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East Coast Biotech Roundup: Sarepta, Aegerion, Syros, Aileron & More

Xconomy Boston — 

All types of scary news in the life sciences world in time for Halloween, from the highest levels of Big Pharma, where Sanofi’s board unceremoniously dumped CEO Chris Viehbacher, to biotech, where three local companies were put in the Wall Street penalty box. Details from the East Coast scene below:

—Shares of Cambridge, MA-based Sarepta Therapeutics (NASDAQ: SRPT) fell sharply this week after the company revealed that it likely won’t file a new drug application with the FDA for its Duchenne Muscular Dystrophy drug, eteplirsen, until mid-2015 at the earliest. The FDA has made a series of new requirements of Sarepta, has questioned its method for tabulating its existing data, and asked for an independent assessment of it. The FDA even uncharacteristically published a response letter to explain its decision and dealings with Sarepta—a response to public pressures from patient advocacy groups.

—Shares of Cambridge-based Aegerion Pharmaceuticals (NASDAQ: AEGR) also plummeted 30 percent after sales of its rare disease drug, lomitapide (Juxtapid), came in well short of expectations and the company significantly lowered its financial projections for 2014. It’s been a precipitous fall for Aegerion. The company traded at close to $90 per share in 2013, but has now lost more than two thirds of its value as lomitapide’s launch has disappointed.

—Cambridge-based Akebia Therapeutics (NASDAQ: AKBA) was the third local biotech to get slammed by Wall Street. Akebia announced positive results for its oral anemia pill, AKB-6548—the drug met the primary endpoint of its Phase 2b study—but that release also revealed some potentially significant safety issues, including one patient death “possibly” related to treatment.

—Cambridge-based Aileron Therapeutics added $33 million to its existing Series E round to help bring its stapled peptide drug targeting the tumor suppressor protein p53 to its first clinical trial. All of Aileron’s existing backers, including the venture arms of GlaxoSmithKline, Novartis, Eli Lilly, and Roche, participated in the funding. I spoke with CEO Joseph Yanchik about the financing, and the company’s plans for the coming study.

—Watertown, MA-based Syros Pharmaceuticals raised $53 million in Series B cash from Polaris Partners, Aisling Capital, Redmile Group, Flagship Ventures, Arch Venture Partners, WuXi PharmaTech Corporate Venture Fund, and Alexandria Venture Investments. Syros CEO Nancy Simonian told Xconomy the cash should be enough to get the company’s first drug into clinical trials; its lead drug blocks a transcriptional kinase called CDK7.

—Cambridge-based Affinivax emerged from stealth with a $4 million seed investment from the Bill & Melinda Gates Foundation. The company, spun out of the work of Richard Malley at Boston Children’s Hospital, is developing a platform for quickly and cheaply developing conjugate vaccines that provoke a broad immune response. I spoke with Malley and CEO Steven Brugger about that technology.

—Shares of New York-based Bristol-Myers Squibb (NYSE: BMY) soared 8 percent after it posted encouraging data from a Phase 2 study of its PD-1 checkpoint inhibitor, nivolumab (Opdivo), in non-small cell lung cancer. Bristol reported 41 percent of the treated patients in its study were alive after one year, beating expectations. Separately, Bristol acquired an option to buy UK-based F-Star Alpha Ltd. and a cancer drug it’s developing for up to $475 million.

—Cambridge-based Mersana Therapeutics said that Millennium Pharmaceuticals, the Boston subsidiary of Takeda, exercised an option to license an antibody-drug conjugate the two have been developing over the past seven months. Terms of the deal weren’t disclosed, however.

—One week after Actavis (NYSE: ACT) nabbed an option to acquire a diabetic gastroparesis drug being developed by Rhythm Pharmaceuticals, the Boston company has pulled itself out of the IPO queue because of the deal. Rhythm filed in late August to go public.

Photo courtesy of Andrea Morlans via Creative Commons.