptor antagonist)
Ariel Pharmaceuticals
Broomfield, CO
migraine
Phase I
AVP-825
(sumatriptan intranasal)
Avanir Pharmaceuticals
Aliso Viejo, CA
migraine
Phase III
www.avanir.com
AZ-104
(Staccato® loxapine low dose)
Alexza Pharmaceuticals
Mountain View, CA
migraine
Phase II completed
www.alexza.com
-------------------------------------Friedreich’s ataxia,
Leigh’s disease
Headache
28
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Headache
Product Name
Sponsor
Indication
Development Phase
BGG492
(selurampanel)
Novartis Pharmaceuticals
East Hanover, NJ
migraine
(see also epilepsy)
Phase II
www.novartis.com
Botox®
onabotulinumtoxinA
Allergan
Irvine, CA
prevention of migraine
(adolescents)
(see also pain, other)
Phase III
www.allergan.com
CL-H1T
(sumatriptan)
Charleston Laboratories
Charleston, SC
migraine
Phase II
www.charlestonlabs.com
doxepin intranasal
Winston Pharmaceuticals
Vernon Hills, IL
chronic daily headache
Phase II
www.winstonlabs.com
DR-105
(ethinylestradiol/levonorgestrel)
Duramed Pharmaceuticals (Teva)
North Wales, PA
menstrual migraine
Phase II completed
www.tevapharm.com
lasmiditan
(serotonin 1F receptor agonist)
CoLucid Pharmaceuticals
Durham, NC
migraine
Phase II
www.colucid.com
LBR-101
(CGRP mAb)
Labrys Biologics
San Mateo, CA
prevention of chronic migraine
Phase I
www.labrysbiologics.com
Levadex®
dihydroergotamine mesilate
inhalation
Allergan
Irvine, CA
MAP Pharmaceuticals
Mountain View, CA
migraine (adults)
application submitted
www.allergan.com
LY2951742
(calcitonin gene-related peptide
antagonist mAb)
Arteaus Therapeutics
Cambridge, MA
Eli Lilly
Indianapolis, IN
prevention of migraine
Phase II
www.arteaus.com
www.lilly.com
NVD-201
(sumatriptan oral spray)
NovaDel
Bridgewater, NJ
migraine
Phase II
www.novadel.com
NXN-188
(nNOS inhibitor/5-HT agonist)
NeurAxon
Mississauga, Canada
acute migraine
Phase II completed
www.neuraxon.com
odansetron/rizatriptan
fixed-dose combination
MonoSol Rx
Warren, NJ
migraine
Phase I
www.monosolrx.com
rizatriptan oral film
IntelGenx
Ville St-Laurent, Canada
RedHill Biopharma
Tel Aviv, Israel
migraine
application submitted
www.intelgenx.com
www.redhillbio.com
RT001
(botulinum toxin A topical)
Revance Therapeutics
Newark, CA
migraine
Phase II
www.revance.com
Medicines in Development
Neurological Disorders 2013
29
Medicines in Development for Neurological Disorders
Headache
Product Name
Sponsor
Indication
Development Phase
SPRIX®
ketorolac intranasal
Regency Therapeutics
Shirley, NY
migraine
Phase III
www.regencytherapeutics.com
TI-001
(oxytocin intranasal)
Trigemina
Moraga, CA
chronic migraine, daily headache
Phase II
www.trigemina.com
ubrogepant
(MK-1602)
Merck
Whitehouse Station, NJ
migraine
Phase II
www.merck.com
zolmitriptan intranasal
Shin Nippon Biomedical Laboratories USA
Everett, WA
migraine
Phase I
www.snblusa.com
zucapsaicin
(TRPV-1 agonist)
Winston Pharmaceuticals
Vernon Hills, IL
cluster headache
(see also pain)
Phase III
www.winstonlabs.com
Huntington’s Disease
Product Name
Sponsor
Indication
Development Phase
EX-527
(SIRT1 protein inhibitor)
Siena Biotech
Siena, Italy
Huntington’s disease
Phase I completed
www.sienabiotech.com
GSK356278
(PDE4 inhibitor)
GlaxoSmithKline
Rsch. Triangle Park, NC
Huntington’s disease
Phase I
www.gsk.com
OMS-824
(PDE10 inhibitor)
Omeros
Seattle, WA
Huntington’s disease
Phase I
www.omeros.com
PBT2
(metal protein-attenuating
compound)
Prana Biotechnology
Parkville, Australia
Huntington’s disease
Phase II
www.pranabio.com
pridopidine
(Orphan Drug)
Teva Pharmaceutical
North Wales, PA
Huntington’s disease
Phase III
www.tevapharm.com
RP103
(mercaptamine bitartrate
delayed release)
Raptor Pharmaceuticals
Novato, CA
Huntington’s disease
Phase II/III
www.raptorpharma.com
SD-809
(VMAT inhibitor)
Auspex Pharmaceuticals
La Jolla, CA
Huntington’s disease
(see also other)
Phase I
www.auspexpharma.com
30
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Multiple Sclerosis
Product Name
Sponsor
Indication
Development Phase
ABT-413
(sphingosine-1 phosphate
receptor modulator)
AbbVie
North Chicago, IL
multiple sclerosis
Phase I
www.abbvie.com
anti-LINGO
(LINGO-1-protein-inhibitor)
Biogen Idec
Weston, MA
multiple sclerosis
Phase II
www.biogenidec.com
ARX424
(peginterferon beta-1a)
Ambrx
La Jolla, CA
multiple sclerosis
Phase I
www.ambrx.com
ATX-MS-1467
(immune tolerizing agent)
EMD Serono
Rockland, MA
multiple sclerosis
Phase I
www.emdserono.com
AZ01
(peginterferon beta)
Allozyne
Seattle, WA
multiple sclerosis
Phase I
www.allozyne.com
BHT-3009
(DNA vaccine)
Bayhill Therapeutics
Palo Alto, CA
relapsing-remitting
multiple sclerosis
Phase II completed
www.bayhilltx.com
Copaxone®
glatiramer acetate for injection
(20 mg)
Teva Pharmaceutical
North Wales, PA
multiple sclerosis
application submitted
www.tevapharm.com
daclizumab
AbbVie
North Chicago, IL
Biogen Idec
Weston, MA
multiple sclerosis, relapsing forms
Phase III
www.abbvie.com
www.biogenidec.com
firategrast
(dual alpha4-integrin antagonist)
GlaxoSmithKline
Rsch. Triangle Park, NC
multiple sclerosis
Phase II
www.gsk.com
GEH-120714
(18F-labeled imaging agent)
GE Healthcare
Waukesha, WI
multiple sclerosis (diagnosis)
Phase I
www.gehealthcare.com
GM6
(peptide therapeutic)
Genervon Biopharmaceuticals
Pasadena, CA
multiple sclerosis
(see also Alzheimer’s, amyotrophic
lateral sclerosis, Parkinson’s, spinal
cord injury, stroke)
Phase I
www.genervon.com
GSK239512
(H3 receptor inhibitor)
GlaxoSmithKline
Rsch. Triangle Park, NC
multiple sclerosis
Phase II
www.gsk.com
GSK2618960
(IL-7 modulator mAb)
GlaxoSmithKline
Rsch. Triangle Park, NC
multiple sclerosis
Phase I
www.gsk.com
idebenone
Santhera Pharmaceuticals
Liestal, Switzerland
U.S. National Institutes of Health
Bethesda, MD
primary progressive multiple sclerosis
(see also other)
Phase I/II
www.santhera.com
Medicines in Development
Neurological Disorders 2013
31
Medicines in Development for Neurological Disorders
Multiple Sclerosis
Product Name
Sponsor
Indication
Development Phase
imilecleucel-T
Opexa Therapeutics
The Woodlands, TX
secondary progressive multiple
sclerosis
(Fast Track)
Phase II
www.opexatherapeutics.com
IR208
(immunostimulant vaccine)
Immune Response BioPharma
New York, NY
multiple sclerosis
Phase II
www.immuneresponsebiopharma.
