August 12, 2013
TransCelerate’s comparator network
now active…2
A CenterWatch Publication
Eli Lilly, Roswell Park Cancer Institute partner in
training program for minority Principal Investigators
T
o increase the number of minority oncologists who also become Principal
Investigators, Eli Lilly and Roswell
Park Cancer Institute (RPCI) have formed a
collaborative training program that includes
a combination of workshops, mentorships,
a focus on investigator retention and annual
gatherings at major cancer conferences.
Currently, there are 10,400 U.S. oncologists, of which 95% (or nearly 9,880) are white,
and1% to 2%, or about 208, are African
American. Another 2% to 3%, or about 312,
are Hispanic.
The goal of the initiative is to train 75 to
150 minority oncologists in the conduct of
clinical trials over the next three years, according to the companies’ announcement.
“By training more oncology minority
investigators, our goal is to reach even more
diverse populations,” said Coleman Obasaju,
M.D., Ph.D., senior medical director at Lilly
Oncology. “Because medicines don’t work the
same for everyone, we need to understand
how medicines work and the safety profile in
patients likely to take them.”
Organizers of the workshops are targeting
minority physicians and senior fellows who
have cancer-related sub-specialties in medical,
hematologic, radiation, pediatric, surgical and
gynecologic oncology. The faculty will come
from those in academic hospital institutions.
Physicians working full time at an academic
institution or practice with a track record of
page 4
»
CRO ProTrials Research sees rapid growth, expansion
P
roTrials Research, a CRO headquartered in Sunnyvale, Calif., has seen
rapid growth during the past year,
adding staff and expanding its operations
in Europe due to increased demand from its
clients, which include a number of mid-sized
companies and startups in the Silicon Valley
area.
The $20 million company, which employs
140 worldwide, reported a 30% increase in
revenue during the past year, largely from
an upsurge in business from existing clients,
and has added senior-level staff members
including a director of quality assurance and
a director of project management. It also
recently established a new subsidiary in the
U.K. to provide clinical monitoring, site management and project management services
Breaking News and
Market Intelligence for
the Clinical Trials Industry
for ongoing trials in Italy, Spain and the U.K.
“A lot of CROs have struggled during
the recession, but we’ve had very steady
growth,” said Inger Arum, president of ProTrials, which she co-founded with CEO Jodi
Andrews in 1996. “We’ve grown organically
and have not taken on any outside venture
capital or debt in the last 17 years. We manage very conservatively and we focus on our
client relationships.”
As the CRO landscape has shifted over
the past five years, with large sponsors
increasingly adopting more integrated and
strategic outsourcing relationships with
top CROs, many smaller CROs have either
merged to create the scale needed to compete against large CROs for the biggest out-
Volume 17, Issue 32. © 2013 CenterWatch. All rights reserved.
page 5
»
Bill would exempt FDA user fees
from sequestration…3
Troubleshooting 101…6
Drug & Device Pipeline News…7
CenterWatch has identified 23 drugs
and devices that have entered a new
trial phase this week.
Trial Results…8
CenterWatch reports on results for
four drugs. Visit www.centerwatch.com
for real-time updates on drugs in
clinical trials.
Biotech Review…9
Biotech briefs from BioWorld Today.
CenterWatch Information Services
The CenterWatch Monthly
A monthly newsletter featuring in-depth stories
on the clinical trials industry and study lead
opportunities. Annual subscriptions start at $399.
JobWatch
A web-based service listing clinical research jobs,
career resources and a searchable resume database.
Clinical Trials Data Library
A valuable online resource providing access to
comprehensive data and charts on the life sciences
and clinical research industry.
Clinical Trials Listing Service™
An international listing service of actively enrolling
clinical trials to support sponsors and CROs in their
patient enrollment initiatives.
Market Intelligence Services
Custom surveys for organizations to gain competitive
insight into the market and their business.
Drugs in Clinical Trials Database
A searchable database of 4,000+ detailed profiles of
new drugs in development. CenterWatch also prepares
custom drug intelligence reports covering a variety of
medical conditions.
Medical Writing Solutions
Comprehensive medical writing services for the
biotechnology, medical device and pharmaceutical
industries.
CenterWatch Publications
CenterWatch publishes a wide range of CME-accredited
training guides, directories, brochures and drug intelligence
information. Visit http://store.centerwatch.com.
