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

4/25/14Follow @benthefidler

It was megamerger week in the pharma world. Novartis, GlaxoSmithKline, and Eli Lilly executed the drugmaking equivalent to the most complex three-way fantasy football trade of all time. Pfizer was rumored to make a kajillion dollar bid for AstraZeneca. Allergan has become the ever-acquisitive Valeant Pharmaceuticals’ latest target. Even a $13 billion med tech buyout was signed. Biotech? Left out of the dealmaking party—for now—but active nonetheless. Your East Coast headlines below.

—Cambridge, MA-based Sarepta Therapeutics’ (NASDAQ: SRPT) rollercoaster ride took a positive turn this week as it came to a deal with the FDA that, at the very least, outlines a potential path to early approval for its Duchenne Muscular Dystrophy drug, eteplirsen. Sarepta has to add more data from existing and future studies to prove its case to the FDA, but the new guidelines were enough for Sarepta to say that it plans to file a new drug application by the end of the year and has a shot to win accelerated approval of eteplirsen next year. This is, of course, by no means a guarantee. The FDA, caught between a potentially promising drug for a debilitating disorder with no effective treatments, pressure from patient advocacy groups, and yet a very small sample size of results, remains skeptical and is treading carefully. But the news nonetheless gives Sarepta a chance to win approval of eteplirsen before completing a long Phase 3 study—sure to be one of the most compelling stories in biotech over the coming year. Shares of Sarepta soared 40 percent, and it immediately capitalized by raising about $101 million in a stock offering.

—There are many ways to build a biotech. Richard Pops, the CEO of Alkermes (NASDAQ: ALKS), chose the deliberate route. Alkermes has used a step-by-step process of partnerships, capital raises, and acquisitions to turn itself from a drug delivery company to a drugmaker. With the company on the precipice of moving its own internal pipeline into the spotlight, I spoke with Pops about his strategy of building Alkermes over the past two decades, and the give and take of relying on pharma partnerships to get there.

—On April 16, we hosted our latest life sciences event, “What’s Hot in Boston Biotech,” at Biogen Idec’s campus in Cambridge, MA, bringing together some of the region’s most intriguing startups, innovators, executives, and venture capitalists. Startups like Editas Medicine and Syros Pharmaceuticals, and more established companies like Alnylam Pharmaceuticals (NASDAQ: ALNY) and Cubist Pharmaceuticals (NASDAQ: CBST), gave attendees an in-depth look at the bold scientific ideas coming out of the local life sciences scene, and the struggles they’re facing to make them a reality. You can read my recap of the festivities here.

—Cambridge-based Zafgen signaled pretty loudly an IPO was coming when it added names like RA Capital, which typically backs public companies, to its investor pool in December. This week, it officially jumped into the queue, filing an S-1 outlining plans to raise up to $86 million and trade on the Nasdaq under the ticker symbol ZFGN. Zafgen, backed largely by Atlas Venture (35.6 percent stake) and Alta Partners (35.4 percent), would use the cash to gear up for a group of mid- and late-stage trials on its fat-burning drug hopeful, beloranib. Rather than target the slightly overweight folks that want to shed a few pounds, Zafgen is trying to position beloranib as a treatment for severely obese people and smaller subsets of patients with rare conditions, like Prater-Willi Syndrome.

—The market for age-related macular degeneration is dominated by injectable treatments. Waltham, MA-based Kala Pharmaceuticals is one of the companies trying to bring an eye drop into the mix, and it grabbed $22.5 million in venture cash this week to fund the effort. New investor Ysios Capital led the round, which also included a new strategic backer—AbbVie Biotech Ventures—and existing investors Lux Capital, Third Rock Ventures, and Polaris Partners. Kala is creating nanoparticle drugs attached to engineered polymers that are supposed to be so small they can slip through the eye’s various defenses, and potentially hit targets in the front, middle, and even back of the eye. I spoke with Kala CEO Guillaume Pfefer about the company’s strategy of managing risk and capital while it tries to develop an eye drop for the wet form of AMD.

—Judging by its swath of creative, and often expensive, early-stage partnerships, Summit, NJ-based Celgene (NASDAQ: CELG) isn’t afraid to make a bold bet. This week, while announcing its latest quarterly earnings, Celgene did just that, announcing a whopping $710 million up front—and potentially another roughly $2 billion in various payments down the road—to Dublin’s Nogra Pharma for the rights to a Crohn’s disease drug prospect on the verge of Phase 3 studies. Celgene is best known for its blood cancer drugs, but it has been trying to grow a franchise in immunology, recently winning FDA approval of its first drug in the space, apremilast (Otezla) a few weeks ago. The Nogra drug, GED-0301, is an antisense drug that binds to the messenger RNA Smad7 that’s being developed for moderate-to-severe Crohn’s and other diseases. Celgene said the drug has completed Phase 2 studies, but the data haven’t been made public yet—they’ll be presented at an upcoming medical meeting. Celgene will start a Phase 3 study by the end of the year. Still, investors didn’t react positively to the news, or Celgene’s earnings—shares immediately traded down 5 percent.

—Biogen (NASDAQ: BIIB) also reported earnings this week, and along with them, news that the oral multiple sclerosis drug dimethyl fumarate (Tecfidera), became a blockbuster drug in its first year, generating close to $1.4 billion in revenue since Biogen began selling it in March 2013. The drug brought in $506 million in the first quarter of 2014, Biogen reported.

— Enanta Pharmaceuticals (NASDAQ: ENTA) got $20 million richer this week, as AbbVie (NYSE: ABBV) sent a check to the Watertown, MA-based company after it submitted a new drug application to the FDA for an all-oral regimen of drugs for the liver disease that includes ABT-450, the protease inhibitor the two companies co-developed. Enanta is in line to pick up much more cash than that from AbbVie if ABT-450 keeps progressing, including $20 million when AbbVie files an application in Europe, which is expected in May. I profiled Enanta’s journey out of obscurity and into the high-stakes hepatitis C race back in January.

—New Brunswick, NJ-based Johnson & Johnson (NYSE: JNJ) won FDA approval of siltuximab (Sylvant) to treat multicentric Castleman’s disease, a rare blood disorder in which white blood cells are over-produced. It’s the first approved treatment in the U.S. for the disease, which affects between 1,100 and 1,300 people in the country.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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