com
laquinimod
Teva Pharmaceutical
North Wales, PA
multiple sclerosis
(Fast Track)
Phase III
www.tevapharm.com
Lemtrada™
alemtuzumab
Bayer HealthCare Pharmaceuticals
Wayne, NJ
Genzyme
Cambridge, MA
multiple sclerosis
(Fast Track)
application submitted
www.bayerpharma.com
www.genzyme.com
masitinib
AB Science USA
Short Hills, NJ
multiple sclerosis, primary
progressive multiple sclerosis,
secondary progressive multiple
sclerosis
Phase II/III
www.ab-science.com
MEDI-551
(anti-CD19 mAb)
MedImmune
Gaithersburg, MD
multiple sclerosis
Phase I
www.medimmune.com
MOR103
(GM-CSF antagonist mAb)
GlaxoSmithKline
Rsch. Triangle Park, NC
multiple sclerosis
Phase I/II completed
www.gsk.com
ocrelizumab
(humanized anti-CD20 mAb)
Biogen Idec
Weston, MA
Genentech
South San Francisco, CA
primary progressive multiple
sclerosis, relapsing-remitting multiple
sclerosis
Phase III
www.biogenidec.com
www.gene.com
ofatumumab
subcutaneous
(CD20 human mAb)
Genmab US
Princeton, NJ
GlaxoSmithKline
Rsch. Triangle Park, NC
relapsing-remitting multiple sclerosis
Phase II
www.genmab.com
www.gsk.com
ONO-4641
(oral S1P receptor modulator)
EMD Serono
Rockland, MA
multiple sclerosis
Phase II
www.emdserono.com
Plegridy™
peginterferon beta-1a
Biogen Idec
Weston, MA
multiple sclerosis, relapsing forms
(Fast Track)
application submitted
www.biogenidec.com
plovamer acetate
(second-generation peptide
copolymer)
EMD Serono
Rockland, MA
multiple sclerosis
Phase I
www.emdserono.com
32
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Multiple Sclerosis
Product Name
Sponsor
Indication
Development Phase
ponesimod
Actelion Pharmaceuticals
South San Francisco, CA
multiple sclerosis
Phase II completed
www.actelion.com
rHIgM22
(myelin protein stimulant mAb)
Acorda Therapeutics
Ardsley, NY
multiple sclerosis
Phase I
www.acorda.com
RPC1063
(S1P1 receptor agonist)
Receptos
San Diego, CA
relapsing multiple sclerosis
Phase II/III
www.receptos.com
RTL1000
(T-lymphocyte modulator)
Artielle ImmunoTherapeutics
San Mateo, CA
multiple sclerosis
Phase I
www.artielle.com
Sativex®
nabiximols
GW Pharmaceutical
Wiltshire, United Kingdom
muscle spasticity in multiple sclerosis
Phase II
www.gwpharm.com
secukinumab
(AIN457)
Novartis Pharmaceuticals
East Hanover, NJ
multiple sclerosis
Phase II
www.novartis.com
siponimod
(BAF312)
Novartis Pharmaceuticals
East Hanover, NJ
multiple sclerosis
Phase III
www.novartis.com
Trimesta™
estriol succinate
Synthetic Biologics
Rockville, MD
multiple sclerosis
Phase II
www.syntheticbiologics.com
TSO
(trichuris suis ova/CNDO-201)
Coronado Biosciences
Burlington, MA
multiple sclerosis
Phase II
www.coronadobiosciences.com
Tysarbi®
natalizumab
Biogen Idec
Weston, MA
multiple sclerosis (first-line therapy)
in patients negative for JCV antibodies
-------------------------------------secondary-progressive
multiple sclerosis
application submitted
www.biogenidec.com
----------------------------------------Phase III
www.biogenidec.com
VX15
(SEMA-4D mAb)
Teva Pharmaceutical
North Wales, PA
Vaccinex
Rochester, NY
multiple sclerosis
Phase I
www.tevapharm.com
www.vaccinex.com
XP23829
(NF-kappa B inhibitor)
Xenoport
Santa Clara, CA
multiple sclerosis
Phase I
www.xenoport.com
Medicines in Development
Neurological Disorders 2013
33
Medicines in Development for Neurological Disorders
Muscular Dystrophy
Product Name
Sponsor
Indication
Development Phase
AAV1-FS344
(gene therapy-delivered myostatin
inhibitor)
(Orphan Drug)
Milo Biotechnology
Cleveland, OH
Duchenne muscular dystrophy
Phase I/II
www.milobiotechnology.com
ataluren
(Orphan Drug)
PTC Therapeutics
South Plainfield, NJ
Duchenne muscular dystrophy
(Fast Track)
Phase III
www.ptcbio.com
drisapersen
(antisense oligonucleotide)
(Orphan Drug)
GlaxoSmithKline
Rsch. Triangle Park, NC
Prosensa
Leiden, Netherlands
Duchenne muscular dystrophy
Phase III
www.gsk.com
eteplirsen
(RNA interference)
Sarepta Therapeutics
Cambridge, MA
Duchenne muscular dystrophy
(Fast Track)
Phase II
www.sareptatherapeutics.com
GNE lipoplex
(muscular dystrophy gene therapy)
Gradalis
Carrollton, TX
hereditary inclusion body
myopathy
Phase I
www.gradalisinc.com
HT-100
(halofunginone)
(Orphan Drug)
Halo Therapeutics
Newton, MA
Duchenne muscular dystrophy
Phase I/II
www.halotherapeutics.com
PF-06252616
Pfizer
New York, NY
muscular dystrophy
Phase I
www.pfizer.com
Product Name
Sponsor
Indication
Development Phase
ABT-639
(T-type calcium channel
antagonist)
AbbVie
North Chicago, IL
diabetic neuropathy
Phase II
www.abbvie.com
ABT-652
(histamine H3 receptor modulator)
AbbVie
North Chicago, IL
diabetic neuropathy
Phase II
www.abbvie.com
AF-219
(purinergic P2 receptor antagonist)
Afferent Pharmaceuticals
San Mateo, CA
osteoarthritis pain
Phase II
www.afferentpharma.com
agomelatine
(NRT-31/36)
Nectid
Princeton, NJ
neuropathic pain, pain
associated with multiple sclerosis
Phase II
www.nectid.com
Pain
34
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Pain
Product Name
Sponsor
Indication
Development Phase
ALO-02
(oxycodone/naltrexone core/
abuse resistant)
Pfizer
New York, NY
moderate to severe chronic pain
Phase III
www.pfizer.com
AmiKet™
amitriptyline/ketamine
EpiCept
Tarrytown, NY
neuropathic pain
(Fast Track)
Phase II
www.epicept.com
ARA 290
(erythropoietin receptor agonist)
Araim Pharmaceuticals
Yorktown, NY
diabetic neuropathy
Phase II
www.araim.org
ARC-2022
(lidocaine topical gel)
Arcion Therapeutics
Baltimore, MD
postherpetic neuralgia
Phase I/II