CenterWatch Main and Editorial Offices
10 Winthrop Square, Fifth Floor, Boston, MA 02110
Tel (617) 948-5100 Fax (617) 948-5101
[email protected] / [email protected]
CWWeekly August 12, 2013
2 of 9
Industry Briefs
Study Conduct
■■
■■
TransCelerate BioPharma’s Clinical Trial
Comparator Network initiative is now
active. Formed to establish reliable, rapid
sourcing of quality drug products for use in
clinical trials, the Comparator Network will
enable accelerated clinical trial timelines and
enhanced patient safety. In July, a master
service agreement, affirming a mutual commitment to offer secure and rapid supply
of comparator drug products for clinical
trials, was executed by several TransCelerate
member companies. The first network-based
transaction has been initiated. Currently, the
mechanisms to acquire clinical trial comparator drugs and co-therapy drugs are inefficient
and unpredictable. Unless a specific agreement is in place between biopharmaceutical
companies, trial sponsors frequently are unable to secure comparators and co-therapies
directly from each other, and therefore must
purchase on the open market. This leads to
uncertainties in obtaining an adequate and
timely supply and can result in supply disruptions. Difficulty in the sourcing of comparator
drugs can result in delays for a clinical study
and delay availability of the drug to patients.
“Locating and accessing these comparators at the right time, in the right quantities
and with the accompanying drug stability
and regulatory information doesn’t always
happen efficiently,” said Dalvir Gill, Ph.D., CEO
of TransCelerate. “Participating TransCelerate companies will be able to source these
comparator drugs directly from each other,
be able to secure supply when they need it
in the quantities they need, have access to
drug data and mitigate the risk of counterfeit
drugs in that clinical trial.”
Faculty of 1000, publisher of a range of
services for life scientists and clinicians, has
launched F1000Trials, a database to enable
rapid discovery and understanding of articles
about clinical trials. The database is a continually updated, comprehensive database of
articles on randomized controlled trials and
systematic reviews, drawn from more than
300 general and specialist medical journals.
There is an increasing need to identify those
studies that should change how physicians
treat their patients. F1000Trials extends the
system of peer-nominated experts identifying
research in biology and medicine pioneered
by F1000Prime. The F1000 faculty members
provide assessment of newly published trial
articles and write short reviews, recommending the most noteworthy by assigning star
ratings and highlighting those that change
clinical practice. F1000Trials provides links to
all publications known about a particular trial
that are included in the life science database
PubMed. Links to trial registration databases
are also included, helping to create an electronic “threaded publication trail.” F1000Trials
allows users to search all known generic and
brand names of drugs, as well as specific
studies, diseases and conditions. F1000Trials
identifies and tags articles that report negative or null results. F1000Trials currently is in
its public “beta” phase, and is free to access to
anyone who registers on the web site.
CROs/Service Providers
■■
Privately-held Oncobiologics and inVentiv
Health have partnered for clinical development of all assets in Oncobiologic’s pipeline.
The partnership initially will focus on biosimilars, and could be expanded to include
innovative molecules. inVentiv will provide
leadership and execution for clinical studies,
as well as the corresponding bioanalytical
support. As the assets are commercialized,
CWWeekly (ISSN 1528-5731)
Cheryl Appel Rosenfeld Editor-in-Chief
Tracy Lawton, Stephanie Hill Drug Intelligence
Melissa Nazzaro Advertising
Holly Rose Production
Send news submissions to Cheryl Appel Rosenfeld
Tel (617) 948-5172 Fax (617) 948-5101
[email protected]
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. the partnership will broaden to include
inVentiv’s commercialization capabilities
in select countries. The current biosimilar
assets include generic versions of Humira,
Rituxan, Avastin, Herceptin and Erbitux.
These biologics represent annual global
revenue of more than $40 billion.
■■
■■
Global CRO Icon and the National Clinical
Trials and Research Center (NCTRC) at
the National Taiwan University Hospital
have collaborated to enhance the setup and
management of clinical studies in Taiwan.
NCTRC and Icon will work together on study
startup, site feasibility, key site and investigator selection and patient recruitment.
Global CRO Clinipace Worldwide has partnered with Finland-based CRO Medfiles.
Medfiles clients gain access to Clinipace’s
proprietary technology, TEMPO eClinical
platform, and Clinipace clients gain access
to Medfiles’ local knowledge, services and
staff across the Nordics and Baltic countries.
Sponsors
■■
The Medicines Company announced
positive results of a phase III clinical trial
of ProFibrix’s lead biologic, Fibrocaps, triggering its previously announced acquisition
of the company. The Medicines Company
entered a deal in June to purchase ProFibrix
subject to its review of the results of the
FINISH-3 trial. It now has purchased all of the
outstanding equity for $90 million and will
pay up to $140 million on U.S. and European
page 3
»
© 2013 CenterWatch, LLC. All rights reserved.
No part of this publication may be distributed or
reproduced in any form or by any means without the
express written consent of the publisher. Permission
requests can be obtained via fax at (617) 948-5101 or
emailed at [email protected].
Single-user annual subscriptions are $249.
Reprints and discounted multi-reader or corporate
subscription rates are available.
Email [email protected] or call (617) 948-5100.
