PRA partners with AMR site network Yale, Mount Sinai expand

October 12, 2015
Breaking News and
Market Intelligence for
the Clinical Trials Industry
Connecticut-based Prometheus Research
awarded more than $250,000 from the
NIH…2
A CenterWatch Publication
Teva Pharmaceutical Industries agrees to
acquire Rimsa…3
CRO Roundup…4
PRA partners with AMR site network
By Ronald Rosenberg
CenterWatch Staff Writer
S
eeking to gain efficiencies, cost savings and faster enrollment timelines
for vaccine clinical trials, North
Carolina-based PRA Health Sciences has
formed a non-exclusive strategic partnership with the Alliance for Multispecialty
Research (AMR), a geographically dispersed
network that represents 17 independently
owned, multi-therapeutic clinical research
investigative sites.
The collaboration—among the first
to engage a single site network for vaccine trials—enables PRA to have detailed
information about the quality and capabilities of each site, with Tennessee-based AMR
providing clinical study services including
vaccines and several bioterrorism-related
government sponsored programs support.
A key goal is to make it easier to identify
appropriate patient populations and implement efficient solutions to smarter vaccine
development, according to Brenda Atchison,
AMR’s chief executive officer.
“We are pretty excited about this collaboration, which we have been working
on for three years,” said Atchison. “Basically
we operate as a core team, with a group of
vetted sites and the advantages of strong
collaborations with CROs and sponsors. But
unlike most CROs, we provide sponsors with
page 5
»
A
pple’s ResearchKit has received a
major boost from two major players
in the academic medical community.
The Yale School of Medicine and
the Icahn School of Medicine at Mount
Sinai are the latest to step up their use of
the mobile software platform—which is
designed to help collect data for medical
research—announcing, respectively, a new
heart condition study and the refinement of
an asthma app.
Researchers at Yale School of Medicine
are developing an app that runs through
ResearchKit and turns the iPhone into a research tool to compile self assessments from
patients with cardiomyopathies—diseases
that limit the heart’s ability to effectively
pump blood through the body.
Called the Yale Cardiomyopathy Index,
the app enables patients to record information about their life, and heart-related
symptoms, plus provide feedback through
iPhones to their physicians about their
physical function and heart rate trends in
real time. They also have an option to perform six-minute walk tests that analyze their
physical abilities and heart rate trends.
Part of the yearlong clinical study, which
is open to anyone between the ages of
2 and 80, involves questionnaires to see
how cardiomyopathy affects different age
Volume 19, Issue 40. © 2015 CenterWatch. All rights reserved.
page 6
Drug & Device Pipeline News…8
Twenty-one drugs and devices have entered
a new trial phase this week.
Trial Results…9
CenterWatch reports on results for three drugs.
Biotech Review…10
Biotech briefs from BioWorld Today.
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Yale, Mount Sinai expand Apple ResearchKit use
By Ronald Rosenberg
CenterWatch Staff Writer
Three Questions…7
Kurt Mussina, vice president and general
manager, Frenova Renal Research
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CWWeekly October 12, 2015
2 of 10
Industry Briefs
Study Conduct
The Patient-Centered Outcomes Research
Institute (PCORI) has awarded the Ohio State
University (OSU) College of Medicine and other
Patient-Centered Network of Learning Health
Systems members—including partners across
six states and nine academic medical centers,
healthcare systems, public health departments
and private health plans—a three-year, $8.6
million grant to participate in a large, national collaborative initiative conducting clinical research
more quickly and less expensively. The National
Patient-Centered Clinical Research Network
(PCORnet) was designed to link researchers, patient communities, clinicians and health systems
in productive research partnerships that leverage
the power of large volumes of health data maintained by the partner networks. PCORnet consists
of two types of networks working toward a
combined goal of clinical data research networks
and patient-powered research networks. The
new network is one of only seven health data
networks approved by PCORI’s board of governors to be added to the PCORnet national health
research resource, which is in its second phase of
development. PCORI is an independent nonprofit
authorized by Congress in 2010 to fund research
that will provide patients, caregivers and clinicians with evidence-based information to make
more informed healthcare decisions.
}}
Prometheus Research, which is based in
New Haven, Conn., has been awarded more
than $250,000 from the NIH to transform the
way electronic data capture (EDC) forms are
configured, integrated and shared for biomedical and behavioral health research. The grant,
part of NIH’s Small Business Innovation Research
(SBIR) program, seeks to empower mental health
researchers to easily share, reuse and locate electronic data capture forms across a variety of EDC
platforms. The outcome of the grant is expected
to have an impact on research areas well beyond
mental and behavioral health. The increased
popularity of patient-reported outcomes,
hospital- and provider-based quality improve}}
ment initiatives, and multisite collaborations has
only exacerbated the prevalent research issue of
EDC interoperability. Prometheus is utilizing the
grant funds to create the Research Instrument
Open Standard (RIOS). RIOS is a publicly available
standard for representing research instruments
and eliminates the need for redundant EDC
form configuration. As part of the NIH grant,
Prometheus Research also is developing a set of
adapters to translate instrument configurations
between RIOS and the formats used by other
popular EDC systems: Research Electronic Data
Capture (REDCap), Qualtrics, and Prometheus’
RexEntry and RexSurvey applications.
