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Sarepta's Marburg Drug Shows High Survival Rates After Intramuscular Delivery in Non-Human Primates

Sarepta's Marburg Drug Shows High Survival Rates After Intramuscular Delivery 
in Non-Human Primates 
CAMBRIDGE, MA -- (Marketwire) -- 03/04/13 --  Sarepta Therapeutics,
Inc. (NASDAQ: SRPT), a developer of innovative RNA-based
therapeutics, today announced positive results from a non-human
primate study of AVI-7288, the Company's lead drug candidate for the
treatment of Marburg virus infection. The data showed that
intramuscular administration of AVI-7288 resulted in survival rates
up to 100 percent in treated subjects, similar to efficacy observed
in previous studies that evaluated the drug when administered by
intravenous injection. Marburg hemorrhagic fever is a severe and
highly lethal disease with no effective treatments, and it has been
classified as a Category A bioterrorism agent by the Centers for
Disease Control and Prevention (CDC). 
"These data reinforce the strong efficacy of AVI-7288, while showing
that the drug can be delivered via a convenient intramuscular
injection," said Chris Garabedian, president and chief executive
officer of Sarepta Therapeutics. "This alternative delivery method to
the intravenous route has the potential to greatly enhance the
practical utility of AVI-7288 in a mass casualty situation and also
serves as a model for delivery of our rapidly adaptable platform for
other therapeutic applications." 
Sarepta is developing AVI-7288 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). Under the contract, Sarepta initiated the study
in non-human primates to evaluate the tolerability, pharmacokinetics
and efficacy of AVI-7288 through intramuscular administration. The
study included four cohorts in which subjects received daily
treatments of AVI-7288 ranging from 7.5 to 30 mg/kg or a placebo
after exposure to the virus. 
In the study, intramuscular injections of AVI-7288 were well
tolerated. Efficacy results showed a high degree of survival between
83 and 100 percent in each of the three treatment groups. No subjects
survived in the placebo-treated control group.  
Under a separate contract with JPM-TMT, Sarepta is developing an
intr
avenous formulation of AVI-7288, which has demonstrated similar
survival rates even when the drug is administered up to four days
after exposure to the Marburg virus. 
The work is a collaborative effort between Sarepta and scientists at
the U.S. Army Medical Research Institute of Infectious Diseases
(USAMRIID), the DoD's leading medical research laboratory for
biological defense, which has the DoD's only maximum containment, or
Biosafety Level 4, capability. 
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 USAMRIID 
U.S. Army Medical Research Institute of Infectious Diseases
(USAMRIID), located at Fort Detrick, Maryland, is the lead medical
research laboratory for the U.S. Department of Defense's Biological
Defense Research Program, and plays a key role in national defense
and in infectious disease research. The Institute conducts basic and
applied research on biological threats resulting in medical solutions
(such as vaccines, drugs and diagnostics) to protect the Warfighter.
While USAMRIID's primary mission is focused on the military, its
research often has applications that benefit society as a whole.
USAMRIID is a subordinate laboratory of the U.S. Army Medical
Research and Materiel Command. For more information, visit
www.usamriid.army.mil. 
About Sarepta Therapeutics 
Sarepta Therapeutics -- formerly AVI BioPharma -- 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 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 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 Contact:
Erin Cox
425.354.5140
ecox@sareptatherapeutics.com 
Sarepta Media Contact:
Jim Baker
617.444.8424 ext. 3124
jbaker@sareptatherapeutics.com 
 
 
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