Sarepta Therapeutics, Inc. 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.
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