R&D Trends
Regeneron Pharmaceuticals is providing
Mitsubishi Tanabe Pharma (MTPC), an Osaka,
Japan-based pharmaceutical company, with exclusive development and commercial rights to fasinumab (REGN475), Regeneron’s NGF antibody
in late-stage development for musculoskeletal
pain. MTPC will obtain exclusive development
and commercial rights to fasinumab in Japan,
Korea and nine other Asian countries, excluding
China. Regeneron President and CEO Leonard
Schleifer, M.D., Ph.D., said, “MTPC has proven
experience marketing biologics for rheumatology and pain management and thus is an ideal
partner in Asia. We look forward to advancing
our NGF program in the coming months with the
goal of bringing this investigational therapy to
patients in serious need.” Regeneron will receive
up to $55 million in up-front and other near-term
payments. The agreement provides for additional
payments to Regeneron of up to $170 million
in R&D reimbursement payments and develop}}
CWWeekly (ISSN 1528-5731)
J. Michael Whalen Managing Editor
Ronald Rosenberg Staff Writer
Tracy Lawton, Stephanie Hill Drug Intelligence
Melissa Nazzaro Advertising
Renee Breau Production
Send news submissions to Managing Editor
Tel (617) 948-5100 Fax (617) 948-5101
editorial@centerwatch.com
© 2015 CenterWatch. Duplication or sharing of this publication is strictly prohibited. ment milestones. Upon commercialization,
Regeneron will supply the product at a range of
purchase prices depending on net sales, such
that Regeneron shares in a significant portion of
any potential profits. Regeneron also is eligible
for additional one-time purchase price adjustment payments of up to $100 million total upon
achievement of specified annual net sales.
The NIH has announced its second wave of
grants to support the goals of the Brain Research
through Advancing Innovative Neurotechnologies (BRAIN) Initiative, bringing the NIH
investment to $85 million in fiscal year 2015.
Sixty-seven new awards, totaling more than $38
million, will go to 131 investigators working at
125 institutions in the U.S. and eight other countries. The awards expand NIH’s efforts to develop
new tools and technologies to understand neural
circuit function and capture a dynamic view of
the brain in action. Projects include proposals to
develop soft self-driving electrodes, ultrasound
methods for measuring brain activity and the
use of deep brain stimulation to treat traumatic
brain injuries. Planning for the NIH component
of the BRAIN Initiative is guided by the long-term
scientific plan “BRAIN 2025: A Scientific Vision,”
which details seven high-priority research areas.
Last year, NIH awarded $46 million to BRAIN
Initiative research.
}}
The NIH Common Fund has awarded more
than $54 million in fiscal year 2015 to launch
projects in four broad scientific areas: the Glycoscience Program, the 4D Nucleome Program, the
Gabriella Miller Kids First Research Program and
the Science of Behavior Change Program. The
}}
page 3
»
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CWWeekly October 12, 2015
3 of 10
Industry Briefs (continued from page 2)
Common Fund planning process identifies major
challenges that impede progress in research
and emerging areas of science. Common Fund
programs that emerge are goal-driven, with
deliverables expected within a five- to 10-year
period. The 2015 awards represented first-year
funding of a multiyear program. The Glycoscience
Program is addressing the difficulty of studying
proteins and lipids that have complex sugars
attached, a problem that stymies researchers
in virtually every arena of biomedical research.
The 4D Nucleome Program is leveraging recent
technological advances to transform understanding of gene regulation. The Gabriella Miller Kids
First Pediatric Research Program will develop a
data resource for the pediatric research community of clinical and genetic sequence data that will
allow scientists to identify genetic pathways that
underlie structural birth defects and childhood
cancer. The Science of Behavior Change Program
aims to implement a mechanism-focused,
experimental medicine approach to behavior
change research.
Ethics/Regulatory
The Medicines and Healthcare products
Regulatory Agency (MHRA) has signed a
Memorandum of Understanding (MOU) with its
counterpart body in India. The agreement will
increase collaboration between the two countries
in the area of medicines and medical devices with
the aim of further improving public safety. It is the
first MOU agreed with the Central Drugs
Standard Control Organization (CDSCO)—
part of the Ministry of Health and Family
Welfare of Republic of India. The central under}}
standings of the agreement include promotion
of each other’s regulatory frameworks, requirements and processes. Significant outcomes
will include the facilitation and exchange of
information and opportunities for technical
cooperation of mutual benefit, helping to ensure
the regulators are better equipped to protect the
health of their respective publics.
Drug Sponsors
OncoResponse, an immuno-oncology company, has been launched jointly by The
University of Texas MD Anderson Cancer
Center and Seattle’s Theraclone Sciences.
OncoResponse will use Theraclone’s I-STAR immune repertoire screening technology to identify
antibodies against novel targets from immunooncology treated patients. I-STAR technology
rapidly screens antibodies made by the human
immune system to identify those with exceptional reactivity that may lead to cancer treatment
development. MD Anderson will provide access
to samples and physiologic, prognostic and genotypic data from patients that have responded
well to cancer immunotherapies, along with
oncology and translational medicine expertise.
