Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative
RNA-based therapeutics, today announced favorable safety results from
the single ascending dose portion of a Phase I study of AVI-7100, the
company’s lead candidate for the treatment of influenza virus, in
healthy volunteers. The clinical trial is being conducted at the
National Institutes of Health (NIH) through a collaboration between the
company and the NIH’s National Institute of Allergy and Infectious
Diseases. AVI-7100 uses Sarepta’s advanced and proprietary PMOplus™
chemistry, which is also the basis of the company’s clinical-stage Ebola
and Marburg drug candidates.
The Phase I clinical study is a randomized, double-blind,
placebo-controlled trial designed to characterize the safety,
tolerability and pharmacokinetics of single and multiple doses of an
intravenous formulation of AVI-7100 in healthy adult volunteers. In the
completed single ascending dose portion, 40 subjects were enrolled in
five cohorts (6 active: 2 placebo) up to the highest dose tested of 8
mg/kg AVI-7100. Results showed that AVI-7100 was well-tolerated with no
reported serious or clinically significant adverse events. The
pharmacokinetic analysis of AVI-7100 reveals a highly similar
dose-dependent profile to that of Sarepta’s Ebola and Marburg PMOplus™
drug candidates. An independent Data and Safety Monitoring Board
reviewed safety results from the study and recommended the study
continue as planned to the multiple dose portion of the study.
“We are very encouraged that our partnership with NIH is generating new
favorable clinical safety data, adding to a growing body of evidence
supporting the safety of Sarepta’s PMO-based chemistry platform across a
broad range of disease targets,” said Chris Garabedian, president and
chief executive officer of Sarepta Therapeutics. “The similar drug-like
characteristics demonstrated across a broad spectrum of targets offers a
versatility that will be especially critical in developing a capability
to rapidly respond and adapt to real-world, global health threats –
whether that is an Ebola outbreak, an influenza pandemic, or a
never-before-seen emerging infectious disease.”
AVI-7100 utilizes a novel mechanism of action to target 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 strains. Seasonal influenza (H3N2) and 2009 H1N1
are both caused by the influenza A virus. Preclinical studies funded by
the U.S. Department of Defense 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® Chemistry
PMOplus® 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® chemistry includes specific molecular
charges positionally inserted into the PMO's inherent charge-neutral
backbone. PMOplus® 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 and follow-on drug
candidates, 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.sarepta.com.
Forward Looking Statements
This press release contains “forward-looking statements” within the
meaning of the safe harbor provisions of the U.S. Private Securities
Litigation Reform Act of 1995. 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 AVI-7100; continuing to the multi-dose portion of the
AVI-7100 study; Sarepta’s partnership with the National Institutes of
Health generating new favorable clinical safety data and adding to a
growing body of evidence supporting the safety of Sarepta’s PMO-based
chemistry platform across a broad range of disease targets; similar
drug-like characteristics demonstrated across a broad spectrum of
targets offering a versatility that will be especially critical in
developing a capability to rapidly respond and adapt to real-world,
global health threats; and the potential of AVI-7100 to act as a broad
spectrum treatment for multiple influenza strains.
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 PMO-based
chemistry platform; AVI-7100 and any of Sarepta's drug candidates,
including those using Sarepta’s PMO-based chemistry may fail in
development, may not receive required regulatory approvals, or may not
become commercially viable; and those additional risks identified under
the heading "Risk Factors" in Sarepta's Quarterly Report on Form 10-Q
for the Quarter ended June 30, 3014 filed with the Securities and
Exchange Commission (SEC) and Sarepta’s other filings with the SEC.
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 the Company's
filings with the SEC. We caution investors not to place considerable
reliance on the forward-looking statements contained in this press
release. Sarepta does not undertake any obligation to publicly update
its forward-looking statements based on events or circumstances after
the date hereof.
Copyright Business Wire 2014