www.arciontherapeutics.com
ARRY-797
(p38 MAP kinase inhibitor)
Array BioPharma
Boulder, CO
osteoarthritis pain
Phase II
www.arraybiopharma.com
AS-3201
(ranirestat)
Eisai
Woodcliff Lake, NJ
diabetic neuropathy
Phase III
www.eisai.com
ATx08 001
(PPAR-gamma agonist)
Aestus Therapeutics
East Windsor, NJ
postherpetic neuralgia
Phase II
www.aestustherapeutics.com
AV-101
(NMDA receptor antagonist)
VistaGen Therapeutics
South San Francisco, CA
neuropathic pain
Phase I
www.vistagen.com
AVP-786
(deuterated dextromethorphan)
Avanir Pharmaceuticals
Aliso Viejo, CA
chronic pain
Phase I
www.avanir.com
AVP-923
(dextromethorphan/quinidine
fixed-dose combination)
Avanir Pharmaceuticals
Aliso Viejo, CA
diabetic neuropathy
(see also Alzheimer’s)
-------------------------------------central neuropathic pain in
multiple sclerosis
Phase III
www.avanir.com
----------------------------------------Phase III
www.avanir.com
BEMA buprenorphine transmucosal
BioDelivery Sciences International
Raleigh, NC
neuropathic pain
Phase III
www.bdsi.com
Botox®
onabotulinumtoxinA
Allergan
Irvine, CA
osteoarthritis pain
(see also headache, other)
Phase II
www.allergan.com
Butrans™ Transdermal
System CIII
buprenorphine transdermal
Purdue Pharma
Stamford, CT
chronic pain (adolescents)
Phase III
www.purduepharma.com
Celebrex®
celecoxib
Pfizer
New York, NY
chronic pain
application submitted
www.pfizer.com
Medicines in Development
Neurological Disorders 2013
35
Medicines in Development for Neurological Disorders
Pain
Product Name
Sponsor
Indication
Development Phase
clonidine topical gel
BioDelivery Sciences
Raleigh, NC
diabetic neuropathy
(Fast Track)
Phase II
www.bdsi.com
CNSB015
(flupirtine-based pain therapy)
Relevare Pharmaceuticals
Melbourne, Australia
neuropathic pain
Phase I/II
www.relevarepharma.com
COL-003
(oxycodone extended release/
abuse-resistant formulation)
Collegium Pharmaceuticals
Canton, MA
chronic lower back pain
Phase III
www.collegiumpharma.com
COL-172
(oxymorphone extended release/
abuse resistant)
Collegium Pharmaceuticals
Canton, MA
chronic lower back pain
(Fast Track)
Phase I
www.collegiumpharma.com
C-peptide long-acting
(CBX129801)
Cebix
La Jolla, CA
diabetic neuropathy
(Fast Track)
Phase II
www.cebix.com
CR845
(opioid kappa receptor agonist)
Cara Therapeutics
Shelton, CT
chronic pain
Phase I
www.caratherapeutics.com
CTP-354
(GABA-A receptor modulator)
Concert Pharmaceuticals
Lexington, MA
neuropathic pain
(see also spasticity)
Phase I
www.concertpharma.com
diclofenac nano-formulation
Iroko Pharmaceuticals
Philadelphia, PA
osteoarthritis pain
Phase III
www.iroko.com
DS-5565
(CACNA2D1 protein modulator)
Daiichi Sankyo
Parsippany, NJ
chronic pain
Phase II
www.dsi.com
Eladur®
bupivacaine transdermal
(Orphan Drug)
DURECT
Cupertino, CA
post-herpetic neuralgia
Phase II
www.durect.com
ELI-154
(oxycodone controlled release)
Elite Pharmaceuticals
Northvale, NJ
chronic pain
Phase II
www.elitepharma.com
ELI-216
(oxycodone/naltrexone
fixed-dose combination)
Elite Pharmaceuticals
Northvale, NJ
moderate to severe chronic pain
Phase II
www.elitepharma.com
EMA401
(angiotensin-type-2 receptor
antagonist)
Spinifex Pharmaceuticals
South Yarra, Australia
postherpetic neuralgia
Phase I
www.spinifexpharma.com.au
Fentora®
fentanyl buccal
Cephalon (Teva)
Frazer, PA
chronic pain
application submitted
www.tevapharm.com
36
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Pain
Product Name
Sponsor
Indication
Development Phase
filorexant
(dual orexin receptor antagonist)
Merck
Whitehouse Station, NJ
diabetic neuropathy
Phase II
www.merck.com
fulranumab
(nerve growth factor inhibitor)
Janssen Research & Development
Raritan, NJ
neuropathic pain
Phase II
www.janssenrnd.com
GRT6005
(ORL-1 receptor agonist)
Forest Laboratories
New York, NY
pain due to osteoarthritis of the knee
Phase II
www.frx.com
hydrocodone extended release
(CEP-33237)
Cephalon (Teva)
Frazer, PA
chronic pain
Phase III
www.tevapharm.com
hydromorphone intrathecal
Mallinckrodt
Hazelwood, MO
chronic pain
Phase III
www.mallinckrodt.com
K-103-IP
(COX inhibitor)
Kowa Research Institute
Morrisville, NC
arthritis pain
Phase I
www.kowaus.com
KRN5500
(protein synthesis inhibitor)
DARA Biosciences
Raleigh, NC
peripheral neuropathy
(Fast Track)
Phase II
www.darabio.com
levorphanol extended release
(abuse-deterrent drug delivery
system)
Relmada Therapeutics
Blue Bell, PA
moderate to severe chronic pain
Phase I
www.relmada.com
Lyrica®
pregabalin
Pfizer
New York, NY
peripheral neuropathic pain
(see also epilepsy)
Phase III
www.pfizer.com
mavatrep
Janssen Research & Development
Raritan, NJ
chronic pain
Phase I
www.janssenrnd.com
meloxicam nano-formulation
Iroko Pharmaceuticals
Philadelphia, PA
osteoarthritis pain
Phase III
www.iroko.com
NCE
Eli Lilly
Indianapolis, IN
osteoarthritis pain
Phase I
www.lilly.com
neublastin
Biogen Idec
Weston, MA
NsGene
Ballerup, Denmark
neuropathic pain
Phase I
www.biogenidec.com
NKTR-181
(mu-opioid agonist)
Nektar Therapeutics
San Francisco, CA
moderate to severe chronic pain
(Fast Track)
Phase II
www.nektar.com
Medicines in Development
Neurological Disorders 2013
37
Medicines in Development for Neurological Disorders
Pain
Product Name
Sponsor
Indication
Development Phase
NP-1998
(capsaicin topical liquid)
Acorda Therapeutics
Ardsley, NY
neuropathic pain
Phase II
www.acorda.com
NXN-462
(nNOS inhibitor)
NeurAxon
Mississauga, Canada
postherpetic neuralgia
Phase II
www.neuraxon.com
oxycodone/naloxone