CWW1732
CWWeekly August 12, 2013
3 of 9
Industry Briefs (continued from page 2)
approvals and milestones, in addition to
$10 million paid by Medicines to secure the
option to acquire Profibrix. Fibrocaps is a dry
powder topical formulation of fibrinogen and
thrombin being developed to aid in hemostasis during surgery. FINISH-3 met all primary
and secondary hemostasis efficacy endpoints
in four distinct surgical indications. The results
of FINISH-3, which was conducted at 65
sites across the U.S. and Western Europe, are
expected to support a biologics license application with the FDA in 2014 and a marketing
authorization application with the EMA by
yearend. The Medicines Company anticipates
peak Fibrocaps revenues of greater than $300
million if approved in major markets.
■■
Gene by Gene, which develops consumer
DNA testing products for ancestry and genealogy applications, has acquired Arpeggi, a
developer of solutions for genome sequencing, data management and computational
analysis. The combined company will enable
acceleration of affordable genetics testing
and diagnostics services for consumers, researchers and healthcare providers. Arpeggi
has developed proprietary sequencing tools,
designed for scale, that enable accurate,
fast and cost-effective analysis of genomes.
Arpeggi will be incorporated into Gene by
Gene, and Arpeggi’s founders will join Gene
by Gene’s management team.
■■
GTCR, a Chicago-based private equity firm,
has partnered with Ed Fiorentino to form
Crealta Pharmaceuticals of Lake Forest, Ill.,
according to BioWorld Today. The company’s
PharmAthene and Theraclone Sciences, a
privately-held monoclonal antibody (mAb)
discovery and development company, will
merge. The all-stock transaction has been
unanimously approved by both boards and
is subject to shareholder and regulatory
approval. PharmAthene will issue shares of
common stock to Theraclone stockholders,
who will own 50% of the combined company. Clifford J. Stocks, CEO of Theraclone,
will be president and CEO. The company will
be a fully-integrated and diversified biologics company with four clinical-stage product
candidates targeting high-value commercial
and government markets. It will combine
vaccine and human monoclonal antibody
expertise with a focus on infectious diseases
and oncology, and will have a discovery
pipeline with four preclinical programs and
multiple discovery candidates, along with
three partnered products, including Theraclone’s collaboration with Pfizer for specific
infectious disease and oncology indications.
Ethics/Regulatory
■■
■■
maceutical Association (GPhA), Medical
Imaging & Technology Alliance (MITA),
PhRMA and trade associations praised the
effort and urged Congress to find a bipartisan solution for releasing sequestered FY13
user fees. “Sequestration is preventing the
FDA from accessing nearly $83 million in
industry-paid user fees in the current fiscal
year,” the bill’s sponsors said. “These user fees
cannot, by law, be used for any other purpose
and their sequestration does not decrease
the nation’s deficit. Preventing the FDA from
fully accessing these user fees only serves to
exacerbate the severe budgetary constraints
of a historically underfunded agency, to the
detriment of patients and public health.”
focus is acquiring specialty pharmaceutical companies and products, including
products already on the market and those
in late-stage development. GTCR may invest
up to $200 million in the new company.
Five senators have introduced a bill to
exempt future FDA user fees from sequestration. A similar bill was introduced in the
House. The Advanced Medical Technology
Association (AdvaMed), BIO, Generic Phar-
■■
In granting certain combination drugs the
five-year market exclusivity reserved for new
chemical entities (NCEs), a House bill could
make it easier for startups and other drugmakers to find investors willing to bet on the
development of combo products that include
already approved components, according to
BioWorld Today. Developing a combination
drug can be costly and time-consuming,
as the FDA requires trials demonstrating
efficacy against each of the components
separately, as well as against placebo. It
limits new combination drugs to three years
of exclusivity if any of the components has
already been approved. The bill would offer
additional incentives for the development of
new combination drugs and grant five-year
exclusivity—provided the drug application
is supported by new clinical trials, including
multifaceted efficacy studies. eCOA FORUM
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW1732
CWWeekly August 12, 2013
4 of 9
Features (continued from page 1)
Training
enrolling patients in cancer trials may also
apply to participate in the program at the RPCI
and Lilly web sites.
The workshops, called “Reducing cancer
disparities through the training of a diverse
workforce,” are slated to begin in the spring of
2014 and run through 2016. They also aim to
overcome continuing difficulties in developing
a minority community of oncologist clinical
investigators.
“Many of the participants who go to the
big cancer conferences and take workshops
that train young oncologists come from big
institutions like Memorial Sloan Kettering
and it’s very competitive, but I always felt it
would be a good idea for a workshop that
helped minorities in clinical trials and focused
on cancer,” said Alex A. Adjei, M.D., Ph.D.,
senior vice president for clinical research and
director of the Center for Drug Development
and Clinical Trials at RPCI.
The joint program launches as clinical trials
are being viewed as changing patient care, by
providing earlier access to new therapeutics.
