Information and data from study will advance
understanding of medical history and medical events in people with
PWS
Zafgen, Inc., (Nasdaq:ZFGN), a clinical-stage biopharmaceutical
company using its proprietary knowledge of MetAP2 systems biology
to help patients affected by a range of metabolic diseases, and the
Foundation for Prader-Willi Research (FPWR), a nonprofit
organization founded to eliminate the challenges of Prader-Willi
syndrome (PWS) through the advancement of research and
therapeutic development, announced today the launch of a
co-sponsored natural history study to advance understanding of the
medical history of and medical events in people with PWS. PATH for
PWS (
Paving the way for
Advances
in
Treatments &
Health for
PWS) is a non-interventional, observational study of serious
medical events in PWS, intended to inform development and clinical
trial design for potential new treatments for PWS, including
ZGN-1258, Zafgen’s new, second-generation program for PWS designed
to decrease hyperphagia, change the way the body metabolizes fat,
and reduce fat mass.
Enrollment for the four-year study is anticipated to begin in
September 2018 through the Global Prader-Willi Syndrome Patient
Registry, which is powered by the National Organization for Rare
Disorders’ (NORD) IAMRARE™ Registry Program.
“While we understand more about Prader-Willi syndrome than ever
before, it is critical that we continue to build on the body of
knowledge to improve care and explore new treatment options,” said
Theresa Strong, Ph.D., Director of Research Programs, FPWR, and the
lead study investigator. “The PATH for PWS study will allow us to
collect and analyze important health information about those with
PWS to better understand how the condition presents and how that
could inform clinical trial design, new and better treatment
options, and even additional resources for families and physicians.
We hope caregivers of adults and children living with PWS will
consider participating in the PATH for PWS to contribute to the
actionable information available to our community.”
Every six months, caregivers of people with PWS enrolled in the
study will be asked to provide an update on any medical issues
experienced and related medical procedures or prescriptions, and
information about conditions often associated with PWS such as
hyperphagia. Clinic visits will not be required, as the information
will be provided through internet-based surveys and
questionnaires. The data will be analyzed to evaluate the
incidence of serious medical events, prescription medication use
associated with medical events of interest, patterns of
hyperphagia-related behaviors, as well as additional areas of
importance to the PWS community such as changes in body weight,
management strategies for hyperphagia, and characterization of
traits and actions common to individuals with PWS. The
analyses will also help Zafgen, FPWR and others in
the Prader-Willi research community identify potential areas for
future study.
“PATH for PWS is a testament to the community-wide commitment to
PWS. This isn’t about a single therapy, but about the power of a
partnership between PWS research and advocacy organizations,
industry and the broader community to transform outcomes for
patients and their families through greater knowledge and
understanding,” said Jeff Hatfield, Chief Executive Officer,
Zafgen.
“Natural history studies are a best practice in rare disease
drug development, and we are looking forward to important insights
that the PATH for PWS study will provide into medical events
experienced by people with PWS,” said Tom Hughes, Ph.D., President
and Chief Scientific Officer, Zafgen. “As we continue work on our
ZGN-1258 program, the PATH for PWS study will be an invaluable
resource to inform its development and potentially other
therapeutic development work for those with PWS.”
To be eligible for the study, patients must have a confirmed
diagnosis of PWS, be at least 5 years of age, live in the United
States, Canada or Australia, and be enrolled or willing to enroll
in the Global PWS Registry. The primary caregiver of the enrolled
person with PWS must have access to the internet to enter study
data and consent to being contacted by registry staff and allowing
registry staff to enter specific data on their behalf.
Participation in clinical trials and other non-interventional
studies will be permitted while enrolled in PATH for PWS.
For more information about PATH for PWS, please visit
www.fpwr.org or www.zafgen.com. Ahead of PATH for PWS
study enrollment opening in September 2018, people with PWS and
their caregivers can enroll in the broader Global PWS Patient
Registry at www.pwsregistry.org.
About Prader-Willi Syndrome (PWS)Prader-Willi
syndrome (PWS) is a rare, genetic form of life-threatening obesity
characterized by unrelenting pathologic hunger (hyperphagia)
leading to dangerous food-seeking behavior, and there is currently
no approved treatment for hyperphagia. People with PWS are also
impacted by slowed metabolism, psychiatric conditions and higher
risk for cardiopulmonary and metabolic co-morbidities. Our best
estimate of prevalence is 1:40,000 people. People with PWS have a
shortened life expectancy of approximately 32 years.
