13 June 2011 CenterWatch Weekly Strategic Partnerships - ICON plc

June 13, 2011
Breaking News and
Market Intelligence for
the Clinical Trials Industry
A CenterWatch Publication
Strategic partnerships: a good deal for CROs,
or just a cost-cutting measure by big pharma?
Strategic partnerships with the top 20
pharma companies are what most sizable
CROs dream of these days.
And recent moves in the market reflect
just that, with mid-tier CROs either buying
one another or being bought by investment
groups almost every week. The strategy: get
large enough that the big pharma companies
can’t ignore you when it’s time to
choose new strategic partners to which they
will funnel the lion’s share of their studies.
Such partnerships are part of a movement
among big pharma to restructure operations
in an effort to show shareholders they can
be efficient and profitable, according to John
Kreger, a financial analyst with William Blair
who has been scrutinizing the clinical research
outsourcing space for almost 20 years.
As sponsors and CROs feel the
pressure to improve clinical trial
efficiency, increase enrollment and
maintain a solid bottom line, it’s critical
that they avoid information overload while
measuring the right performance indicators
as data from multiple sources is streamlined.
That’s the tall—and growing—order
of clinical trial portals, now used by study
managers, data managers and medical, safety
and regulatory staff to help plan, conduct,
manage and review trials.
“The idea of a clinical trials portal has been
around, but only now are people understanding
the necessity,” said Susan MH Lewenz,
“There’s a tremendous amount of cynicism
among stockholders that internal R&D
infrastructure is completely broken and dysfunctional
and inefficient, and they put tremendous
pressure on management to fix it
and downsize,” he said. “The big 20 pharma
companies have been almost in a state of
panic about restructuring their model, and
outsourcing is being seized upon as a great
way to cut unnecessary R&D spending and
get more efficient.”
Enter the strategic partnership agreement,
in which a very small handful of CROs
are chosen to do most, if not all, of a drug
maker’s R&D work for a specific, usually
long-term time frame. The pharma company
chooses the best CROs for the job(s), then
page 4 »
More sites turn to clinical trials portals, dashboards
to improve management and communication
CEO of Axxiem Web Solutions, a Hastingson-Hudson,
N.Y. company that provides a
secure way to efficiently distribute and track
time-sensitive documents and training materials
to users around the world.
The company’s AxxiTRIALS global portal—
a secure, customizable, private web-based
platform—acts as a central point of communications
for all members of a trial. It offers a
central space for posting patient recruitment
metrics and sharing best practices to create a
sense of community between investigators,
study coordinators and sponsor/CRO project
management teams involved in the trial.
page 5 »
Two researchers indicted
for falsifying data…2
Parexel details early-phase cuts…3
The Pulse on Recruitment…6
Drug & Device Pipeline News…7
CenterWatch has identified 15 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.
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Volume 15, Issue 24. Copyright © 2011 by CenterWatch. All rights reserved.

CWWeekly June 13, 2011 2 of 9
Industry Briefs
Study Conduct
■■
■■
Two former employees of Kansas-based
Lee Research Institute have been indicted
for falsifying study data in a clinical trial,
according to the Kansas City Business Journal.
Dr. Wayne Spencer, 73, and Lisa Sharp, 48,
were charged with conspiracy, mail fraud
and falsifying information to the FDA.
Institute officials said they terminated
Spencer and Sharp after learning of their
alleged actions in June 2010. They then
conducted an internal investigation and
cooperated with both the FDA and sponsor
Schering-Plough. “The company has been
advised by the investigating officers that no
wrongdoing of any sort has been found on
the part of the company,” the institute said in
a statement. Schering-Plough, a subsidiary
of Merck, contracted with Lee Research
last year to do clinical trials on a new allergy
drug. Spencer was the P.I. and Sharp was
lead CRC and director of clinical trials at Lee
Research. The study called for subjects age
50 or older suffering from ragweed-induced
allergy symptoms, and prohibited employees
of Lee Research from participating. The
indictment alleges two of the eight subjects
were institute employees and were given
false names and birth dates to hide that they
were younger than 50. It also claims the pair
falsely reported the ineligible subjects had
physical examinations, gave false statements
to the FDA and tried to hide the subjects’
ineligibility by scheduling required office
visits when the executive director was out.
Schering-Plough paid the institute $30,000
for the study. If convicted, Spencer and
Sharp could face up to five years in federal
prison and $250,000 in fines for conspiracy;
20 years and $250,000 for each mail fraud
charge; and three years and $10,000 for
providing false information to the FDA.
