Sarepta Therapeutics Enters Into Collaboration for the Development of
Additional Exon-Skipping Product for Duchenne Muscular Dystrophy
Collaboration Represents Significant Progress in Sarepta's Path to
Develop Treatments for Broader DMD Population
CAMBRIDGE, MA -- (Marketwire) -- 11/26/12 -- Sarepta Therapeutics
(NASDAQ: SRPT), a developer of innovative RNA-based therapeutics,
announced today a collaboration for the development of an additional
exon-skipping drug targeting exon 53, its fourth drug in development,
in support of Sarepta's broad-based program for the treatment of
Duchenne muscular dystrophy (DMD). Sarepta's collaboration is with
University College London's (UCL) scientist, Professor Francesco
Muntoni, MD, the Dubowitz Neuromuscular Centre, the Institute of
Child Health and other scientists from the EU and US. The EU Health
Innovation-1 2012 Collaborative research grant will support certain
IND-enabling activities and clinical proof of concept studies for an
exon 53-skipping therapeutic. Sarepta recently announced positive
results from its extension study of its Phase IIb trial of
eteplirsen, its exon 51-skipping therapeutic candidate for the
treatment of DMD. Sarepta is also developing other PMO-based
exon-skipping drug candidates for exons 45 and 50.
"The recent compelling clinical data on eteplirsen targeting exon 51,
which started with our work on the Phase I study in the UK, provides
a strong foundation for using Sarepta's technology against exon 53,"
said Francesco Muntoni, professor of pediatric neurology and head of
the Dubowitz Neuromuscular Centre at the UCL Institute of Child
Health, London. "We are pleased to be working with Sarepta to bring
this exciting exon-skipping therapeutic approach to Europe and the
broader DMD population."
This program will be based on Sarepta's advance proprietary RNA-based
platform, Phosphorodiamidate Morpholino Oligomers (PMOs), which is a
novel backbone chemistry that provides enhanced target tissue
specificity, increased potency to allow for more efficient dosing,
and greater uptake within a cell to further increase protein
production. Targeting exon 53 with this technology will potentially
address one of the most prevalent sets of mutations in DMD that are
amenable to exon-skipping (deletion of exons 42-52
, 45-52, 47-52,
48-52, 49-52, 50-52, or 52) by restoring the cellular machinery's
ability to produce a functional dystrophin protein.
"The initiation of this program, along with our other collaborations
for exons 45 and 50, continues to advance Sarepta's strategy in
pursuing exon-skipping therapeutics for all of the DMD patients who
may benefit from this drug technology," said Chris Garabedian,
President and CEO of Sarepta Therapeutics. "Our goal of demonstrating
that the success of eterplirsen can be reproduced across other
exon-skipping targets is a critical step in being able to treat more
boys and young men affected with this devastating disease."
About EU Health Innovation-1 2012
The aim of the EU investment in health research is to improve the
health of European citizens, to address global health issues and to
boost the competitiveness of European health-related industries. Each
year the European Commission publishes calls for proposals to which
researchers can apply for collaborative research grants.
The European Commission implements a thorough and transparent
evaluation process to identify proposals worthy of funding. The
process is firmly grounded in the principle of access for all and the
codes of fairness, impartiality, confidentiality, efficiency and
ethics. For more information please visit
About Dubowitz Neuromuscular Centre and UCL Institute of Child Health
The Dubowitz Neuromuscular Centre (DNC) provides a multidisciplinary
service as a leading clinical and research center specializing in
neuromuscular disorders affecting individuals in the pediatric age.