}}
Biotechnology Industry Organization
(BIO) President and CEO Jim Greenwood has
released a statement expressing disappointment
in the Trans-Pacific Partnership (TPP) agreement. “We are very disappointed in reports from
Atlanta that suggest trade ministers have failed
to include 12 years of data exclusivity for biologics
in the Trans-Pacific Partnership agreement,” said
Greenwood. “BIO strongly believes that 12 years
}}
of data exclusivity is a prerequisite to attract
the investment required to continue medical
innovation and develop new biological cures and
therapies. The current 12-year period of exclusivity in the U.S. was carefully crafted by a bipartisan
majority of the Congress after a thorough and
thoughtful debate and deliberation. ...We believe
the failure of our Asian-Pacific partners to agree
to a similar length of protection is remarkably
shortsighted and has the potential to chill global
investment and slow development of new breakthrough treatments for suffering patients.”
Teva Pharmaceutical Industries has agreed
to acquire Representaciones e Investigaciones
Médicas (Rimsa), a pharmaceutical manufacturing and distribution company in Mexico, along
with a portfolio of products and companies, intellectual property, assets and pharmaceutical patents in Latin America and Europe in a debt-free,
cash free set of transactions, for an aggregate of
$2.3 billion. Rimsa had revenue in 2014 of $227
million with an annual growth, year over year of
10.6% since 2011. The company has an extensive
portfolio of specialty products, including fixeddose combination products which have fueled its
growth. Rimsa’s well-established sales footprint
is expected to provide a platform for additional
Teva products. The acquisition was unanimously
approved by Teva’s board of directors, led by the
chairman, professor Yitzhak Peterburg. Teva expects to close the transactions next year by early
first quarter. The acquisition is not expected to
impact 2016 non-GAAP earnings and is expected
to be accretive starting Q1 2017. The transactions
will be funded through a combination of cash on
hand and lines of credit. }}
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4 of 10
CRO Roundup
LSK selects InForm
to speed clinical trials
LSK Global Pharma Services has adopted
cloud-based Oracle Health Science’s InForm
to improve its clinical development processes,
streamline data capture and make trial timelines
faster. LSK Global offers task flow, product
development, regulatory consulting, project
development, clinical research, data management and other CRO services. The company also
conducts clinical trials for many local pharmaceutical firms. LSK Global looked to Oracle for
a solution that would enable its R&D team to
access real-time clinical trial data, reduce invalid
data entries and expedite the availability of
drug trial information. LSK Global plans to utilize
Oracle’s best-in-class technology to gain deeper
and faster insight into its clinical data, which will
help the company save time and effort. In turn,
the CRO will be able to help sponsors improve
the speed, quality and efficiency of their clinical
studies. Oracle Health Sciences InForm offers
comprehensive support for industry standards,
such as CDISC, to increase efficiency and time
for trial design and setup and enhance data
collection and reporting quality.
Chiltern expands
development services
Chiltern is expanding its clinical development
services in endocrinology and metabolism and
has named Randy Anderson, Ph.D., as senior
vice president of scientific affairs. With nearly
30 years of industry experience, Anderson is an
expert in diabetes and other endocrine/metabolism disorders. A board director for JDRF International, he also serves on multiple scientific
advisory boards for pharmaceutical companies
with endocrine products. He joins Chiltern after
18 years with another major clinical research
organization, where he provided scientific
leadership for the endocrinology and metabolism therapeutic area and was a contributor on
hundreds of trials and products. In addition to
being the therapeutic area leader for endocrinology and metabolism, Anderson will serve
as Chiltern’s administrative leader for its other
scientific affairs experts across all therapeutic
areas. The endocrinology and metabolism area
at Chiltern includes diseases of the endocrine
organs and related complications: type 1
diabetes, type 2 diabetes, diabetes complications, obesity, dyslipidaemias, adrenal disorders,
thyroid disorders, pituitary disorders, calcium
homeostasis disorders, cardiovascular disease
prevention, growth disorders, sex androgen
dysfunction and other rare metabolic disease
indications such as lysosomal storage disorders,
Prader-Willi Syndrome and Fabry disease.
INC gains iCardiac Site Certification
INC Research Holdings has been certified
to use the iCardiac Early Precision QT approach for evaluating the cardiac safety of new
compounds in phase I clinical development.
INC’s use of iCardiac Early Precision QT may
help customers reduce costs, and increase the
accuracy, reliability and speed of cardiac safety
testing in clinical research. Since 2005, the FDA
has required all new compounds under development to be tested to determine their impact
on the QT interval—the time between the start
of the Q wave and the end of the T wave during
the heart’s electrical cycle. A lengthened QT
interval indicates potential safety risk factors for
a new drug. With the technology in place, INC
may be able to eliminate the need for a separate, dedicated Thorough QT (TQT) study, which
can cost between $2 million and $5 million. By
applying the approach, INC can help companies
spend only a fraction of that amount and collect
cardiac safety data during phase I trials rather
than during later stage trials.