The University of Geneva (UNIGE), Geneva
University Hospitals (HUG) and the Swiss
Institute of Bioinformatics (SIB) have
formed a strategic collaboration with Roche
in translational medical research. The initial
focus will be on basic research, bioinformatics
and biomarker development in cardiology,
hematology, pathology and applied
human toxicology. Translational medical
research translates basic scientific research
into medical practice with the ultimate
objective to deliver personalized healthcare
to patients. Roche has established such collaborations
with distinguished universities in
the U.S., Asia and Europe.
Technology
■■
■■
Medidata Solutions faces a patent infringement
lawsuit from smaller competitor
Datatrak International. Cleveland-based
Datatrak sued Medidata for alleged unauthorized
use of its intellectual property. Datatrak
said Medidata’s Rave product infringes
on its patent for “methods and systems for
unifying data from a plurality of heterogeneous
databases with each having businesscontext
related data and a data access
mechanism.” According to FierceBiotechIT,
the suit comes as New York-based Medidata
continues to distance itself from competitors
in the electronic data capture (EDC) business.
Its customer base has grown from 92 in 2008
to 232 at the end of the first quarter of 2011,
as more drug developers and life sciences
companies use its web-based system for
managing data in clinical trials. Datatrak
seems poised for a legal battle with its larger
competitor. “After months of attempting,
with little success, to engage proactively
with Medidata and its counsel, it has become
clear that the only way we will be able to
obtain any relief from this apparent infringement
is through litigation,” Laurence Birch,
chairman and CEO, said in a statement.
Boston, Mass.-based Radiant Sage, a provider
of on-demand clinical trial image management
solutions, has officially launched.
The company has developed Software-as-a-
Service (SaaS) clinical trial imaging management
solutions that address clinical trial
sponsors’ needs for rapid trial starts, increased
efficiency, the ability to retain control of trials,
real-time access to and integration of images,
complete transparency and elimination
of capital infrastructure investments. “We
believe this will revolutionize the way images
are used in drug discovery and research,” said
Chander Jain, vice president of development
and services. Core-Lab-in-a-Box and RadVista
Viewer enable rapid trial start and increased
overall efficiency of the collection, distribution
and processing of images, and require no
capital infrastructure investments.
R&D Trends
■■
The Ministry of Health and Social Development
of the Russian Federation (MoH)
approved 95 new clinical trials of all types
including local studies during the first quarter
of 2011, 29% less than in the year-ago
quarter, according to a quarterly analytical
report produced by Moscow-based CRO
Synergy Research Group (SynRG). The
quarterly report on Russia’s clinical trial
market is part of SynRG’s goal to improve the
research attractiveness of Russia for foreign
sponsors. Of the 95 new studies, 74 were
multi-national, multi-center clinical trials,
down 11% from the first quarter of 2010.
The number of local clinical trials conducted
page 3 »
CWWeekly (ISSN 1528-5731)
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CWWeekly June 13, 2011 3 of 9
Industry Briefs (continued from page 2)
in Russia by domestic and foreign sponsors
was also down, from 36 to 18. The trials were
sponsored by companies from 15 countries:
22 were initiated by U.S. sponsors; Russian
sponsors had 19 studies; Swiss sponsors had
15; and U.K. companies had 10. The number
of subjects planned for enrollment in phase
II-IV trials was 11,123 subjects, 15% less than
a year ago. GlaxoSmithKline is sponsoring
eight new studies, while Novartis has six new
trials, AstraZeneca and Roche each has five,
and Sanofi has four. Of the new studies, 74%
were in five therapeutic areas: 29 were initiated
in Oncology; 12 in Respiratory diseases;
11 in Endocrinology; seven in Cardiovascular
diseases and six in Musculoskeletal diseases.
CROs
■■
Parexel has revealed details of its previously
announced plan to cut early-phase capacity
by 30%. It is closing four facilities and
downsizing another five in the restructuring,
according to Outsourcing-Pharma. Parexel
believes there are irreversible changes in the
sector. Biopharm is focusing on making an
early assessment of proof-of-concept and
then going back to do other work on the
most promising compounds. Adoption of
this strategy has resulted in fewer studies
per molecule and overcapacity in the
early-phase sector. At its nine sites in Europe,
the U.S. and South Africa, the measures will
cut the bed count by 28% and cut about
300 jobs. Global overcapacity has led to
competitors offering 20% discounts, said
Mark Goldberg, Parexel’s COO. The company
■■
said making the cuts and focusing resources
on growth in early-phase areas such as trials
in patients will help return the unit to profitability.