However, ethnic and racial minorities are
grossly underrepresented in clinical trials—
even with the FDA and the National Institutes of Health encouraging the creation of
clinical trial data that better reflects real-world
diversity. African Americans are underrepresented in important medical research to find
treatments for the very diseases that affect
them, notably diabetes, cardiovascular hypertension, HIV/AIDS and lung cancer, according
to the National Medical Association. Also,
variations in genetic coding can make a cancer
“By training more oncology
minority investigators, our
goal is to reach even more
diverse populations.”
—Coleman Obasaju, M.D., Ph.D., senior
medical director, Lilly Oncology
treatment more toxic in one ethnic group than
it would be in another.
The push for training minority investigators
also is seen as a critical way to increase trial
participation by minority patients and help
them to overcome fear, mistrust of the medical
community and the burden associated with
trial participation.
“A minority patient is going to be much
more comfortable participating in a clinical trial
if he is asked by a minority physician working
in the community as opposed to someone
from outside,” said Adjei. “It’s ‘Do I really believe
that my physician has my interests at heart and
is recommending something that is best for
me or with an outside doctor is he just trying to
fill slots for a trial program and is pushing me
to accept?’ Those issues always come up.”
Despite the disparities, minority groups are
supportive of clinical trials. A national public
opinion poll in July showed within the general
population, 61% of African Americans, 57% of
Hispanics and 50% of Asians said it is important to participate as a volunteer in a clinical
trial to improve the health of others, compared
to 47% of non-Hispanic whites, according to
Research! America. But the poll also found only
about a quarter of African Americans, Hispanics
and Asians had heard about clinical trials from
their doctors or other healthcare providers.
The Lilly/Roswell program follows the shutdown of two major programs to train minority
physicians as trial investigators, due to lackluster results and loss of financial sponsorship.
The leader of Project IMPACT (Increase Minority
Participation and Awareness of Clinical Trials), a
National Medical Association project to increase
minority physician and patient involvement in
clinical trials, cited poor results due to several
barriers, including lack of awareness and lack of
access to clinical research coordinators.
“Clinical trial training programs alone are
not enough,” wrote James Powell in the Journal of the National Medical Association. “The
future also should include more attention to
the inclusion of clinical trial experiences in the
formal medical education of physicians and
greater access to clinical trials information for
both physicians and patients.”
Similarly, a Hispanic investigator training
program from the National Hispanic Research
Network reported that of the 170 novice
researchers who applied for investigator roles,
less than 5% were approved. The poor results
prompted program sponsor AstraZeneca to
cease funding.
“I think the Lilly/Roswell program is a step
in the right direction,” said Rebecca Budd,
managing director of Navita Clinical Strategy Group, which works with pharmaceutical
companies to improve their minority patient
enrollment. “But it may not make long-lasting
change in minority enrollment.”
—Ronald Rosenberg
Dirty,
dirty
data!
Shame
on
you.
>
on
3me
data.
Shamelessly
clean.
Dedicated.
Accurate.
Experienced.
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. 513.247.5500
CWW1732
CWWeekly August 12, 2013
5 of 9
Features (continued from page 1)
ProTrials
sourcing opportunities, closed their doors
or pursued niche or specialty strategies to
become more transactional providers.
Yet ProTrials has maintained steady
growth, even during the economic downturn, by remaining an independent, fullservice CRO that offers a comprehensive suite
of services and global support for phase I-IV
clinical trials to the pharmaceutical, biotechnology and medical device markets. At the
same time, the company has experience in a
broad range of therapeutic areas, including
oncology, cardiology, ophthalmology, infectious diseases and neurosciences.
While sponsors typically look to CROs
with relevant experience in their indications,
Arum believes it’s equally important for a
CRO to combine therapeutic expertise with
the ability to manage a study and deliver
high-quality data. She said ProTrials can
deliver on both fronts because of its highly
skilled teams, including project managers
who have an average of 14 to 16 years of
operational experience and monitors with
at least 10 years of experience; the company
also reports a 90% employee retention rate.
“We truly are generalists,” said Arum. “We
have a very strong operational team and that
really allows us to go in and operationally
execute almost any type of trial. We will lose
out to those companies who are looking
for therapeutic expertise or strong connections within the FDA or EMA. That’s not our
specialty. But we can work with partners or
“We truly are generalists… Our
clients don’t mind working with
multiple vendors as long as
they know the communication,
the transparency and the
accountability is good.”
—Inger Arum, president, ProTrials
alliances that do have that expertise and we
can bring the strong project management
and monitoring piece. Our clients don’t mind
working with multiple vendors as long as
they know the communication, the transparency and the accountability is good.”
Developing long-term relationships with
its sponsors, especially smaller pharmaceutical and biotech companies in the Silicon Valley area, has been key to ProTrials’ growth in
recent years. “When they are lucky and have
success, we grow with them,” said Arum.