Frontage doubles U.S.
bioanalytical lab capacity
Frontage has expanded its bioanalytical
laboratories to meet increased demand for its
services from existing and new biopharmaceutical clients. As a provider of bioanalytical services for small molecules, large molecules and
biologics, including the bioanalysis of Antibody
Drug Conjugates (ADCs) by Mass Spectroscopy
and Ligand Binding platforms, Frontage has
completed an expansion of its operations in
Exton, Pa. With an additional 10,000 square
feet of laboratories, the expansion allows for
the installation of 20 more state-of-the-art
mass spectrometers. The company also added
laboratories that expand its biologics service offerings for ECL and ELISA-based assays, and its
growing DMPK operations supporting discovery-stage pharmacokinetics and developmentstage metabolism studies. Frontage has hired
21 additional scientific personnel, including
eight Ph.D. chemists. Frontage is continuing
its efforts to improve client satisfaction and
turn-around times for method development,
validation and analysis of regulated bioanalytical samples with an increased capacity of
50,000 samples per month.
400 Columbia Drive
Suite 111
West Palm Beach, FL 33409
CLINICAL TESTING SPECIALISTS
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w w w.PalmBeachCRO.com
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PRA/AMR
(continued from page 1)
due diligence on these specific sites before
receiving the actual study, which helps
sponsors with feasibility, so they don’t have
to worry about picking and evaluating sites.”
AMR believes that its sponsors meet
better enrollment timelines. Atchison cited
subject retention rates between 93% and
100% for its previous clinical trial studies,
which allows for successful support of small
to mid-size vaccine trials.
For PRA, the collaboration is a chance to
identify patient populations needed for vaccine studies and work with one of the most
cost-effective clinical research site networks,
noted Frank Hijek, PRA senior vice president
for strategic drug development.
“Together, we can help sponsors get vaccines to the people who need them,” Hijek
said in a prepared statement. He added that
PRA has provided support for 10 approved
vaccines in the past five years.
PRA’s vaccine experience includes phase
I through phase IV adult and pediatric studies involving nearly 213,000 participants
at more than 3,000 sites around the world,
while AMR member-centers have enrolled
nearly 50,000 volunteers across all age
ranges in vaccine trials alone and including several bioterrorism-related programs
conducted under government contracts.
Under the strategic partnership, AMR—
which is an alliance, rather than a site
“We are pretty excited about
this collaboration, which we
have been working on for
three years. Basically we
operate as a core team,
with a group of vetted
sites and the advantages
of strong collaborations
with CROs and sponsors.”
—Brenda Atchison, CEO,
Alliance for Multispecialty Research
management organization—provides, at no
cost to PRA, self-coordination services that
maximize consistency across all the sites. Instead, the network members pay yearly dues
to AMR, which helps all the sites with the
study design development and with writing
the protocols. For each clinical trial involving
the alliance, AMR assigns an internal chairperson who provides internal oversight.
“What we are creating is a formalized,
longstanding collaborative relationship with
PRA,” said Atchison. “We work on behalf
of our sites on immediate program development, proposal development, budget
assistance and management assistance.
Before CROs’ bids are ever in, we participate
actively on behalf of our members. And we
are involved from day one, and not just in
vaccine trials but with other trials—all at
very early stages that no one else does.”
AMR, which recently completed a major
Ebola study that enrolled 150 patients at
multiple sites, also has provided support
for more than 10 approved vaccines in the
past five years. Although the majority of
vaccines are for children, there are industry
projections that the adult vaccine market
is expected to double by 2020—especially
in the area of infectious diseases that affect
people globally.
In a joint statement, both PRA and AMR
stated the collaboration is expected to identify patient populations needed for vaccine
studies with the ultimate goal of getting
vaccines to market more quickly than in the
past.
“This is an ideal collaboration,” said Hijek.
“It combines the expertise of PRA, one of the
world’s leading contract research organizations, and AMR, one of the most effective clinical research site networks in the
industry. Together, we can help sponsors get
vaccines to the people who need them.”
Email comments to Ronald at
ronald.rosenberg@centerwatch.com.
Follow @RonRCW
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ResearchKit
(continued from page 1)
groups, with parents completing answers
for the youngest group of children. An
education part of the app includes resources
enabling participants to understand cardiomyopathies.
A typical question is: “Do you feel your
heart is beating really fast when you are
NOT active—such as when you are studying, listening to music or hanging out?” The
four answer choices are “no,” “once or twice
a month,” “once or twice a week” or “most
days.”
“We’re excited about this because, with
ResearchKit, we are able to reach out to a
large number of interested families and patients around the country,” said E. Kevin Hall,
M.D., one of the Yale School of Medicine’s
research developers of the app. He is an assistant professor of cardiology and director
of the Pediatric Heart Failure Program.
“It helps us learn how to improve patients’ quality of life and provides a better
understanding of how heart issues affect
kids and young adults,” Hall added, as the
smartphone has sensors that could help in
studies looking at a patient’s gait, motor
impairment, fitness, speech and memory.
“We see ResearchKit as a shovel, where you
can use it for many things. Its power is the
ability to access large numbers of patients
and be able to collect and communicate the
data. For us, this is a significant change in
how research can be done.”
The Cardiomyopathy Index follows an
earlier heart health study program from
Stanford University School of Medicine,
which last March launched a free smartphone app called MyHeart Counts. Its
purpose is to enable users to help advance
their understanding of the health of the human heart by collecting data about physical
activity and cardiac risk factors. In the future,
the app will be utilized to study various
methods for using smartphones and other
“[ResearchKit’s] power is
the ability to access large
numbers of patients and be
able to collect and communicate
the data. For us, this is a
significant change in how
research can be done.”