About Zafgen Zafgen (Nasdaq:ZFGN) is
a clinical-stage biopharmaceutical company leveraging its
proprietary MetAP2 biology platform to develop novel therapies for
patients affected by complex metabolic
diseases. Zafgen has pioneered the study of MetAP2
inhibitors in both common and rare metabolic disorders and is
currently advancing programs for type 2 diabetes, Prader-Willi
syndrome and liver diseases. The Company’s lead product candidate,
ZGN-1061, a MetAP2 inhibitor for difficult-to-control type 2
diabetes, has successfully completed the initial part of a Phase 2
clinical trial. Learn more at www.zafgen.com.
About Foundation for Prader-Willi Research
(FPWR) FPWR is composed of thousands of parents,
family members, researchers, and others who are interested in
addressing the many issues related to PWS, including childhood
obesity, developmental delays, psychiatric disorders, and autism
spectrum disorders. The mission of FPWR is to eliminate the
challenges of Prader-Willi syndrome through the advancement of
research and therapeutic development. FPWR supports cutting edge
research studies around the world to advance the understanding of
PWS, and collaborates with research institutions, pharmaceutical
companies and patient advocacy groups to advance new
treatments that will help those with PWS. To date, FPWR has
funded over $10 million in PWS research. For more information
please visit www.fpwr.org.
Safe Harbor Statement Various statements in
this release concerning Zafgen’s future expectations, plans and
prospects, including without limitation, Zafgen’s expectations
regarding the collection of medical history and medical events from
PATH for PWS participants to inform development and clinical trial
design for potential new treatments for PWS, including ZGN-1258,
and the use of ZGN-1258, ZGN-1061 and other
second-generation MetAP2 inhibitors as treatments for metabolic
diseases including Prader-Willi syndrome, type 2 diabetes, liver
diseases and obesity and Zafgen’s expectations with respect to the
timing and success of its ability to collect and analyze PATH for
PWS data for development and clinical trial design and with respect
to its nonclinical studies and clinical trials
of ZGN-1258, ZGN-1061 and its other product
candidates, may constitute forward-looking statements for the
purposes of the safe harbor provisions under The Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
Forward-looking statements can be identified by terminology such as
“anticipate,” “believe,” “could,” “could increase the likelihood,”
“estimate,” “expect,” “intend,” “is planned,” “may,” “should,”
“will,” “will enable,” “would be expected,” “look forward,” “may
provide,” “would” or similar terms, variations of such terms or the
negative of those terms. Actual results may differ materially from
those indicated by these forward-looking statements as a result of
various important factors, including, without limitation, Zafgen’s
ability to successfully collect and analyze medical history and
medical events from PATH for PWS participants, the capacity for
such data to inform clinical trial design and potential areas for
future study, Zafgen’s ability to successfully demonstrate the
efficacy and safety of ZGN-1258, ZGN-1061 and its
other product candidates and to
differentiate ZGN-1258, ZGN-1061 and its other
product candidates from first generation MetAP2 inhibitors, such as
beloranib, the nonclinical and clinical results
for ZGN-1258, ZGN-1061 and its other product candidates,
which may not support further development and marketing approval,
actions of regulatory agencies, which may affect the initiation,
timing and progress of nonclinical studies and clinical trials of
its product candidates, Zafgen’s ability to obtain, maintain and
protect its intellectual property, Zafgen’s ability to enforce its
patents against infringers and defend its patent portfolio against
challenges from third parties, competition from others developing
products for similar uses, Zafgen’s ability to manage operating
expenses, Zafgen’s ability to obtain additional funding to support
its business activities and establish and maintain strategic
business alliances and new business initiatives when needed,
Zafgen’s dependence on third parties for development, manufacture,
marketing, sales and distribution of product candidates, and
unexpected expenditures, as well as those risks more fully
discussed in the section entitled “Risk Factors” in Zafgen’s most
recent Annual Report on Form 10-K filed with the Securities and
Exchange Commission, as well as discussions of potential risks,
uncertainties, and other important factors in Zafgen’s subsequent
filings, including without limitation Zafgen’s Quarterly Reports on
Form 10-Q, with the Securities and Exchange Commission. In
addition, any forward-looking statements represent Zafgen’s views
only as of today and should not be relied upon as representing its
views as of any subsequent date. Zafgen explicitly disclaims any
obligation to update any forward-looking statements, whether as a
result of new information, future events or otherwise.
Media/Investor Relations
Contacts: Zafgen, Inc.
Patricia Allen Chief Financial Officer 617-648-9792
Media Krystle Gibbs Ten Bridge Communications
krystle@tenbridgecommunications.com 508-479-6358
Investors John Woolford Westwicke Partners
John.woolford@westwicke.com 443-213-0506
Foundation for Prader-Willi Research John
WalterChief Executive Officerjohn.walter@fpwr.org 888-322-5487
Zafgen (NASDAQ:ZFGN)
Historical Stock Chart
From Mar 2024 to Apr 2024
Zafgen (NASDAQ:ZFGN)
Historical Stock Chart
From Apr 2023 to Apr 2024