[Updated, 2:45 pm ET, see below] Ed Kaye never thought he’d run a biotech —until waking up one day in 2015 realizing not only that he could, but that Sarepta Therapeutics, where he was chief medical officer at the time, may fall apart if he didn’t.
In March 2015, Kaye (pictured) was one of many high-level executives ready to leave the Cambridge, MA-based company, which was developing a Duchenne muscular dystrophy drug called eteplirsen (Exondys 51). Kaye and many others, he says, were no longer willing to work with then-CEO Chris Garabedian. Garabedian had resurrected Sarepta (NASDAQ: SRPT), but Sarepta had also gotten into an ugly, public dispute with the FDA over eteplirsen under Garabedian’s watch. Reported board squabbles emerged, and Sarepta missed development deadlines, angering investors. Sarepta declined to comment for this story, but there was widespread coverage in the Wall Street Journal and elsewhere about the turmoil within the company. Kaye was looking for houses in San Francisco, ready to take another job. “People were very frustrated,” he says. “It was not a good situation.”
[Updated with response from Chris Garabedian] In response to a request for comment shortly after publication, Garabedian offered this response: “There’s always more than one side to every story and it is unfortunate that I was not contacted by Xconomy to present my views on the circumstances described in the article. That said, I will simply state that I disagree with many of the conclusions and characterizations outlined in the article and the state of the company and my leadership at the time of my departure as described by Ed Kaye. I’m very pleased with Sarepta’s success to date and proud of my contributions as its CEO from 2011 to 2015.”
Kaye says relayed his concerns and plans to leave the company to Sarepta’s board of directors. They came back with a request: what would it take to keep you? His answer: the top seat at Sarepta.
On March 31, 2015, that’s what Kaye got, and the rest is history. Kaye led Sarepta’s controversial quest to win FDA approval of eteplirsen, largely off of evidence from a tiny, 12-patient trial. Sarepta would’ve had to scrap eteplirsen and restructure if the effort had failed. Instead, during a year in which the FDA rejected two other Duchenne drugs, eteplirsen succeeded and became the first-ever approved treatment in the U.S. for the disease. The decision caused a rift within the agency and remains a polarizing topic within biotech circles.
After spending a year launching eteplirsen and battling with insurers to cover the pricey drug, which costs an average of $300,000 per year, Kaye abruptly stepped aside in April. For his work turning Sarepta around and getting eteplirsen to market, Kaye was named a 2017 co-winner of Xconomy’s CEO Award.
The 68-year-old Kaye is just resurfacing now as the CEO of a startup called Stoke Therapeutics based on the work of Cold Spring Harbor Laboratory researcher Adrian Krainer. Krainer is one of the inventors of Biogen’s (NASDAQ: BIIB) nusinersen (Spinraza), the first-ever spinal muscular atrophy drug. The Apple Tree Partners-backed company is developing treatments for rare genetic diseases based on RNA regulation, and will likely be announcing more news soon.
Xconomy spoke with Kaye about his rise through the industry, the eteplirsen saga, Stoke, how the pricing of rare disease drugs is changing, and how he would do clinical development differently. Edited excerpts from the conversation follow below.
Xconomy: You started your career in academia. Why did you make the transition to industry in the first place?
Ed Kaye: I was trying to develop gene therapies for diseases of the central nervous system, but it became very apparent to me that … Next Page »