East Coast Biotech Roundup: Zafgen, Decibel, Voyager & More

Xconomy Boston — 

Biotech investing isn’t for the faint of heart. Just ask those who put money into Zafgen this week. The company lost 60 percent of its value in two days without reporting any news, gained some of it back, and then plummeted again. How about investors of PTC Therapeutics? On a failed trial, shares initially fell 20 percent, but then quickly rebounded and opened Friday up double digits. It can happen that quickly in biotech. Your East Coast headlines below.

—It was a crazy week for Cambridge, MA-based Zafgen (NASDAQ: ZFGN). First, reports surfaced that Zafgen cancelled some investor meetings. Zafgen went radio silent for two days, and shares plummeted more than 60 percent as investors feared the worst. Zafgen finally broke its silence on Wednesday, and disclosed that a Prader-Willi Syndrome patient in its Phase 3 trial of obesity drug beloranib had died. Shares climbed for a few days, as Zafgen hadn’t yet known whether the patient who died was on beloranib or not, but then fell again after the company confirmed that was indeed the case—and that the FDA had placed a partial clinical hold on the drug.

—Third Rock Ventures and SR One launched Boston-based Decibel Therapeutics with a big $52 million Series A round and a plan to get at the underlying biological problems that cause hearing loss. I spoke with CEO and Third Rock partner Kevin Starr, and one of Decibel’s scientific founders, M. Charles Liberman, about the company’s plans, which include fixing a “broken” connection between two key elements of the inner ear—sensory cells, and nerve fibers.

—Cambridge, MA-based gene therapy startup Voyager Therapeutics outlined plans to go public and fund its gene therapy work for a variety of neurological diseases, which include Parkinson’s, Huntington’s, and amyotrophic lateral sclerosis. Voyager’s prospectus gave some good insight into the challenges it’ll face trying to deliver genetic instructions directly into the spine or brain, which I detailed in this piece about the offering.

—New York-based Bristol-Myers Squibb (NYSE: BMY) doubled down on its alliance with the Bay Area’s Five Prime Therapeutics (NASDAQ: FPRX), paying $350 million up front to grab rights to a cancer drug it aims to use in a combination regimen with its own immuno-oncology treatment, nivolumab (Opdivo). Bristol and Five Prime first cut a deal last year, with Bristol taking a small stake in the company as part of an effort to work together on cancer drugs.

—South Plainfield, NJ-based PTC Therapeutics (NASDAQ: PTCT) presented mixed results from a Phase 3 study of its Duchenne muscular dystrophy drug, ataluren (Translarna). PTC’s drug failed the main goal of its study, but succeeded in other measures. The question is whether, given the serious need for Duchenne treatments, regulators will approve ataluren anyway. PTC aims to file an FDA application by the end of the year.

—Speaking of Duchenne, Cambridge-based Sarepta Therapeutics (NASDAQ: SRPT) said that an FDA advisory panel is expected to review its Duchenne drug, eteplirsen, on Jan. 22. An FDA panel will weigh in on a rival drug from BioMarin Pharmaceutical (NASDAQ: BMRN) on Nov. 24.

FierceBiotech reported that Moderna Therapeutics chief scientific officer and president of R&D Joseph Bolen left the company. His duties are being assumed by Moderna president Stephen Hoge and chief medical officer Tal Zaks, according to the report.

—The Wyss Institute spun out its second startup in two weeks, as Arch Venture Partners, Hansjoerg Wyss, and some unspecified private investors put $5.15 million into a company called UltiVue. The company has developed a technology that’s supposed to give reserachers detailed looks at cellular behavior and disease progression. Last week, the Wyss spun out another company, Opsonix, with an $8 million Series A.

Photo of Ambrose at the South Street Seaport courtesy of Joe Mabel.