ProTrials established its U.K. subsidiary at
the request of its small and mid-sized clients
who want to conduct multi-national and
multi-site clinical trials in Europe for access
to selected patient populations and to get
drugs to market more quickly, but who
didn’t want to work with large, global CROs.
Through the subsidiary, ProTrials Global Limited, the CRO can extend its clinical monitor-
EMBRACE THE CHALLENGE
GET THE RECOGNITION YOU DESERVE
ing, site management and project management services in Europe without having to
partner with larger CROs, giving it better
access to regulatory authorities overseas.
“It’s a way of branching out our services
to our clients beyond North America and
into a larger scope in Europe,” said Arum. “A
lot of our clients in the U.S. ended up going
with the larger CROs when they moved
outside of North America. Because of the
space we are in—the smaller, mid-sized
companies—often they weren’t seeing the
same service from us that they were in North
America. It was very frustrating for them
because they would get lost in the much
bigger environment. They kept saying we
should move into Europe, get some folks
on the ground there and start to build our
services there like we’ve done here in North
America.”
The company also has operations in
South America and Asia yet, looking ahead,
ProTrials is not planning any further global
expansion. Its growth strategy calls for bringing data management under the company
umbrella.
“We’ve worked with many data management companies and it’s worked well for
us. But having data mangers in-house will
be the next step, so that we can work more
closely with the many EDC products out
there,” said Arum. “We’ve seen a huge shift
away from paper, and it makes it simpler for
us to bring the data management in-house.”
—Karyn Korieth
US CLINICAL
RESEARCHER OF THE
YEAR COMPETITION
E N T E R N O W AT:
W W W.U S C L I N I C A L R E S E A R C H E R .C O M
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW1732
CWWeekly August 12, 2013
Troubleshooting 101
6 of 9
By Beth Harper
O
ne of the least appreciated opportunities to enhance clinical trial
performance is the area of informed
consent. Sure, we all know the informed consent
document itself is unwieldy. Numerous articles,
industry presentations and initiatives have
discussed the need to find a balance between
the legal, regulatory and ethical considerations
for ensuring the adequate informed consent of
study participants. And we all know that while
the document must be written in language
understandable to the patient, often, for myriad
reasons, it is not. A discussion of the GCP-ramifications of informed consent is beyond the scope
of this article. That said, gaining an appreciation
for why patients decline study opportunities
beyond having to deal with an intimidating
informed consent document can be instructive.
When troubleshooting the challenge of high
consent decline ratios, we can turn to our trusty
5-Why’s methodology. As this is a particularly
complex topic, it can be useful to first categorize
the reasons, or “buckets,” for why patients might
decline the opportunity to participate in a clinical trial. I have outlined five key categories:
■■ Personal or relationship-related issues
■■ Physical or safety-related reasons
■■ Emotional, educational or communication-
related issues
Financial or schedule-related reasons
■■ Other reasons
Once the core categories are mapped out,
we can continue asking “why” a patient might
decline participation within each of these
buckets. Assuming, for example, that we have
a GCP-compliant and understandable consent
form, what could drive patient willingness to
engage or not engage in the study? Are patients
concerned about study risks? Is it primarily a logistical issue, such as fitting study visits into their
work schedules? Does it have more to do with
their comfort level with the site staff? Are they
struggling to understand all of the complexities
of the trial? Or are important influencers discouraging their participation? It could be more than
one of the above. In order to start a diagnosis,
you must know the reasons. Ensuring your
screening logs capture at least some of the basic
categories above can go a long way toward
streamlining the root cause analysis.
It is essential to have a very clear picture of all
of the factors contributing to the primary study
performance issue or symptom (in this case,
a higher-than-expected number of patients
declining to participate). As part of our study
“rescue” strategy, we could put in place a very
■■
comprehensive patient education program
complete with interactive videos and other
e-consent solutions, only to find out the biggest barrier to participation has to do with the
amount of the stipend being offered or the fact
that the patients’ primary care physicians are
discouraging their participation.
Before investing in more sophisticated patient educational materials, it may be more important to re-visit the patient stipend and create
a primary physician education and engagement
program. And no intervention will be successful
if the sites are not comfortable with the protocol
or communicating the protocol. Site re-training
interventions may be necessary in this case.
The graphic is a starting point for conducting the root cause analysis. For each factor, one
could go several more layers deep. But it can
kick-start the process should you find yourself
faced with consenting challenges. Beth Harper is president of Clinical Performance
Partners, a clinical research consulting firm specializing in enrollment and site performance management. She tackles different issues, with a focus on
understanding root cause and suggesting possible
solutions, based on 30 years of experience. Email
[email protected].