controlled release
(OXN)
Purdue Pharma
Stamford, CT
severe chronic lower back pain
Phase II
www.purduepharma.com
PA65020
(aspirin 650mg/omeprazole 20mg
fixed-dose combination)
POZEN
Chapel Hill, NC
chronic pain
Phase I
www.pozen.com
Pennsaid® 2%
diclofenac transdermal
second-generation
Nuvo Research
Mississauga, Canada
osteoarthritis pain
application submitted
www.nuvoresearch.com
PF-05089771
(Nav1.7 sodium channel inhibitor)
Pfizer
New York, NY
chronic pain
Phase I
www.pfizer.com
PF-06273340
Pfizer
New York, NY
chronic pain
Phase I
www.pfizer.com
PF-06305591
Pfizer
New York, NY
chronic pain
Phase I
www.pfizer.com
PLX1100
(ibuprofen/phosphatidylcholine)
PLx Pharma
Houston, TX
osteoarthritis pain
Phase II
www.plxpharma.com
pregabalin controlled release
Pfizer
New York, NY
postherpetic neuralgia
(see also epilepsy)
Phase III
www.pfizer.com
PTI-202
(abuse-resistant opioid)
Pain Therapeutics
Austin, TX
moderate to severe chronic pain
Phase I completed
www.paintrials.com
PTI-721
(abuse-resistant opioid)
Pain Therapeutics
Austin, TX
moderate to severe chronic pain
Phase I completed
www.paintrials.com
Qutenza®
capsaicin 8% patch
Astellas Pharma
Tokyo, Japan
NeurogesX
San Mateo, CA
HIV-associated neuropathy
(Fast Track)
application submitted
www.astellas.com
www.neurogesx.com
----------------------------------------Phase III
www.astellas.com
www.neurogesx.com
38
-------------------------------------diabetic neuropathy
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Pain
Product Name
Sponsor
Indication
Development Phase
Remoxy™
oxycodone controlled release
(Mu-type opioid receptor agonist/
abuse resistant)
Pain Therapeutics
Austin, TX
Pfizer
New York, NY
moderate to severe chronic pain
application submitted
www.pfizer.com
rezatomidine
(alpha adrenergic receptor agonist)
ACADIA Pharmaceuticals
San Diego, CA
Allergan
Irvine, CA
diabetic neuropathy
Phase II
www.acadia-pharm.com
www.allergan.com
S-117957
Purdue Pharma
Stamford, CT
Shionogi
Florham Park, NJ
neuropathic pain
Phase I
www.purduepharma.com
www.shionogi.com
SAR292833/GRC15300
(TRVP3 antagonist)
Glenmark Pharmaceuticals
Mumbai, India
Sanofi US
Bridgewater, NJ
neuropathic pain, osteoarthritis pain
Phase II
www.sanofi.com
SD-254
(deuterium-substituted
venlafaxine analogue)
Auspex Pharmaceuticals
La Jolla, CA
neuropathic pain
Phase I
www.auspexpharma.com
SEP-228432
(SRNDI inhibitor)
Sunovion Pharmaceuticals
Marlborough, MA
neuropathic pain
Phase I
www.sunovion.com
SKL-NP
SK biopharmaceuticals
Fair Lawn, NJ
neuropathic pain
Phase II
www.skbp.com
sufentanil
(transdermal delivery system)
DURECT
Cupertino, CA
chronic pain
Phase II
www.durect.com
TAK-428
(neurotrophic factor production
accelerator)
Takeda Pharmaceuticals International
Deerfield, IL
diabetic neuropathy
Phase II
www.takeda.com
TEM
(senrebotase)
Allergan
Irvine, CA
postherpetic neuralgia
Phase II
www.allergan.com
V116517
Purdue Pharma
Stamford, CT
pain due to osteoarthritis of the knee,
postherpetic neuralgia
Phase II
www.purduepharma.com
V158866
(FAAH inhibitor)
Vernalis
Winnersh, United Kingdom
neuropathic pain
Phase II
www.vernalis.com
Medicines in Development
Neurological Disorders 2013
39
Medicines in Development for Neurological Disorders
Pain
Product Name
Sponsor
Indication
Development Phase
VBY-036
(protease inhibitor)
Virobay
Menlo Park, CA
neuropathic pain
Phase I
www.virobayinc.com
verapamil
Calosyn Pharma
Sharon, MA
osteoarthritis pain
Phase I/II
www.calosynpharma.com
Vicodin® CR
hydrocodone/paracetamol
controlled release
AbbVie
North Chicago, IL
back pain, osteoarthritis pain
application submitted
www.abbvie.com
VM202
(modified hepatocyte growth
factor gene therapy)
ViroMed
Seoul, South Korea
diabetic neuropathy
Phase II
www.viromed.co.kr
XEN402
Teva Pharmaceutical
North Wales, PA
Xenon Pharmaceuticals
Burnaby, Canada
postherpetic neuralgia
Phase II
www.tevapharm.com
www.xenon-pharma.com
YKP-509
(carisbamate)
SK biopharmaceuticals
Fair Lawn, NJ
neuropathic pain
Phase II
www.skbp.com
YKP-3089
SK biopharmaceuticals
Fair Lawn, NJ
neuropathic pain
(see also epilepsy)
Phase II
www.skbp.com
Z160
(N-type calcium channel
antagonist)
Zalicus
Cambridge, MA
neuropathic pain,
postherpetic neuralgia
Phase II
www.zalicus.com
Z944
(T-type calcium channel
antagonist)
Zalicus
Cambridge, MA
neuropathic pain
Phase I
www.zalicus.com
Zohydro ER™
hydrocodone extended-release
Zogenix
San Diego, CA
moderate to severe chronic pain
application submitted
www.zogenix.com
zucapsaicin
(TRPV-1 agonist)
(Orphan Drug)
Winston Pharmaceuticals
Vernon Hills, IL
chronic pain, postherpetic neuralgia
(see also headache)
application submitted
www.winstonlabs.com
40
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Parkinson’s Disease
Product Name
Sponsor
Indication
Development Phase
AFQ056
(mavoglurant)
Novartis Pharmaceuticals
East Hanover, NJ
drug-induced dyskinesia in
Parkinson’s disease
Phase II
www.novartis.com
AQW051
(alpha7 nicotinic acetylcholine
receptor agonist)
Novartis Pharmaceuticals
East Hanover, NJ
drug-induced dyskinesia
in Parkinson’s disease
Phase II completed
www.novartis.com
autologous stem cell therapy
NeuroGeneration
Los Angeles, CA
Parkinson’s disease
Phase I
www.neurogeneration.com
AVE-8112
(PDE4 inhibitor)
Sanofi US
Bridgewater, NJ
The Michael J. Fox Foundation
for Parkinson’s Research
New York, NY
Parkinson’s disease
Phase I
www.sanofi.com
www.michaeljfox.org
AZD3241
(myeloperoxidase inhibitor)
AstraZeneca
