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Don’t Panic! Novartis Still Invested
In CAR-T
CTL019’s exclusion from Novartis’s
future potential blockbuster list has
explanation (p3)
Data
Expert View
Opdivo fallout: rivalry in PD-1
market to continue and more trials
could mean more surprises (p8)
Pharma does not need to make
wearables; instead it needs to figure
out how best to use the data these
technologies can provide (p20)
Scrip
19 August 2016
No. 3816
Pharma intelligence | informa
Shutterstock: T.Dallas
s cripintell ig e n c e .c om
Boehringer’s Oncology Strategy:
Build From Niche Lung Cancer Base
EMILY HAYES [email protected]
With Gilotrif sales building over time, private Boehringer Ingelheim turns its focus
to broad development program for its
third-generation EGFR inhibitor olmutinib, with ambition to become a leader in
lung cancer.
A
s it moves into its next stage of life
as an oncology player, Boehringer
Ingelheim GMBH is prioritizing lung
cancer, with hopes of building a strong franchise upon a base of targeted EGFR drugs –
Gilotrif and olmutinib, the third-generation
follow-on drug in-licensed from Hanmi
Pharmaceutical Co. Ltd.
Approval of Boehringer’s Gilotrif/Giotrif (afatinib) in July 2013 for NSCLC with EGFR mutations marked Boehringer’s entry into the
oncology market after 20 years of investment
and research and development in the space.
Gilotrif, a once-daily, irreversible blocker
of the ErbB family that specifically inhibits
EGFR, HER2 and ErbB4, faced a big commercial challenge from the start, competing with Roche/Astellas Pharma Inc.’s entrenched Tarceva (erlotinib). Furthermore,
AstraZeneca PLC’s EGFR inhibitor Iressa (gefitinib) was approved by FDA in June 2015,
paving the way for reintroduction into the
US market and global approvals.
Boehringer had suggested that afatinib might have advantages over others in the class based on the mechanism
of action, which is irreversible compared
to reversible competitors, but at the time
of approval, head-to-head data was not
available. It got a boost in January when
Boehringer announced that Gilotrif had a
modest efficacy advantage compared to
Iressa in the Phase II LUX-Lung 7 study in
non-squamous NSCLC.
Gilotrif received approval for secondline treatment of squamous NSCLC in
April, with a modest overall survival benefit compared to chemotherapy, but the
market has been shifting toward PD-1
inhibitors like Bristol-Myers Squibb Co.’s
Opdivo (nivolumab).
NEWCOMER’S PERFORMANCE
Boehringer has not publicly released sales
figures for afatinib, but Jörg Barth, corporate senior vice president and therapy
area head in oncology, commented in an
interview that there has been a nice trend
toward growth globally and the company
presumes based on strong data that this
will continue.
According to IMS Health Inc., the drug
had US sales of about $513,000 in the third
quarter of 2013, its first quarter on the market. It estimates US sales of $5.3m for 2013,
$73.6 m in 2014 and $114.3m in 2015,
with $33.5 m in the first quarter of 2016. In
the third quarter of 2015, its US re-launch
period, IMS Health data show Iressa had
$709,000 in US sales, with yearly sales of
$3.2m. In the first quarter of this year, Iressa
had sales of about $4.9m.
BI is a relative newcomer to the oncology space, and it will take some time beCONTINUED ON PAGE 6
BROUGHT TO YOU BY THE EDITORS OF PHARMASIA NEWS, START-UP AND SCRIP INTELLIGENCE
IN THIS ISSUE
Continuation of
MONARCH 2 study may
delay filing and tougher
competitive outlook
against Pfizer’s Ibrance
and Novartis’ ribociclib
13
from the managing editor
[email protected]
Cast your eyes over the Olympics medal table and
you will see something remarkable. Great Britain,
as a diplomat friend put it, is “doing quite well.”
For now, Team GB is in second place, ahead of
China. Amazing considering that in Atlanta ‘96 it
ranked 36th.
How so this swift rise up the ranks? The answer,
it seems, is cold hard cash and a dose of tough
love. Since the UK’s humiliation of the late 1990s,
National Lottery funding has poured in, but good
money is never thrown after bad. If your sport does
well, funding is increased. If not, it is cut. Cycling
and rowing have struck gold, but basketball lost
out and the team failed even to qualify for Rio.
It’s a brutal but effective system that works well
in drug development too. If it’s gold you’re after, invest hard in core areas and know when to cut your
losses. But in both cases there is a risk that rough
diamonds in unfashionable fields may get lost. How
to capture these assets is the next challenge.
Despite the
political turmoil,
Sanofi appears
keen to invest in
the manufacturing
of an insulin
product in Turkey
16
COVER / Boehringer’s Oncology Strategy:
Build From Niche Lung Cancer Base
3 Don’t Panic! Novartis Still Invested In CAR-T Despite
2Q Silence
4 Novartis Korea May Face Business Suspension
In Rebate Probe
5 Pfizer Sees Bright Long Term Biosimilar Future In China
7 Business Bulletin
8 Opdivo Fallout: Rivalry In PD-1 Market To Continue
10 Medivation Sees Potential For Combinations Out Of
Opdivo Failure
11 Allergan Sees Growth From New Launches,
Not Big M&A
12 R&D Bites
13 Lilly’s Abemaciclib Hit Leaves It Down But Not Out
14 Another Late-Stage Failure For AstraZeneca’s Selumetinib
14 CAR-T Update: Kite’s Filing Coming Soon
15 Valeant’s New Strategic Direction:
Clean Break Or Simply A Paint Job?
16 Pricing Concerns Delaying Sanofi’s Turkey Insulin
Investment?
17 Policy & Regulation Briefs
exclusive online content
Surprise! It’s A Phase III Failure
18 If Egalet Wins At FDA, Arymo ER Still Might Not
In Opioid Market
19 Stockwatch: Landscaping Earnings Announcements
At Valeant And Endo
When Phase III clinical trial failures happen it is a painful blow
– to the drug manufacturer, to investors and to patients. Scrip
takes a look at some of the biggest Phase III surprises since 2010.
http://bit.ly/2bt3bwc
20 Expert View: Wearables: A World Of Pharma Partnership
And Potential
Partnerships Required For Emerging Infectious
Diseases
23 Appointments
22 Pipeline Watch
Bringing drugs and vaccines for emerging infectious diseases
to the marketplace requires playing the long game and
collaborations if efforts are going to be successful.
http://bit.ly/2aQZAuj
2 | Scrip intelligence | 19 August 2016
@ s cri pn e w s
/s cri pi nte l l i genc e
/s cri pi nte l l i g en ce
/s cri pi nte l l i genc e
© Informa UK Ltd 2016
HEADLINE NEWS
Don’t Panic! Novartis Still Invested In CAR-T Despite
2Q Silence
Novartis AG’s CAR-T cell therapy CTL019, set to be the first in this class to achieve a US FDA filing, might not be an
immediate blockbuster owing to its tricky treatment logistics, small initial indication and a lack of physician and
patient exposure, but it is still a game changer in immuno-oncology, says Dr. Oz Azam, Novartis’s head of cell therapies.
LUCIE ELLIS [email protected]
N
ovartis AG’s cell therapy CTL019, a CAR-T (chimeric antigen
receptor T-cells) product, caused a stir last month when the
big pharma neglected to include the pipeline-leading therapy on a list of its upcoming potential blockbuster drugs during its
second-quarter earnings presentation. But Dr. Oz Azam, global head
of cell and gene therapies at Novartis, told Scrip this was not a big
deal and indeed there were a number of explanations for the medicine’s exclusion.
While Azam said he couldn’t comment directly on why Novartis’s
senior management team did not include CTL019 on a slide presented to investors last month called ‘11 potential blockbusters in
innovative medicines’; he noted the company had previously been
very “open about the fact we are filing this product next year and
that we see huge potential for it.”
He also highlighted that Novartis’“rich pipeline” has plenty of upcoming products to talk about across multiple therapy areas.
However, in a discussion with Scrip, Azam highlighted the challenges facing Novartis as it pursues its mission to be first to market
with a CAR-T therapy in the US.
UNKNOWN PATIENT POPULATION
Currently cells therapies are only being used for a modest number of
indications, with skin and blood stem cells representing the majority
of therapeutic uses – but the pipeline has increased exponentially in
recent years. According to Informa Pharma’s Biomedtracker there are
22 CAR-T therapy programs in the pipeline from 16 companies: nine
clinical-stage assets and 13 preclinical programs. There are also two
clinical programs being led by the National Institutes of Health.
Still, companies will remain uncertain on the potential reach of
their new therapies until more treatments make it to market, particularly in the oncology space where there are already a growing
number of therapeutic immuno-oncology options.
If Novartis is first to market with its CAR-T offering, Azam admitted the company was unsure on the extent of its initial uptake because it is an entirely new treatment. “To be very candid, we think
there is a sizable opportunity here but I don’t have values and numbers to share with you. We might be treating thousands of patients
or tens of thousands of patients. We just don’t know yet,” Azam said.
He added that physicians need more exposure for CTL019 or
other CAR-T therapies post-approval so they can decide how and
when best to use these options. Furthermore, Novartis is initially
targeting a third-line use indication for CTL019 in a discrete patient
population – pediatric acute lymphoblastic leukemia (ALL). So
judging the number of patients who will ultimately be eligible for
CTL019 therapy is tricky.
Azam isn’t worried about the future of Novartis CAR-T therapy
though: “With such a transformative and powerful therapy it’s okay
scripintelligence.com
to start with a distinct population first and demonstrate that you
have got that right,” he said. Novartis will look at the wider indications following CTL019’s introduction to the US market. “Cell
therapy is an important piece of the puzzle for Novartis’s overall
oncology strategy,” Azam said.
REGULATORY PATHWAY TO BE FORGED
Novartis will present its most recent clinical trial data for CTL019 at
the American Society of Hematology’s annual meeting in December
this year, following on from data presented last year by the company’s development partner on the CAR-T drug, the University of Pennsylvania. The pharma then plans to submit its data package to the
FDA in early 2017.
However, Azam said the company was aware that any CAR-T
therapy seeking approval will be scrutinized by US regulators because of the novel nature of the products and the risks they carry,
plus a few recent cases of clinical trial holds on CAR-T drugs due to
adverse events.
The FDA imposed a clinical hold on a trial forJuno Therapeutics
Inc.’s CAR-T therapy candidate JCAR015 in early July in response to
three deaths in the study. However, the hold was lifted less than
a week later. Juno speculated that the three cases of lethal cerebral edema observed in the Phase II ROCKET study were caused
by fludarabine, which was added to cyclophosphamide to prime
the immune systems of adults with relapsed or refractory B-cell
ALL. The FDA responded quickly to the company’s request to resume enrolling patients in ROCKET as long as the trial’s protocol
required pre-conditioning with a cyclophosphamide-only chemotherapy regimen.
“The FDA is going to be in a very detailed and data orientated
mood when assessing CTL019,” Azam said, “but it should be because this filing may well represent the introduction of a totally
new therapy.”
He said Novartis was preparing “the best package possible” for
CTL019 to reassure regulators it had a full data set, that the company had analyzed the benefit/risk profile of the treatment and that
it had all the fundamentals in place for a successful commercial
launch in 2017. “It’s not just a scientific and data analysis, the FDA
will want to know our treatment will be available once approved
and that the service and manufacturing is going to be done to a
high standard to meet their guidelines,” Azam said.
Novartis has invested in a centralized manufacturing site in Morris Plains, New Jersey, which it believes will fulfil its current needs
for providing CTL019 to patients. The Swiss pharma acquired the
Morris Plains site for $43m from Dendreon in 2012. It also has access to a manufacturing site in Europe. “Novartis has been doing a
CONTINUED ON PAGE 4
19 August 2016 | Scrip intelligence | 3
HEADLINE NEWS
Novartis Korea May Face Business
Suspension In Rebate Probe
Novartis’ South Korean subsidiary could face a business suspension and
other administrative measures after prosecutors indicted several employees
without detention on charges of illegal rebate payments.
JUNG WON SHIN [email protected]
S
outh Korean prosecutors have indicted six current and former employees
of Novartis Korea without detention
on charges relating to their alleged involvement in illegal “rebate” payments to
doctors in return for prescribing the company’s drugs.
According to prosecutors, the South
Korean subsidiary of Switzerland-based
Novartis AG allegedly paid a total of
KRW2.59bn ($2.4m) in rebates to doctors
through five pharmaceutical journals and
an academic journal publishing firm over
the period from January 2011 to around
January this year.
Prosecutors indicted a total of 34 people,
including the head of Novartis Korea and five
other current or former company officials,
without detention for their alleged participation in the criminal acts.
Prosecutors have also asked the Ministry of
Health and Welfare and Ministry of Food and
Drug Safety to take administrative measures
against Novartis Korea, including business
suspension, relevant drug price cuts, and
reimbursement suspensions. The ministries
have also been requested to suspend the licenses of the doctors suspected of receiving
rebates from the company.
Prosecutors didn’t reveal the specific drugs
involved in the alleged rebate payments.
