Sarepta Therapeutics Enters Into Clinical Trial Agreement With

Sarepta Therapeutics Enters Into Clinical Trial Agreement With the
National Institutes of Health for Further Development of Influenza
CAMBRIDGE, MA -- (Marketwire) -- 12/21/12 --  Sarepta Therapeutics,
Inc. (NASDAQ: SRPT), a developer of innovative RNA-based
therapeutics, today announced that the Company has entered into a
Clinical Trial Agreement (CTA) with the National Institute of Allergy
and Infectious Diseases (NIAID), part of the National Institutes of
Health (NIH), to conduct a Phase I study with AVI-7100, the Company's
lead drug candidate with a novel mechanism of action and potentially
broad-spectrum activity against influenza viruses, including
Tamiflu-resistant virus strains.  
"We are very excited and honored to work with the NIH, which has a
long-standing commitment to fighting pandemic and seasonal
influenza," said Chris Garabedian, President and CEO. "Our agreement
signifies the importance of developing new treatment options for
influenza with novel mechanisms of action given the limitations of
currently available influenza antivirals. Our combined effort with
the NIH will further increase our understanding of AVI-7100 and its
underlying platform chemistry, which we are also applying to other
infectious disease targets."  
The agreement establishes a formal collaboration between NIAID and
Sarepta to allow NIAID researchers to proceed with a Phase I,
double-blind, placebo-controlled, dose-escalating study to assess the
safety, tolerability and pharmacokinetics of single and multiple
doses of an intravenous formulation of AVI-7100 in healthy
volunteers. The trial is being conducted at the NIH Clinical Center
in Bethesda, MD, under the direction of Richard Davey, M.D., of
NIAID's Division of Intramural Research (
Identifier: NCT01747148). Per the terms of the agreement, Sarepta
will provide AVI-7100 to NIAID. In return, Sarepta will have the
right to use the data from this clinical study to support future
development of AVI-7100. 
About AVI-7100 
AVI-7100 is Sarepta's lead therapeutic candidate for the treatment of
influenza. AVI-7100 targets a well-conserved region of the influenza
A virus, affording it the potential to act as a broad-spectrum
treatment for multiple influenza strains, including Tamiflu-resistant
flu str
ains. Seasonal influenza (H3N2) and the more recently emergent
swine origin influenza virus (SOIV), H1N1, are both caused by the
influenza A virus. AVI-7100 employs the Company's patented PMOplus(R)
technology that selectively introduces positive charges to a
phosphorodiamidate morpholino oligomer (PMO) backbone to improve
selective interaction between the drug and its target. AVI-7100 was
preclinically developed and identified as the lead candidate with
support from the U.S. Department of Defense's Joint Project Manager
Transformational Medical Technologies (JPM-TMT) under contract
HDTRA1-09-C-0046. Preclinical studies funded under JPM-TMT contract
HDTRA1-10-C-0079 demonstrated that AVI-7100 improved clinical
symptoms and reduced viral titers in animal models infected with
pandemic H1N1 or H3N2 viruses, and had statistically significant
activity as compared to saline and Tamiflu controls.  
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
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 
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 AVI-7100 or any of Sarepta's drug candidates and/or
Sarepta's antisense-based technology platform; development of
AVI-7100 may not result in funding; 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
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