—E. Kevin Hall, M.D., of the
Yale School of Medicine
wearable devices to enhance heart-healthy
habits, according to Stanford University.
At the New York-based Icahn School of
Medicine at Mount Sinai, LifeMap Solutions
has been developing the Asthma Health
app, which uses ResearchKit to make it possible for asthma sufferers to participate in research studies via their iPhones. Along with
Weill Cornell Medical College, the research
project seeks to track individuals’ asthma
symptom patterns and potential triggers to
improve existing treatments.
So far, more than 8,600 research participants have been using the Asthma Health
app without direct in-person contact. It
also is one of the first five apps to launch
on ResearchKit since the medical research
platform was officially launched about six
months ago.
“When we began our partnership with
Mount Sinai and Icahn, we saw opportunities to create new mobile health apps that
enable individuals with asthma to participate in a large-scale medical research study
using their iPhones,” said Corey Bridges, CEO
of LifeMap Solutions, a digital-health subsidiary of BioTime, which collaborated with
Mount Sinai to develop the free Asthma
Health app, starting at the request of Apple
shortly before HealthKit’s official release.
© 2015 CenterWatch. Duplication or sharing of this publication is strictly prohibited. The app is a personalized tool that helps
individuals gain greater insight into their
asthma, avoid triggers and adhere to treatment plans. Having gathered six months’
worth of data and feedback, LifeMap
expanded its capability by enabling the
sharing of data gathered with the app by
physicians and including a “Doctor Dashboard” to display data on a patient’s asthma
condition, symptom control and activity to
their physician healthcare provider.
“We also found that many participants
were showing their health data, from the
Asthma Health app to their physicians to
start anecdotal conversations,” said Bridges.
“So we worked closely with Mount Sinai
to create the dashboard where the patient
just presses a button in the app and hands
their iPhone to the doctor to provide a quick
sense of the patient’s asthma symptom
control and activity. To have initial findings
in just six months and an impact on people’s
health, and then adjust it to put in additional functionality, is certainly exciting.”
The rich trove of data from thousands of
participants also has led to interesting clinical outcomes, according to Yu-Feng Yvonne
Chan, M.D., Ph.D., director of digital health
and personalized medicine at the Icahn Institute for Genomics and Multiscale Biology
at Mount Sinai.
“Further, many of our study participants
wrote to inform us that the app was serving
as more than a research tool, or an educational tool, but was actually helping them better
understand and manage their condition and
feel better overall,” Chan said in a prepared
statement. “This potential impact of our research study on the participants has inspired
our team to introduce new features and
enhancements to the Asthma Health app.”
Email comments to Ronald at
ronald.rosenberg@centerwatch.com.
Follow @RonRCW
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CWWeekly October 12, 2015
7 of 10
Three Questions
Kurt Mussina, Frenova Renal Research
CWWeekly presents this feature as a way to
put the spotlight on issues faced by executives
in the clinical trials space. Kurt Mussina is vice
president and general manager at Frenova
Renal Research in Waltham, Mass.
Q
With one of the largest renal databases
integrated as part of Fresenius Medical
Care North America (FMCNA), a healthcare
provider, what are some of the “real” protocol
efficiencies and feasibilities—beyond faster
startup—that provide speedier recruitment,
thought leadership and quicker completion of
clinical trials? Also, what are
some of the concerns/limitations?
A
Real protocol efficiencies extend well beyond
faster startup. With access
to FMCNA’s data, we inform
better clinical trial design.
We can take a protocol’s inclusion/exclusion
criteria, layer it on top of our patient data and
conduct some rather interesting sensitivity
analyses. Also, not only can we determine the
number of our patients who are eligible, but
we can determine where those patients are
and whether they are near one of our research
facilities. So, by undertaking a much deeper
exploration of a protocol and better informing
that protocol, we set up everyone for success
when that study starts.
Additionally, when it comes to study conduct, we can monitor data in near-real time
to identify patients who may become eligible
for a trial. We can then notify investigators
immediately, which makes recruitment much
more efficient and targeted.
While we are able to inform protocol
design by determining the number of eligible
patients and projected enrollment rates, a
database-driven feasibility assessment cannot
completely tell us which patients, or even
which investigators, would be interested in
participating in the study. This is likely where
traditional site surveys are best suited.
and how do sponsors articulate what’s needed
to weigh the economic value of a recruited patient that also will benefit other stakeholders?
A
Targeted patient recruitment is grounded
in data and data mining, particularly
What has changed in recruiting renal
in the data we are able to access. I am not
patients, in terms of the types of patients sure the industry or even medical science is
over the last 10 years?
anywhere near to sorting out a multi-study
patient participation approach to clinical
The trends in recruiting patients who
research. Determining the economic value of a
suffer with renal impairment have folrecruited patient is just one of many questions
lowed trends in the types of studies, whether
sponsors would need to consider. However, I
drug or device, clinical development phase,
would propose that an even more interesting
and the specific disease conditions being
question is the economic value of the patient
treated. At the moment, for example, there are who has completed the study.