Wilmington, DE
Parkinson’s disease
Phase II
www.astrazeneca.com
CERE-120
(AAV-NTN)
Ceregene
San Diego, CA
Parkinson’s disease
Phase II
www.ceregene.com
CVT-301
(levodopa inhalation)
Civitas Therapeutics
Chelsea, MA
Parkinson’s disease
Phase II
www.civitastherapeutics.com
davunetide intranasal
Allon Therapeutics
Vancouver, Canada
Parkinson’s disease
(see also Alzheimer’s, other)
Phase I
www.allontherapeutics.com
dipraglurant
(ADX48621)
(immediate release)
Addex Therapeutics
Geneva, Switzerland
levodopa-induced dyskinesia in
Parkinson’s disease
Phase II
www.addextherapeutics.com
DM-1992
(carbidopa/levodopa
controlled-release)
Depomed, Inc.
Newark, CA
Parkinson’s disease
Phase II
www.depomed.com
Duopa®
levodopa/carbidopa
intestinal gel (LCIG)
(Orphan Drug)
AbbVie
North Chicago, IL
advanced Parkinson’s disease
(Fast Track)
application submitted
www.abbvie.com
fipamezole
(alpha-2 adrenergic receptor
antagonist)
Santhera Pharmaceuticals
Liestal, Switzerland
Parkinson’s disease
(Fast Track)
Phase II completed
www.santhera.com
Medicines in Development
Neurological Disorders 2013
41
Medicines in Development for Neurological Disorders
Parkinson’s Disease
Product Name
Sponsor
Indication
Development Phase
GM6
(peptide therapeutic)
Genervon Biopharmaceuticals
Pasadena, CA
Parkinson’s disease
(see also Alzheimer’s, amyotrophic
lateral sclerosis, multiple sclerosis,
spinal cord injury, stroke)
Phase II
www.genervon.com
GZ404477
(AAV-hAADC gene therapy)
Genzyme
Cambridge, MA
Parkinson’s disease
Phase I
www.genzyme.com
HT-1067
(MOA-B reversible inhibitor)
Dart NeuroScience
San Diego, CA
Parkinson’s disease
Phase I
www.dartneuroscience.com
KW-6002
(istradefylline)
Kyowa Hakko Kirin America
Princeton, NJ
Parkinson’s disease
application submitted
www.kyowa-kirin.com
levodopa/carbidopa
extended release
IMPAX Laboratories
Hayward, CA
Parkinson’s disease
application submitted
www.impaxlabs.com
NAV5001
(123I-labelled imaging agent)
Navidea Biopharmaceuticals
Dublin, OH
Parkinsonian disorder (diagnosis)
(see also Alzheimer’s)
Phase III
www.navidea.com
Nurelin™
ADS-5102/amantadine ER
Adamas Pharmaceuticals
Emeryville, CA
levodopa-induced dyskinesia
in Parkinson’s disease
Phase III
www.adamaspharma.com
NuroPro®
neurotrophic factor companion
diagnostic
Amarantus BioSciences
Sunnyvale, CA
Parkinson’s disease (diagnosis)
Phase II
www.amarantus.com
opicapone
(COMT inhibitor)
Bial
Lisbon, Portugal
Parkinson’s disease
Phase I
www.bial.com
OS-320
(levodopa/carbidopa)
Osmotica Pharmaceutical
Wilmington, NC
Parkinson’s disease
Phase III
www.osmoticausa.com
Posiphen®
R-phenserine
QR Pharma
Berwyn, PA
Parkinson’s disease
(see also Alzheimer's)
Phase II
www.qrpharma.com
PYM-50028
Phytopharm
Huntingdon, United Kingdom
early stage Parkinson’s disease
Phase II
www.phytopharm.com
safinamide
Newron Pharmaceuticals
Bresso, Italy
Zambon
Bresso, Italy
Parkinson’s disease
Phase III
www.newron.com
www.zambonpharma.com
tozadenant
(adenosine A2A receptor
antagonist)
Biotie Therapies
South San Francisco, CA
UCB
Brussels, Belgium
Parkinson’s disease
Phase II/III
www.biotie.com
www.ucb.com
42
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Parkinson’s Disease
Product Name
Sponsor
Indication
Development Phase
XP21279
(dopamine receptor agonist)
Xenoport
Santa Clara, CA
Parkinson’s disease
Phase II
www.xenoport.com
Product Name
Sponsor
Indication
Development Phase
CK-2127107
Cytokinetics
South San Francisco, CA
muscular atrophy associated with
neuromuscular dysfunction,
muscular weakness, and/or muscle
fatigue
Phase I
www.cytokinetics.com
CTP-354
(GABA-A receptor modulator)
Concert Pharmaceuticals
Lexington, MA
muscle spasticity
(see also pain)
Phase I
www.concertpharma.com
Dysport®
abobotulinumtoxinA
Ipsen Biopharmaceuticals
Basking Ridge, NJ
focal spasticity of upper and lower
limb in adults, adolescents and
children
Phase III
www.ipsen.com
OS-440
(arbaclofen extended release)
Osmotica Pharmaceutical
Marietta, GA
muscle spasticity due to
multiple sclerosis
Phase III
www.osmotica.com
SUN 09
(baclofen extended release)
Sun Pharma Advanced Research
Mumbai, India
muscle spasticity due to
multiple sclerosis
Phase III
www.sunpharma.in
Xeomin®
incobotulinumtoxinA
Merz Pharmaceuticals
Greensboro, NC
post-stroke spasticity
Phase III
www.merzusa.com
Product Name
Sponsor
Indication
Development Phase
AC105
(nervous system modulator)
Acorda Therapeutics
Ardsley, NY
spinal cord injury
(Fast Track)
Phase II
www.acorda.com
ATI355
(anti-NOGO-A mAb)
(Orphan Drug)
Novartis Pharmaceuticals
East Hanover, NJ
spinal cord injury
Phase I
www.novartis.com
Spasticity
Spinal Cord Injury
Medicines in Development
Neurological Disorders 2013
43
Medicines in Development for Neurological Disorders
Spinal Cord Injury
Product Name
Sponsor
Indication
Development Phase
autologous stem cell therapy
DaVinci Biosciences
Costa Mesa, CA
spinal cord injury
in clinical trials
www.dvbiosciences.com
BA-210
(Rho GTP-binding
protein-inhibitor)
(Orphan Drug)
BioAxone BioSciences
Ft. Lauderdale, FL
spinal cord injury
Phase II
www.bioaxonebio.com
GM6
(peptide therapeutic)
Genervon Biopharmaceuticals
Pasadena, CA
spinal cord injury
(see also Alzheimer’s, amyotrophic
lateral sclerosis, multiple sclerosis,
Parkinson’s, stroke)
Phase I
www.genervon.com
HuCNS-SC®
adult neural stem cell therapy
StemCells
Newark, CA
chronic spinal cord injury
(see also genetic)
Phase I/II
www.stemcellsinc.com
NEU2000
GNT Pharma
Yongin, South Korea
