East Coast Biotech Roundup: Sarepta, Zalicus, Alkermes, & More

Xconomy Boston — 

It’s been an optimistic year for biotech. IPOs have come fast and furious, and have made a lot of money for investors. The Nasdaq Biotechnology Index is up big. But this week offered a cold reminder of the high risk that comes with those potentially high rewards. Those stories and more below:

—Cambridge, MA-based Sarepta Therapeutics’ (NASDAQ: SRPT) chances of getting accelerated approval based on a promising, 12-patient data set of patients with Duchenne Muscular Dystrophy has been one of the year’s most closely watched biotech stories. Unfortunately for the company, the FDA squashed those hopes earlier this week. The FDA told the company its new drug application for eteplirsen is “premature.” While Sarepta still hopes to change the FDA’s mind, it will likely now have to go the traditional route and put together a confirmatory, Phase III, placebo-controlled study that could take a few years to set up, enroll patients in, and complete. With eteplirsen’s approval delayed by two years or more, and the uncertainty surrounding the trial design, Sarepta’s shares plummeted more than 60 percent.

—Be sure to check out Brad Loncar’s guest editorial on Sarepta as well.

—Cambridge-based Zalicus (NASDAQ: ZLCS) had been waiting for years to find out whether the reformulation work it did to a failed pain drug would pay dividends. Its day of reckoning came Monday, and the results weren’t good. That drug, Z160, failed both of its mid-stage clinical trials. It’s a big setback for Zalicus, which will now turn its attention to another pain drug that is much earlier in development. Investors aren’t waiting around to find out how that turns out—they sent Zalicus stock freefalling by about 75 percent.

—Today, Dublin and Waltham, MA-based Alkermes (NASDAQ: ALKS) is rolling along with an emerging pipeline and a market value approaching $5 billion. But just over 10 years ago, it was on the brink of disaster. Xconomy’s National Biotech Editor, Luke Timmerman, spoke to Alkermes CEO Richard Pops about how the company survived its own brush with death in 2002, when the FDA rejected a drug New Brunswick, NJ-based Johnson & Johnson (NYSE: JNJ) developed using the company’s drug delivery technology.

—Johnson & Johnson won FDA approval this week of ibrutinib (Imbruvica), a highly-anticipated blood cancer drug it co-developed with Sunnyvale, CA-based Pharmacyclics (NASDAQ: PCYC). Ibrutinib is approved to treat patients with mantle cell lymphoma, a small patient group, but the two companies are also testing the drug in a wide range of cancers. All-told, the expectations for ibrutinib are sky-high. JP Morgan analyst Cory Kasimov is estimating more than $6 billion in potential peak annual sales for the drug.

—Cambridge-based Catabasis Pharmaceuticals is signaling that an IPO is on its long-term to-do list. The biotech has raised a $32.4 million Series B round from its existing investors (SV Life Sciences, Clarus Ventures, MedImmune Ventures, and Advanced Technology Ventures), a new investor (Lightstone Ventures), and importantly, an unspecified public crossover fund. Catabasis CEO Jill Milne acknowledged that an IPO is a possibility, but that the company wants to get its lead drug, CAT-2003, further into clinical trials and develop some of its other assets before it takes the leap.

—New Enterprise Associates has been investing in Boston biotech for many years without having a physical footprint. That changed this week, as the investment firm opened a new office in Kendall Square. General partner David Mott told me that the time was right for such a move, given that NEA is more active now in Boston than at any point in its 36-year history.

—Cambridge-based Zafgen revealed the full results from the Phase II study of its injectable fat-zapping drug, beloranib, and the numbers held up from the top-line figures it announced earlier this year. Patients taking the highest dose of beloranib (2.4 milligrams) lost an average of about 24 pounds (10.9 kg) over the course of the 12-week study. Patients in the mid-dose group (1.2 mg) shed about 15 pounds on average, while those getting the low dose (0.6) lost an average of about 12.4 pounds over the course of treatment. Zafgen tested 147 patients in the trial—mostly obese women about 48.4 years old—and 122 of them completed the study. The most common side effects seen in patients taking the drug were nausea, vomiting, diarrhea, and trouble sleeping.

—The Broad Institute of MIT and Harvard got a new $100 million gift from philanthropists Eli and Edythe Broad. The Broads have now donated about $700 million to the institute since its founding in 2003. Just a few weeks ago, the Broad Institute got a $74 million gift from the Carlos Slim Foundation to advance research in genomic medicine.

—The FDA has given a fast-track designation to Cambridge-based Alnylam Pharmaceuticals’s (NASDAQ: ALNY) experimental RNA-based drug, patisiran, for transthyretin familial amyloid polyneuropathy, meaning it will get a speedier review from the agency than it would have otherwise. Alnylam is currently enrolling patients in a Phase III trial.

—Waltham-based GeNo Healthcare this week became the latest biotech to file for an IPO. The company, which is developing a new way to deliver inhaled nitric oxide, plans to raise up to $50 million from public investors. Founder, executive chairman and chief scientific officer David Fine is by far the company’s largest shareholder, owning 58.2 percent of GeNo’s stock. Jefferies LLC, Stifel, Nicolaus & Co., and Canaccord Genuity are underwriting the offering.

—The FDA has approved Marlborough, MA-based Sunovion Pharmaceuticals’s anti-seizure drug, eslicarbazepine (Aptiom), as an add-on therapy for patients with epilepsy. Sunovion is a unit of Dainippon Sumitomo Pharma. When known as Sepracor, the company was acquired by the Japanese pharma giant for $2.6 billion.