In any case, whether we ever
see
patients participating in
“I think we are beginning to see patients
more than one study screening
becoming more interested and willing
at a time may not be decided by
to at least consider participating in
sponsors or regulatory agenclinical research.”
cies, but by patients themselves.
Kurt Mussina, VP and GM, Frenova Renal Research
It may not be tomorrow or in
the immediate future, but as
patients become more aware,
a number of studies in anemia underway—
knowledgeable and empowered, such deciand, presumably, later phase studies being
sions will likely rest with them. I am actually
planned in both the non-dialysis-dependent
having this conversation with my phone
and dialysis-dependent chronic kidney disease propped upon a book titled “The Patient Will
patient populations.
See You Now,” by Eric Topol. Within that book
Fortunately, I think we are beginning to see are some of the concepts, especially concernpatients becoming more interested and willing information asymmetry, that I am referring
ing to at least consider participating in clinical
to here.
research. This is partially the result of efforts
We start by educating patients on clinical
made by patient advocacy organizations in
research and the opportunities to participate.
the nephrology and related disease communi- Armed with that education and awareness,
ties. While this is good to see, we can’t ignore
patients will begin to understand just how
a trend that is troublesome: Protocols have
valuable—and I mean “valuable” in the
become far more complex than they were 10
economic sense—they are to sponsors and
years ago. Unless protocols are designed from
to healthcare providers. Once that concept
the start with the patient in mind, we risk plac- takes hold with patients, how long will it be
ing an undue burden on patients attempting
before patients want to negotiate a better
to adhere to those complicated protocols.
deal than what’s on the table? I have recently
asked a few investigators about this. None
You’ve stated that among the most
have seen any evidence yet. But I think that
disruptive recruitment strategies will be
is only because there is a lack of awareness,
systems that allow patients to participate in
and perhaps even a lack of interest, in certain
multi-study screenings. How will they evolve,
patient populations. Q
A
Q
© 2015 CenterWatch. Duplication or sharing of this publication is strictly prohibited. CWW1940
CWWeekly October 12, 2015
8 of 10
Drug & Device Pipeline News
Company
Adocia, Eli Lilly
Drug/Device
BioChaperone
Lispro
Medical Condition
type 1 diabetes
Ascend
Biopharmaceuticals
Seattle Genetics
ASN-002
basal cell carcinoma
relapsed or refractory
Hodgkin lymphoma
GTx
Adcetris
(brentuximab
vedotin), Opdivo
(nivolumab)
enobosarm
(GTx-024)
advanced, androgen receptor
positive, triple negative breast
cancer
enobosarm
estrogen receptor positive,
(GTx-024)
androgen receptor positive
breast cancer
REP 2139-Mg/REP
HBeAg negative chronic
2165-Mg, Viread and hepatitis B infection
Pegasys or Zadaxin
ARX-04
moderate-to-severe acute pain
associated with trauma or injury
Firdapse
congenital myasthenic
(amifampridine
syndromes
phosphate)
OTI-15-01
chronic diabetic
foot ulcers
ABTL0812
pediatric cancer
neuroblastoma
guadecitabine
cancer
(SGI-110)
RGX-111
mucopolysaccharidosis
type I
avelumab
metastatic merkel
cell carcinoma
TXA127
Duchenne muscular
(angiotensin 1-7)
dystrophy
514G3
Staphylococcus
aureus infections
Aristada
schizophrenia
(aripiprazole
lauroxil)
Opdivo (nivolumab), BRAF V600 wild-type unresectable
Yervoy (ipilimumab) or metastatic melanoma
PD-L1 IHC 22C3
advanced non-small cell lung
pharmDx
cancer
Letairis
pulmonary arterial hypertension
(ambrisentan),
tadalafil
MorphaBond
abuse-deterrent ER-morphine
(morphine sulfate)
Keytruda
metastatic non-small cell lung
(pembrolizumab)
cancer
GTx
Replicor
AcelRx
Pharmaceuticals
Catalyst
Pharmaceuticals
Osiris Therapeutics
Ability
Pharmaceuticals
Astex
Pharmaceuticals
Regenxbio
Merck
Tarix Orphan
XBiotech
Alkermes
Bristol-Myers
Squibb
Dako
Gilead Sciences
Inspirion Delivery
Technologies
Merck
© 2015 CenterWatch. Duplication or sharing of this publication is strictly prohibited. Status
phase Ib trials initiated
enrolling 36 subjects in
Germany
phase I/IIa trials initiated
enrolling 36 subjects
phase I/II trials initiated
enrolling 80 subjects
in the U.S.
