Sarepta Therapeutics Initiates Dosing in Phase I Multiple Ascending Dose Study of Drug for Treatment of Marburg Virus

Sarepta Therapeutics Initiates Dosing in Phase I Multiple Ascending Dose Study 
of Drug for Treatment of Marburg Virus 
CAMBRIDGE, MA -- (Marketwired) -- 05/07/13 --  Sarepta Therapeutics,
Inc. (NASDAQ: SRPT), a developer of innovative RNA-based
therapeutics, today announced that it has initiated dosing in a Phase
1 multiple ascending dose (MAD) clinical trial of AVI-7288, the
Company's lead drug candidate for the treatment of Marburg virus
infection. The Phase I MAD study is designed to characterize the
safety, tolerability and pharmacokinetics of AVI-7288 after repeat
dosing in healthy adult volunteers. The initiation of this study
follows the successfully completed Phase 1 single ascending dose
study, which showed AVI-7288 was well tolerated in healthy
volunteers. Sarepta is developing AVI-7288 under a contract from the
U.S. Department of Defense through the Joint Project Manager
Transformational Medical Technologies (JPM-TMT) Project Management
Office. AVI-7288 utilizes Sarepta's advanced and proprietary
PMOplus(R) chemistry.  
"The initiation of this safety study, which assesses the safety of
our compound with repeated dosing, will help us understand the
potential therapeutic window of our proprietary PMOplus(R)
chemistry," said Chris Garabedian, President and CEO. "Together with
our non-human primate efficacy studies, this safety study will help
us determine the appropriate dose of this drug for the potential use
as a medical counter measure against this lethal hemorrhagic fever
virus." 
The randomized, double-blind, placebo-controlled MAD study will be
overseen by an independent Data and Safety Monitoring Board, who will
review safety and clinical laboratory data after each dose cohort
prior to enrolling the next highest dose cohort. Thirty-two
volunteers will be enrolled in one of four cohorts made up of eight
subjects each. The cohorts will include six subjects who receive the
therapeutic, and two who will receive a placebo. In completed single
ascending dose studies of Sarepta's Marburg and Ebola drug
candidates, which both utilized the PMOplus(R) chemistry, no safety
issues were identified from a total of 48 subjects receiving doses up
to 9 mg/kg of either drug candidate.  
Data from preclinical studies demonstrated that AVI-7288 provides
post-exposure efficacy in infected non-human primates with survival
rates between 83 and 100 percent when the drug is administered up to
four days after exposure to the Marburg virus. 
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 is a material
threat to national security and public health as determined 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 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, 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 or methods of administration;
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 may not become commercially viable.
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. 
"Safe Harbor" Statement under the Private Securities Litigation
Reform Act of 1995: The statements that are not historical facts
contained in this release are forward-looking statements that involve
risks and uncertainties, including, but not limited to, the results
of research and development efforts, the results of preclinical and
clinical testing, the effect of regulation by the FDA and other
agencies, the impact of competitive products, product development,
commercialization and technological difficulties, and other risks
detailed in the company's Securities and Exchange Commission filings. 
Sarepta Investor Contact:
Erin Cox
857.242.3714
ecox@sareptatherapeutics.com 
Sarepta Media Contact
Jim Baker
857.242.3710
jbaker@sareptatherapeutics.com 
 
 
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