In biotech this Monday, all eyes were on an FDA advisory panel in Hyattsville, MD, where a throng of patient advocates some 800 strong gathered in support of approval for an experimental drug for Duchenne muscular dystrophy, a fatal genetic disease with no cure. The results of the hearing didn’t go the way the crowd was hoping, as a negative vote that was long expected came down. But the door isn’t completely shut on the drug, known as eteplirsen, as you’ll read below along with the rest of this week’s headlines.
—At the end of a dramatic, 11-hour meeting, a group of FDA advisors recommended against accelerated approval of eteplirsen, a drug developed by Sarepta Therapeutics (NASDAQ: SRPT) for Duchenne muscular dystrophy. Though the FDA usually follows the advice of its advisory panels, the results aren’t binding. Indeed, despite the negative vote, some investors are still betting on a positive FDA ruling—by Thursday Sarepta’s stock price had nearly recovered from the 30 percent drop following the Monday panel decision. (here’s more on a possible positive outcome from TheStreet.com.). The agency is expected to make a decision by May 26.
—Some 10 years ago, Bedford, MA-based Hologic (NASDAQ: HOLX) bought Suros Surgical Systems, a maker of minimally invasive breast biopsy tools. Since then, Suros’s former executives have taken on a new venture, an Indianapolis startup called Nico with a much tougher task—to change how certain types of brain surgery are done. I spoke with Nico executives about their journey from Suros to Nico, and the crucial clinical trial they’re running to try to prove the worth of their brain surgery device, “Brain Path.”
—Back in 2012, a gout drug called lesinurad (Zurampic) was so intriguing to pharma giant AstraZeneca and former CEO David Brennan that the company paid nearly $1.3 billion to buy the drug’s developer, Ardea Biosciences of San Diego. Since then, under CEO Pascal Soriot, AstraZeneca’s strategic focus has changed, one of the reasons it just handed U.S. rights to lesinurad to Cambridge, MA-based Ironwood Pharmaceuticals (NASDAQ: IRWD) in a deal worth as much as $265 million. The FDA approved lesinurad in December, but AstraZeneca hasn’t launched it yet. Approval came with a boxed warning about the risk of kidney failure—one of the challenges Ironwood will face trying to market the drug going forward. BioWorld has more on the deal here.
—After a roughly year-long search, Cambridge, MA-based Biogen (NASDAQ: BIIB) has named a replacement for former R&D chief Doug Williams, who left the company last July to run startup Codiak Biosciences. Biogen has hired Michael Ehlers as its executive vice president of R&D, a position he’ll begin late next month. Ehlers will join Biogen from Pfizer, where he had been chief scientific officer of the pharma company’s neuroscience and pain research unit, and the senior VP of its BioTherapeutics R&D division. Incidentally, when Ehlers signed on for that role last year, he took over for another executive who’d left a big company to run a startup—Jose-Carlos Gutierrez-Ramos, now the CEO of Synlogic.
—Intellia Therapeutics inched closer to becoming the second publicly traded drug developer based on CRISPR-Cas9 gene editing technology. The Cambridge company set the terms for its coming IPO, in which it aims to sell 5 million shares at between $16 and $18 apiece. Insiders have committed to buying $30 million of Intellia’s shares, and the company’s drug development partners Regeneron Pharmaceuticals (NASDAQ: REGN) and Novartis (NYSE: NVS) have committed to buy $55 million worth of Intellia stock in concurrent, separate deals. All this basically ensures that Intellia will get at least $85 million regardless of what happens in its IPO. The deal is expected to go down next week.
—Lebanon, NH-based Adimab announced more partnerships this week, as Takeda, iTeos Therapeutics, and Kodiak Sciences will all tap into the company’s discovery platform to try to unearth antibody drugs. Adimab already has collaborations in place with more than 35 life sciences companies.
—Massachusetts Gov. Charlie Baker and Lt. Governor Karyn Polito announced $20 million in tax incentives for 28 life sciences companies in the Bay State through a series of awards that are expected to create 1,325 new jobs this year. Some of the recipients are Alnylam Pharmaceuticals (NASDAQ: ALNY)—which just opened a new facility in Norton, MA—Enanta Pharmaceuticals (NASDAQ: ENTA), and Moderna Therapeutics.
—Summit, NJ-based Celgene (NASDAQ: CELG) was among the investors in the $50 million Series B round for FLX Bio. The South San Francisco, CA startup was formed out of the drug programs that Bristol-Myers Squibb (NYSE: BMY) didn’t acquire when it bought Flexus Biosciences last year. (Here’s more from Alex Lash on Flexus and the strategy that led to FLX Bio.)