THE ALLEGATIONS
South Korea has vowed to step up its fight
against illegal activities involving rebates (irregular payments in return for favors) said to
be rampant in the local pharma industry, as
the government aims to raise the industry’s
global competitiveness and nurture the
sector as an economic growth engine.
After the latest probe, prosecutors confirmed that multinational firms - which have
been stressing their ethical management
- “aren’t free from the temptation of rebate
payments.” They have also confirmed that
pharma journals are serving as “agencies” for
companies’ rebate payments and that doc-
4 | Scrip intelligence | 19 August 2016
tors in university and general hospitals have
been receiving rebates.
Novartis Korea is said to have indirectly
provided rebates to doctors by having the
journals and an academic publishing company invite a number of doctors to fine
restaurants including hotels to have them
discuss Novartis drugs, activities for which a
certain amount of money would be paid to
the doctors. The journals and publisher essentially served as agencies in exchange for
“advertisement” fees.
The multinational pharma firm also allegedly provided expenses to doctors to attend
overseas conferences as guest reporters for
the pharma journals, prosecutors said.
NOVARTIS RESPONSE
Meanwhile, Novartis Korea said it regrets
that some of its employees had violated
rules against corporate culture and social
expectations for the company and the pharma industry by holding small-scale medical
meetings through pharma journals.
In addition, it confirmed that some employees had provided support to some
medical professionals to attend overseas
conferences in a way that violated the Fair
Competition Code of the Korea Researchbased Pharma Industry Association (KRPIA),
which represents foreign pharma firms operating in the country.
But it did not agree that such acts had
been conducted with the approval of Novartis Korea’s management, said Novartis
Korea. “Novartis doesn’t tolerate illegal activities. Based on our internal probe, we have
been taking measures to improve this. We
will thoroughly review details of the indictment and consider coming up with followup measures,” it said.
Established in 1997, Novartis Korea is the
second-largest multinational pharma operating in South Korea, posting KRW455.3bn in
sales last year.
From the editors of PharmAsia News.
Published online 10 August 2016
CONTINUED FROM PAGE 3
lot of planning around this for the last few
years, it’s not something you do overnight,”
he said. “We have concrete and robust
plans to invest and expand our capacity as
patient populations mature.”
NEW COMMERCIAL MODEL
NEEDED
As well as planning for manufacturing infrastructures that will be required for the commercial launch of CTL019, Novartis has been
preparing a new commercial model for this
type of treatment. Unlike a small molecule or
biologic product, Azam highlighted that cell
therapy treatments were more like “services.”
As such, Novartis has been taking lessons
from its Alcon Inc. unit, particularly from its
device and medtech products, in order to
design its commercial model for CTL019.
“CTL019 is going to need a service type
of model, not a traditional pharma sales
model,” Azam noted. “In a device setting,
you hand-hold a physician through device
training more than you would for a new
drug product.” For example, Azam said that
practitioners would need to be able to provide a “cell therapy service” to patients. The
patients need to be managed throughout
the process of harvesting blood cells, those
cells being reengineered at a manufacturing site and then reintroduced. Doctors will
need to be aware and cautious of possible
side effects and patients will need follow-up
checks after treatment.
CLT019 is Novartis’s focus in the cell and
gene therapy space for the rest of 2016, but it
has two other pipeline programs: FCR001 for
use in renal transplants and HSC835 which
is a stem cell transplant program. Azam told
Scrip that the company had not ruled out
further investment in this unit. “Cell therapy
is one of the pillars of Novartis’s immuno-oncology strategy. The company is very active
in this space but its CAR-T component is the
most real and most mature right now.”
Still, as the field develops in size and by
the number of targets available, he said
Novartis was an agnostic company that
would look at broadly at opportunities.
“We have some amazing pipeline CAR-Ts
but this doesn’t preclude us from looking
at other cell and gene therapies in the future. If there is good science we will follow
it and that might lead to future business
development opportunities,” he said.
Published online 8 August 2016
© Informa UK Ltd 2016
HEADLINE NEWS
Pfizer Sees Bright Long Term Biosimilar Future In China
Despite shorter term general cost pressures and the time needed for approval reviews, Pfizer Inc. sees a strong future
for its biosimilar operations in China, as it recorded a solid second quarter performance for its traditional franchises
and unveiled a series of new health alliances in this key emerging market.
YING HUANG [email protected]
P
fizer Inc. is betting long in China,
where it sees significant market opportunities for its biosimilar business
in particular, a potential growth strategy
which the US giant highlighted in its recent
second quarter earnings call.
This key emerging market, where Pfizer
made its biggest capital investment in the
quarter, contributed double-digit growth
in both the cardiovascular and anti-infective franchises. “China continues to perform
well, even in the face of some anticipated
cost pressures,” John Young, group president for Pfizer Essential Health, noted in
the call. “We continue to see some strong
volume growth overall. Our business grew
around about 11% in the quarter.”
Pfizer remains “very positive” about the
opportunities for its business in China,
Young said, and overall its franchises are
growing strongly. “It [the business] is very
well-aligned with the priorities of the Chinese government, and we’re working very
closely with them to strengthen primary
care services and management for chronic
diseases, which we believe will do good
things for the Chinese healthcare system
and for patients, and obviously are very
well-aligned with our portfolio as well,”
Young noted.
China was also a key driver of broader
emerging markets performance. “In emerging markets, operational revenue growth
of $116m or 4% was driven by legacy Hospira Inc. operations and certain Essential
Health products, primarily in China,” Pfizer’s
chairman and chief executive officer Ian
Read noted in the call.
The company did not break out China
figures but noted that Legacy Hospira operations contributed $78m across emerging markets in the quarter.
NEW STRATEGIC ALLIANCES
As part of its effort to align with needs
and better help Chinese patients with
chronic diseases, Pfizer formed two strategic alliances with local partners in the
second quarter.
scripintelligence.com
In May, it signed a memorandum with
the China Cardiovascular Association to
form an in-depth and broad collaboration, including high-quality professional
competence demonstration exchange
programs for cardiology, primary chronic
disease management, a primary chest pain
center construction project, a cardiovascular health indicators project based on big
data management, and the establishment
of a volunteer service team.
Another strategic partnership was set up
with the National Center for Cardiovascular
Disease in June, which covers cardiovascular disease prevention policy research and
specification establishment, prevention
and control technology development, and
academic research in disease trends based
on big data.
Most recently, Pfizer and the Guizhou provincial government signed a major strategic
collaboration framework agreement for developing “Big Health” on July 8. The concept
of Big Health encompasses a wide range of
healthcare industries including pharmaceuticals, healthcare products, medical equipment and healthcare big data.
Through the alliance, Pfizer will support
the construction of an emergency network
system and the establishment of stroke
and chest pain centers, as well as exploring the application of big data to establish
disease management model.
COST PRESSURES?
The cost pressures Young alluded to may
include new investigations into pricing that
are apparently being planned by the regulatory authorities in China.
In May, the country’s state-owned newswire China Daily reported that the National
Development and Reform Commission,
China’s top medicines price regulator, may
require more drug companies to provide
information under soon to be launched
“large-scale and systematic” antitrust investigations into the pharma industry.
The planned move comes after Chinese
Premier Li Keqiang said during an ex-
ecutive meeting of the State Council that
healthcare reform had entered a crucial
stage, with a reduction in drug prices listed
among the key tasks going forward. Pfizer
and a number of distribution companies
and medical device manufacturers have
been summoned to provide data and information, the newswire said, citing sources close to the regulator.
BUILDING UP BIOSIMILARS
Biosimilars present a very attractive market
opportunity for Pfizer in China as it is now
the worldwide leader in the sector, the executives said. Last September, Pfizer completed the acquisition of Hospira, a leading
provider of injectable drugs and infusion
technologies with a strong global presence
in biosimilars.
However, “In China, whilst we see significant potential from the Hospira portfolio,
it will take time to realize that just given
by the time of regulatory approval,” Young
cautioned.
In June, Pfizer announced the investment of $350m to develop a global biotechnology center in the Hangzhou Economic Development Area in China. “This
innovative facility will ensure we develop
and manufacture the high-quality, affordable biosimilar medicines that have the
potential to benefit patients both in China
and throughout the world,” Read said.
Even though the penetration of biologic medicines generally into China is
low compared with most benchmarks internationally, Pfizer believes that post the
expiry of any relevant patents in China,
there will be significant opportunities, according to Young.
The Hangzhou center is expected to be
completed in 2018, and the company will
work with the China FDA and relevant authorities to bring biosimilar molecules and
other products coming out of that facility
to the Chinese market as soon as possible
after that, he added.
From the editors of PharmAsia News.
Published online 9 August 2016
19 August 2016 | Scrip intelligence | 5
HEADLINE NEWS
Datamonitor
Healthcare had been
projecting $590m in
worldwide sales for
Gilotrif in 2016
But lung cancer specialist Howard Jack
West commented that use of afatinib after erlotinib in non-squamous NSCLC is “a
testament to the tenacity and ignorance
of physicians and patients over actual
evidence.” West is medical director of the
thoracic oncology and genitourinary oncology programs at the Swedish Cancer
Institute in Seattle. “There is no compelling evidence to suggest this is a remotely
good idea, and I strongly suspect that a
trial comparing second-line afatinib to
chemo in patients with acquired resistance
to erlotinib would be far more favorable for
chemotherapy,” he said in an interview.
6 | Scrip intelligence | 19 August 2016
As for afatinib’s approved indications,
West notes that the head-to-head data
against Iressa in the LUX-Lung 7 study was
not widely publicized and, in his opinion,
awareness is low, particularly among community oncologists.
In late July, the company announced
some setbacks in its plans to expand Gilotrif into head and neck cancer. It stopped
the LUX-Head & Neck 2 trial in patients
with locally advanced head and neck cancer who have no evidence of disease after
treatment with chemotherapy and radio-
in July 2015 for global markets except for
China, Hong Kong and South Korea, where
the drug is already approved based on
Phase I/II data from the HM-EMSI-101 study.
In conjunction with the American Society of Clinical Oncology annual meeting
in June, the company unveiled a comprehensive pivotal development program for
olmutinib called ELUXA, including monotherapy and combination studies.
Regulatory filings could start with ELUXA
1, a Phase II trial in EGFR-positive lung cancer with T790 mutations in patients already
therapy, as well as the LUX-Head & Neck
4 trial in Asia, based on the advice of an
independent data monitoring committee,
which determined during a pre-planned
interim analysis that it was “highly unlikely”
the LUX-Head & Neck 2 trial would show
an efficacy advantage for afatinib. The LUXHead & Neck 3 trial in Asian patients with
recurrent and/or metastatic head and neck
cancer will continue as planned, consistent
with the DMC’s recommendation.
Datamonitor Healthcare had been projecting $590m in worldwide sales for Gilotrif/Giotrif in 2016, prior to the head &
neck failure, rising to almost $1bn in 2023,
including new indications.
treated with an EGFR inhibitor; the trial has
completed enrollment. The other studies,
which will kick off this year, include headto head trials against Gilotrif.
Shutterstock: Lightspring
CONTINUED FROM COVER
fore they really see sustained forward momentum there, Datamonitor Healthcare
analyst Justin Burns told Scrip. “The next
few years will be especially critical for them
as their pipeline moves from early-to-mid
to a more mid-to-late stage of candidate
development,” Burns noted.
Based on a March 2016 survey, Kantar
Health’s CancerMPact Treatment system
suggests that Gilotrif was the first choice
for use in second-line non-squamous
NSCLC patients with EGFR mutations, but
not T790m mutations (not an approved indication), and had a 15% share of the firstline EGFR mutant population.
Stephanie Hawthorne, vice president of
clinical and scientific assessment at Kantar
Health, notes that a subset analysis of the
LUX-Lung-3 trial that supported approval
showed that Gilotrif was able to confer
an overall survival benefit in patients with
a specific EGFR mutation (exon 19 deletion, or del19); this is the only randomized
data for an EGFR inhibitor showing an OS
benefit compared to chemotherapy in this
treatment setting and patient population.
“It is possible that the utilization that Gilotrif has gained in first-line EGFR mutants
represents select use in some del19 patients,” Hawthorne told Scrip.
FULL STEAM AHEAD ON
OLMUTINIB
Boehringer is now very focused on the
late-stage development of olmutinib (BI
1482694), a third-generation EGFR inhibitor.
The company licensed rights to the drug
from Hanmi, where it was named HM61713,
HOW TO DIFFERENTIATE?
But as with its second-generation Gilotrif,
Boehringer is once again playing followthe-leader. AstraZeneca PLC’s Tagrisso
(osimertinib) nabbed the first approval for
EGFR-positive patients with T790 mutations.
Asked how olmutinib will be differentiated from Tagrisso and other third-generation EGFR inhibitors, Boehringer’s Barth
commented: “We are still on the learning
curve there, at the moment it looks similar
[to] Tagrisso but we think there are opportunities over time.”
Boehringer notes that it wasn’t initially
apparent that Gilotrif would show a survival
advantage in EGFR patients with del19 mutations, the specific mutation that helped it
differentiate from the other EGFR TKIs.