Up to one-third of new early-phase
business at Parexel is for patient-healthy
volunteer hybrid work, said Goldberg, and
the company plans to refine its early-phase
business to “straddle phase I and II.” Expanding
relationships with external early-phase
sites is one strategy. Parexel seeks to keep a
solid presence in early phase, as it views the
unit as important to being a full-service provider.
Parexel referred to increased interest in
early-phase strategic partnerships and needs
to keep scale to compete for this work.
Quintiles has begun its Egyptian contract
sales and marketing partnership, further
expanding the firm’s presence in what
it says is one of the world’s most rapidly
expanding regional markets, according to
Outsourcing-Pharma. The joint venture, announced
last October, teams Quintiles with
Egypt’s Kare Pharma International (HKPI)
and Ramco Import and Export to create a
contract marketing organization. Quintiles
will bolster Ramco’s 40-member sales team
with marketing capabilities developed by
its global teams. The local knowledge its
partners provide is key, said Jim Feathersone,
Quintiles’ vice president of product
development and commercialization. “We
believe we can provide the best solutions
for our customers by partnering with bestin-class,
local companies that have intimate
knowledge, experience and expertise in
delivering commercial solutions locally.”
■■
■■
Cetero Research has signed an earlystage
preferred provider agreement and
is eyeing other deals as sponsors look to
extend the partnership model into phase
I, according to Outsourcing-Pharma. Most
strategic relationships with CROs focus on
phase II to IV clinical trials, but the model
is now extending into early phase. With
1,500 early-phase beds in North America,
Cetero is well placed to enter into these
deals. With a large network and considerable
experience, Cetero is confident of its
position. A number of large CROs have
made early-phase cuts and spoken of
overcapacity. But Cetero CEO Troy McCall
said Cetero has fared better because of its
focus on phase I.
U.S.-based Aptiv Solutions, a new
adaptive clinical trial-focused CRO, has
officially begun operations, according to
Outsourcing-Pharma. The organization is
a conglomerate formed in February by
six CROs: Averion International, Trio
Clinical Research, ClinResearch, Niphix,
Addplan and Fulcrum Pharma. The
firm’s approach is to combine software
and expertise in the execution of adaptive
clinical trials, specialized medical device
development, a dedicated focus in Oncology
and the flexibility of an international
CRO. Speaking at the official launch, CEO
Patrick Donnelly said, “Aptiv Solutions
is the only CRO to offer design, simulation
and execution of adaptive clinical
trials—and the only partner to make fully
functional adaptive trials a reality.”
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CWWeekly June 13, 2011 4 of 9
Features (continued from page 1)
Partnerships
works only with them. The CROs receive a
steady stream of work.
But are these arrangements actually good
for the CROs chosen? According to a report by
William Blair analysts about Pfizer’s recently
announced strategic partnerships with Parexel
and Icon, during their ramp-up phase,
these deals can actually impair CROs a bit.
“Given the size and complexity of the
implementation, start-up costs and lack of
near-term revenue as Pfizer transitions away
from its broad list of other clinical CRO vendors,
we would not be surprised if this award
pressures [Parexel’s] and [Icon’s] margins in
the short term,” the report said.
But once fully operational, the analysts
predicted those particular deals should be
profitable. The report estimated the agreement
could increase Parexel’s revenue and
earnings base by roughly 11% to 12%, and
Icon’s base by 13% to 15%.
Still, some critics say these deals may seem
like prizes but in reality they force the CRO to
offer painfully deep discounts in exchange
for a steady flow of work. August Troendle,
co-founder, president and CEO of Medpace,
is one such critic. Medpace, which just sold
an 80% stake to CCMP Capital Advisors,
does not jockey for strategic partnerships or
preferred provider status with big pharma.
“Frankly, we don’t have to sit down at the
table and talk about the 15% to 18% price
reduction that a preferred provider has to
do,” said Troendle. “I’d rather be getting the
work that’s the exception to their preferred
provider lists than to be on the preferred
provider lists.”
Instead, he bucks the trend of CROs
scrambling to buy one another and grow big
enough to attract the attention of large sponsors.
“All these companies line up to get revenue
from large pharma, and it’s very compelling
to them, but they pay a significant price.
Look at INC and Kendle and PharmaNet—all
of them will be left at the lower end of the
majors and have tremendous difficulty getting
on those lists, and at great price concessions,
while having to integrate acquisitions.”
