Sarepta leaps into Ebola spotlight with a shelved therapy

Sarepta CEO Chris Garabedian

Back in the fall of 2012, Sarepta Therapeutics ($SRPT) took a hit when the Department of Defense decided to stop funding its work on an RNA-silencing treatment for Ebola. But there was a little-noticed footnote to that setback: The feds reserved an option to restart the work if other companies in the field couldn't complete the job. And with Tekmira's ($TKMR) alternative-track treatment on hold, Sarepta CEO Chris Garabedian has jumped into the media blitz surrounding the Ebola outbreak in Africa, offering to provide the small stock still on the shelves and order up fresh supplies of the Phase I drug.

Sarepta's Ebola program was built around its work on RNA, looking to slam the brakes on viral replication. The DOD had provided a lucrative contract to Sarepta for its work back in 2010, putting up $80 million for the early R&D work in a contract potentially worth $291 million in total. But the feds went with Tekmira after cutting back, and Tekmira was forced to put its Ebola program on hold last month to check out concerns about signs of cytokine release in a Phase I study involving healthy volunteers, a syndrome that can spur a deadly fever in patients.

"We have a drug that could be deployed and shipped," Garabedian told Barron's. But evidently, there's not much of it. Garabedian told the Boston Globe that there was enough AVI-7537 on hand to treat a couple of dozen patients. And he offered to restart production, supplying enough doses of the drug to treat about 100 patients in the next few months.

A spokesperson for the company tells FierceBiotech today that the DoD reached out to the company to "inquire about manufacturing capabilities," offering to cover any costs. "The company has expressed a willingness to help," he added, "but to be sure, Duchenne is the company's top priority." That said, Sarepta "would be interested in restarting" the $291 million contract "in support of national security and public health."

In a study involving rhesus monkeys the drug was credited with a 60% to 80% survival rate. The drug has been through a Phase I trial for healthy volunteers but hasn't advanced into field trials involving infected patients.

Ebola research has largely been a side show in biotech, warranting little careful attention. The virus has been restricted so far to poor countries in Africa, leaving commercial players largely uninterested, while biotechs would only agree to pursue new therapies if they won a government contract or NIH grant to pay for it. After an American physician and missionary were plucked from Liberia following a recent outbreak, their story has suddenly placed Ebola in the center ring of a global media circus, and a variety of biotechs have been paying close attention.

Overnight the small biotech Mapp Biotherapeutics found itself in the limelight after its experimental therapy evidently helped the two Americans recover. And now biotechs of many stripes are looking to take advantage of the media blitz. For Sarepta, the sudden attention could help revive a dormant program, as well as a big federal contract.

- here's the Barron's piece
- and the Boston Globe story