Sarepta Therapeutics Receives Notice From Department of Defense

Sarepta Therapeutics Receives Notice From Department of Defense for
Termination of Ebola Therapeutic Program 
CAMBRIDGE, MA -- (Marketwire) -- 10/03/12 --  Sarepta Therapeutics,
Inc. (NASDAQ: SRPT), a developer of innovative RNA-based
therapeutics, received notice from the U.S. Department of Defense
(DoD) that the Ebola portion of the Company's contract for the
advanced development of hemorrhagic fever virus therapeutics was
terminated for the convenience of the government due to funding
constraints. The DoD's Joint Project Manager Transformational Medical
Technologies (JPM-TMT) awarded this contract in July 2010 to advance
a platform capability for treating both Marburg virus and Ebola
virus. The Marburg portion of the contract remains in effect. 
"Sarepta is moving with all practical speed to advance our platform
capability through our Marburg program," said Chris Garabedian,
President and CEO. "It is unfortunate that DOD's near-term
programmatic and funding issues may hinder their ability to reap the
long-term strategic and economic benefits of using a single, common
platform to address multiple threats. The unprecedented success of
our PMOplus(R) chemistry against Marburg virus is helping pave the
way toward our goal of providing a nimble response capability that
can be applied to other biothreats, both known and unknown."  
Sarepta previously received a stop-work order for the Ebola portion
of the contract which was in effect from August 2, 2012 through the
termination on October 2, 2012 while JPM-TMT conducted an evaluation
of its Ebola medical countermeasure candidate development efforts.
JPM-TMT selected an alternative contractor's candidate for continued
development. However, upon entry into a settlement agreement between
the Company and DoD regarding costs associated with the termination,
the government may reserve the right under the settlement agreement
to reinstate the Ebola portion of Sarepta's contract if Sarepta's
Ebola therapeutic becomes the only alternative under which the
government can fulfill its requirement because the other company
fails in developing its Ebola therapeutic or is in default of
contract requirements, provided that the Marburg portion of the
contract is continuing at that time, or if DoD subsequently
identifies additional funding to enable both companies' Ebola medical
countermeasure efforts to continue in parallel.  
Earlier this year, Sarepta completed Phase I single ascending-dose
studies in healthy adult volunteers with its drug candidates for the
treatment of Ebola virus and Marburg virus demonstrating positive
safety data for each therapeutic candidate. In September, Sarepta
announced both lead candidates received Fast Track designation from
the FDA. 
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 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 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 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 in
Marburg 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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