William Blair analysts alluded to such
price concessions in their report on the
Pfizer deals, saying, “... there are risks that go
along with working with a sponsor with the
purchasing muscle of Pfizer.”
Parexel now has four announced strategic
partnerships, as well as some that are not
public. Founder, president and CEO Josef von
Rickenbach said the negative and the positive
of these deals seem to even out.
“We’ve done comprehensive analyses on
the quality and benefits of strategic partnerships,
and for us it hasn’t worked out any
worse than regular business from a profitability
point of view,” he said. “I don’t believe
these deals are going to have profitability issues.
I also don’t believe they will boost your
profitability miraculously. But they do really
have a lot of cost advantages that normally
wouldn’t be there. For instance, you don’t
have to go through this bidding all the time,
which is very expensive.”
Said Peter Gray, CEO of Icon—which also
now has four public partnership deals in
place as well as some that are not public—
rendering these tight arrangements profitable
can be done, it just takes effort, focus
and patience.
“We believe the process of making these
relationships work in a way that’s profitable
takes time, but the whole premise behind
these deals is that you align all your processes,
and build and grow together, and the
way you begin to interact soon enables CROs
to be profitable and also allows the client to
get good value for their money,” said Gray.
Those who criticize these deals just may
be resentful about not being invited to the
party, he added. “Perhaps there are some
sour grapes involved. But they are failing to
recognize that the pharma industry is seeking
to transform itself, and you can either
choose to be a part of that or you can stand
on the sidelines and wail about it happening.
We’ve chosen to get in the game.”
Said Kreger, more deals are definitely in the
works. “This is the direction the industry is moving,
and we’re still in the early innings; there are
more pharma companies that don’t have these
agreements than those that do,” he said.
Who might be next to choose CRO strategic
partners? Amid wide speculation, Kreger said
many are eyeing Merck, which, having bought
Schering-Plough in 2009, now seems ripe for
a strategic outsourcing marriage or two.
—Suz Redfearn
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CWWeekly June 13, 2011 5 of 9
Features (continued from page 1)
Portals
For drug developers, Lewenz said, portals
save time and resources by distributing documents
digitally and dispersing notifications
globally, sharply reducing stacks of paper
while also enabling global compliance that
can include automatic reminders. Comparative
enrollment and retention statistics are
highlighted to motivate recruitment, along
with alerts, notifications and the availability
of training materials.
For investigators, the portal engages and
motivates them by creating a private online
community that can include such social features
as a sponsor-monitored blog and forums
to enable good multi-level communications.
“A well-designed clinical trials portal
accounts for the human psyche in order to
motivate users,” Lewenz wrote in a recent blog.
“Certainly money can be a good motivator,
but it must be used to promote quality results.
Being part of a community, part of something
that really matters, and being recognized as
an individual and a member of an important
team—those are very powerful motivators.”
Similarly, other developers of clinical
trial portals point to improved communication
and collaboration among study sites
dispersed geographically, which can reduce
errors at individual sites.
“Clinical trial portals are like any other
technology—it is wonderful when it is used,
but one of the limitations in this industry is
that it has been slow to change things and
no one wants to change unless 10 others do
it first,” said Michael Rosenberg, M.D., CEO
and founder of Durham, N.C.-based Health
“Being part of a community, part
of something that really matters,
and being recognized as an
individual and a member of an
important team—those are very
powerful motivators.”
—Susan MH Lewenz, CEO,
Axxiem Web Solutions
Decisions. The adaptive trials CRO is committed
to shortening development timelines and
maximizing pipeline value for pharmaceutical,
biotech and medical device companies.
Acknowledging that portals are finally
catching on, Rosenberg said the major drawbacks
are on the data entry side, in which
a CRO or sponsor needs one or two staffers
to transcribe hand-written materials—a
process that can result in errors and interrupt
workflow. His company conquers that
limitation in several ways, including providing
staffers with digital pens that act like
cameras and capture key strokes, which they
convert to letters and numbers.
For sponsors, the benefit of potentially
more information available in one place is
currently offset by relatively little information
from all the data—a situation that will change
as technology improves, Rosenberg said.
For patient volunteers, some portals offer
a section in which they can discuss the disease
and treatment experience, which can
help boost compliance.
At an industry conference last year, Rosenberg
estimated about 20% to 30% of CROs
and sponsors are using clinical trial portals.
“The need to exchange information and
communicate at multiple levels given the
time-consuming and complex nature of this
[clinical trial multi-step] process, as well as
trials becoming multi-center, the need for a
system with centralized accessibility at every
level, is the need of the hour,” noted Global
Industry Analysts in its recent report on
Clinical Trial Management Systems.