spinal cord injury
(see also stroke)
Phase I completed
www.gntpharma.com
stem cell therapy
TCA Cellular Therapy
Covington, LA
spinal cord injury
(see also amyotrophic lateral
sclerosis)
Phase I
www.tcacellulartherapy.com
SUN13837
(FGF-receptor agonist)
Daiichi Sankyo
Parsippany, NJ
spinal cord injury
Phase II
www.dsi.com
Product Name
Sponsor
Indication
Development Phase
3K3A-APC
(recombinant human
activated protein C)
ZZ Biotech
Houston, TX
stroke
Phase I
www.zzbiotech.com
ALD-401
(bone marrow-derived adult
stem cell therapy)
Cytomedix
Gaithersburg, MD
stroke
Phase II
www.cytomedix.com
allogeneic mesenchymal
bone marrow cell therapy
Stemedica Cell Technologies
San Diego, CA
ischemic stroke
Phase I/II
www.stemedica.com
Ampyra®
fampridine sustained-release
Acorda Therapeutics
Ardsley, NY
post-stroke deficits
(see also other)
Phase II
www.acorda.com
Stroke
44
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Stroke
Product Name
Sponsor
Indication
Development Phase
betrixaban
Portola Pharmaceuticals
South San Francisco, CA
prevention of stroke in patients with
atrial fibrillation
Phase II
www.portola.com
CNTO-0007
(cell therapy)
Janssen Research & Development
Raritan, NJ
stroke
Phase I/II
www.janssenrnd.com
desmoteplase
Lundbeck
Deerfield, IL
stroke
(Fast Track)
Phase III
www.lundbeck.com
GM6
(peptide therapeutic)
Genervon Biopharmaceuticals
Pasadena, CA
stroke
(see also Alzheimer’s, amyotrophic
lateral sclerosis, multiple sclerosis,
Parkinson’s, spinal cord injury)
Phase II
www.genervon.com
GSK249320
(myelin-associated glycoprotein
mAb)
GlaxoSmithKline
Rsch. Triangle Park, NC
stroke
Phase II
www.gsk.com
methamphetamine intravenous
Sinapis Pharma
Jacksonville, FL
stroke
(see also brain injury)
Phase I completed
www.sinapispharma.com
MP-124
(PARP inhibitor)
Mitsubishi Tanabe Pharma America
Jersey City, NJ
acute ischemic stroke
Phase I
www.mt-pharma.co.jp
MultiStem®
stem cell therapy
Athersys
Cleveland, OH
stroke
Phase II
www.athersys.com
NA-1
(signal transduction
pathway inhibitor)
NoNO
Toronto, Canada
acute ischemic stroke
Phase II completed
www.nonoinc.ca
NEU2000
GNT Pharma
Yongin, South Korea
stroke
(see also spinal cord injury)
Phase I completed
www.gntpharma.com
PF-03049423
Pfizer
New York, NY
stroke recovery
Phase II
www.pfizer.com
RP-1127
(glibenclamide)
Remedy Pharmaceuticals
New York, NY
stroke
(see also brain injury)
Phase II
www.remedypharmaceuticals.com
SAR126119
(TAFIa inhibitor)
Sanofi US
Bridgewater, NJ
acute ischemic stroke
Phase I
www.sanofi.com
SB623
(stem cell therapy)
SanBio
Mountain View, CA
stroke
Phase I/II
www.san-bio.com
Medicines in Development
Neurological Disorders 2013
45
Medicines in Development for Neurological Disorders
Stroke
Product Name
Sponsor
Indication
Development Phase
TS01
(recombinant complement
C1-inactivator-protein)
Thrombolytic Science International
Cambridge, MA
stroke
Phase I
www.tsillc.com
Product Name
Sponsor
Indication
Development Phase
18F-AV-133
imaging agent
Avid Radiopharmaceuticals
Philadelphia, PA
neurodegenerative disorders
(diagnosis)
Phase I/II
www.avidrp.com
Abilify®
aripiprazole
(once-weekly)
Otsuka America Pharmaceutical
Rockville, MD
Tourette’s syndrome
Phase III
www.otsuka.com
ABT-354
(serotonin-6 receptor antagonist)
AbbVie
North Chicago, IL
neurological disorders
Phase I
www.abbvie.com
ABT-384
AbbVie
North Chicago, IL
neurological disorders
(see also Alzheimer’s)
Phase I completed
www.abbvie.com
Ampyra®
fampridine sustained release
Acorda Therapeutics
Ardsley, NY
cerebral palsy
(see also stroke)
Phase II
www.acorda.com
AP-1101
Ariel Pharmaceuticals
Broomfield, CO
neuroprotection against
postoperative cognitive decline and
memory loss in patients undergoing
extracorporeal
cardiopulmonary bypass
Phase II
Botox®
onabotulinumtoxinA
Allergan
Irvine, CA
juvenile cerebral palsy
(see also headache, pain)
Phase III
www.allergan.com
BYM338
(type II-B activin receptor
modulator mAb)
Novartis Pharmaceuticals
East Hanover, NJ
inclusion body myositis
Phase II
www.novartis.com
CX1739
(AMPA receptor agonist)
Cortex Pharmaceuticals
Glen Rock, NJ
central sleep apnea
Phase I
www.cortexpharm.com
davunetide intranasal
(Orphan Drug)
Allon Therapeutics
Vancouver, Canada
progressive supranuclear palsy
(Fast Track)
(see also Alzheimer’s, Parkinson’s)
Phase II/III
www.allontherapeutics.com
Other
46
Medicines in Development
Neurological Disorders 2013
Medicines in Development for Neurological Disorders
Other
Product Name
Sponsor
Indication
Development Phase
ecopipam
(D1 dopamine receptor antagonist)
Psyadon Pharmaceuticals
Germantown, MD
Tourette’s syndrome
(see also genetic)
Phase I/II
www.psyadonrx.com
EVP-0334
(HDAC inhibitor)
EnVivo Pharmaceuticals
Watertown, MA
neurodegenerative disorders
Phase I
www.envivopharma.com
Firdapse™
amifampridine
(Orphan Drug)
Catalyst Pharmaceutical Partners
Coral Gables, FL
Lambert-Eaton myasthenic syndrome
Phase III
www.catalystpharma.com
Gilenya®
fingolimod
Novartis Pharmaceuticals
East Hanover, NJ
chronic inflammatory
demyelinating
polyradiculoneuropathy
Phase III
www.novartis.com
glycerol phenylbutyrate
(Orphan Drug)
Hyperion Therapeutics
South San Francisco, CA
hepatic encephalopathy
Phase II completed
www.hyperiontx.com
human immune globulin
(subcutaneous)
CSL Behring
King of Prussia, PA
chronic inflammatory
demyelinating
polyradiculoneuropathy
Phase III
www.cslbehring.com
idebenone
(Orphan Drug)
Columbia University
New York, NY
