Sarepta Is Surging After Good News For Its Ebola Treatment

Sarepta Therapeutics could become an even bigger part of the Ebola trade.

On Thursday, the company announced that two studies involving its Ebola treatment candidates, "demonstrated no clinical or toxicologic safety concerns with the company's drug candidates for the treatment of Ebola."

In pre-market trade on Friday, shares of Sarepta - which has a market cap a bit over $900 million - were soaring, gaining as much as 11%.

In a statement, Michael Wong, senior medical director at Sarepta said, "We believe these promising early clinical safety results, coupled with the strong safety and efficacy data generated from animal studies for all four PMO compounds, reinforce the use of our PMOplus® chemistry platform to pursue potential treatments for deadly infectious diseases such as Ebola and Marburg."

The results of the study are set to be published in the November issue of the American Society for Microbiology's journal, "Antimicrobial Agents and Chemotherapy."

Sarepta is engaged in the development of treatments for rare and infectious diseases, and has been part of the "Ebola trade" that has taken place since the summer and includes stocks like Tekmira Pharmaceuticals, BioCryst Pharmaceuticals, as well as protective gear makers Lakeland Industries and Alpha Pro Tech.

Back in July, shares of Sarepta plunged after results from a trial involving the company's drug eteplirsen for the treatment of patients with Duchenne muscular dystrophy disappointed.

Year-to-date, Sarepta shares are up about 9%, and just this week the stock was up 18% before Friday's pre-market gains.

Here's Thursday's full release from Sarepta:

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 16, 2014-- Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced the publication of results from two single ascending-dose studies that demonstrated no clinical or toxicologic safety concerns with the company's drug candidates for the treatment of Ebola and Marburg virus, respectively. The study results are to be published in the November issue of the American Society for Microbiology's journal, Antimicrobial Agents and Chemotherapy and are available online at dx.doi.org/10.1128/AAC.03442-14.

AVI-6002 for the treatment of Ebola is a combination therapy of two phosphorodiamidate morpholino oligomers (PMOs AVI-7537 and AVI-7539), which target the viral matrix proteins VP24 and VP35, respectively. AVI-6003 for the treatment of Marburg is a combination therapy of two PMOs, (AVI-7287 and AVI-7288), which target the viral proteins VP24 and NP, respectively. These drug candidates use Sarepta's advanced and proprietary PMOplus^®chemistry, which is also the basis of the company's clinical-stage influenza drug candidate, AVI-7100. Results from previous viral challenge studies of AVI-6002 and AVI-6003 in non-human primates demonstrated prevention of disease development and death following exposure to Ebola or Marburg virus. Subsequent animal studies demonstrated that for each combination therapy, only one oligomer contributed to efficacy, and therefore, the lead drug candidates for Ebola and Marburg have since become the single compounds AVI-7537 and AVI-7288.

"We believe these promising early clinical safety results, coupled with the strong safety and efficacy data generated from animal studies for all four PMO compounds, reinforce the use of our PMOplus® chemistry platform to pursue potential treatments for deadly infectious diseases such as Ebola and Marburg," said Michael Wong, senior medical director, infectious diseases at Sarepta Therapeutics. "We are particularly encouraged to see results such as these in the healthy human volunteers to what we have learned to be the effective agents, AVI-7537 and AVI-7288. These compounds have protected up to 80-100 percent of the non-human primates to Ebola and Marburg virus challenge infections, respectively."

The two Phase I clinical studies were randomized, double-blind, placebo-controlled trials designed to characterize the safety, tolerability and pharmacokinetics of single doses of intravenous formulations of AVI-6002 or AVI-6003 in healthy adult volunteers. In each study, 30 subjects were enrolled in six cohorts receiving up to 9 mg/kg of the combination drug candidates (4 active:1 placebo per cohort) for a total of 60 subjects. Results showed the compounds to be well-tolerated with no dose limiting level demonstrated. No clinically significant or dose-dependent effects were observed at any of the safety endpoints evaluated. The safety and pharmacokinetics of the four PMOplus^® compounds comprising the two combination therapies were similar, regardless of the target RNA sequence.

A previously reported Phase I MAD study of AVI-7288 for the treatment of Marburg found no clinically significant or dose-dependent effects on any of the safety endpoints evaluated when tested at up to 16 mg/kg/day for 14 days in healthy adult volunteers. The results of these clinical studies add to a growing body of evidence supporting the safety of Sarepta's PMO-based chemistry platform across a broad range of disease targets.

This work was conducted under contract with the Joint Product Management Office of BioDefense Therapeutics (BD-Tx).