Published online 10 August 2016
© Informa UK Ltd 2016
BUSINESS BULLETIN
Valeant’s Dermatology Sales
Strategy: Teenage Zits
Valeant Pharmaceutical International
Inc.’s dermatology business is in trouble. But CEO Joseph Papa said the company is counting on teenagers eager
to have clear skin when they return to
school in the fall to boost prescriptions
of the firm’s acne medicine Solodyn (minocycline). “As many of the teenagers go
back to school, that’s an important time
period for acne and other parts of the
dermatology franchise,” Valeant CEO
Joseph Papa told investors and analysts
during the company’s Aug. 9 secondquarter earnings conference call. Sales
of Valeant’s Solodyn, a tetracycline-class
drug indicated to treat inflammatory lesions of non-nodular moderate to severe
acne vulgaris in patients 12 years or older,
plummeted 74% in the second quarter,
compared to a year earlier. Papa noted
Valeant has instituted a couponing program for independent pharmacies for the
company’s dermatology products, which
the firm is hoping will help push sales of
its medicines, including Solodyn.
[email protected], 10 August 2016
Chi-Med Approaches
Inflection Points
Hutchison China MediTech Ltd., better known as Chi-Med, is uniquely positioned as a global pharma R&D company
based in China, supported by a profitable
domestic prescription and OTC drugs
business, with listings on both London’s
AIM market and Nasdaq. Led for 16 years
by British CEO Christian Hogg, the firm
counts AstraZeneca PLC and Eli Lilly &
Co. as partners and has global ambitions
for its R&D pipeline, built around selective kinase inhibitors for cancer and inflammatory indications. Some of those
ambitions are approaching key inflection
points, with major data publication catalysts expected for four of its candidates
by the end of the first quarter of 2017. If
all goes according to plan, the firm could
be filing its lead product fruquintinib for
approval in China, and moving AZ-partnered savolitinib as well as unpartnered
scripintelligence.com
BMS Reacquisition Bulks Up ASLAN’s
Deal Chest
Bristol-Myers Squibb Co. is paying Aslan Pharmaceuticals Pte. Ltd. $10m
upfront to reacquire at an early stage the Asian rights to a first-in-class oncology molecule it originally licensed to the Singapore-based oncology venture back in 2011. ASLAN will also be eligible for development and regulatory milestones “in excess of” a further $50m, plus royalties on BMS’ future
worldwide sales of the drug, ASLAN002 (also known as BMS777607), while
the US major will also assume all development and commercialization costs.
The new funds will add to the $34m raised through ASLAN’s recent Series C
funding, which took the venture’s total to $69m, and provide new capacity
for planned licensing deals to bolster ASLAN’s remaining pipeline assets.
“We absolutely intend to pursue new agreements and in fact have already
been in discussions,” ASLAN CEO Dr. Carl Firth revealed to Scrip. ASLAN
originally acquired rights to BMS777607 in China, South Korea, Taiwan,
Australia and other selected Asian countries in November 2011 for undisclosed financial terms, with BMS holding on to the drug in the rest of the
world as part of the deal. The small molecule acts as an inhibitor of RON and
related cMET receptor tyrosine kinases and is currently in Phase II at ASLAN
for second-line use in gastric and breast cancer. While these programs have
focused on monotherapy use, potential is also seen for combination use with
anti-PD1 and other immune checkpoint inhibitors.
[email protected], 10 August 2016
epitinib into pivotal studies, all by the
middle of next year. The recent amendment to the firm’s co-development deal
with AstraZeneca for its c-Met inhibitor
savolitinib is indicative of Chi-Med’s desire to step up its investment. It is paying
an extra $50m over three years to help
fund a Phase III study in c-Met-driven
papillary renal cell carcinoma that is expected to start soon, and will get a five
percentage point increase in the global
(non-China) tiered royalty rate payable
on savolitinib sales for all indications,
bringing it up to 14-18%.
[email protected], 8 August 2016
Diabetes Delivers China
Strength For Merck
China continued to provide robust double-digit growth in the second quarter
for Merck & Co. Inc., and in spite of increased generic and brand name competition for its best sellers including Januvia
(sitagliptin) for diabetes, the US firm saw
key brands keep growing across emerg-
ing markets. In the primary care Januvia
franchise, strong double-digit growth in
emerging markets and China is going to
continue over time, Adam Schechter, executive vice president and president for
Global Human Health, predicted during
an earnings call. “We really have not gotten National Reimbursement Drug List
[NRDL] approval for Januvia in China
yet. So once that’s achieved, I think that
that represents another opportunity for
us for growth of the Januvia franchise
outside of the US,” he noted. Inclusion
in the list would provide wider reimbursed access through China’s national
health insurance schemes for Januvia,
which was launched in China in March
2010. In China, the US firm is taking various steps to improve disease awareness
and the availability of modern therapies
in the diabetes sector. It held the Sixth
Incretin Forum in Shenzhen in March,
gathering experts to discuss China’s diabetes market and address precise and localized treatment solutions for Chinese
patients.
[email protected], 10 August 2016
19 August 2016 | Scrip intelligence | 7
HEADLINE NEWS
Opdivo Fallout: Rivalry In PD-1 Market To Continue
Opdivo’s Phase III disappointment in first-line non-small cell lung cancer (NSCLC) is not the end of the world for BristolMyers Squibb, according to Datamonitor Healthcare analyst Dustin Phan. It also may not be the last surprise in a field
with so many contenders and so many ongoing trials, as data from Pharmaprojects shows.
LUCIE ELLIS [email protected]
B
8 | Scrip intelligence | 19 August 2016
2
Keytruda
1
10
8
1
1
21
4
Opdivo
1
8
15
2
1
27
1
1
Tecentriq
ristol-Myers Squibb Co. shocked the market late last week
when it announced a Phase III trial exploring its market leading PD-1 inhibitor Opdivo (nivolumab) as a first-line treatment
for NSCLC had missed its primary endpoint. However, Datamonitor
Healthcare analyst Dustin Phan told Scrip Opdivo will still pose a barrier to market entry for many late-phase pipeline therapies because
of its strong uptake as a second-line treatment.
Topline data from the pivotal Phase III CheckMate 026 trial revealed that Opdivo failed to meet its primary endpoint of progression-free survival (PFS) in previously untreated NSCLC. Patients in
this study were randomized to either Opdivo monotherapy or a
physician’s choice platinum-based chemotherapy regimen. All
patients were screened for PD-L1 expression, which remains a
biomarker with debatable utility. BMS, which has approval for allcomers in second-line NSCLC, used a threshold of just ≥5% for the
primary analysis.
Opdivo’s low cutoff for PD-L1 expression is likely the most significant contributor to the drug’s negative Phase III efficacy data in the
first-line setting, Phan noted in an analysis for Datamonitor Healthcare. There are additional cut-points for PD-L1 levels in CheckMate
026, presumed to be 10%, 20% and 50%, which could easily show
efficacy. Merck & Co. Inc.’s first-line NSCLC story for its PD-1 inhibitor
Keytruda, KEYNOTE-024, showed PFS and overall survival using a
50% threshold for PD-L1 expression, and the drugs are widely regarded as similar.
The high regard for BMS’ drug should help it “remain dominant,”
Phan noted. The analyst pointed out that Opdivo’s “blockbuster
status across the entire oncology treatment space gives it physician comfort and familiarity that will be difficult to overcome.” BMS
reported Opdivo held an 80% share of the PD-1 market and was
the firm’s top-seller during the second quarter, with $840m in sales.
Opdivo has already secured more approvals than its two major
rivals, Merck’s Keytruda (pembrolizumab) and Roche’s PD-L1 inhibitor Tecentriq (atezolizumab), and Opdivo has more active trials ongoing than the two other PD-1/L1 therapies (see chart). According
to the Pharmaprojects database there are 26 clinical trial programs
ongoing for Opdivo, as well as one preclinical study. Meanwhile,
Merck has 20 ongoing clinical programs for Keytruda and one preclinical study and Roche is exploring Tecentriq in 16 clinical and
two preclinical programs.
With so many competitive studies ongoing in the PD-1/L1 space,
it is not unlikely that some produce surprising results – much like
Opdivo’s miss in first-line NSCLC – despite the commercial success
of these products in other areas or different settings in the same
indication. While full data from the CheckMate 026 study have not
yet been presented, initial thoughts have put the missed endpoint
down to trial design and not lack of response with the drug. The
full information could help improve the design of future clinical
studies for PD-1/L1 products.
6
3
6
2
18
0
2
4
6
8
10
ApprovedIndications
PhaseI
12
14
16
Pre-Registration
Preclinical
18
20
22
PhaseIII
24
26
PhaseII
Total
Source:CitelinePharmaprojects
Pharmaprojects
Meanwhile, Phan highlighted that Opdivo’s Phase III failure will
likely only delay and not prevent the drug’s entry into the first-line
NSCLC treatment space, as the ongoing CheckMate 227 trial will
also investigate Opdivo plus BMS’s own Yervoy (ipilimumab) as well
as Opdivo plus chemotherapy in the first-line setting. “In addition,
patients in this study will undergo immunohistochemical testing
for PD-L1 expression, so we can expect subgroup analysis that will
provide insight into the efficacy of these regimens across PD-L1
expression levels,” he said.
Still, combinations featuring Opdivo will have to demonstrate
efficacy in both PD-L1-positive and -negative patients, or demonstrate considerably larger survival improvements in PD-L1-positive
patients than PD-1 inhibitor monotherapy, if Opdivo is to overcome Keytruda’s first-to-market advantage. Keytruda could achieve
a first-line label expansion in 2017.
© Informa UK Ltd 2016
HEADLINE NEWS
Ongoing Trials For Approved PD-1/L1 Drugs By Indication
bladder
Phase III
DRUG NAME & LEAD COMPANY
prostate
Phase II
KEYTRUDA (PEMBROLIZUMAB) MERCK & CO.
peritoneal
Phase II
DISEASE
DISEASE STATUS
fallopian tube
Phase II
melanoma
launched
ovarian
Phase II
NSCLC
launched
pancreatic
Phase II
head and neck
pre-registration
solid, unspecified
Phase II
renal
Phase III
breast
Phase II
bladder
Phase III
lymphoma, non-Hodgkin’s
Phase II
urethral
Phase III
colorectal
Phase II
gastrointestinal, stomach
Phase III
lymphoma, B-cell
Phase II
esophageal
Phase III
leukemia, chronic myelogenous
Phase II
breast
Phase III
mesothelioma
Phase II
myeloma
Phase III
cervical
Phase II
colorectal
Phase III
endometrial
Phase II
lymphoma, Hodgkin’s
Phase III
sarcoma, soft tissue
Phase II
liver
Phase III
myeloma
Phase I
endometrial
Phase II
Infection, hepatitis-C virus
Phase I
lymphoma, B-cell
Phase II
leukemia, chronic lymphocytic
Preclinical
lymphoma, unspecified
Phase II
TECENTRIQ (ATEZOLIZUMAB) ROCHE
solid, unspecified
Phase II
DISEASE
DISEASE STATUS
ovarian
Phase II
bladder
launched
peritoneal
Phase II
NSCLC
pre-registration
fallopian tube
Phase II
renal
Phase III
prostate
Phase II
breast
Phase III
pancreatic
Phase I
urethral
Phase III
leukemia, chronic lymphocytic
Preclinical
head and neck
Phase III
OPDIVO (NIVOLUMAB) BRISTOL-MYERS SQUIBB
colorectal
Phase III
DISEASE
DISEASE STATUS
lung, small cell
Phase III
melanoma
launched
solid, unspecified
Phase II
NSCLC
launched
sarcoma, synovial
Phase II
renal
launched
sarcoma, lipo
Phase II
lymphoma, Hodgkin’s
launched
melanoma
Phase I
head and neck
pre-registration
gastrointestinal, stomach
Phase I
gastrointestinal, stomach
Phase III
lymphoma, B-cell
Phase I
esophageal
Phase III
lymphoma, non-Hodgkin’s
Phase I
brain
Phase III
myeloma
Phase I
lung, small cell
Phase III
myelodysplastic syndrome
Phase I
liver
Phase III
ovarian
preclinical
squamous cell
Phase III
prostate
preclinical
urethral
Phase III
Opdivo, Keytruda and Tecentriq will continue to battle for market share for the foreseeable future as all three drugs are targeting the same cancer indications. Just looking
at ongoing Phase III studies there is a lot of
crossover on the indications being targeted
scripintelligence.com
Source: Citeline Pharmaprojects
by BMS, Merck and Roche, including for bladder, stomach, esophageal, urethral, breast,
colorectal and liver cancers (see table).
However, Merck is the most advanced
company with a PD-1 drug targeting myeloma, as it currently has a Phase III program
running for this indication. BMS and Roche’s
drugs are only in Phase I studies for myeloma.
In total, Pharmaprojects lists 63 pipeline
PD-1, PD-L1 or PD-L2 drugs, from preclinical to Phase III.
Published online 11 August 2016
19 August 2016 | Scrip intelligence | 9
HEADLINE NEWS
Medivation Sees Potential For Combinations Out Of
Opdivo Failure
EMILY HAYES [email protected]
Failure of Bristol’s PD-1 inhibitor Opdivo in first-line lung cancer
may open doors for combination use with drugs with immuneactivating properties, like prostate cancer drug Xtandi and PARP
inhibitor talazoparib.