Santhera Pharmaceuticals
Liestal, Switzerland
MELAS syndrome
(see also multiple sclerosis)
Phase II
www.santhera.com
interleukin-7
Cytheris
Rockville, MD
progressive multifocal
leukoencephalopathy
Phase II
www.cytheris.com
IPX159
IMPAX Pharmaceuticals
Hayward, CA
restless legs syndrome
Phase II
www.impaxpharma.com
MK-8616
(sugammadex)
Merck
Whitehouse Station, NJ
neuromuscular blockade
application submitted
www.merck.com
NBI-98854
(VMAT2 inhibitor)
Neurocrine Biosciences
San Diego, CA
drug-induced dyskinesia
(Fast Track)
Phase II
www.neurocrine.com
NH001
(apomorphine subcutaneous)
(Orphan Drug)
NeuroHealing Pharmaceuticals
Waban, MA
coma
(Fast Track)
Phase II
www.neurohealing.com
Medicines in Development
Neurological Disorders 2013
47
Other
Product Name
Sponsor
Indication
Development Phase
NNZ-2566
(cytokine inhibitors/neuropeptide
receptor modulator)
Neuren Pharmaceuticals
Bethesda, MD
Rett syndrome
(Fast Track)
(see also brain injury)
Phase I
www.neurenpharma.com
OCR-002
(ornithine phenylacetate)
(Orphan Drug)
Ocera Therapeutics
San Diego, CA
hepatic encephalopathy in patients
with acute liver failure (iv)
(Fast Track)
-------------------------------------hepatic encephalopathy in patients
with liver cirrhosis (oral)
(Fast Track)
Phase II
www.ocerainc.com
----------------------------------------Phase I
www.ocerainc.com
placulumab
Cephalon (Teva)
Frazer, PA
sciatica
Phase I/II
www.tevapharm.com
pomaglumetad
(LY2140023 monohydrate)
Eli Lilly
Indianapolis, IN
CNS disorders
Phase I
www.lilly.com
SD-809
(VMAT inhibitor)
Auspex Pharmaceuticals
La Jolla, CA
drug-induced dyskinesias,
Tourette’s syndrome
(see also Huntington’s)
Phase I
www.auspexpharma.com
Soliris®
eculizumab
(Orphan Drug)
Alexion Pharmaceuticals
Cheshire, CT
myasthenia gravis, severe and relapsing neuromyelitis optica
Phase II
www.alxn.com
SPI-017
Sucampo Pharmaceuticals
Bethesda, MD
lumbar spinal stenosis
Phase II
www.sucampo.com
SPI-3608
Sucampo Pharmaceuticals
Bethesda, MD
spinal stenosis
Phase I
www.sucampo.com
Tardoxyl™
pyridoxal phosphate
Medicure Pharma
Somerset, NJ
drug-induced dyskinesia
(Fast Track)
Phase II
www.medicure.com
uridine triacetate
(PN401)
Wellstat Therapeutics
Gaithersburg, MD
neurodegenerative disorders
Phase I
www.wellstattherapeutics.com
vigabatrin
(GABA-AT inhibitor)
Catalyst Pharmaceutical Partners
Coral Gables, FL
Tourette’s syndrome
Phase I/II
www.catalystpharma.com
48
Medicines in Development
Neurological Disorders 2013
Glossary
Alzheimer’s disease—The most common form of dementia, characterized by
progressive and chronic deterioration of
cognitive functions, including memory,
thinking and reasoning. Early manifestations include forgetfulness, impaired
ability to focus, and changes in mood and
personality. As the disease progresses,
there is a loss of computational ability, in
addition to word-finding problems and difficulty with ordinary activities. Ultimately,
the disease leads to severe memory loss,
complete disorientation, social withdrawal,
loss of independence, and is fatal.
amyotrophic lateral sclerosis (ALS)—
Also known as Lou Gehrig’s disease, the
most common of the motor neuron diseases, a group of rare disorders in which
the nerves that control muscular activity
degenerate within the brain and spinal
cord causing weakness and wasting of
the muscles.
application submitted—An application
for marketing has been submitted by the
company to the Food and Drug Administration (FDA).
attention deficit/hyperactivity disorder
(ADHD)—ADHD is a complex neurological impairment that results in an overactive behavior pattern and a difficulty
concentrating. While it primarily affects
children, a growing number of adults are
being diagnosed with the disorder. Boys
are affected about three times as often
as girls. Children with ADHD are fidgety,
impulsive, reckless, irritable, emotionally
immature and sometimes aggressive.
Because their attention span is short,
they do not conform to orderly routine.
ADHD often leads to anti-social acts and
difficulty learning, although IQ is normal.
No definite cause has been established,
but some researchers now believe genetics plays a role.
cerebral palsy—A general term for disorders of movement and posture resulting
from damage to the brain in pregnancy,
during birth, in the newborn period or in
early childhood.
dementia—Degeneration of central
nervous functions, such as memory and
Medicines in Development
learning capacity. The natural decline of
these functions with age is grossly exaggerated in dementia.
problems such as clumsy or awkward
movements and unsteadiness, occurs in
many different diseases and conditions.
Duchenne muscular dystrophy—An
inherited disorder that involves rapidly
worsening muscle weakness. Other
muscular dystrophies get worse much
more slowly. Duchenne’s is caused by a
defective gene. Because of the way the
disease is inherited, males are more likely
to develop symptoms than are women.
glioblastoma multiforme—The most
common and most malignant of the astrocytomas. The tumor grows so fast that
it increases pressure in the brain, producing headaches, slowed thinking, and if
severe enough, sleepiness and coma.
epilepsy—Recurrent seizures—transient
neurological abnormalities caused by
abnormal electrical activity in the brain—
or temporary alteration in one or more
brain functions. Seizures are a symptom
of brain dysfunction and can result from
a wide variety of diseases or injury.
