HACKENSACK, N.J. and
CAMBRIDGE, Mass., Sept. 7, 2018 /PRNewswire/ -- Parent Project
Muscular Dystrophy (PPMD), a nonprofit organization leading the
fight to end Duchenne muscular dystrophy (Duchenne), announced
plans today to launch a patient- and physician-reported outcomes
data warehouse – the Duchenne Outcomes Research Interchange (the
Interchange) – which will combine data from The Duchenne Registry
with clinician entered data provided by its inaugural partner,
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development
of precision genetic medicines to treat rare neuromuscular
diseases. The Duchenne Outcomes Research Interchange, scheduled to
launch in 2019, will aggregate patient and clinician entered data
obtained through various means, providing critical information to
clinicians, sponsors, payers, regulators, and the patients/families
who participate in PPMD's Duchenne Registry.
In parallel to PPMD establishing a new patient engagement
platform, Sarepta will initiate their post-approval patient
registry which will collect clinician-reported outcomes data from
those with Duchenne on EXONDYS 51®
(eteplirsen). The registry will later expand to the broader
Duchenne community. These data will flow into the
Interchange, governed by a steering committee and PPMD and become
part of a comprehensive data set, which is intended to inform the
understanding of the long-term impact of the therapies developed by
Sarepta. PPMD plans to invite industry, advocacy, and academic
partners to join the Interchange, following the successful
integration with Sarepta.
For the last several years, PPMD has been pursuing enhancements
of The Duchenne Registry, a patient reported outcomes (PRO)
registry. The need to combine patient-reported outcomes data with
clinician-reported outcomes data was clear, but with a new
therapeutic landscape, the goal is to not only combine these data
sets but to also incorporate electronic health record data (EHR)
into a combined, centralized data interchange that could benefit
the entire community. The Interchange will provide this central
repository for all types of Duchenne data and 'real world evidence'
to be collected and analyzed in one place.
The Duchenne Registry has been an invaluable resource for all
Duchenne stakeholders, since its inception over 10 years ago,
thanks to the participation of thousands of families who keep their
records up-to-date. The creation of the Interchange, and premier
partnership with Sarepta, firmly places PPMD's registry at the
center of the Duchenne data world. For patients and families who
currently engage with PPMD's Duchenne Registry, the transition will
be relatively seamless, providing enhanced access, as well as new
and improved features and real life conveniences for a more
engaging and valuable user experience.
Knowing the importance of 'real-world evidence' to regulators
and payers, PPMD has been collecting patient-reported outcome data
for over a decade, informing the natural history of Duchenne. PPMD
will now build upon this knowledge by combining it with
clinician-reported outcome data to better meet the needs of the
patient, research, and industry communities, enhancing the value of
patient-reported data in a post-marketing surveillance space.
PPMD and Sarepta are working with preeminent companies involved
in data acquisition and data aggregation to ensure that the
Interchange partners chosen understand the needs of the Duchenne
community and can offer the most advanced technology possible.
"Since a small group of parents and grandparents formed PPMD
almost 25 years ago, we have believed in the importance of
innovation and patient data in the fight to end Duchenne," said
Pat Furlong, Founding President
& CEO of PPMD. "The Duchenne Registry, now powered with
10 years of robust data thanks to our incredible community, is
ready to expand and evolve. Partnering with Sarepta, the first
company in the U.S. to receive an approval, to launch the Duchenne
Outcome Research Interchange made perfect sense, and they will
contribute their own powerful data set. We look forward to creating
a tool that multiple stakeholders can turn to in an effort to
accelerate the clinical trial process and bring safe and effective
therapies to families faster."
"We could not be more proud to be partnering with PPMD on this
important initiative," stated Doug
Ingram, Sarepta's president and chief executive officer.
"The potential of the Duchenne Outcomes Research Interchange is
immense and the impact profound, serving as a trusted resource for
patient and clinician reported outcomes. The repository of data
culled from the Interchange holds the potential to improve the way
in which the science and clinical development programs advance and
ultimately how the best therapies reach patients with
expediency."
About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs people of
their muscle strength. Parent Project Muscular Dystrophy (PPMD) is
the largest most comprehensive nonprofit organization in
the United States focused on
finding a cure for Duchenne muscular dystrophy—our mission is to
end Duchenne.
We invest deeply in treatments for this generation of people
affected by Duchenne and in research that will benefit future
generations. We advocate in Washington,
DC, and have secured hundreds of millions of dollars in
funding. We demand optimal care, and we strengthen, unite and
educate the global Duchenne community.
Everything we do—and everything we have done since our founding
in 1994—helps people with Duchenne live longer, stronger lives. We
will not rest until every person has a treatment to end Duchenne.
Go to www.ParentProjectMD.org for more information or to learn how
you can support our efforts and help families affected by Duchenne.
Follow PPMD on Facebook, Twitter, and YouTube.