T
he disappointing results for Bristol-Myers Squibb Co.’s Checkmate 026 trial are more of a setback for Opdivo than a failure,
but with the expectation that the PD-1 inhibitor will need to
be used in combination or in patients with higher levels of PD-L1
expression – other companies are seeing opportunity.
Medivation Inc. is among those, highlighting the potential for its
blockbuster prostate cancer drug Xtandi and PARP inhibitor talazoparib to play a complementary role to immunotherapy during the company’s second-quarter earnings call.
Bristol reported on Aug. 5 that Opdivo (nivolumab) monotherapy
missed the progression-free survival endpoint in the first-line CheckMate 026 study, looking at patients with at least 5% expression of
PD-L1. Merck & Co. Inc. had success in this indication with its competing Keytruda (pembrolizumab), but enrolled patients with 50%
or greater expression.
‘PARP inhibition in NSCLC is a less
crowded space and we intend to pursue
this opportunity’
Even before this failure, the limitations of PD-1 inhibitors have
been widely acknowledged. While PD-1 inhibitors have dramatically
changed the oncology treatment landscape, typically only 20%-30%
get a response. The CheckMate 026 failure further underscored the
need to develop other kinds of immunotherapies to improve results.
The gaps may open doors for development of Xtandi and talazoparib, both of which could have effects that complement immunotherapy, Medivation suggested during its Aug. 9 call.
Medivation reported $330m in US sales for Xtandi for the second
quarter. Including sales reported by Astellas Pharma Inc., the drug had
a total of $595m for this period. Medivation noted that the drug is on
track to be the sixth-best-selling oncology drug in 2016, with $2.8bn
in projected sales.
The company could see expanded labeling for Xtandi, which is approved in metastatic castration-resistant prostate cancer, in late October. The supplementary filings are based on the Phase II TERRAIN and
STRIVE studies, which tested the drug head to head against bicalutamide. The STRIVE study included non-metastatic as well as metastatic prostate cancer patients.
An expansion could be a needed boost. In an Aug. 10 note, Leerink Swann analyst Jeffrey Porges noted the flat, sequential growth for
Xtandi in recent quarters. “This quarter marks the third consecutive
10 | Scrip intelligence | 19 August 2016
quarter of 3%-4% organic volume growth for Xtandi in the US, and we
do not see any near-term catalysts that could materially increase this
steady, low-single digit demand growth rate before the potential label
expansion in Q4,” he noted.
Meanwhile, hopes are high for talazoparib, which is in a Phase III
study of genetically targeted breast cancer. It is also in development for
prostate cancer, small cell lung cancer and ovarian cancer. The company is starting five trials in four indications this year and will pursue two
more large indications – glioblastoma and non-small cell lung cancer
– next year, CEO David Hung told the call. “We’re investing pretty thoroughly in this asset,” the exec added. The poly ADP ribose polymerase
(PARP) inhibitor class got a boost with stellar results for Tesaro Inc.’s niraparib in June. Medivation is positioning talazoparib as a drug that is
much more potent at trapping PARP than niraparib.
IMMUNO-OPPORTUNITIES
During the earnings call, Medivation execs played up the immuneactivating potential of Xtandi and agreed with analyst suggestions
that the failure of Bristol’s CheckMate 026 study could create new
opportunities for drug development.
“Clearly the ability to up-regulate PD-L1 is an important feature
about this drug. And it could make it really synergistic with [a] PD-1
agent,” Hung said. “We think it would be very interesting to study this in
other tumors where PD-1 may have less than stellar results. I think that
certainly does open a door.”
Prostate cancer is one of the tumor types that has been left untouched by the revolution of PD-1 inhibitors. In the past, PD-1 inhibitors have not been associated with response in prostate cancer.
Jennifer Bishop, writing in the journalOncotarget in January 2015,
reported significant up-regulation of PD-L1 expression after enzalutamide treatment, the company noted during the call.
More recently, Julie Graff of the Oregon Health & Science University and colleagues reported promising initial data from a Phase
II study of Xtandi with Merck’s Keytruda in metastatic castrationresistant prostate cancer in Oncotarget on July 12. In the first 10
patients enrolled, three (30%) had a response. A patient with liver
metastases was one of the responders and these patients are usually very difficult to treat.
Medivation also believes that there is potential for talazoparib in
combination with immunotherapy, particularly in NSCLC. PARP inhibitors inhibit DNA damage response, which is thought to be complementary to checkpoint inhibition. AstraZeneca PLC has the only
marketed PARP inhibitor – Lynparza (olaparib) – and is studying it in
combination with its investigational PD-L1 inhibitor durvalumab on
the theory it could prime patients for response.
“We do think that PARP inhibition would be very exciting in nonsmall cell lung. And it is one of the indications that we’re targeting. We
do think that that space is a little bit less crowded. And we do intend to
pursue that opportunity,” Hung said.
Published online 11 August 2016
© Informa UK Ltd 2016
HEADLINE NEWS
Allergan Sees Growth From New Launches,
Not Big M&A
MANDY JACKSON [email protected]
A
llergan PLC has $27.6m in cash after closing the $40.5bn sale of its
generics business to Teva Pharmaceutical Industries Ltd. and paying down
$9.3bn debt, but the company will rely on
blockbuster sales gains and recent launches
for revenue and earnings growth, not a big
acquisition.
Allergan PLC has $27.6m in cash after
closing the $40.5bn sale of its generics
business to Teva Pharmaceutical Industries
Ltd. and paying down $9.3bn debt, but the
company will rely on blockbuster sales gains
and recent launches for revenue and earnings growth, not a big acquisition.
CEO Brent Saunders stuck with his first
quarter earnings message during Allergan’s
second quarter conference call on Aug.
8, repeating the drug maker’s plan to buy
companies or license products that represent stepping stones on the path to sales
growth, rather than pursue large, transformational mergers and acquisitions. The road
to rising revenue also will be paved with
double-digit growth from Allergan’s key
franchises, including Botox (onabotulinumtoxinA) and Restasis (cyclosporine), as well
as new products across the company’s core
therapeutic areas.
Leerink analyst Jason Gerberry pointed
out in an Aug. 8 research note that “management’s commentary on the call suggested
the company is focusing on its existing business and possible tuck-in deals rather than
a larger transformational deal, effectively
dismissing media speculation that Allergan
may be interested in acquiring or merging
with Biogen Inc.”
After selling its generic medicines to Teva,
which completes Allergan’s transition to a
branded product company, Saunders said
during the second quarter call that “we are
well-positioned to continue to deliver strong
results, powered by our ‘Growth Pharma’
model. This model is built around five key
elements: top-line growth, with the goal of
double-digit growth; category leadership
in each of our seven therapeutic areas; customer intimacy; ‘Open Science’ R&D to fuel
innovation; and operational excellence.”
scripintelligence.com
Allergan reported $3.7bn in brandname product revenue for the second
quarter, which was a 2% gain versus the
same period in 2015, or a 9% gain excluding three big impacts: divested assets,
foreign currency exchange rates, and
sharply declining sales for Namenda (memantine) immediate-release (IR), which
is facing generic competition. Non-GAAP
diluted earnings per share (EPS) dropped
over the same time frame by 9% to $3.35.
Revenue fell short of analyst consensus
of $4bn in sales, but beat EPS expectations of $3.33.
Sales for the wrinkle-reducer and migraine headache-reliever Botox rose 14%
year-over-year to $719.7m in the second
quarter, while sales for the dry eye drug
Restasis grew 20.2% to $390.6. However,
sales for the former Alzheimer’s disease
blockbuster Namenda IR plunged 98.2%
to $4.1m while Namenda extended-release
(XR) sales fell 18.7% to $166.5m and revenue
from Namzaric, a combination of Namenda
XR and the Pfizer Inc. drug Aricept (donepezil), totaled $12.8m.
In addition to buying back $5bn to
$10bn in stock from its shareholders and
paying down some of its $33.3bn in debt,
Allergan will use the proceeds from the
Teva deal and cash flow from product sales
to boost sales top-selling assets, support
recently launched products, and invest in
growth through research and development as well as acquisitions – just not big
M&A deals.
And the company’s $27.6bn cash balance is set to grow soon, since Allergan
also recently agreed to sell its drug distribution business known as Anda Inc. to Teva
for $500m.
COMMERCIAL CHIEF
OPTIMISTIC ABOUT LAUNCHES
Chief commercial officer William Meury
said Allergan was happy with sales to date
from recent launches, but noted investments that are being made to help those
products grow and to protect established
assets. The number of prescriptions and
prescribers for Viberzi (eluxadoline), which
is approved to treat irritable bowel syndrome with diarrhea (IBS-D), “are climbing at a high rate consistently week-overweek,” Meury said. Sales growth for Viberzi
is similar to the growth rate for Linzess (linaclotide), which was approved in 2012 to
treat IBS with constipation (IBS-C) and is
marketed in partnership with Ironwood
Pharmaceuticals Inc., despite the fact that
Viberzi costs almost three times as much
as Linzess.
Linzess sales increased 37% year-overyear to $155.1m in the second quarter, while
Viberzi sales totaled $20.4m for the April-toJune period.
Saunders mentioned the difference
between Viberzi and Linzess pricing in response to a question about payer pressures
on drug costs during Allergan’s second
quarter conference call. The CEO noted
that payers are willing to negotiate a favorable price for novel, differentiated therapies,
but pricing discussions are more difficult in
crowded markets.
“If there are four drugs launching with
relatively similar mechanisms and indications over a 12-month period, pricing dynamics are more challenging than they
have been. If you’re launching something
like Viberzi, where we can get a price that’s
almost three times the price of Linzess, because it’s a one-of-kind and novel mechanism, [discussions are] pretty straightforward,” he said.
Payer negotiations are not an issue
for Kybella (deoxycholic acid), however,
which is approved to reduce the appearance of a double chin. Like Allergan’s Botox Cosmetic product, Kybella is sold by
plastic surgeons and dermatologists to
cash-paying customers. Net revenue for
the chin-thinning drug totaled $12.7m in
the second quarter.
Published online 9 August 2016
CLICK
To read more online:
http://bit.ly/2aUjJed
19 August 2016 | Scrip intelligence | 11
R&D BITES
Oncolytics Biotech reported less than
impressive Phase II NSCLC data for
its cancer drug Reolysin (pelareorep)
last week – and despite the company’s
best attempt to find statistical significant data in subgroup analyses, analysts are not convinced. The Canadian
firm reported on Aug. 10 data split
by gender from the Phase II IND211
study, which enrolled patients with
both non-squamous (adenocarcinoma) and squamous cell histology.
Progression-free survival (PFS) was
significantly better for female patients
in the test arm of the IND211 study
(n=20) than for those in the control
arm (n=16); median PFS was 5.39
months compared with 3.02 months,
respectively (p=0.0201). The company also promoted a “strong evolving
trend” towards overall survival benefit
for female patients in the test arm.
However, no median PFS or p-value
was provided by the company for the
overall patient population, leaving analysts to contemplate how successful
this study actually was. Datamonitor
Healthcare analyst Dustin Phan noted
that the company’s decision to report
statistical analysis across gender lines
to achieve statistical significance was
“rather odd,” suggesting as it does
that “there may not have been an improvement in progression free survival in the overall (male and female)
population.”
[email protected], 11 August 2016
Advaxis Advances
Personalized Cancer Vaccines
The struggle for personalized cancer
therapies – and the downfall for one
high-profile company – has been costly,
complicated and time-consuming manufacturing, but one of the potential
advantages of Advaxis Inc.’s vaccines
targeting patient-specific neoepitopes
is a simplified manufacturing process.
The Princeton, New Jersey-based company recently entered into a collabo12 | Scrip intelligence | 19 August 2016
Spark Therapeutics Now Targets 2017
For First Retinal Gene Therapy Approval
Spark Therapeutics Inc. has announced a slight delay to completing the US
submission of what is expected to the first BLA involving a gene therapy for
a genetic disease, from the end of 2016 to early in 2017. The company has
also released clinical data on a further nine patients treated with its potential gene therapy, voretigene neparvovec (SPK-RPE65) for inherited retinal
disease (IRD) caused by mutations in the RPE65 gene, a rare blinding condition, and preliminary data from a Phase I/II study of a potential gene
therapy, SPK-9001 for hemophilia B. Voretigene neparvovec is not going to
be the first gene therapy to be developed anywhere, as uniQure NV gained
an EU approval for the first gene therapy in the Western world, Glybera (alipogene tiparvovec), back in 2012 for the treatment of lipoprotein lipase
deficiency. More recently, GlaxoSmithKline PLC gained approval in Europe
in May 2016 for Strimvelis, an ex vivo autologous hematopoietic stem cell
gene therapy to treat severe combined immunodeficiency due to adenosine
deaminase deficiency. Nonetheless, Philadelphia-based Spark has US breakthrough therapy designation for both voretigene neparvovec and SPK-9001
from the FDA, and a deep-pocketed big pharma, Pfizer Inc., as a partner for
its hemophilia gene therapy program. Approval of Spark’s groundbreaking
ophthalmic therapy could only be a matter of months away.