Sponsor Contact
www.lilly.com
phase II trials initiated
enrolling 55 subjects
globally
phase II trials initiated
www.gtxinc.com
phase IIb trials initiated
info@replicor.com
phase III trials initiated
www.acelrx.com
phase III trials initiated in
pediatric patients
(305) 529-2522
info@catalystpharma.com
phase III trials initiated
enrolling 224 subjects
Orphan Drug designation
granted by the FDA
Orphan Drug designation
granted by the FDA
Orphan Drug designation
granted by the FDA
Fast Track designation
granted by the FDA
Fast Track designation
granted by the FDA
Fast Track designation
granted by the FDA
FDA approved
(443) 545-1800
osiris@osiris.com
contact@abilitypharma.com
FDA approved
www.bms.com
FDA approved
www.agilent.com
FDA approved
www.gilead.com
FDA approved
(845) 589-0277
info@inspirionrx.com
www.merck.com
FDA approved
+61 3 8606 3488
info@ascendbiopharma.com
(425) 527-4000
contact@seagen.com
www.gtxinc.com
www.astx.com
www.regenxbio.com
www.emdgroup.com
www.tarixorphan.com
(512) 386-2900
info@xbiotech.com
www.alkermes.com
CWW1940
CWWeekly October 12, 2015
9 of 10
Trial Results
Dermatology
Novan Therapeutics has announced positive phase IIb study results of SB204 for the
treatment of acne vulgaris. In the doubleblind, vehicle-controlled study conducted
across 20 sites in the U.S., 213 subjects were
randomized to five treatment arms: SB204
2% twice daily, SB204 4% once daily, SB204
4% twice daily and vehicle once or twice
daily and treated for up to 12 weeks. All
doses of SB204 showed statistically significant reductions in the percent change
of non-inflammatory and inflammatory
lesions compared to vehicle at the 12-week
endpoint (intent-to-treat population). At the
end of treatment, the only dose group to
be statistically significant in both absolute change and percent change for both
lesion types was the SB204 4% once-daily
treatment arm. The absolute change from
baseline in inflammatory lesions was -11.3
(42%) for 4% once daily and -5.8 (19%) for
vehicle (p=0.004) and the absolute change
from baseline in non-inflammatory lesions
was -14.1 (37%) for 4% once daily and -7.6
(17%) for vehicle (p=0.032). In a time-toevent analysis, the 4% once-daily treatment
demonstrated a time-to-median improvement of 4.1 weeks compared to 11.6 weeks
for Vehicle (p=0.014 as defined by a 35%
reduction in inflammatory lesions). A 6% difference between the pooled SB204-treated
subjects and vehicle-treated subjects was
observed in the Investigator Global Assessment (IGA) endpoint. While not statistically
significant, the difference in IGA enables the
power calculations for future phase III pivotal studies with a 95% confidence interval.
At the end of 12 weeks of treatment, <5%
of the subjects had a cutaneous tolerability score of “moderate” for any of the local
tolerability assessments (erythema, dryness,
scaling, itching, burning/stinging) with zero
}}
reported subjects having any severe local
application site reactions. Based on those
results and having completed an end-ofphase II meeting with the FDA, Novan plans
to initiate two pivotal phase III trials with
SB204 once daily in the first quarter of 2016,
targeting enrollment of 1,300 subjects per
trial.
Pulmonary Diseases
Afferent Pharmaceuticals has released
results of a phase IIb study of AF-219 in
chronic cough patients. The 29-patient, randomized, double-blind, placebo-controlled
crossover study was conducted at 10 clinical
sites in the U.S. Those in the treatment
group received AF-219 50mg, followed by a
titration up to 100mg, 150mg and 200mg,
with each dose given twice daily for four
days. Patients were then crossed over to the
alternate arm of the study and treated with
either AF-219 or placebo for 16 more days.
All AF-219 doses, including the lowest dose
of 50mg twice daily, demonstrated a statistically significant reduction in awake cough
frequency compared to placebo (p=0.002).
AF-219 was generally well-tolerated. The
incidence of decreased taste acuity, as
observed in the first study at 600mg twice
daily, was much less at the 50mg dose. Only
one patient discontinued treatment at any
dose in the current study, due to the taste
effect.
}}
Immunology
DBV Technologies has announced results
of Viaskin Peanut for peanut allergy. OLFUS
is an ongoing, open-label, follow-up study
to VIPES, the company’s phase IIb clinical
trial with Viaskin Peanut. OLFUS enrolled
171 subjects who previously had received either placebo or one of three 12-month dose
}}
© 2015 CenterWatch. Duplication or sharing of this publication is strictly prohibited. regimens administered during VIPES. During
the first year of OLFUS, patients were to
receive a daily application of Viaskin Peanut
50µg, Viaskin Peanut 100µg or Viaskin Peanut 250µg for 12 months. Baseline response
levels in OLFUS were based on the results
of the last double-blind, placebo controlled
food challenge (DBPCFC) in VIPES, and adjusted by the number of patients enrolling
in OLFUS. As in VIPES, a responder in the OLFUS trial was defined as a subject who could
reach a peanut protein eliciting dose equal
to or greater than 1,000mg peanut protein
during the 12-month DBPCFC or a subject
with a ≥10-fold increase of the eliciting dose
compared to the initial eliciting dose after
12 months of treatment. Patients enrolled
in OLFUS who received placebo in VIPES
were analyzed separately from subjects who
initially received Viaskin Peanut. During the
first 12 months of OLFUS, no drug-related
epinephrine use or serious adverse events
(SAEs) due to Viaskin Peanut were reported.
The study’s median compliance rate, which
was maintained at 96%, also was consistent
with previously reported results. A preliminary analysis of the OLFUS data showed
that 12 additional months of therapy with
Viaskin Peanut 250μg increased the number
of patients benefiting from treatment to
70% in OLFUS from 50% in VIPES, with 80%
of children (ages 6 to 11 at entry in VIPES)
responding to therapy after 24 months.