Fast Track—A process designed to
facilitate the development and expedite
the review of drugs to treat serious
diseases and fill an unmet medical need.
The status is assigned by the U.S. Food
and Drug Administration. The purpose
is to get important new drugs to the
patient earlier. Fast Track addresses a
broad range of serious diseases. Generally, determining factors include whether
the drug will have an impact on such
factors 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.
Filling an unmet medical need is defined
as providing a therapy where none exists
or providing a therapy which may be
potentially superior to existing therapy.
Once a drug receives Fast Track designation, early and frequent communication
between the FDA and a drug company is
encouraged throughout the entire drug
development and review process. The
frequency of communication assures that
questions and issues are resolved quickly,
often leading to earlier drug approval
and access by patients.
Friedreich’s ataxia—An inherited
disease that causes progressive damage to the nervous system resulting in
symptoms ranging from gait disturbance
and speech problems to heart disease.
“Ataxia,” which refers to coordination
Neurological Disorders 2013
glioma—A type of brain tumor arising
from the supporting glial cells within
the brain. Gliomas make up about 60
percent of all primary brain tumors.
Huntington’s disease—Huntington’s
chorea is an uncommon, inherited disease in which degeneration of the basal
ganglia (structures deep in the brain)
results in chorea (rapid, jerky, involuntary
movements) and dementia (progressive
mental impairment).
Lennox-Gastaut syndrome—Characterized by seizures and mental retardation
in infants and young children.
metastases/metastatic—Areas of secondary cancer that have spread from the
primary or original cancer site.
migraine—Severe headache resulting from an abnormal dilation of blood
vessels deep within the brain. It can last
from two hours to several days and is
often accompanied by nausea, vomiting
and sensitivity to noise and/or light.
multiple sclerosis (MS)—Progressive
disease of the central nervous system in
which scattered patches of the covering
of nerve fibers (myelin) in the brain and
spinal cord are destroyed. Symptoms
range from numbness and tingling to
paralysis and incontinence.
muscular dystrophy—Inherited muscular disorder of unknown cause in which
muscle fibers slowly degenerate. Duchenne MD is the most common type.
myasthenia gravis—A chronic autoimmune neuromuscular disease characterized by varying degrees of weakness of
the skeletal (voluntary) muscles of the
body. The hallmark of myasthenia gravis
49
Glossary
is muscle weakness that increases during
periods of activity and improves after
periods of rest.
neuroblastoma—A tumor of the adrenal
glands or sympathetic nervous system
(the part of the nervous system responsible for certain automatic body functions, such as the control of heart rate).
Neuroblastomas are the most common
extracranial (outside the skull) solid
tumors of childhood.
neuropathic pain—Caused by disease,
inflammation, or damage to the peripheral nerves, which connect the central
nervous system (brain and spinal cord) to
the sense organs, muscles, glands, and
internal organs.
neuropathy—Disease, inflammation, or
damage to the peripheral nerves, which
connect the central nervous system to
the sense organs, muscles, glands, and
internal organs.
Orphan Drug—A drug to treat a disease
that has a patient population of 200,000
or less, or a disease that has a patient
population of more than 200,000 and a
developmental cost that will not be recovered from sales in the United States.
Parkinson’s disease—Chronic neurologic
disease of unknown cause, characterized
by tremors, rigidity and an abnormal gait.
Phase 0—First-in-human trials conducted in accordance with FDA’s 2006 guidance on exploratory Investigational New
Drug (IND) studies designed to speed up
development of promising drugs by establishing very early whether the tested
compound behaves in human subjects as
was anticipated from preclinical studies.
Phase I—Safety testing and pharmacological profiling in humans.
Phase II—Effectiveness testing in
humans.
Phase III—Extensive clinical trials in
humans.
postherpetic neuralgia—A burning pain
that may recur at the site of an attack
of shingles months or even years after
the illness.
prodrug—An inactive medicine that
becomes active inside the body through
metabolic processes. Prodrugs can be
used to improve how a specific medicine is absorbed, distributed, metabolized or excreted.
restless legs syndrome—Restless legs
syndrome is an overwhelming urge
to move the legs usually caused by
uncomfortable or unpleasant sensations
in the legs.
spinal cord injury—Damage to the spinal
cord which can cause loss of sensation,
muscle weakness or paralysis.
stroke—Usually caused by atherosclerosis. It results in death or serious brain
damage, such as paralysis or loss of
speech. An ischemic stroke is caused
by blocked or narrowed arteries that
prevent sufficient blood and oxygen from
reaching the brain.
Tourette syndrome (TS)—A neurological disorder characterized by repetitive,
involuntary movements and vocalizations
called tics. The early symptoms of TS are
typically noticed first in childhood, with
the average onset between the ages of
3 and 9. TS occurs in people from all
ethnic groups; males are affected about
three to four times more often than
females. It is estimated that 200,000
Americans have the most severe form
of TS, and as many as 1 in 100 exhibit
milder and less complex symptoms such
as chronic motor or vocal tics. Although
TS can be a chronic condition with
symptoms lasting a lifetime, most people
with the condition experience their worst
tic symptoms in their early teens, with
improvement occurring in the late teens
and continuing into adulthood.
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 July 18, 2013. 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 © 2013 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, Washington, DC 20004
50
Medicines in Development
Neurological Disorders 2013
The Drug Discovery, Development and Approval Process
Developing a new medicine takes an average of 10-15 years;
For every 5,000-10,000 compounds in the pipeline, only 1 is approved.
PRECLINICAL
5,000 – 10,000
CLINICAL TRIALS
FDA REVIEW LG-SCALE MFG
5
250
ONE FDAAPPROVED
DRUG
3 – 6 YEARS
PHASE
1
PHASE
3
PHASE
2
NUMBER OF VOLUNTEERS
20 –80
100 – 300
1,000 – 3,000
6 – 7 Y EARS
NDA SUBMIT T ED
COMPOUNDS
IND SUBMIT T ED
P R E - DI S COVERY
DRUG DISCOVERY
LON G , RISKY ROAD
0. 5 – 2
Y EARS
PHASE 4: POST-M AR K ET I N G SU RVEI L L A N C E
Drug Discovery and Development: A
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 experimental drug to travel from lab to U.S. patients,
according to the Tufts Center for the Study of
Drug Development. Only five in 5,000 compounds that enter preclinical testing make it to
human testing. And only one of those five is
approved for sale.
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 recent study by the Tufts Center
for the Study of Drug Development.
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 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 80 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
300, 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 3,000 patients
(but sometime many more thousands), to gener-
ate 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 $48.5 billion in research and development in 2012.