5-Why’s root cause analysis
Patients may also include caregivers, family members or other important “influencers”
“Influencers” discouraging participation
Personal/relationship
Not comfortable with person conducting consent
Other study or treatment options more attractive
Physical/safety
Concerns about product safety or placebo
Patients
declining
study
opportunity
Emotional/educational/
communication
Patient doesn’t understand or isn’t
comfortable with study expectations
Visits, procedures burdensome, inconvenient
Financial, schedule
Can’t afford travel to site or stipend not sufficient
No transportation to site
Other
Informed consent
too long/confusing
Supplemental
educational materials
don’t exist or not
patient-friendly
Site personnel
not comfortable
communicating
protocol
Sites not adequately
trained on protocol
Sites have not rehearsed
conducting consent
Materials/consent
not provided in
native language/
poor translation
Lack of care transition or follow-up treatment
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW1732
CWWeekly August 12, 2013
7 of 9
Drug & Device Pipeline News
Company
Drug/Device
Medical Condition
Rebiotix
RBX2660
ImmunoGen
AllaChem
IMGN289
AV4025
Cleveland BioLabs,
Incuron
CBL0137
recurrent Clostridium
IND approved by the FDA
difficile infection
tumors
IND approved by the FDA
chronic hepatitis C infection Phase I trials initiated enrolling 30
subjects
metastatic or unresectable Phase I trials initiated in the U.S.
advanced solid cancers and
lymphomas
Rexahn
Pharmaceuticals
ImmunoCellular
Therapeutics
gIcare Pharma
RX-5902
solid cancer tumors
Phase I trials initiated
ICT-121
Phase I trials initiated in the U.S.
RedHill Biopharma
RHB-104
recurrent glioblastoma
multiforme
opioid for colonoscopy
patients
rheumatoid arthritis
OncoGenex
Pharmaceuticals
Karyopharm
Therapeutics
Ultragenyx
Pharmaceutical
Aeterna Zentaris
OGX-427
non-squamous non-small
cell lung cancer
oral SINE Selinexor advanced soft tissue or
(KPT-330)
bone sarcomas
triheptanoin
glucose transporter type-1
deficiency syndrome
zoptarelin
endometrial cancer
doxorubicin
Phase II trials initiated enrolling
155 subjects in the U.S.
Phase IIb trials initiated
Phase III trials initiated enrolling
500 subjects in North America,
Europe and Israel
(908) 626-5428
www.aezsinc.com
Biotest
Civacir
www.biotest.com
Sanofi Pasteur
Clostridium
difficile vaccine
Clotbust ER
Phase III trials initiated enrolling 90
subjects in the U.S. and Canada
Phase III trials initiated enrolling
15,000 subjects globally
Phase III trials initiated enrolling
800 subjects globally
Phase III trials initiated enrolling
840 women globally
Orphan Drug designation granted
by the E.C.
E.C. approved
Cerevast
Therapeutics
Nektar Therapeutics
GIC-1001
post-transplant hepatitis
C virus
Clostridium difficile
infection
acute ischemic stroke
Vital Therapies
etirinotecan pegol metastatic breast cancer
(NKTR-102)
C3A cells
acute liver failure
Aegerion
Pharmaceuticals
Takeda
Novartis
LOJUXTA
(lomitapide)
Nesina (alogliptin)
Menveo
Oncos Therapeutics
CGTG-102
homozygous familial
hypercholesterolemia
type 2 diabetes
pediatric meningococcal
disease
soft tissue sarcoma
Cangene
IB1001
hemophilia B
Convergence
Pharmaceuticals
CNV1014802
trigeminal neuralgia
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. Status
Phase IIa trials initiated
Phase IIa trials planned
Phase II trials planned
Approved in China
FDA approved indication
expansion
Orphan Drug designation granted
by the EMA
FDA hold lifted
Orphan Drug designation granted
by the FDA
Sponsor Info
www.rebiotix.com
www.immunogen.com
http://allachem.com/
(716) 849-6810
www.cbiolabs.com
+7(495)974-74-01
[email protected]
(240) 268-5300
[email protected]
(818) 264-2300
[email protected]
(514) 508-7014
[email protected]
+972 (0)3 541 3131
[email protected]
(425) 686-1500
www.oncogenex.com
(508) 975-4820
http://karyopharm.com
(415) 483-8800
[email protected]
(800) 622-0724
www.sanofipasteur.us
(425) 821-1939
[email protected]
(415) 482-5300
[email protected]
(858) 673-6840
[email protected]
(855) 305-2347
[email protected]
www.takeda.com
(888) 669-6682
www.us.novartis.com
[email protected]
(204) 275-4200
www.cangene.com
+44 (0)1223 755 501
[email protected]
CWW1732
CWWeekly August 12, 2013
8 of 9
Trial Results
Gynecology
■■
Pharmanest released results of a phase II
study of SHACT for the treatment of pain
in connection with intrauterine device
insertion. SHACT is a product based on a
formulation of lidocaine, an anesthetic, and
a proprietary application device developed
to simplify topical application in the cervix
and uterus. The randomized, double-blind
trial involving 218 women between ages
18 and 45 showed women receiving SHACT
during IUD insertion experienced a more
than 30% reduction in pain, measured on a
visual analogue scale, compared to patients
who received placebo. This effect was
statistically significant (p < 0.0001). Patients
who received SHACT also experienced less
discomfort (p < 0.05) than women who
received placebo. Women who received
SHACT reported similar adverse events, in
terms of type and frequency, as women
who received placebo treatment.
