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East Coast Biotech Roundup: Forum, Neon, Sarepta, Accelerator & More

Xconomy Boston — 

Things aren’t always pretty in biotech, though it may have seemed like they were over the past few years. Now the raging biotech bull market has slowed, and billions of dollars in value has seemingly disappeared overnight. Is this a blip or a long-term correction? Is the IPO window on the verge of closing? Will the dollars still be there for startups? All worth watching as we march towards the end of 2015. Your East Coast headlines below.

—Waltham, MA-based Forum Pharmaceuticals recently disclosed that the FDA put a clinical hold on two trials it’s been running for its lead drug, encenicline, a prospective treatment Alzheimer’s disease, schizophrenia, and potentially several other neurological disorders. CEO Deborah Dunsire told me the hold is due to a “very small number of gastrointestinal-related safety events” in a Phase 3 Alzheimer’s trial that she called “somewhat varied in presentation.” It’s unclear at this point whether encenicline is to blame. But I polled a group of Alzheimer’s experts to see what might’ve happened in the study, given what we know so far about encenicline and the type of health problems Alzheimer’s patients typically deal with.

—Third Rock Ventures and Len Blavatnik, the richest man in the U.K., poured $55 million in Series A cash into a a new Cambridge, MA-based immuno-oncology startup called Neon Therapeutics. Neon is pursuing a new type of cancer vaccine that would train the immune system to spot so-called “neoantigens,” or, pieces of tumor cells that result from new mutations as the tumor grows. The company has a high-profile group of scientific founders, among them Eric Lander, co-founder of the Broad Institute, and James Allison, whose work in immuno-oncology just won him the prestigious Lasker award. Alex Lash has more here.

—Seattle’s Accelerator Corp. raised $11.7 million and added AbbVie and WuxiTech to its pool of backers, closing out its fourth fund at $62.8 million. The announcement comes 14 months after Accelerator announced it was opening a New York branch. CEO Thong Le told me this week that while Accelerator hasn’t closed any deals in New York as of yet, “several” startups will be announced by the end of the year—both in the Big Apple and Seattle.

—Shares of Cranbury, NJ-based Amicus Therapeutics (NASDSAQ: FOLD) immediately plunged more than 50 percent after the company said that, based on new feeback from the FDA, it likely won’t be able to file for U.S. approval of its Fabry disease drug migalastat by the end of the year as previously expected. The path forward now seems much murkier. According to Amicus, the FDA has essentially asked for more data, and Amicus is now “further evaluating several U.S. pathways for approval.” Amicus said the timing for filing an application will depend on “the determination of the optimal regulatory pathway.”

—The drama continues to build for Sarepta Therapeutics (NASDAQ: SRPT), which is heading towards an expected FDA advisory panel in November for its Duchenne Muscular Dystrophy drug, eteplirsen. This week, Sarepta revealed some additional data on eteplirsen—it compared the patient data it has against an “external control,” as in, untreated Duchenne patients from other studies. Judging the data from the 12 patients in Sarepta’s tiny, Phase 2b study, that means eteplirsen patients, after three years of treatment, walked an average of 151 meters farther than control patients on the standard 6 minute walk test. Shares of Sarepta climbed 22 percent. Here’s more on the data from TheStreet.com.

—Separately, Sarepta inked a research deal with Murdoch University researchers Steve Wilton and Sue Fletcher to look at using its technology to find disease targets for cystic fibrosis and spinal muscular atrophy.

—The biotech slump continued this week as the sector’s recent downturn impacted a few IPOs. Among those offerings was the IPO of Berkeley Heights, NJ-based Edge Therapeutics (NASDAQ: EDGE), which had hoped to raise $85 million by selling 5.7 million shares at $14 to $16 apiece, but settled for the $80 milion it could raise by selling 7,315,151 shares at $11 apiece.

—Boston-based Verastem (NASDAQ: VSTM) stopped a clinical trial of its lead drug VX-6063 in mesothelioma because it wasn’t producing a “sufficient level of efficacy.” The Phase 2b study was Verastem’s biggest test to date, and investors responded to the flop by sending shares spiraling down more than 70 percent.

—New York-based Bristol-Myers Squibb (NYSE: BMY) won FDA approval to use a combination of two immuno-oncology drugs—ipilimumab (Yervoy) and nivolumab (Opdivo)—to treat certain types of patients with metastatic melanoma. The regimen is the first of its kind, and it carries an exorbitant price tag: according to the Wall Street Journal, $256,000, per-patient, per year.

—Genzyme opted into rights to an experimental RNA interference drug called ALN-AT3 that Alnylam Pharmaceuticals (NASDAQ: ALNY) is developing as a treatment for hemophilia. Genzyme was given the option as part of the wide-ranging alliance the two forged in 2014. ALN-AT3 is currently in Phase 1 testing.

—In other Alnylam news,the company won a long-standing suit against University of Utah professor Brenda Bass over RNAi patents known as “Tuschl II” that it licensed several years ago. Bass had been seeking to be named a co-inventor of the patents. Here’s more on the suit from the Boston Globe.

Photo of Boston skyline courtesy of flickr user Bill Damon via Creative Commons.