About Sarepta Therapeutics
Sarepta Therapeutics is a commercial-stage biopharmaceutical
company focused on the discovery and development of precision
genetic medicine to treat rare neuromuscular diseases. The Company
is primarily focused on rapidly advancing the development of its
potentially disease-modifying Duchenne muscular dystrophy (DMD)
drug candidates. For more information, please visit
www.sarepta.com.
About EXONDYS 51
EXONDYS 51 uses Sarepta's proprietary phosphorodiamidate
morpholino oligomer (PMO) chemistry and exon-skipping technology to
skip exon 51 of the dystrophin gene. EXONDYS 51 is designed to bind
to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this
exon during mRNA processing in patients with genetic mutations that
are amenable to exon 51 skipping. Exon skipping is intended to
allow for production of an internally truncated dystrophin
protein.
Important Safety Information About EXONDYS 51
Hypersensitivity reactions, including rash and urticaria,
pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension,
have occurred in patients who were treated with EXONDYS 51. If a
hypersensitivity reaction occurs, institute appropriate medical
treatment and consider slowing the infusion or interrupting the
EXONDYS 51 therapy.
Adverse reactions in DMD patients (N=8) treated with EXONDYS 51
30 or 50 mg/kg/week by intravenous (IV) infusion with an incidence
of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were
(EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%,
0%) and contact dermatitis (25%, 0%). The most common adverse
reactions were balance disorder and vomiting. Because of the small
numbers of patients, these represent crude frequencies that may not
reflect the frequencies observed in practice. The 50 mg/kg once
weekly dosing regimen of EXONDYS 51 is not recommended.
In the 88 patients who received ≥30 mg/kg/week of EXONDYS 51 for
up to 208 weeks in clinical studies, the following events were
reported in ≥10% of patients and occurred more frequently than on
the same dose in Study 1: vomiting, contusion, excoriation,
arthralgia, rash, catheter site pain, and upper respiratory tract
infection.
For further information, please see the full Prescribing
Information.
About The Duchenne Registry
The Duchenne Registry (formerly DuchenneConnect) began in 2007,
when a group of thought leaders in the Duchenne and Becker muscular
dystrophy community began discussing the need for a new kind of
resource that would connect and serve the needs of the entire
community. What they envisioned was a central hub that would bring
together those living with Duchenne or Becker, along with their
families and caregivers, to connect them with medical research,
clinical care, clinical trials, and each other. At the same time,
it would also be a resource for researchers and industries with an
interest in Duchenne, allowing access to aggregate, de-identified
data provided by patients and their families — information that
could prove vital to advances in care and treatment. Today, the
result of this endeavor is The Duchenne Registry, the largest, most
comprehensive registry for Duchenne and Becker muscular dystrophy,
as well as female carriers.
Ann Martin, Director of The
Duchenne Registry, adds that the success of this platform is a
direct result of community participation: "When families join and
update their account in The Duchenne Registry, they are
strengthening the power of a 10-year-old network of patient-powered
data that will be used to improve care for people living with
Duchenne and increase understanding of the disorder. Families
become citizen scientists by contributing to real scientific
research, without ever leaving their homes."
To learn more about The Duchenne Registry, visit our
website.
Forward-Looking Statements
This press release contains "forward-looking statements." Any
statements contained in this press release that are not statements
of historical fact may be deemed to be forward-looking statements.
Words such as "believes," "anticipates," "plans," "expects,"
"will," "intends," "potential," "possible" and similar expressions
are intended to identify forward-looking statements. These
forward-looking statements include statements regarding PPMD's plan
to launch the Interchange in 2019; Sarepta's plan to initiate its
post-approval patient registry, and the plan to expand the registry
to the broader Duchenne community and to flow these data into the
Interchange, with the intention to inform the understanding of the
long-term impact of the therapies developed by Sarepta; PPMD's plan
to invite industry, advocacy, and academic partners to join the
Interchange, following the successful integration with Sarepta; the
expectation that the transition from Duchenne Registry to the
Interchange will be relatively seamless; and the potential and the
expected impact of the interchange.
These forward-looking statements involve risks and
uncertainties, many of which are beyond Sarepta's control. Known
risk factors include, among others: the expected benefits and
opportunities related to the partnership between PPMD and Sarepta
may not be realized or may take longer to realize than expected;
and those risks identified under the heading "Risk Factors" in
Sarepta's most recent Annual Report on Form 10-K for the year ended
December 31, 2017 and most recent
Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) as well as other SEC filings made by
Sarepta which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect
Sarepta's business, results of operations and the trading price of
Sarepta's common stock. For a detailed description of risks and
uncertainties Sarepta faces, you are encouraged to review Sarepta's
2017 Annual Report on Form 10-K and most recent Quarterly Report on
Form 10-Q filed with the Securities and Exchange Commission (SEC)
as well as other SEC filings made by Sarepta. We caution investors
not to place considerable reliance on the forward-looking
statements contained in this press release. Sarepta does not
undertake any obligation to publicly update its forward-looking
statements based on events or circumstances after the date
hereof.
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SOURCE Parent Project Muscular Dystrophy