Oncology
■■
GlaxoSmithKline issued results of a randomized, double-blind, phase III, placebocontrolled trial of pazopanib monotherapy
in women with epithelial ovarian, fallopian
tube or primary peritoneal cancer whose
disease had not progressed after completing
standard debulking surgery and first-line
chemotherapy. After completion of five or
more cycles of platinum-taxane chemotherapy, 940 patients were randomized 1:1
to receive 800mg pazopanib once daily
or placebo for up to 24 months (median
time from diagnosis to randomization was
approximately seven months). Pazopanib
treatment reduced the risk of disease
progression or death by 23% (HR = 0.77;
95% CI: 0.64-0.91; p = 0.0021). The incidence
of serious adverse events was higher in the
pazopanib group compared to the placebo
group (26% v. 11%). Regulatory applications
will be submitted before 2014.
■■
Immunomedics reported results of a phase
Ib study of 90Y-labeled-clivatuzumab
for the treatment of metastatic pancreatic
cancer in 58 patients who had received at
least two prior treatments. Patients were
randomized to receive either 90Y-labeledclivatuzumab once a week for three weeks at
6.5mCi/m2 with gemcitabine 200mg/m2
given weekly for four weeks (Arm A) or
90Y-labeled-clivatuzumab alone (Arm B).
This treatment cycle was repeated every four
weeks until unacceptable toxicity, patient
deterioration or patient withdrawal. Patients
were followed for one year or until death.
The median overall survival (OS) for Arm A
(N=27) was 119 days, an improvement over
Arm B (N=26), with a median OS of 80 days
(P=0.04). For the 23 patients who received
multiple cycles of therapy, the median OS increased to 157 days in Arm A compared with
103 days in Arm B. Survival also was related
to patients’ Karnofsky Performance Status
(KPS) scores at study entry, increasing from a
median of 79 days for patients with 80% KPS
to 119 days for patients with 90% to 100%
KPS. In contrast, increased number of prior
treatments is a negative prognostic indicator
for survival. The median OS decreased from
90 to 82 to 73 days for patients who received
2, 3 or 4 to 5 prior treatments, respectively.
Phase III trials are planned for 2013 or the
beginning of 2014 in the U.S. and the E.U.
Respiratory Disease
■■
Novavax released results of a phase I trial
of its respiratory syncytial virus (RSV)
vaccine candidate in 220 healthy elderly
adults (age 60 or older, with a mean age
of 68). All subject groups receiving the
recombinant fusion (F) protein nanoparticle vaccine candidate exhibited antibody
responses against RSV at 28 and 56 days
post-immunization, with risesin serum antiF immunoglobulin G (IgG) antibody levels.
Subjects received either 60μg or 90μg of the
RSV F vaccine candidate, with or without
adjuvant, or a placebo. The overall immune responses were greater in the groups
receiving the 90μg dose of RSV F vaccine
compared to the groups dosed with 60μg.
The 90μg RSV F adjuvanted vaccine group
experienced a 5.6-fold rise in anti-F IgG
and a sero-response rate of 79% at day 56.
Subjects receiving 90μg RSV F vaccine with
adjuvant reached levels of competitive antibodies equivalent to 186μg/mL of palivizumab. Levels of antibodies specific for the
antigenic site II peptide, representing the
neutralizing epitope on the RSV F protein
recognized by palivizumab, rose 5.6- to 12.5fold, with best responses again in the 90μg
RSV F adjuvanted vaccine group. Job seekers know JobWatch!
• 17k+ registered clinical research professionals
• Nearly 8k searchable candidate resumes
• 25k+ avg. unique monthly visitors
• 80k+ avg. unique monthly page views
• 40k+ avg. monthly job searches
• 7k+ social media followers
Post your jobs, search candidate resumes and more!
Contact [email protected], (617) 612-5101.