The DNC provides clinical assessment, diagnostic services, advice on
treatment and rehabilitation, and is involved in clinical trials and
in basic research focused on understanding the cause of neuromuscular
diseases in childhood, and identifying novel therapeutic
intervention. Excellence in this field is demonstrated not only by
the National Commissioning Group (NCG) status achieved in 2000 to
provide a national specialist diagnostic and assessment service for
Congenital Muscular Dystrophies and Congenital Myopathies, but also
by the designation as a muscle center by the Muscular Dystrophy
The DNC is part of the UCL Institute of Child Health and Great Ormond
Street NHS Trust and is a member of the recently created MRC
Neuromuscular Translational Research Centre at UCL. In addition to
the service provided for all pediatric neuromuscular disorders, the
DNC is involved with clinical, molecular and cell biological research
mostly focused on the genetic basis of congenital muscular
dystrophies; on muscle stem cell and on experimental therapies for
Duchenne muscular dystrophy. For more information please visit
About Duchenne Muscular Dystrophy and Eteplirsen
Duchenne muscular dystrophy (DMD) is an X-linked rare, degenerative
neuromuscular disorder causing severe, progressive muscle loss and a
premature death. One of the most common fatal genetic disorders, DMD
affects approximately one in every 3,500 boys worldwide. A
devastating and incurable muscle-wasting disease, DMD is associated
with specific inborn errors in the gene that codes for dystrophin, a
protein that plays a key structural role in muscle fiber function.
Progressive muscle weakness eventually spreads to the arms, neck and
other areas. Eventually, this progresses to complete paralysis and
increasing difficulty in breathing due to respiratory muscle
dysfunction requiring ventilatory support, as well as cardiac muscle
dysfunction leading to heart failure. The condition is terminal, and
death usually occurs before the age of 30.
Eteplirsen is Sarepta's lead drug candidate that is designed to
address the underlying cause of DMD by enabling the production of a
functional dystrophin protein. Data from clinical studies of
eteplirsen in DMD patients have demonstrated a broadly favorable
safety and tolerability profile and restoration of dystrophin protein
expression. Eteplirsen uses Sarepta's novel phosphorodiamidate
morpholino oligomer (PMO)-based chemistry and proprietary
exon-skipping technology to skip exon 51 of the dystrophin gene
enabling the repair of specific genetic mutations that affect
approximately 13 percent of the total DMD population. By skipping
exon 51, eteplirsen may restore the gene's ability to make a shorter,
but still functional, form of dystrophin from messenger RNA, or mRNA.
Promoting the synthesis of a truncated dystrophin protein is intended
to improve, stabilize or significantly slow the disease process and
prolong and improve the quality of life for patients with DMD.
About Sarepta Therapeutics
Sarepta Therapeutics is focused on developing first-in-class
RNA-based therapeutics to improve and save the lives of people
affected by serious and life-threatening rare and infectious
diseases. The Company's diverse pipeline includes its lead program
eteplirsen, for Duchenne muscular dystrophy, as well as potential
treatments for some of the world's most lethal infectious diseases.
Sarepta aims to build a leading, independent biotech company
dedicated to translating its RNA-based science into transformational
apeutics for patients who face significant unmet medical needs.
For more information, please visit us at www.sareptatherapeutics.com.
Forward-Looking Statements and Information
In order to provide Sarepta's investors with an understanding of its
current results and future prospects, this press release contains
statements that are forward-looking. 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 about the development of eteplirsen and its efficacy,
potency and utility in the treatment of DMD and the potential for the
creation of novel dystrophin to lead to significant clinical benefit
over a longer course of treatment.
These forward-looking statements involve risks and uncertainties,
many of which are beyond Sarepta's control. Known risk factors
include, among others: clinical trials may not demonstrate the safety
and efficacy of eteplirsen and/or Sarepta's antisense-based
technology platform; treatment of patients with DMD using eteplirsen
over a longer duration may not lead to significant clinical benefit;
and any of Sarepta's drug candidates, including eteplirsen, may fail
in development, may not receive required regulatory approvals, or be
delayed to a point where they do not become commercially viable.
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 the
Company's filings with the Securities and Exchange Commission.
Sarepta does not undertake any obligation to publicly update its
forward-looking statements based on events or circumstances after the
Sarepta Media and Investor Contact:
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