Patients who received placebo for one year
in VIPES and received Viaskin Peanut for 12
months in OLFUS showed a 50% response
rate, which was consistent with findings
from VIPES. DBV expects to launch its phase
III Viaskin Peanut trial at 35 sites in North
America, Australia, Ireland and Germany
in the fourth quarter. DBV also is in the
development process for other Viaskin treatments, such as its treatment currently being
trialed for milk.
CWW1940
CWWeekly October 12, 2015
10 of 10
Biotech Review
Bowing to the inevitable, industry bodies
in the U.K. have collaborated with payers and regulators in publishing a guide
to biosimilars that informs staff in the
National Health Service (NHS) on their
adoption. The guide, described by the NHS
as the “authoritative source of reference,” is
claimed to be the first consensual summary
on the properties of biosimilars and factors
to consider in their use to be published at a
country level. Its publication comes as the
first biosimilar of a big-selling monoclonal
antibody, Remicade (infliximab, Johnson &
Johnson), is starting to transform market
dynamics. The chief pharmaceutical officer
for NHS England, Keith Ridge, said as the
number of biosimilars increases, the health
service needs to plan for their timely, appropriate and cost-effective introduction.
In addition to clinical decision-making, the
guide is specifically intended to inform
finance and procurement decisions. While
the industry previously has dragged its heels
on the introduction of biosimilars, raising
questions of bioequivalence, substitutability
and interchangeability, it is now starting to
support the NHS in building understanding
of those products. Among other influences
is the fact that biologics originator companies such as Amgen have moved into
biosimilars. Along with the Association of
the British Pharmaceutical Industry, the
Bioindustry Association and the British
Generic Manufacturers Association on
the industry side, the preparation of the
guide was supported by the Medicines and
Healthcare Products Regulatory Agency,
National Institute for Health and Care
Excellence and Royal Pharmaceutical
Society.
}}
Public and private biotech companies
are still having no difficulty raising cash
despite the horrendous performance of the
capital markets during the third quarter. The
sector collectively raised a whopping $34
billion, an amount swollen by three major
}}
debt offerings. Private companies enjoyed
another highly successful quarter attracting venture investments that totaled more
than $2.2 billion to bring the year-to-date
total for that category of financing to more
than $7 billion, an amount that establishes
a new high-water mark for private financing
by the industry. In the third quarter, private
global biotechs concluded a total of 59 deals
compared to 66 deals completed in the third
quarter of 2014. Despite the lower number,
the amount raised was almost 86% higher
than the amount raised in the period last
year. Interestingly, the number of overseas
biotech companies raising private capital
has remained steady year-over-year, according to BioWorld Snapshots data, at around
30%. In fact, taking the top spot for the
amount raised during the period was Oxford, U.K.-based Immunocore, which broke
the existing European record for a private
round, raising $320 million to fund the rapid
acceleration of its T-cell receptor immunooncology portfolio—again reinforcing the
intense global interest investors have in the
space.
Pharmalink has raised SEK100 million
($12 million) from existing investors to prepare the ground for phase III development
of its lead program, Nefecon, and is shaping
up for an IPO in 2016. The mezzanine round
is on the back of an interim analysis of the
phase IIb trial of Nefecon, a treatment for
immunoglobulin A nephropathy (IgAN),
published in April. “We are topping up the
cash to get ready for phase III, which we
have been working on since we got the
phase IIb data. We need to expand staff
numbers and prepare stuff, and this money
will pay for that,” said Johan Haggblad,
Pharmalink’s managing director. Following
on from that, Stockholm, Sweden-based
Pharmalink intends to list on the main
market of the Swedish exchange sometime
next year, to pay for the phase III trial. “It
will depend on market circumstances, but
}}
© 2015 CenterWatch. Duplication or sharing of this publication is strictly prohibited. we are planning to raise enough to bring
us through phase III, which will take two to
three years,” Haggblad told BioWorld Today.
While the design of the phase III is yet to be
finalized, the aim is that a single trial can
meet the requirements of the FDA and the
EMA. “We know what we want to accomplish,” said Haggblad.
The EMA will welcome back Guido Rasi as
executive director almost 12 months after
suspending him from the post when his
appointment was annulled three years into
a five-year term. Last November, the E.U.
Civil Service Tribunal found Rasi’s selection
in 2011 was invalid because the recruitment
process was not objective. Now, following a
new process, the EMA management board
once again has nominated him for the post.
Rasi will get his old job back subject to satisfying the European Parliament Committee
dealing with health issues, which he hopes
to do at an Oct. 13 hearing. Kent Woods,
chair of the EMA management board, said
the decision “is the result of a robust recruitment process,” adding, “We look forward to
professor Rasi resuming his leadership of
the agency.” Andreas Pott, deputy executive
director, has nominally been leading the
EMA since the tribunal ruling. But although
suspended from the executive director post,
Rasi has continued to work at EMA and to
represent it on panels and at conferences.
As principal advisor in charge of strategy,
he was de facto host of the EMA’s 20th anniversary conference in March, opening the
meeting and setting down key challenges
for the agency in future. }}
The stories included in Biotech Review have been
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BioWorld Today and its publisher, Thomson Reuters,
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