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. centerwatch.com/jobwatch
CWW1732
CWWeekly August 12, 2013
9 of 9
Biotech Review
■■
■■
Biopharma dealmaking was up 39% during
the first half of 2013, with 368 licenses and
joint ventures (JVs) compared to 265 during
the first six months of 2012. Thomson
Reuters Recap captured 1,234 life science
deals during the first half of the year, with
licenses and JVs representing the largest portion, at 30% of the total. With 152
disclosed deals, biopharma mergers and
acquisitions (M&As) represented 12%, while
78 asset purchases made up 6% of transactions. Recap reported a total deal value
of $73.3 billion based on a fraction of the
transaction universe: 277 deals with publicly
disclosed terms, excluding expansions of
existing deals. Dollar values were heavily
skewed toward M&As—$52.6 billion, or
72%. Licenses and JVs represented $9.4
billion, or about 13%, while asset purchases
were $7.1 billion, or about 10%. Licensing
and JV deals through June suggested 2013
will far outpace 2012, which saw only 565
deals, down from 662 in 2011. But disclosed
deal values were lower than in the previous two years, leveling out at an aggregate
value of about $4.7 billion per quarter
compared to average aggregate values of
$7.8 billion per quarter in 2011 and $5.8
billion per quarter last year. Although early
to mid-stage partnering remained strong,
the number of licensing deals for discovery
assets increased 13%, while lead/preclinical
asset deals grew 71% and phase I transactions spiked 77%. Cancer dominated
licensing trends, accounting for 36% of 271
licenses for which the therapeutic area was
disclosed, compared to 33% during the first
half of 2012. Neurology/CNS were 15% of
deals, followed by infectious disease at 10%,
autoimmune/ inflammatory at 9% and endocrine/metabolic at 6%. The average total
potential deal size inched up 3%, to $229
million, from $223 million in 2012.
The Australian Therapeutics Goods
Administration (TGA) in its latest guidance
on follow-on biologics made it clear there
would be no automatic substitution for
biosimilars. Despite their “conceptual parallels,” biosimilars are in no way, shape or form
to be confused with generics. The guidance,
modeled largely on European guidelines
for evaluating biosimilar applications, shuts
the door on interchangeability—a comparability standard many biosimilar sponsors
are aiming for in the U.S. as it could allow
pharmacists to automatically substitute a
cheaper follow-on for a prescribed brand
biologic. Such substitution would make it
easier, and cheaper, to market a biosimilar.
To make sure prescribers and patients
understand biosimilars are not generics,
the TGA also expects sponsors to include
a paragraph in the product information
indicating the level of comparability shown
isn’t sufficient to designate a biosimilar as
a generic version of the reference biologic.
And while biosimilar sponsors may be able
to extrapolate safety and efficacy data for
other indications in certain cases, they will
generally be required to justify or demonstrate the data separately for each claimed
indication. When it comes to post-registration requirements and pharmacovigilance,
sponsors must develop a comprehensive
risk management plan (RMP) outlining its
pharmacovigilance procedures.
■■
A study by the Coalition of Small Business
Innovators (CSBI) showed several proposed
changes designed to help small R&D-intensive startups better use existing tax provisions and incentivize investment in small
business innovation would have the dramatic effect of increasing total investment
in small businesses by $20.6 billion. In the
second quarter, private biotechs developing
therapeutics raised almost $1.3 billion—a
massive 190% increase over the amount
raised in the first quarter and a 153% jump
over the $510 million raised in the second
quarter of 2012. But many biotech execs
feel venture capital is not flowing as fast as
it should. According to BioWorld Snapshots,
© 2013 CenterWatch. Duplication or sharing of this publication is strictly prohibited. in July global private biotechs raised just
over $300 million from 17 deals, compared
with the $325 million from 13 deals in the
same period last year, an 8% decrease. For
the five-year period from 2006 to 2010, the
average amount of private capital raised in
July was $421 million; the average amount
raised in July for the past three years has
dropped to $311 million. The dramatic fall
is a reflection of a VC industry consolidation
and their smaller fund sizes. CSBI would like
to change current tax incentives to encourage spending on R&D. CSBI consists of 17
organizations, including BIO, dedicated
to stimulating sustained, private investment in small, highly innovative companies
developing new technologies. Its study
considered the economic impact of three
potential tax changes: An R&D partnership
structures proposal allowing qualifying
startups to raise money for specific projects,
and their investors would be able to use
tax losses and credits generated by those
projects on a current basis; reform net
operating loss restrictions allowing growing
biotechs to maintain the value of their NOLs
during transactions; extend and expand the
qualified small business stock provision to
permanently extend the 100% capital gains
exclusion from the sale of qualified small
business stock. The report said if enacted together, the three proposals would increase
total private sector R&D spending by 6% in
the long run, and would increase total R&D
investment by $20.6 billion, resulting in an
estimated 623,000 jobs. The stories included in Biotech Review
have been provided to CenterWatch with
full permission of BioWorld Today and its
publisher, AHC Media LLC, 3525
Piedmont Road, Building 6, Suite 400,
Atlanta, GA 30305. Call (800) 688-2421
or (404) 262-5476 for more information,
www.bioworld.com.
©2013 AHC Media LLC
CWW1732