[email protected], 11 August 2016
ration with Amgen Inc. that’s worth
$65m up front and up to $475m in
milestone fees to develop and commercialize ADXS-NEO, an immunotherapy
that’s designed to target the mutations
or neoepitopes specific to an individual
patient’s tumor cells. The treatments
uses live attenuated Listeria monocytogenes (Lm) that are bioengineered
to produce and deliver tumor antigens
to antigen-presenting cells in order to
provoke a T-cell response.
[email protected],
11 August 2016
Glucokinase Activation Gets
Boost With vTv Phase II Data
Shutterstock: Pressmaster
Oncolytics’ Subgroup Data
Unlikely To Save Reolysin
will be developed and manufactured
using the same technology underlying
Advaxis’s off-the-shelf cancer vaccines.
Advaxis CEO Daniel O’Connor told
Scrip the company expects to get from
tissue sample testing to a two-year supply of vaccine treatments within six
weeks for each patient. Advaxis has
three vaccines in the clinic in multiple
indications, all of which are based on
the company’s Lm Technology, which
Initial data from the Phase IIb AGATA
trial reveal that vTv Therapeutics Inc.’s
selective glucokinase activator TTP399
was well tolerated and produced a significant change from baseline in HbA1c levels at six months, the primary endpoint,
at the higher dose tested. President and
CEO Steve Holcombe declared that the
company was “extremely pleased” with
the findings and enthusiastic about advancing the product into the next stage
of development. However, that will await
a partner, although a number of possible
partners are in the offing.
[email protected],
11 August 2016
© Informa UK Ltd 2016
HEADLINE NEWS
Lilly’s Abemaciclib Hit Leaves It Down But Not Out
EMILY HAYES [email protected]
for the second quarter and is now Pfizer’s top-selling cancer drug.
A filing for ribiciclib, which was recently awarded breakthrough
therapy designation, is expected by the end of this year.
Lilly has been positioning abemaciclib as being best-in-class and
has suggested the drug has an advantage when it comes to dosing
over Ibrance because it has a milder safety profile and therefore
does not require dose interruptions or reductions.
Continuation of Phase III MONARCH 2 study, with no early termination for efficacy, may mean a delay in filing and a tougher
competitive outlook against Pfizer Inc.’s Ibrance and Novartis
AG’s ribociclib.
N
Shutterstock: sergign
ot only does Eli Lilly & Co.’s announcement that its breast
cancer drug abemaciclib did not show enough efficacy to
terminate the pivotal MONARCH 2 study early throw its filing
plans into doubt, it may also dash the commercial prospects for the
cyclin-dependent kinase (CDK)4/6 inhibitor, which now seems destined to be third to market.
Hopes for an early filing of abemaciclib were dashed on Aug. 10,
when Lilly announced that the MONARCH 2 study, which included
669 patients, would continue after a preplanned interim analysis
by a data monitoring committee. Final results are due in the first
half of 2017.
The company said that it “will await further data and continue to
work with the FDA to inform its submission plan for single-agent
abemaciclib,” based on the single-arm, Phase II MONARCH 1 study.
During its second-quarter earnings call, Lilly had affirmed plans
to file abemaciclib with FDA in the third quarter using the MONARCH 1 study of the drug as a single agent, following an interim
read out from MONARCH 2, which tests the drug in combination
with the aromatase inhibitor fulvestrant in women with refractory
hormone-receptor positive, HER2-negative locally advanced or
metastatic breast cancer.
While abemaciclib does have breakthrough therapy designation with the FDA and the agency has accepted single arm data for
oncology accelerated approvals, especially when a Phase III trial is
ongoing, the application would have been much stronger with a
positive interim analysis from MONARCH 2.
Lilly’s stock price was trading down by 1.34% to $80.43 on the
news and closed at $80.55 (down 1.17%) on Aug. 10.
Investors had expected that the MONARCH 2 study would be terminated early for efficacy at the time of the interim analysis. After all,
Pfizer Inc.’s Phase III PALOMA-3 study of the first-in-class CDK4/6 inhibitor Ibrance (palbociclib) and Novartis AG’s Phase III MONALEESA-2
study of ribociclib (LEE011) were both terminated early for efficacy.
Ibrance won accelerated approval in early 2015 and went on to
become a huge success in breast cancer, bringing in $514 in sales
scripintelligence.com
THIRD TO MARKET
Expectations that abemaciclib will be third to market is “secured” by
the news that MONARCH 2 study will continue, Bernstein Research
analyst Tim Anderson said in an Aug. 10 note.
“It is a bit unfair – the threshold for significance is intentionally set
high at the interim analysis, and difference in design/accrual/events
can easily a cause a strong signal to fall short,” but nevertheless the
outcome will raise questions over whether there is a difference in
efficacy compared to Ibrance or ribociclib, the analyst observed.
In the MONARCH 1 study, the drug demonstrated an objective
response rate of 19.7%, but as Anderson pointed out, it “technically
missed its endpoint.”
The lack of interim data from MONARCH 2 means that MONARCH
1 will have to stand on its own and approvability based on this trial
is “borderline,” the analyst concluded. Results from MONARCH 2 are
expected in the first half of 2017.
“Some may wonder if the news foreshadows a real difference
in activity – drawing a link between frequent low grade diarrhea
to lowered persistence to compromised activity,” Anderson said,
as the side effect had been a concern in the shorter MONARCH
1 that could be exacerbated with the continuation of MONARCH
2. Furthermore, the efficacy of oncology drugs can drop in larger,
later-stage trials, so the MONARCH 2 data could disappoint when
the trial is complete.
Bernstein is forecasting $710m in abemaciclib sales in 2020, assuming “third-to-market status, no real differentiation, and no usage
in other tumors like KRAS [mutant] NSCLC (Phase III data in 2018),”
Anderson said. But, he added, “if MONARCH 1 leads to early approval
in 2017, and if the Phase III program shows a more competitive profile relative to Ibrance, then estimates in 2020 could increase substantially. For now, we maintain our conservative assumptions.”
Leerink Swann analyst Seamus Fernandez similarly concluded in
an Aug. 10 note that it’s unlikely that the company will be able to
file the drug as a monotherapy as planned this year, and that the
market opportunity now “becomes even narrower.”
BMO Capital Markets analyst Alex Arfaei said that he expects a
positive outcome in MONARCH 2 and “conservatively” forecasts a
mid-2018 launch.
The lack of an early stoppage does “cast some uncertainty over
the efficacy of abemaciclib and its competitive profile given that
it is significantly behind Pfizer’s Ibrance,” but the first-line MONALESSA-2 study of ribociclib is “not completely comparable to MONARCH 2, which also included patients who received prior endocrine
therapy,” Arfaei said.
Published online 10 August 2016
19 August 2016 | Scrip intelligence | 13
HEADLINE NEWS
Another Late-Stage Failure For AstraZeneca’s Selumetinib
ALEX SHIMMINGS [email protected]
AstraZeneca PLC’s novel targeted anticancer selumetinib has
missed its endpoints in a Phase III study in lung cancer, the second late-stage failure for the product, which last year disappointed in uveal melanoma.
T
op-line data from the 510-patient Phase III SELECT-1 trial of
AstraZeneca PLC’s MEK 1/2 inhibitor failed to show any benefit on the primary endpoint of progression-free survival
(PFS) when used in combination with docetaxel chemotherapy for
the second-line treatment of patients with KRAS mutation-positive
(KRASm) locally advanced or metastatic non-small cell lung cancer
(NSCLC). There was also no sign of any benefit on overall survival (OS).
This is in contrast to its performance at Phase II in 87 previously
treated patients with KRAS-positive NSCLC that found the addition of selumetinib to docetaxel therapy statistically improved PFS
and objective response rate. Selumetinib also improved OS to 9.4
months as opposed to 5.2 months seen in patients only treated
with docetaxel; however, this result was not statistically significant.
“It is disappointing for patients that these results have not been
confirmed in Phase III,” commented AstraZeneca’s Sean Bohen, executive vice president, global medicines development and chief
medical officer.
The SELECT-1 setback seems to spell the end for selumetinib
in this indication, with AstraZeneca’s attention now turning to
other therapeutic strategies in NSCLC: Bohen said the company
remained “committed to further developing treatments in the
lung cancer setting, such as our immunotherapy combinations
and targeted EGFR treatments.” Indeed the product seems to have
got rather lost by the wayside at AstraZeneca,when compared
with the development paths for the TKI Tagrisso (osimertinib) and
anti-PDL1 product durvalumab. Selumetinib is a highly selective
oral inhibitor of MEK1/ 2, which play a critical role in the RAS-ERK
pathway, which is activated during cancer growth, including in patients with KRASm NSCLC. This was a unique mechanism of action
in NSCLC, but the product did have competition in KRAS+ disease
in the form of Eli Lilly & Co.’s investigational CDK4/6 dual inhibitor
abemaciclib, and the newer immune targeted products, among
other already marketed products.
“Selumetinib likely wouldn’t have been a blockbuster in
NSCLC, as it would have entered into an increasingly crowded
second-line setting and would have had to compete with immunotherapies as well as drugs already like Cyramza [Lilly’s
ramucirumab] and Vargatef [Boehringer Ingelheim GMBH ‘s
nintedanib]. However, we did have it projected to be the first
targeted drug approved for KRAS mutation-positive NSCLC patients. This patient group has historically been difficult to treat,
so the approval of selumetinib could have been a significant
clinical advancement in NSCLC,” said Datamonitor Healthcare
lead analyst Hardik Patel.
Failure in the trial comes just after a year since selumetinib flopped
at the Phase III stage in its original lead indication, uveal melanoma.
AZ had originally planned to file for uveal melanoma in 2015 and
later for KRASm NSCLC in 2017.
Published online 9 August 2016
CAR-T Update: Kite’s Filing Coming Soon
With all eyes on the race to get the autologous cell immunotherapies to market, Kite Pharma Inc. announces
it could soon see late-stage data on its lead candidate.
EMILY HAYES [email protected]
K
ite Pharma Inc. a major player in autologous chimeric antigen
receptor T-cell (CAR-T) therapies aimed at B-cell malignancies,
is expecting late-stage data for its lead candidate, KTE-C19, in
diffuse large B-cell lymphoma very soon.
The company indicated during its second-quarter earnings call
that it will be getting top-line data from the ZUMA-1 study of KTEC19 in diffuse large B-cell lymphoma, a pivotal trial, by the end of
the third quarter. Kite, Novartis AG and Juno Therapeutics Inc. have
been in a race to get competing complex, autologous immunotherapies to market. With high stakes for the personalized medicines, there’s been intense scrutiny on every comment, or absence
of comment, from the sponsors.
CAR-T therapies have demonstrated impressive efficacy in hematological malignancies, but have largely been relegated to later
lines of therapy of B-cell malignancies due to the challenges of targeting, which can result in severe adverse events.
14 | Scrip intelligence | 19 August 2016
David Chang, executive vice president of R&D and chief medical
officer, said during an Aug 8 call that the company continues to advance trials of KTE-C19, which are expected to enroll more than 330
patients in 50 sites in the US. The company will maintain its focus
on patients with refractory B-cell malignancies with few treatment
options, Chang said. Kite is planning a registrational filing by the end
of 2016 and 2017 in EU.
Chang said that the company also initiated a technology collaboration with UCLA for the development of off-the-shelf, allogeneic
T-cell therapies from renewable pluripotent stem cells.
Kite said that it is also investigating KTE-C19 in earlier lines of
DLBCL and a range of new indications, including follicular lymCLICK
phoma and chronic lymphocytic
Read full story at:
leukemia.
http://bit.ly/2b3UZlJ
Published online 9 August 2016
© Informa UK Ltd 2016
HEADLINE NEWS
Valeant’s New Strategic Direction:
Clean Break Or Simply A Paint Job?
Trying to get out from under the shadow of ongoing investigations into its pricing and questionable business
practices, which have led to its shares diving 90% in the past year, Valeant Pharmaceuticals International Inc. laid out
its strategy for becoming a “new” company. But one analyst questioned whether the changes being made were simply
“new paint on the same old shed.”
DONNA YOUNG [email protected]
I
nsisting it was now a “new” company – or
at least on its way to becoming one – Valeant Pharmaceuticals International Inc.
on Aug. 9 unveiled plans to reorganize its
business, reaffirmed its 2016 full year guidance and vowed to aggressively bring down
its $30bn in debt by renegotiating its terms
and selling off noncore assets.
“We are setting the company on a new
path with new strategic imperative, changes to the management team and structure
and new business segments and a new direction,” said Valeant CEO Joseph Papa.
The company has been plagued by
scandals in the past year over significantly
hiked up drug prices and its questionable
relationship with the specialty pharmacy
Philidor Rx Services Inc.
“Valeant will be embarking on a new
vision and mission,” Papa told investors
and analysts during the company’s Aug.
9 second-quarter earnings call. “We want
to be a trusted healthcare partner and to
improve people’s lives with our healthcare
products.”