Already, a wide range of clinical
trial technology companies are in various
stages of developing and marketing clinical
trial portals and dashboards. Much like the
gauges and displays in a car or an airplane
enable navigation, clinical trial dashboards
enable the project manager, along with the
data manager, medical monitor and CRA, to
view a snapshot of the data relevant to their
functions in the clinical trial process.
“Each member of my team has a dashboard
with data that relates to their particular
role,” said Wendell Guthrie, Chief Operating
Officer of Evofem, a small, privately
held reproductive and personal healthcare
products company for women in La Jolla,
Calif. The dashboards were developed using
Health Decision’s HD360° Clinical Management
platform. “They [team members] no
longer have to wait for a report, and then
mine the data for their particular needs. The
HD system allows them to react and communicate
faster and more efficiently because
they have the data in minutes, not weeks.”
—Ronald Rosenberg
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CWWeekly June 13, 2011 6 of 9
The Pulse on Recruitment By Cara Danchak
When developing a patient
recruitment radio or television
commercial, there are
many factors to consider. You want the
ad to be effective and deliver a strong
response, so it’s easy to get somewhat
caught up in the content. Will this message
resonate with audiences? Have you
clearly explained the trial and the purpose
of the ad? Does the ad mention the
benefits of trial participation? If so, you
have prepared an effective recruitment
ad. However, if you have overlooked the
media’s rules and regulations, there is
a very good chance your ad will never
make it to the airwaves.
TV and radio stations in the United
States certainly are not strict, compared
to many other countries. However, they
do have standard commercial guidelines
that must be followed. Failure to follow
these regulations results in exclusion
from their stations until new ad copy
is produced and approved. Given the
lengthy turnaround of the IRB process,
revising ad materials is not a quick solution.
Therefore, it’s imperative that study
teams understand media requirements
from the beginning so that proper ad
materials are developed.
According to the Code of Federal
Regulations (CFR), the Federal Communications
Commission (FCC) has mandated
that advertisements include sponsorship
information. This means an advertiser must
be mentioned in clinical trial patient recruitment
ads, as a means of identification. The
first solution would be to include the name
of the trial sponsor, the pharmaceutical
company. However, most sponsors are not
inclined to do this, as they tend to prefer anonymity
in patient recruitment campaigns.
One way around this is to include the names
of the participating sites in the ad, since
they are the ones actually conducting the
research. This inclusion satisfies the media’s
requirement of sponsorship identification
On the air
The following is an example of a radio ad produced by
Clinical Trial Media for a Stress Urinary Incontinence
trial in 2009:
Attention Women:
Does exercising, coughing, sneezing, laughing or
any other sudden movement cause you to lose
bladder control?
It may be Stress Urinary Incontinence—and you
are not alone… so please listen closely to the
following message.
A research study is being conducted in our area
to examine an investigational medication for
the treatment of Stress Urinary Incontinence. To
qualify, you must be a woman over the age of
18 and experiencing symptoms of Stress Urinary
Incontinence.
Qualified participants will receive all study-related
care and medication at no cost and may also be
compensated for time and travel. Health insurance
is not needed to participate.
So if you’ve ever thought you just had a “weak
bladder” because you lost control during coughing,
sneezing or other sudden movements, you
may have Stress Urinary Incontinence. Call (123-
456-7890) to see if you qualify for this study, now
being conducted in our area.
Call (insert site name here) now at (123-456-
7890).That’s (123-456-7890).
and also pleases the sites, as it gives them
some acknowledgment in the ad.
The media also will pay close attention
to the subject of your ads. What is the
study indication? What are the symptoms
you will be discussing? If it’s a sensitive
ailment, something personal or private, the
media expects ad materials to be equally
sensitive. Broadcast TV and radio stations
definitely will pull your ad if it’s found to be
offensive or vulgar.
For example, a couple of years ago I was
working on a trial for Stress Urinary Incontinence.
Prior to IRB approval, we ran the ad
copy by several stations for feedback,
knowing this was a sensitive topic. Of
five stations, only one would accept our
copy. Based on this, we modified the
ad with alternative verbiage that was
slightly more vague, yet still effective.
If we had not done this due diligence
first, the ads would have gone through
IRB approval only to be rejected later by
the media.
