Despite CEO's Comments, Experimental Sarepta Drug Protects Lab Monkeys From Ebola

Published: Feb 11, 2015

Takeda COO Was Approached for Sanofi CEO Job But Declined
February 11, 2015
By Riley McDermid and Jessica Wilson, BioSpace.com Breaking News Staff

An experimental medication, called AVI-7537, protected 75 percent of monkeys from Ebola in a study conducted by the U.S. Army, Sarepta Therapeutics , and researchers at the University of Washington and Oregon State University. The success of the drug must be ironic for Sarepta CEO Chris Garabedian, who only days ago told an investor conference that investing in Ebola research wasn't worthwhile for the company's shareholders.

“Sarepta had its best stock day when we cancelled our Ebola program [in 2012]. Companies need governments to cover the costs for new drug development, and as a public company, I just don’t see investor interest in advancing Ebola drugs,” said Garabedian in New York on Monday. “Contract business alone is not enough to move the needle. Until we actually see those dollars flowing, investors are going to be skeptical on development.”

Still, AVI-7537's efficacy is remarkable. The results of the study were published yesterday in mBio, the online journal of the American Society for Microbiology.

AVI-7537 belongs to a class of drugs known as phosphorodiamidate morpholino oligomers (PMOs), which are synthetic antisense molecules that target the genetic code within Ebola and similar viruses to prevent viral reproduction. An antisense molecule “silences” a gene. AVI-7537 specifically silences genes need to create the protein VP24.

Previous research has shown that drugs targeting genes that code for protein VP24 and VP35 protected monkeys from Ebola. The significance of this new study, however, is that it showed targeting VP24 alone provided protection, whereas targeting VP35 alone did not.

“The study demonstrates that we can protect non-human primates from Ebola virus, using only a single antisense agent,” said lead study author Travis Warren, a principal investigator in the Molecular and Translational Sciences Division at the U.S. Army Medical Research Institute of Infectious Diseases in Fort Detrick, Md., in a statement.

This is good news because one medication versus a combination of two would be easier to get approved and cheaper to manufacture. Sarepta has a “straightforward” manufacturing process for AVI-7537, Steve Brozak, an analyst and president of WBB Securities, told Reuters.

ZMapp from Mapp Biopharmaceutical and a compound from Tekmira Pharmaceuticals Corp. are the two other agents “shown to cure non-human primates given otherwise-lethal injections of Ebola virus,” according to Reuters. AVI-7537’s straightforward manufacturing process isn’t the only advantage the drug holds over those two agents, however. Sarepta announced in October 2014 the results of a Phase I study that “demonstrated no clinical or toxicologic safety concerns” in human volunteers administered AVI-6002, a combination therapy of the PMOs AVI-7537 and AVI-7539.

Sarepta had a $300 million contract with the U.S. Department of Defense to develop drugs against Ebola and the Marburg virus, both of which are filoviruses that cause severe hemorrhagic fever; the contract ended in 2012 because of government funding cuts, according to Reuters. The study published yesterday was completed before the funding cut, but not published until the uptick in interest for Ebola drugs spurred on by the outbreak in West Africa, Warren told Reuters.

“I think [the Ebola crisis] has been a real wake-up call that there is another distinct way DoD [Department of Defense] should be prepared for filoviruses to enter the United States and potentially infect medical personnel and the population at large,” Warren said in an interview published on the Department of Defense website in October 2014.

Garabedian raised some eyebrows among biotech investors Monday, after saying at a conference that it is not in shareholder interest to keep investing the firm’s money in finding therapies to treat the deadly Ebola virus, despite its continued spread and devastating reach in 2014.

Garabedian quickly faced backlash from social media, with an almost immediate response rippling across Twitter.

“Did I hear ‘good citizenship’ cited as reason for companies to fight poor-market diseases?” tweeted closely-watched biotech consultant Wayne Koberstein in response, among other commenters aghast at Garabedian’s comments.

Garabedian made the comments as part of a panel at the 17th Annual BIO CEO and Investor Conference at the Waldorf-Astoria in New York City.

Sarepta originally had fast-track designation for its AVI-7537, which saw a 60 to 80 percent success rate when used in primates. But the development of the drug was stopped two years ago amid cost concerns, and although it may be revived as the Ebola health crisis continues, it is not a primary concern for the company, said Garabedian.

“Unfortunately, we can’t operate as a biotech and advance an Ebola drug without government support,” he told the audience. “Let’s hope this is a wake-up call because we need at least two years of planning to roll out the creation and manufacturing, etc.”

Indeed, Garabedian took a dim view of most of Big Pharma’s massive efforts to end the disease, saying that despite a roster of drugs and companies currently targeting Ebola, the virus has continued to be a formidable opponent.

“Even if all biotech investigational drugs deployed against Ebola, we couldn't stop it,” he said. “Ebola is here to stay.”

Other members of the panel, “Getting Ahead of Ebola and Other Infectious Threats—Overturning Assumptions,” seemed taken aback by Garabedian’s comments, with Peter Khoury, senior program officer at the Bill & Melinda Gates Foundation, quickly marshalling a litany of statistics about Ebola’s deadliness.

“I hear it all the time, ‘Ebola, isn't that over?’" said Khoury. “But this past week there has been a concerning increase of 124 new cases while 22,500 have had Ebola during the outbreak, with at least 9,500 deaths. Of those, 488 were healthcare workers, out of 822 who became infected," he said.

“Those are scarily high death rates,” said Khoury, who added, “We've seen the corner turned on funding for Ebola from the American government and private health foundations, with the Bill & Melinda Gates Foundation donating $55 million in September alone."


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