But skeptical Wells Fargo analyst David
Maris questioned whether things were really changing with Valeant or if the Canadian drug company was simply putting “new
paint on the same old shed.”
He concluded Valeant’s new actions
were far from enough to change his mind
– declaring the company’s business trends
were weak, its debt was high, its key management were leaving, congressional and
Securities and Exchange Commission investigations were ongoing and the firm
remained a “price-driven model in a payer
market that has gotten smarter.”
But investors were pleased with Valeant’s
plan for a turnaround – pushing shares up
nearly 26% before closing at $28.16, a gain
of $5.74, or 25.4%.
Papa, who has been trying to turn
things around for Valeant since he was
installed as CEO in May – replacing Mike
scripintelligence.com
Pearson, who was essentially fired by the
firm’s board in March but stayed on until a replacement could be found – said
the company’s reorganization currently
doesn’t involve a name change, although
he said it’s “something we’ll always continue to evaluate.”
Nonetheless, he said the new Valeant
will consist of three reorganized business
segments: Bausch & Lomb Inc. ophthalmology products and international business; branded prescription medicines,
including dermatology, gastrointestinal
and women’s health drugs; and the company’s US diversified products, including
the firm’s neurology and generic drugs,
its Solta medical aesthetic device systems
and its Obagi skin care line.
The latter segment, said TD Securities
analyst Lennox Gibbs, appears to be earmarked for divestment.
Indeed, Papa said Valeant was looking
at alternatives for a number of its “noncore
businesses and geographies that represent revenue greater than $2bn,” although
he said the company thinks it can get up
to $8bn, which Gibbs noted would go a
long way towards paying down the firm’s
$30bn debt.
Papa said Valeant has received “indications of interest on these assets and we
have engaged respected banks and advisors to assist us in exploring our options.”
About a third of the offers that have
come in have been unsolicited, he said.
Valeant officials noted the company already has taken steps to streamline its portfolio in the second quarter by agreeing to
return its European rights to its plaque psoriasis drug brodalumab toAstraZeneca PLC,
divest its Synergetics USA OEM business
and sell back its rights to its recombinant
human C1 esterase inhibitor Ruconest to
Pharming NV, which brought in $181m in
cash and the potential for up to $329m for
in approval and sales milestones.
Those actions, said Rodman & Renshaw
analyst Raghuram Selvaraju, “constitute
logically structured transactions that do
not, in our view, impair Valeant’s base
business. In our view, Valeant is taking a
measured and rational approach to targeted divestitures of non-core businesses,” he said.
“We fully intend to make decisions regarding our asset base in the best longterm interest of our shareholders,” Papa
said. He said Valeant expects to “simplify
the business and reduce our debt through
strategic measures and cash generation
over the next 12 months to 18 months.”
Papa noted that Valeant already had paid
$1.29bn towards its permanent debt so far
and has completed all 2016 scheduled
amortization payments and the payment
for the first quarter of 2017. It has about
$475m in remaining mandatory term loan
amortization for next year, he said.
But Papa said that while Valeant continues to be in compliance with its financial
maintenance covenants under its bank
debt through 2016, “our cushion is not as
large as I would like it to be.”
So the company is seeking to modify its
interest coverage financial maintenance
covenant with its lenders and plans to
launch an amendment process shortly.
“We plan to modify the interest coverage, financial maintenance covenants as
well as a couple of other small changes,”
explained Linda LaGorga, senior vice
president and treasurer at Valeant. “This
is all opportunistic and focus on getting
us the flexibility to up-size our capital
structure. So while the interest coverage
financial maintenance covenant is a bit
tight right now with additional push, we
think we’ll be very comfortable going
forward is just able to focus on the strategy of the business and talking about the
business.”
Published online 9 August 2016
19 August 2016 | Scrip intelligence | 15
HEADLINE NEWS
Pricing Concerns Delaying Sanofi’s Turkey Insulin
Investment?
AHMET SEVINDIK
of its diabetes product in Turkey, with the company not yet satisfied over this. The Sanofi executive clearly expressed his opinion
at the Frankfurt meeting that prices in the country are “too low”
and not sustainable for high-value products.
Despite the political turmoil, Sanofi appears keen to invest in the
local manufacturing of an insulin product in Turkey if the government “opens the way,” but is holding off over pricing worries.
S
anofi said at the beginning of this year that it had been negotiating with the Turkish government over the possible local
production of one of its insulin preparations, and although
there have been no further concrete statements on the move, negotiations are apparently still moving forward despite the political
turmoil in the country.
The French company last month took some well-known Turkish
columnists and journalists to Frankfurt, where one of its major insulin production facilities is located and Turkey country president Fabrizio Guidi gave some further hints about the planned investment.
Talking about the economic and social burden that diabetes is
placing on Turkey and the expertise and technological superiority
of Sanofi in the field of insulin production, he emphasized that the
firm does indeed want to increase the local production of valueadded products. Sanofi is ready to invest if the government “opens
the way” and the project would include importing an active ingredient from Germany and formulating it into a finished product for
local distribution, he said.
Shutterstock: Aleksey Klints
PROJECT NOT FINALIZED
Which specific Sanofi insulin preparation might be manufactured in
Turkey is still not clear, and the company has been careful not to give
away any details. However, the most obvious candidate would seem
to be its long-acting basal product Lantus (insulin glargine).
In addition, although Sanofi declared months ago that it was
looking for a local partner in the diabetes project - which the government encourages and would prefer as a matter of policy - the
company also has the option to stay in-house if no suitable collaborator can be found.
When Sanofi acquired Czech generic drug maker Zentiva BV back
in 2009, the deal gave it a factory in Lüleburgaz, Turkey, in which the
French Group has since invested more than €600m ($665m) and
globally has now become its third biggest production facility.
The site earlier this year began exporting sterile antibiotic
preparations to Japan and there are plans to begin shipments
to China and Taiwan, and it may well be able to handle the diabetes product.
While Turkey’s Ministry of Health would be very happy with such
an investment for the local manufacturing of a high-tech product
in line with its policy aims, and would probably be ready to provide
purchasing guarantees in return, Guidi referred to the country’s
pricing policies as a “road block” making it difficult to move forward
at this stage.
Downward pressure on prices has emerged as one of the
main concerns for the pharma industry in Turkey, being blamed
by the major local association last year for adversely affecting
profitability and dragging the market into contraction when adjusted for inflation. Given this, it is perhaps not surprising that
the Sanofi negotiations have been focusing on the likely pricing
16 | Scrip intelligence | 19 August 2016
POLITICAL FACTORS?
Of course, Sanofi’s investment plans were first discussed before the
July 15 coup attempt, which despite obvious political tensions nobody really saw coming. A state of emergency is now in place and
the situation has sparked serious concerns globally about Turkey’s
political and economic stability.
Currently, 2,000-3,000 soldiers, prosecutors and judges are in
jail and 50,000-60,000 public servants and bureaucrats have been
sacked, while the owners of some big companies have been
detained because of alleged ties to the religious group, Fethullah Community, held responsible by the government for the attempted coup.
When asked whether the uncertainty might have any impact on
its planned diabetes manufacturing investment, Sanofi Turkey declined to answer. Several other multinationals have already issued
statements stressing their continued belief in the future of Turkey
and that investment and business plans are going ahead (even if
this might just be lip service), but interestingly, Sanofi hasn’t made
any similar declarations so far.
At the moment at least, the incumbent government seems to
be in control, and for pharma companies Turkey is still a profitable market where they can do business, despite the challenges.
Reassurances on pricing will therefore probably open the way to
Sanofi’s new investment, barring any unexpected political turns.
From the editors of PharmAsia News.
Published online 9 August 2016
© Informa UK Ltd 2016
P O L I C Y & R E G U L AT I O N B R I E F S
NICE Backs Ticagrelor
Use With Aspirin
AstraZeneca PLC’s Brilique (ticagrelor)
has won initial backing by NICE for use
at a lower 60 mg dose and in combination with aspirin by people who have
had a heart attack and have completed
an initial 12-month treatment period
using a higher 90 mg strength of the
anti-clotting drug, also with aspirin. The
National Institute for Health and Care
Excellence (NICE) on Aug 12 said it was
recommending such patients take ticagrelor 60mg, which costs about £1 per
tablet, with aspirin twice a day for up
to three years, adding that there should
no interruption between treating them
at the higher 90mg dose and the lower
60 mg dose. The aim is to reduce their
risk of a further heart attack or stroke.
Because there are limited data on ticagrelor’s efficacy and safety, particularly
the risk of bleeding, beyond three years,
the draft guidance does not recommend
treatment with ticagrelor beyond that
period. Ticagrelor is an oral antagonist
of the P2Y12 adenosine diphosphate receptor that inhibits platelet aggregation
and thrombus formation in atherosclerotic disease. It won marketing authorization in February 2016 as a continuation therapy at 60mg for the prevention
of atherothrombotic events in adult
patients with a history of myocardial infarction and a high risk of developing an
atherothrombotic event.
[email protected], 11 August 2016
NICE Clears Amgen’s Imlygic
At Second Attempt
Amgen Inc.’s oncolytic virus-based
immunotherapy Imlygic (talimogene
laherparepvec) has been cleared for
use in melanoma patients by the UK’s
National Institute for health and Care
Excellence (NICE), at the second time
of asking. The recommendation in
NICE’s final draft guidance came after Amgen put in place a patient access
scheme (PAC) in which the drug will be
supplied at an undisclosed discount to
its current price of £1,670 ($2,166) per
scripintelligence.com
Daiichi Arbitration Case Bares Trail Of
Deception At Ranbaxy
Incriminating details in an arbitration order against the former Ranbaxy
top brass led by the Singh brothers has put the spotlight back on allegations of misrepresentation of critical information concerning the US Department of Justice (DoJ) and FDA investigations against the Indian company at the time of its takeover by Daiichi Sankyo. The Singh brothers, who
have challenged the award, though, have cried foul, questioning the timing
of the apparent leak of the “confidential” award ahead of a court hearing. The order, details of which were reported in the local media, suggests
that Ranbaxy’s top brass may have been well aware of certain compliance
deviations way back in 2004. The Indian Express newspaper reported that
a Self Assessment Report (SAR) prepared by Ranbaxy’s then R&D head
and presented at a 2004 meeting which had the firm’s top brass in attendance listed over 200 drugs for which the company was alleged to have used
fabricated data to get regulatory approvals in several countries. The SAR’s
contents were pivotal in the launch of investigations against Ranbaxy by
the US authorities, but the Indian firm’s top brass apparently withheld the
report leaving the Japanese company in the dark. An industry source well
in the know of the past goings on at Ranbaxy, told Scrip that prima facie,
going by the report, it appears that the CEO and the board of the Ranbaxy
“were complicit in misleading Daiichi Sankyo.” Others, such as an expert
with a foreign drug firm, suggested that there was little doubt that at least
some directors of the board were made aware of the malpractices, but they
chose to be “economical with the truth” for reasons best known to them.
[email protected], 11 August 2016
1 ml vial to England’s National Health
Service. One vial is used initially followed by a second at three weeks and
then every two weeks. It’s the second
piece of good news Amgen has received
from NICE in recent months. The company’s PCSK9 inhibitor Repatha (evolocumab) was recommended by NICE
in June 2016 for use in patients with
primary hypercholesterolemia and
mixed dyslipidemia, again with a PAC
agreed. NICE’s final draft guidance on
Imlygic was released Aug. 9 and the
final guidance is expected to be published in September.
[email protected], 9 August 2016
Heron’s Sustol Faces
Competition
The long, tormented regulatory journey Heron Therapeutics Inc. has been
on over the past nearly seven years
with its chemotherapy-induced nausea and vomiting (CINV) drug Sustol
(granisetron) has finally come to a
somewhat successful end, with the
FDA granting its go-ahead for the
product to be marketed in the US, although not for the broad use the company had sought. US regulators said
Sustol, a serotonin-3 (5-HT3) receptor
antagonist, could be sold as a therapy
in combination with other antiemetics to prevent acute and delayed nausea and vomiting associated with initial and repeat courses of moderately
emetogenic chemotherapy (MEC) or
anthracycline and cyclophosphamide
(AC) combination chemotherapy regimens. But the FDA determined Heron
had insufficient cisplatin patients in
its Phase III trial to include the broader indication of highly emetogenic
chemotherapy (HEC) beyond AC in
Sustol’s labeling.
[email protected], 10 August 2016
19 August 2016 | Scrip intelligence | 17
HEADLINE NEWS
If Egalet Wins At FDA, Arymo ER Still Might Not In
Opioid Market
Potential sales impact of even broadest possible label for oral, intranasal and intravenous abuse remains a question
mark given that abuse-deterrent products remain only a small percentage of market.
SUE SUTTER [email protected]
E
galet Corp. is hoping Arymo ER (morphine sulfate extended-release) will
garner the broadest abuse-deterrent
labeling among long-acting morphine products following a positive FDA advisory committee review. However, the potential sales
impact of such a label remains a question
mark given the current market dynamics.
On Aug. 4, the FDA’s Anesthetic and Analgesic Drug Products and Drug Safety and Risk
Management advisory committees voted
18-1 for approval of Egalet’s long-acting opioid for management of pain severe enough
to require daily, around-the-clock, long-term
opioid treatment and for which alternative
treatment options are inadequate.