Finally, the media enforces very strict
limitations on ad lengths. Whatever the
ad length (30 seconds, 60 seconds, etc.),
it is absolutely imperative that your ad
is timed exactly to meet that length. Far
too often, clients come to me with ad
scripts that fill 50 seconds and ask if we
can read it slowly and “stretch it out” to
a full minute. This is never a good idea.
First, why waste 10 seconds that could
potentially contain valuable information?
Second, if the copy is read too
slowly, the ad will not sound good and
the media may not run it simply because
it will annoy their audiences. Similarly,
if an ad is too long and read very fast to
meet the time restriction, the media also
may make you redo it. Stick exactly to
the time length required.
The media has an obligation to protect
their audiences by maintaining the
quality of the content of their broadcasts.
By understanding and following
their guidelines, you will save yourself a
lot of time, as well as money, and hopefully
your ads will lead to a successful recruitment
campaign.
Cara Danchak is a vice president at Clinical Trial
Media, a global patient recruitment and retention
company. She has spent nearly a decade
working with various companies in the patient
recruitment space including sponsors, sites,
CROs, patient recruitment firms, call centers,
AMCs, ad agencies, public relations firms and
technology companies. Email comments and
suggestions to cara@clinicaltrialmedia.com.
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CWWeekly June 13, 2011 7 of 9
Drug & Device Pipeline News
Company Drug/Device Therapeutic Area Status Sponsor Info
Tocagen Toca 511 High-grade glioma Phase I trials initiated in the U.S. (858) 412-8400
www.tocagen.com
Vaxart
H5N1 avian
influenza vaccine
H5N1 avian influenza Phase I trials initiated in the U.S. (415) 437-0132
www.vaxart.com
Adamis
Pharmaceuticals
APC-100 prostate cancer IND approved by the FDA, phase I/IIa trials
planned
N/A
www.adamispharmaceuticals.com
Alkermes ALKS 33-BUP cocaine abuse Phase I/II trials planned for 30 subjects in
the U.S.
(781) 609-6000
www.alkermes.com
FerroKin
BioSciences
FBS0701
pediatrics with
transfusional iron overload
Phase II trials planned for 30 subjects in
North America and Italy
(650) 218-5710
www.ferrokin.com
Array BioPharma Arry-797 osteoarthritis of the knee Phase II trials initiated enrolling 150
subjects in the U.S.
Galapagos GLPG0634 rheumatoid arthritis Phase II trials initiated enrolling 36
subjects in Eastern Europe
(303) 381-6600
www.arraybiopharma.com
+32 15 34 29 00
www.glpg.com
MolMed NGR-hTNF malignant pleural
mesothelioma
Phase II trials initiated enrolling 100
subjects in Italy
+39 0221277.1
www.molmed.com
Morphotek MORAb-004 metastatic melanoma Phase II trials initiated enrolling 80
subjects in the U.S., Europe and Australia
(610) 423-6100
www.morphotek.com
Sound
Pharmaceuticals
SPI-1005
hearing loss induced by
loud music
Phase II trials initiated enrolling in the U.S. (206) 634-2559
www.soundpharmaceuticals.com
Telik Telintra myelodysplastic syndrome Phase II trials initiated enrolling 117
subjects in the U.S.
Onconova rigosertib pancreatic cancer Phase II/III trials initiated enrolling 650
subjects in the U.S.
BioLineRx BL-1020 schizophrenia Phase IIb/III trials initiated enrolling 435
subjects
(650) 845-7700
www.telik.com
(267) 759-3680
www.onconova.com
+972-2-548-9100
www.biolinerx.com
GTx Ostarine muscle wasting/non-small
cell lung cancer
Phase III trials planned for 600 subjects
internationally
(901) 523-9700
www.gtxinc.com
Johnson CWWeekly_Chiltern_Gallery_Banner & ustekinumab Crohn's 10/3/11 disease 11:49 Phase Page III 1 trials planned for 1,200 subjects (732) 524-0400
Johnson
www.jnj.com
The Fine Art of Clinical Research
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CWWeekly June 13, 2011 8 of 9
Trial Results
Musculoskeletal
■■
Geron issued interim results from a phase
I trial of GRNOPC1, a human embryonic
stem cell-based therapy for spinal cord
injuries. Data are from two subjects with
neurologically complete American Spinal
Injury Association Impairment Scale grade
A thoracic spinal cord injuries between
spinal segments T3 and T10. The subjects
received GRNOPC1 at a dose of two
million cells delivered by injection into
the lesion site, administered between
seven and 14 days after injury. Low-dose
tacrolimus was given for temporary
immune-suppression from the time of
injection through 60 days. There were
no surgical complications during or after
either procedure. There was no evidence
of cavitation in the spinal cord at the
injury site on MRI through Day 180. Initial
CW banner:Layout 1 5/20/11 11:05 AM Page 1
analyses showed no evidence of immune
responses to GRNOPC1 through Day 90,
which includes 30 days after complete
withdrawal of immune-suppression. No
adverse events have occurred. Enrollment
has been expanded to include subjects
with injuries at spinal segment T11.
