Sarepta Therapeutics' Lead Therapeutic Drugs for Ebola and

Sarepta Therapeutics' Lead Therapeutic Drugs for Ebola and Marburg
Viruses Receive FDA Fast Track Designation 
CAMBRIDGE, MA -- (Marketwire) -- 09/18/12 --  Sarepta Therapeutics,
Inc. (NASDAQ: SRPT), a developer of innovative RNA-based
therapeutics, today announced that the U.S. Food and Drug
Administration (FDA) has granted Fast Track status for the
development of its lead infectious disease drug candidates, AVI-7288
and AVI-7537, for the treatment of Marburg virus and Ebola virus,
respectively. Sarepta has been developing these platform-based
therapeutics under a U.S. Department of Defense (DoD) contract
managed by the Joint Project Manager Transformational Medical
Technologies (JPM-TMT) Project Management Office, a component of the
Joint Program Executive Office for Chemical and Biological Defense
(JPEO-CBD).  
The FDA's Fast Track program is designed to facilitate the
development of, and expedite the review of, new drugs that have shown
the potential to address an unmet medical need in a serious or
life-threatening disease. Fast Track designated drugs qualify for
Priority Review, an expedited review process available to drugs that
offer major advances in treatment or provide a treatment where no
adequate therapy exists. 
"This is a significant milestone for Sarepta," said Chris Garabedian,
President and CEO. "The FDA recognizes the significant unmet medical
need for these highly lethal hemorrhagic fever viruses and the
necessity to move the development of these drugs forward as fast as
possible to ensure effective medical countermeasures for the
Warfighter. We believe Sarepta has the only drug candidates for
Marburg and Ebola with Fast Track designation and expect this
designation will provide an expedited approval process to help DoD
achieve its mission of protecting the nation from biothreats." 
Sarepta continues to evaluate the safety and efficacy of AVI-7288 for
the treatment of Marburg virus following FDA's Animal Rule and next
plans to initiate a Phase I multiple ascending dose study to
characterize the safety, tolerability and pharmacokinetics of
AVI-7288 after repeat dosing in healthy adult volunteers. Currently,
the JPM-TMT's Ebola programs, including development of AVI-7537,
remain under a temporary stop-work order due to funding constraints. 
About Marburg Virus 
Marburg hemorrhagic fever is a severe and potentially fatal disease
in humans first recognized in 1967. It is caused by an RNA virus of
the Filoviridae family and is understood to be endemic to Africa. The
Marburg virus is classified as a Category A bioterrorism agent by the
Centers for Disease Control and Prevention, or CDC, and was
determined to pose a material threat to national security and public
health by the Secretary of Homeland Security in 2006. Onset of the
disease is often sudden, and the symptoms include fever, chills,
nausea, vomiting, chest pain and diarrhea. Increasingly severe
symptoms may also include massive hemorrhaging and multiple organ
dysfunctions. There are currently no treatments for Marburg virus
infection beyond supportive care. 
About Ebola Virus 
The hemorrhagic fever caused by the Ebola virus is severe and often
fatal in humans. The disease was first recognized in 1976 and is one
of two members of a family of RNA viruses called Filoviridae. The
disease is generally understood to be endemic to parts of Africa.
Onset of illness from Ebola virus is abrupt with symptoms that
include fever, headache, muscle ache, vomiting and stomach pain.
Internal and external bleeding may also be observed in some patients.
There are currently no treatments for Ebola virus infection beyond
supportive care and the mortality rate is very high. 
About Sarepta's PMOplus(R) Chemistry 
PMOplus(R) chemistry is an advanced generation of Sarepta's
phosphorodiamidate morpholino oligomer, or PMO, technology pioneered
by Sarepta. The PMO platform is designed to provide a stable
chemistry backbone with superior drug-like characteristics for
Sarepta's advanced RNA-based therapeutics. PMOplus(R) chemistry
includes specific molecular charges positionally inserted into the
PMO's inherent charge-neutral backbone. PMOplus(R) has potentially
broad therapeutic applications and has thus far shown to be
particularly effective in increasing the potency of PMO-based
oligomers. 
About JPM-TMT 
JPM-TMT is a component of the U.S. Department of Defense's Joint
Program Executive Office for Chemical and Biological Defense
(JPEO-CBD). JPM-TMT aims to protect the Warfighter from emerging
infectious diseases, genetically altered, and unknown biological
threats. Through strategic investments and partnerships with
innovative biotech firms, pharmaceutical corporations, other
government agencies, and academic institutions, JPM-TMT facilitates
the advanced development and acquisition of adaptable platform
technologies, broad-spectrum medical countermeasures, and innovative
systems to enhance our nation's biodefense response capability. For
more information, visit www.jpmtmt.mil. 
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
therapeutics 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 Sarepta's product candidates,
their efficacy, potency and utility in the treatment of rare and
infectious diseases, their potential to treat a broad number of human
diseases and Sarepta's studies. 
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 safety and
efficacy of any of Sarepta's drug candidates and/or Sarepta's
antisense-based technology platform; development of AVI-7288 may not
result in funding from JPM-TMT in the anticipated amounts or on a
timely basis, if at all; and any of Sarepta's drug candidates 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
Sarepta's reports filed 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
date hereof. 
Sarepta Investor and Media Contact:
Erin Cox
425.354.5140
ecox@sareptatherapeutics.com 
 
 
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