In separate votes, the panel also supported labeling as an abuse-deterrent
product by the oral (16-3), nasal (18-1) and
intravenous (18-1) routes of abuse.
If approved with abuse-deterrent labeling by its Oct. 14 user fee date, Arymo
would join six other currently approved
extended-release/long-acting opioids, including two morphine sulfate products,
with labeling that describes studies conducted to support abuse-deterrent properties pursuant to FDA guidance.
POTENTIALLY BROADER LABEL
“It’s our hope and expectation that we will
achieve abuse-deterrent claims in the label
for the intravenous route, oral route and
intranasal routes of abuse,” Egalet President
and CEO Robert Radie said during a second
quarter earnings call that followed the advisory committee meeting. “This would then
be the broadest set of claims [relative to]
the comparisons of the labels for the other
two currently approved abuse-deterrent,
extended-release morphine products.”
Pfizer Inc.’s Embeda (morphine sulfate/
naltrexone extended-release) was approved
in 2009 and added labeling on oral and intranasal abuse deterrence in October 2014.
However, Embeda was unavailable for several years. It was withdrawn from the market
in March 2011 due to stability issues and ap-
18 | Scrip intelligence | 19 August 2016
proved with a manufacturing supplement
in November 2013.
Inspirion Delivery Technologies’ Morphabond (morphine sulfate extended-release)
was approved in October 2015 with deterrence labeling against the intranasal and
intravenous forms of abuse.
In its briefing book for the advisory committee meeting, Egalet noted that morphine is the most commonly prescribed
ER/LA opioid analgesic, with approximately 6.4m prescriptions filled at outpatient
pharmacies in 2015.
FEW INROADS FOR DETERRENT
PRODUCTS
However, abuse-deterrent formulations
have made little inroads in this category.
During the first four months of 2016, 98.5%
of prescriptions filled for morphine extended-release were for products with no abusedeterrent properties, Egalet said, citing IMS
Health data.
‘It’s our hope and
expectation that we will
achieve abuse-deterrent
claims in the label for the
intravenous route’
The difficulties abuse-deterrent formulations have had in capturing market share is
not limited to the morphine category. At a
May advisory committee review of the ER/
LA Risk Evaluation and Mitigation Strategy,
an industry representative said abuse-deterrent formulations account for approximately 22% of the market share of ER/LA opioids.
The bulk of that share is represented
by Purdue Pharma LP’s reformulated OxyContin (oxycodone extended-release).
The new formulation was approved in
2010 and added deterrence claims for
intravenous and intranasal abuse in 2013,
which led the FDA to ban generics of the
original formulation.
The market dynamics raise the question
of how much a third labeled route of abuse
deterrence for Arymo would help it gain a
commercial foothold and how much marketing muscle Egalet will have to put behind the product.
SALES FORCE MIGHT EXPAND
If approved, Arymo will be detailed by
Egalet’s existing sales force. The 71-person
team currently details two immediaterelease analgesics: Sprix (ketorolac nasal
spray) and Oxaydo (oxycodone). Combined net product sales for the second
quarter were $3.5m.
Radie said Egalet’s sales force would detail Arymo to the same 11,500 high-decile
physicians prescribing pain medications
that the company is currently reaching
with Sprix and Oxaydo.
Egalet is currently evaluating the size and
structure of its sales force for the three products, Radie said, predicting the size would
probably expand to about 100 with the approval of Arymo. Assuming approval by the
user fee goal date, active promotion is expected to begin in the first quarter of 2017.
Oxaydo, licensed from Acura Pharmaceuticals Inc., is formulated with an adversive
agent intended to make sniffing the drug
unpleasant. The drug was approved in June
2011, and labeling includes a description of
data from a human intranasal abuse liability
study reflecting less drug liking with Oxaydo
compared to immediate-release oxycodone.
However, the FDA does not consider
Oxaydo to carry abuse-deterrent labeling
claims pursuant to a guidance document
finalized in 2015.
Published online 8 August 2016
CLICK
To read more online:
http://bit.ly/2aU6Jpf
© Informa UK Ltd 2016
S T O C K WAT C H
Landscaping Earnings Announcements At Valeant
And Endo
ANDY SMITH
Endo, Valeant, Mylan and Perrigo all reported second-quarter results last week.
Only maintenance of full-year guidance
was needed for stock price outperformance but guidance cuts for all look like
just a matter of time.
A
fter three consecutive quarterly
earnings disappointments, including the disastrous first-quarter 2016
report that included such a drastic cut in
full-year guidance that its share price fell
about 20% on the day, second-quarter expectations for Endo International PLC were
not high. In the event, both second-quarter
sales and earnings beat analysts’ (recently
deforested) consensus estimates and benefitted from that most sporadic of commercial drivers – wholesaler restocking – while
full-year guidance was not cut further. The
result was a relief rally for Endo’s share price
which finished the week up 32.5%, trouncing the NYSE Arca Pharmaceutical Index
which finished last week down 0.5%. It
wasn’t just me who was left wondering if
the management of Endo should be basking in the glow of a 71.5% share price fall in
the last year: the title of the analysts from
Citigroup’s note was “Absence of a negative.
Finally a relatively uneventful quarter.” Between the lines of its earnings announcement, the underlying pressures seem to remain. Endo expects generic price deflation
to continue from this year’s 5% decline to
double that in 2017. I found myself asking
the same rhetorical question as Cowen analysts. “At what point will improved performance lead to significant debt retirement
in the face of still large [vaginal] mesh liability payments?”
In a reversal of the roles of master and
student, Valeant Pharmaceuticals International Inc. mimicked Endo by not changing
its full-year guidance after slashing it twice
already this year: in mid-March and then
at its delayed first-quarter earnings report
(Also see “Valeant’s New Strategic Direction:
Clean Break Or Simply A Paint Job?” - Scrip,
9 Aug, 2016.). For once, that was where the
similarity between the two companies end-
scripintelligence.com
ed with Valeant missing analysts’ consensus
estimates of sales and earnings by 2% and
5% respectively, with a continuation of the
headwinds of the first quarter. In the same
way that no further cut in full-year guidance
propelled Endo’s share price, Valeant’s rose
by over 25% on the day to finish the week
up over 13%. With a continuing degradation of its business, Valeant’s shareholders
seemed to be distracted by the commentary on its efforts to repay its $31bn debt
mountain. While the sale of non-core assets
has been mentioned by both Valeant and
Endo, I seemed to be the only one who remembered both companies acquiring wall
flower assets in which no other acquirer
was interested and then cutting R&D and
raising prices, transforming them into even
less palatable assets. Valeant also discussed
transforming its agreement with Walgreens
Boots Alliance Inc. This resulted in Valeant
losing money on most of its prescriptions
as Walgreens was paid to dispense Valeant’s
branded generic products which were not
reimbursed by the patients’ insurers. If Walgreens was a not for profit institution there
may be a possibility that it would donate its
profits to Valeant or wrangle branded prices
out of insurers for Valeant’s products that
could easily be substituted by a generic –
as its captive pharmacy Philidor Rx Services
Inc. was doing until earlier this year – but
neither is likely.
Even without the reminder that Federal
prosecutors are continuing their criminal
investigation of Valeant’s relationship with
Philidor, there were enough hints in Valeant’s second-quarter earnings commentary
to suggest that like Endo, the full-year guidance cuts from earlier this year would not be
the last. Valeant disclosed that it was seeking another relaxation on the covenants
over its debt. This disclosure should have
worried Valeant’s investors more than any
other part of its earnings announcement,
firstly because like Walgreens, debt holders are not charities and will require a cash
compensation for relaxing the covenants.
That cash compensation will reduce Valeant’s ability to pay down the principal on
its debt. Secondly, of the two components
in the interest cover ratio that is part of Valeant’s debt covenants, it is more likely to
be lower earnings before interest and tax
rather than higher interest that would be
cause for concern and lower earnings imply
lower sales. This is not the higher sales and
earnings for the second half of the year that
maintenance of full-year guidance after a
slower second-quarter implies.
Mylan NV also reported second-quarter
results last week, beating analysts’ consensus earnings estimates, a maintenance of
full-year guidance but a sales miss driven by
US generic price erosion. That was echoed
by Impax Laboratories Inc. and Perrigo Co.
PLC results where earnings were well below
analysts’ expectations and full-year guidance was lowered, in Perrigo’s case for the
second time this year driven by (yes, you
guessed it) a deteriorating generic pricing
environment.
The end of August will mark 300 years
since the birth of the celebrated landscape
designer Capability Brown. Landscape design has been described as the transformation or taming of nature whilst making
the outcome look natural. In the same way
the absence of full-year guidance cuts in
Endo’s and Valeant’s results last week supposedly transformed their investment landscapes from bomb sites into stately homes.
However, with the added complication of
a foundation of debt to the deteriorating
generic pricing and pre-authorization environment, nature will probably reclaim that
work fairly quickly.
Andy Smith is chief investment officer of
Mann Bioinvest. Mann Bioinvest is the investment adviser for the Magna BioPharma
Income fund which has no position in the
stocks mentioned, unless stated above. Dr
Smith gives an investment fund manager’s
view on life science companies. He has been
lead fund manager for four life science–specific funds, including International Biotechnology Trust and the AXA Framlington Biotech Fund, and was awarded the Technology
Fund Manager of the year for 2007.
Published online 15 August 2016
19 August 2016 | Scrip intelligence | 19
EXPERT VIEW
Wearables: A World Of Pharma Partnership
And Potential
MELANIE SENIOR
Pharma does not need to make wearables. Instead, it needs to
figure out how best to use the data that these technologies can
provide, how to integrate wearables into product offerings and
how to persuade payers to foot the bill. Investment in this field is
increasing as the potential of mobile and wearable technologies
to transform therapeutic development and healthcare delivery
becomes clear.
A
s the commercial and scientific potential of wearable devices
in medicine becomes clearer, not least to slow the competition-driven price slide typically seen in consumer tech, both
established technology giants and start-ups are vying for some of
the action. This presents pharma firms with new potential partners
– and potential competitors – in the likes of Google, Apple, and Samsung as well as in smaller specialists like Empatica and AliveCor.
Device makers can be split into five broad categories, encompassing established medical devices players, large software and
consumer technology firms, and a range of start-ups and specialists
targeting the medical/healthcare market. Pharma does not need
to make wearables. Instead, it needs to figure out how best to use
the data that these technologies can provide, and how to integrate
wearables into product offerings. “Our energy is spent more on
making meaning out of the data that wearables are able to generate [rather than on building the tools themselves],” says Pfizer Inc.’s
head of clinical innovation Craig Lipset.
Makers Of Wearables
TYPE OF COMPANY
EXAMPLE
Established medical devices firms
Philips, Medtronic
Consumer technology giants
Apple, Samsung
IT/data companies
Google, Qualcomm, Microsoft,
IBM
Medical technology specialists
Mega Electronics
Start-ups (healthcare/medical
devices)
Vital Connect, Empatica, MC10
Start-ups (consumer technology)
Withings, Empatica
Source: Datamonitor
Since most wearable devices are freely available on the consumer market, “whatever we see out there, we buy and try it. We
have a cupboard full of wearables. Sometimes I have four or five on
together, to test them,” says Kristian Hart-Hansen, chief executive
officer of LEO Innovation Lab. “We’re looking at how to incorporate
them into our solutions and thinking, but I don’t think we’ll be the
people developing them.”
A handful of pharma firms have acquired technology partners,
preventing competitors from tapping the same technology or approach. In 2015, Teva Pharmaceutical Industries Ltd. bought smart
20 | Scrip intelligence | 19 August 2016
inhaler firm Gecko Health Innovations, for example, following a
similar move by diversified healthcare group Opko Health Inc. Also,
AstraZeneca PLC made a small investment in New Zealand-based
smart inhaler company Adherium Ltd during 2015, having shortly
before announced a long-term commercialization agreement.
PHARMA-TECHNOLOGY PARTNERSHIPS
Indeed, partnerships are for now the most popular arrangement
between biopharma companies and technology firms. Partnerships
offer both sides flexibility and ensure that technological innovation
can continue freely; in the case of larger tech groups, acquisition is
generally not an option in any case.
Hence partnerships are proliferating as the potential of mobile
and wearable technologies to transform therapeutic development and healthcare delivery becomes clear, and as both sides
seek the other’s expertise to make that happen. Biogen has tied
up with Google’s Verily, Novartis AG has deals with Microsoft and
Qualcomm, and Pfizer has joined forces with IBM to use remote
monitoring devices to investigate Parkinson’s disease. Meanwhile,
other firms are experimenting in focused areas with smaller players
like MC10 or Vital Connect.
Technology firms seeking to penetrate the healthcare market also
require input from clinicians and drug developers. “We give device
makers lots of feedback on what we think their products should be
able to do,” says Mike Capone, chief operating officer at clinical trial
solutions provider Medidata. “We have met with Apple extensively,”
echoes Adriana Karaboutis, executive vice-president of technology
and business solutions at Biogen. “They’re trying to understand our
requirements for, as an example, what might be required for tremor
or voice tests in Alzheimer’s or Parkinson’s. We’re working informally
to help give them insights into what we’re looking for.”