Oncology
■■
Algeta and Bayer reported interim results
from a phase III trial of radium-223
chloride for the treatment of prostate
cancer that has metastasized to the bone.
This randomized, double-blind, multidose,
placebo-controlled trial, ALSYMPCA,
■■
enrolled 922 subjects with symptomatic
castration-resistant/hormone-refractory
prostate cancer that had spread to the
bone. The subjects received radium-223
or placebo plus current standard of care.
The primary endpoint was overall survival.
The overall survival result was statistically
significant, with a median overall survival
of 14 months for radium-223 chloride
and 11.2 months for placebo (two-sided
p-value =0.0022). Based on these results,
the study was stopped and subjects in
the placebo arm will be offered treatment
with radium-223 chloride.
Endocyte issued results from a phase
IIb trial of EC145 for the treatment of
platinum-resistant ovarian cancer. This
randomized, open-label trial, dubbed
PRECEDENT, enrolled 149 women who
received the combination of EC145 and
Pegylated Liposomal Doxorubicin (PLD)
or PLD alone until disease progression
or death. The primary endpoint was progression
free survival (PFS). The median
PFS for the entire population, regardless
of folate receptor expression, was five
months in the EC145/PLD arm compared
to 2.7 months for the PLD single agent
arm (p=0.031). The overall response rate
was 28% in the EC145 combination group
versus 16% in the PLD-alone group. The
study also utilized EC20, an investigational
companion imaging diagnostic
designed to identify subjects with folate
receptor positive tumors. In the subgroup
identified as most positive for the folate
■■
receptor, PFS increased by four months,
from 1.5 months to 5.5 months (p=0.018),
representing more than a 60% reduction
in the risk of progression. The overall
response rate was 22% in the EC145
combination group versus 7% in the PLDalone
group. A phase III trial, PROCEED, is
currently underway in the U.S.
Pfizer released results from a phase I
trial of crizotinib for the treatment of
ALK-positive non-small cell lung cancer
(NSCLC). Data are from 82 ALK+ subjects.
The data was compared to phase I historical
controls from 37 NSCLC ALK+ subjects
who were not treated with crizotinib
(ALK+ controls), as well as 253 NSCLC
ALK-/EGFR- subjects (ALK- controls). In the
82 ALK+ subjects treated with crizotinib,
the one year overall survival was 77%
and two year overall survival was 64%.
The median overall survival has not been
reached. Among the ALK+ controls, the
one- and two-year overall survival was
73% and 33%, respectively, and median
overall survival was 20 months. Subsets of
ALK+ subjects were also evaluated by line
of therapy. Survival of 32 subjects treated
with second- or third-line crizotinib was
significantly longer than that of 24 ALK+
controls (p=0.004): one-year overall
survival was 71% versus 46%, two-year
overall survival was 61% versus 9%. For
123 ALK- controls in the 2nd line setting,
the one- and two-year overall survival was
49% and 33%, respectively. An NDA is currently
under review by the FDA.
Join us for a sneak peek
inside a confidential IRB meeting.
Thursday, June 16th at 4PM, Le Meridien Hotel, Cambridge, MA
Register now! Go to www.neirb.com today to see the
FREE
meeting agenda and submit your FREE registration!
Copyright © 2011. Duplication of this publication is prohibited.
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CWWeekly June 13, 2011 9 of 9
Biotech Review
■■
AVEO Pharmaceuticals licensed exclusive
worldwide development and commercialization
rights to the company’s pre-clinical
antibodies targeting the RON (Recepteur
d’Origine Nantais) receptor to Centocor
Ortho Biotech of Horsham, Pa., a wholly
owned subsidiary of Johnson & Johnson, for
$15 million up front, as much as $540 million
in milestones and double-digit royalties
on worldwide net sales. Centocor Ortho will
be responsible for all clinical development,
manufacturing and commercialization,
and also will fund certain research by AVEO
including translational research studies to
identify biomarkers for patients most likely
to benefit from treatment with RON-targeted
antibodies. Cambridge, Mass.-based
AVEO will receive the first half of the $15
million from Centocor, with the second half
raised through an equity private placement
and the sale of new shares to Johnson &
Johnson Development. Last November
AVEO announced pre-clinical data from its
anti-RON antibody program that showed
the successful identification of antibodies
with potent antitumor activity.
