Sarepta Therapeutics Announces Favorable Safety Results from Phase I Clinical Study of Influenza Drug Candidate
September 30 2014 - 7:30AM
Business Wire
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.
Sarepta Media Contact:Tony Plohoros,
908-591-2839tplohoros@6degreespr.comorSarepta Investor
Contact:Stephanie Ascher, 212-362-1200stephanie@sternir.com
Sarepta Therapeutics (NASDAQ:SRPT)
Historical Stock Chart
From Aug 2024 to Sep 2024
Sarepta Therapeutics (NASDAQ:SRPT)
Historical Stock Chart
From Sep 2023 to Sep 2024