APPLE: AN EMERGING HEALTHCARE COMPETITOR
Apple’s ubiquitous iPhone and the newer Apple Watch are rapidly
making this corporate giant a healthcare player in its own right.
Apple has made no secret of its intention to break into the sector
and become a centerpiece of mobile health data collection and presentation. Its HealthKit platform aggregates and presents consumers’
health data from across a variety of clinical apps and devices (including non-Apple ones). Additionally, ResearchKit, launched in March
2015, is an open-source software framework designed to enable scientists and medical researchers to build apps that can easily gather
health data from Apple devices (and other gadgets connected via
HealthKit) that may be spread nationwide or beyond. The newest
software addition, CareKit, is smaller-scale: it allows developers to
build healthcare apps that gather individual health data for personal
use or for sharing with physicians or provider networks.
Apple is also building its relationships with key healthcare stakeholders: it is reported to have had regular talks with the US Food
and Drug Administration (FDA) and other regulators, and has tied
© Informa UK Ltd 2016
EXPERT VIEW
up with the likes of Epic, the largest vendor of electronic health
records. The Apple Watch, launched in early 2015 and with an estimated 5–10 million quarterly sales, is Apple’s first wearable offering:
the watch tracks heart rate, activity data (including whether you are
standing or sitting), and collates information from a growing suite
of other health and fitness apps and devices keen to be associated
with the popular brand.
The watch also provides an attractively large, ready-made customer base for multiple health-related accessories, including many
seeking clinical application. The Kardia Band, designed as a strap for
the Apple Watch by San Francisco-based medtech start-up AliveCor Inc. provides an electrocardiogram reading from a thumb pad,
and generates detailed heart rhythm data that can be used to help
patients detect episodes of atrial fibrillation. The device is pending 510k FDA clearance. Other apps on the Apple Watch help you
remember to drink enough water, move around, eat healthily, and
take your medication properly.
Google has also joined the health wearables bandwagon. It has
already licensed its blood glucose-testing contact lens technology
to Novartis, and has developed a wristband to track heart rate, temperature, and other external variables like light and noise, which it
intends to have licensed as a medical device for use in clinical trials
or as part of physician-prescribed regimens.
FOSTERING AND FUNDING DIGITAL HEALTH
INNOVATION
Another way for pharma to embrace technology is by fostering entrepreneurial, innovative health-tech start-ups – just as several Big
Pharma companies have done to ignite creative drug discovery. In
2015, Danish dermatology group Leo Pharma AS launched LEO Innovation Lab, a series of units across Europe and North America focused on developing apps, digital platforms, and other initiatives to
better meet the needs of people living with chronic skin diseases.
“Wearables…are driving a lot of innovation within the pharmaceutical-digital space,” enthuses Kristian Hart-Hansen. LEO Pharma is also,
since January 2016, investing in health-tech focused start-ups seeking novel solutions for psoriasis sufferers, by way of the newly created
LEO Ventures.
Pharmaceutical firms have long been investing in health technology: Merck & Co. Inc.’s $500m Global Health Innovation Fund
has supported over 20 digital health companies since 2010, although few if any appear to be developing wearables. Bayer AG’s
Grants4Apps program attracts digital health start-ups for funding, office space, and mentoring in exchange for innovative ideas
across digital health, including wearables. “It’s a great way to bring
digital expertise into the Bayer community,” says Bayer’s digital development head Jessica Federer about the program. One of the
start-ups Bayer has chosen to support in its Grants4Apps Accelerator program is FabUlyzer, which is a wearable nano-sensor device that, when breathed or blown on, measures how much fat is
burned during exercise.
DIGITAL HEALTH INVESTMENTS
Pharma’s foray into wearable technologies is happening as part of its
broader drive to embrace digital technologies and patient-centricity.
Most have brought on technology expertise to help both R&D and
commercial teams understand and exploit new opportunities.
scripintelligence.com
GlaxoSmithKline PLC recently formalized a multi-disciplinary
innovation unit whose mission is to modernize clinical trials using digital technologies; the Clinical Innovation and Digital Platforms group has a dedicated team and budget to ensure that
initiatives move beyond experimental pilots toward more widespread application.
Bayer’s digital development head Jessica Federer was appointed in July 2014 to take the entire organization digital, across all
its divisions, thereby helping foster closer integration between
the various segments (pharmaceuticals, crop sciences, and consumer) as well.
Novartis has set up a digital medicines team to think about how
to use beyond-the-pill technologies to improve outcomes, and has
a digital development group within the team to look specifically at
how to bring new technologies into the R&D organization. Many in
the team are technologists with high scientific aptitude, often from
the venture capitalist community. “We’re looking at how to absorb
these technologies into regular operations, and to scale them up,”
explains Vas Narasimhan, global head of drug development and
chief medical officer at Novartis. “That’s how they can substantially
improve the efficiency of the whole process.” He’s referring to technologies across the board, including software linking to searchable
electronic health records to help identify trial patients, e-consent
systems, and data entry, as well as telemedicine and wearables to
allow virtual trials.
Integrating wearables systematically across an R&D organization requires not just testing the wearables themselves, but an
entire infrastructure of people, process, and technology. “Over the
last 2–3 years, we’ve made specific investments…to use these
novel data capture instruments in a more consistent manner,” explains Pfizer’s Craig Lipset. That includes making sure that they
are understood by, and accessible to, all study teams, that they
generate meaningful data that impact development, and that the
data interfaces are optimized for the patient experience. It means
ensuring compliant usage, and an understanding of patient preferences. It also means changing the approach to running trials
and generating data. “It’s not just about strapping wearables on
patients in [an] existing study setup. You need a different approach,” insists Lipset.
Neither Lipset nor Narasimhan, like most of their counterparts at
other pharma companies, will quantify how much they are investing in digital technologies and related expertise. But Novartis’s Narasimhan is clear on the timeframe for returns. “We’ve bet ourselves
that within five years we’ll see a step change in R&D efficacy,” he
says, “though we’re still trying to dimensionalize it.” As the technologies scale up, he expects quality to improve first, with efficiency
and cost gains coming later.
It is still too early to identify a common investment approach.
Bayer’s Federer says only that the group’s digital transformation
should not cost a lot of money, but instead take advantage of existing expertise. The Grants4Apps program, fostering and supporting
innovative health-tech start-ups onsite to bring in creativity and
opportunities for cross-pollination, is not a big-budget item: each
group gets €50,000.
This article is part of a Datamonitor Healthcare report: Wearables are
Transforming R&D and Care Delivery. The full report is available online.
Published online 8 August 2016
19 August 2016 | Scrip intelligence | 21
P I P E L I N E WAT C H
Scrip’s weekly Pipeline Watch tabulates the most recently reported
late-stage clinical trial and regulatory developments from the more
than 10,000 drug candidates currently under active research worldwide.
CLICK
Visit scrip intelligence.com
for the entire pipeline with
added commentary.
Late-stage clinical developments for the week 5–11 August 2016
LEAD COMPANY
PARTNER COMPANY
DRUG
INDICATION
MARKET
Heron Therapeutics Inc.
–
Sustol (granisetron) extendedrelease sc injection
chemotherapy-induced nausea and
vomiting
Takeda Pharmaceutical
Co. Ltd.
–
Ninlaro (ixazomib) capsules
multiple myeloma
Xydalba (dalbavancin)
skin and skin structure infections
Jardiance (empagliflozin)
CV death reduction in type 2 diabetes
–
Keytruda (pembrolizumab)
head and neck squamous cell carcinoma
US
Merck & Co
Samsung Bioepis Co. Ltd.
biosimilar insulin glargine
(MK-1293)
type 1 and 2 diabetes
US
Eisai Co. Ltd.
–
Halaven (eribulin)
breast cancer
Marathon
Pharmaceuticals LLC
–
deflazacort
Duchenne muscular dystrophy
US
–
Aristada (aripiprazole lauroxil)
Inj.
schizophrenia
US
–
masitinib
amyotrophic lateral sclerosis
EU
–
biosimilar pegfilgrastim
(CHS-1701)
neutropenia
US
Seroquel XR (quetiapine
fumarate) extended-release
tablets
bipolar depression
–
deflazacort
Duchenne muscular dystrophy
US
Ascendis Pharma AS
–
TransCon Growth Hormone
(long acting and unmodified)
pediatric growth deficiency
–
Kissei Pharmaceutical
Co. Ltd.
JCR Pharmaceuticals
Co. Ltd.
biosimilar darbepoetin
alfa (JR-131)
renal anemia
–
vadadustat
anemia in chronic kidney disease
–
–
PRO 140
HIV/AIDS
–
–
Vonvendi (von Willebrand
factor)
von Willebrand disease
US
REGULATORY APPROVAL
US
Canada
SUPPLEMENTAL REGULATORY APPROVAL
Cardiome Pharma Corp.
Allergan PLC
Boehringer Ingelheim
(Canada) Ltd.
Eli Lilly Canada Inc.
EU
Canada
ACCELERATED/CONDITIONAL APPROVAL
Merck & Co. Inc.
REGULATORY FILING ACCEPTED
China
SUPPLEMENTAL REGULATORY FILING ACCEPTED
Alkermes PLC
ORPHAN DRUG DESIGNATION
AB Science
REGULATORY FILING
Coherus BioSciences Inc.
SUPPLEMENTAL REGULATORY FILING
Astellas Pharma Inc.
AstraZeneca PLC
Japan
PRIORITY REVIEW
Marathon
Pharmaceuticals LC
PHASE III TRIAL INITIATION
Akebia Therapeutics Inc.
CytoDyn Inc.
Mitsubishi Tanabe
Pharma Corp.
PRODUCT LAUNCH
Shire PLC
Source: Biomedtracker
22 | Scrip intelligence | 19 August 2016
© Informa UK Ltd 2016
APPOINTMENTS
Biotech company REGENXBIO Inc. has
appointed Daniel J. Abdun-Nabi to its
board of directors – effective Aug. 5, 2016.
Abdun-Nabi was president and CEO of
Emergent BioSolutions Inc. since 2012
and has held various leadership positions
at the company since he joined in 2004,
including chief operating officer and
general counsel. Prior to this, he was general counsel of IGEN International Inc., and
senior vice president, legal affairs, general
counsel and secretary at North American
Vaccine Inc.
Andrew Hirsch is joining Agios Pharmaceuticals Inc. as chief financial officer (CFO)
– effective Sept. 19, 2016. With over 20 years’
experience in strategic and operating roles
in the biotech sector, Hirsch was recently
president and CEO of BIND Therapeutics.
Hirsch also held various other leadership
positions at BIND including chief operating
officer and CFO and prior to this he was CFO
at Avila Therapeutics until its acquisitions by
Celgene. Hirsch was also vice president of
corporate strategy and M&A and program
executive for the Tecfidera development
team at Biogen.
Arena Pharmaceuticals Inc. has appointed Vincent Aurentz executive vice
president and chief business officer. Au-
rentz carries more than 27 years’ experience in the pharma and biotech sector
and prior to Arena, he was chief business
officer for Epirus Biopharmaceuticals. Previously to this, he was president of HemoShear Therapeutics and before this, he was
executive vice president and member of
the executive management board at Merck KGaA. Aurentz is also a former executive
vice president at Quintiles.
Wilson Therapeutics has appointed Vincent Metzler vice president commercial
planning & launch strategy. Metzler is a
senior marketing and commercial operations executive with 16 years’ experience
in the pharma and biotech industries He
most recently was senior director marketing at Alexion and before this he held
various sales and marketing positions at
Miltenyi Biotec, Roche and Amgen.
Olivier Litzka, partner at Edmond
de Rothschild Investment Partners
(EdRIP) and member of the supervisory
board of Probiodrug AG, will step down
from his board position as of Sept. 12, 2016.
Chief business officer Mark Booth will also
be leaving the company Aug. 15, 2015 with
CEO of Probiodrug Konrad Glund taking
over his responsibilities.
BioLineRx Ltd. has appointed Phillip A.
Serlin CEO of BioLineRx and will be succeeding Kinneret Savitsky – effective
Oct. 10, 2016. Savitsky will serve as scientific advisor to the company until March
2017. Serlin joined the company in 2009
as chief financial and operating officer
and previously was chief financial and
operating officer of Kayote Networks Ltd.
Celldex Therapeutics Inc. has promoted
Elizabeth Crowley to the newly created
position of senior vice president, chief
product development officer. Crowley was
previously senior vice president of product
development of Celldex and joined the
company in 2009 as vice president, clinical
development. Prior to this she held various
senior level roles at CuraGen Corporation,
most recently serving as the vice president
of development operations.
Recordati has appointed Alberto Recordati chair of its board of directors and
Andrea Recordati vice chair and CEO following the death of its chair and CEO,
Giovanni Recordati. Giovanni had been
CEO of the company since 1990 and chair
of its board since 1999. Andrea, who has
been chief operating officer since 2013,
has been given the powers for the ordinary and extraordinary management of
the company.
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JOSEPH HAAS
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