■■
■■
Aegera Therapeutics and its clinical-stage
oncology programs were acquired by generic
pharmaceutical company Pharmascience,
both based in Montreal. Pharmascience
said the acquisition is part of the company’s
diversification and expansion strategy,
adding it will keep Aegera’s 20 employees.
Aegera has two oncology programs in clinical
development. AEG35156 is in phase II for the
treatment of solid tumors and hematological
malignancies. AEG40826 (HGS1029) is in
phase I for treatment of solid and lymphoid
tumors in collaboration with Human Genome
Sciences of Rockville, Md. Pharmascience
was founded in 1983 and has more
than 1,300 employees.
Despite a stock rally following the news of
Optimer Pharmaceuticals’ approval for
macrolide antibiotic Dificid (fidaxomicin),
■■
■■
the company’s challenge might be getting
its drug widely—and quickly—accepted in
the market. Dificid is the first antibiotic in 25
years for Clostridium difficile-associated diarrhea
(CDAD). The San Diego-based company
will add 100 sales reps, most in place by
Dificid’s launch, expected in about six weeks,
said president and CEO Pedro Lichtinger. Optimer
plans to position Dificid in both firstline
and second-line treatment and said the
drug should reduce average hospital stays of
CDAD patients, as well as reduce costs. CDAD
costs the U.S. healthcare system about $3.8
billion a year, he added. Optimer set a price
of $2,800 for a 10-day course of Dificid.
Shares of Affymax have yet to recover from
the beating they took about a year ago when
phase III data for peginesatide (formerly
Hematide) fell short of investor expectations,
but that hasn’t stopped the Palo Alto,
Calif.-based biotech and partner Takeda
Pharmaceutical from moving forward with
a new drug application. The companies filed
for approval of peginesatide to treat anemia
associated with chronic renal failure in dialysis
patients. They originally hoped to file for
approval in dialysis and nondialysis patients
simultaneously, last year’s data showed unexpected
safety issues in the nondialysis group.
Affymax said there are currently no plans to
move forward with the predialysis indication
in the U.S., but Takeda is pursuing it in Japan.
As the process of winding down the company
continues, Renovo CEO Mark Ferguson
estimated there will be $54.3 million left
once the process is complete and said the
company has decided to see ongoing clinical
trials through to conclusion. That move will
increase the potential value to be extracted
from the assets of Manchester, U.K.-based
Renovo as it looks for a buyer following the
catastrophic failure of its lead wound-healing
product Juvista. A 90-day consultation with
staff, aimed at shedding more than 100 of
its 110 employees, is drawing to a close. The
■■
■■
company will be reduced to a stock market
listing, cash in hand and its remaining clinical
programs. Adaprev, for the reduction of
tendon adhesions, is being developed in Europe
as a medical device. Prevascar reported
statistically significant phase II efficacy data
in improving the appearance of scarring. The
company will let the trial run to its planned
conclusion in the first half of 2012.
Despite hailing two six-month pivotal studies’
successes for Relovair in chronic obstructive
pulmonary disease (COPD), South San
Francisco-based Theravance saw its shares
hurt as investors focused on one endpoint
that fell short of statistical significance.
Theravance and partner GlaxoSmithKline
said top-line results from the two studies
supported Relovair’s overall development
program. A once-daily inhaled combination
treatment comprising long-acting beta
agonist vilanterol and inhaled corticosteroid
fluticasone furoate, Relovair is positioned to
replace GSK’s twice-daily COPD and asthma
drug Advair (fluticasone/salmeterol), which
had sales of nearly $7 billion in 2009.
Seaside Therapeutics of Cambridge, Mass.,
will begin a phase III trial to evaluate the
efficacy of a drug candidate against the
core symptoms of orphan disease Fragile
X syndrome. Fragile X is the most common
inherited form of intellectual disability and
the largest known cause of autism.
The stories included in Biotech Review
have been provided to CenterWatch with
full permission of BioWorld Today and its
publisher, AHC Media LLC. Copyright
©2011 AHC Media LLC.
BioWorld is located at 3525 Piedmont
Road, Building 6, Suite 400, Atlanta, GA
30305. U.S.A. Please call (800) 688-2421 or
(404) 262-5476 for more information. Or
visit www.bioworld.com.
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