East Coast Life Sciences Roundup: IPO Madness, Amicus, & More

6/21/13Follow @benthefidler

The white-hot biotech sector kept humming along this week— particularly on the East Coast, where companies tripped over one another on their way to the Nasdaq. We’ve got that and a whole lot more below:

—Cambridge, MA-based Bluebird Bio (NASDAQ: BLUE) soared onto the stock market on Wednesday and saw its share price immediately take off. Bluebird priced its IPO on Tuesday, selling 5.94 million shares at $17 apiece and raising about $101 million. Those numbers blew past the targets it had set in May, when it aimed to sell 5 million shares at between $14 and $16 apiece. Bluebird is developing gene therapies for orphan diseases and severe genetic disorders such as childhood cerebral adrenoleukodystrophy, beta-thasselemia, and sickle cell disease, but has yet to begin clinical trials for its therapies. The company’s shares closed at $26.92 apiece after its first day of trading, an increase of nearly 60 percent.

—South Plainfield, NJ-based PTC Therapeutics (NASDAQ: PTCT) quickly followed Bluebird into the public markets, pricing about 8.4 million shares at $15 apiece and raising about $125 million. PTC expects to see about $114.2 million in proceeds after certain deductions. PTC priced near the high end of its $13 to $16 per share range and upsized from about 6.9 million shares it initially hoped to sell to investors, despite the fact that its lead drug, ataluren, has already failed two late-stage clinical trials. PTC is targeting subgroups of patients with Duchenne Muscular Dystrophy, a degenerative muscle disease, or cystic fibrosis, in cases where the disease is caused by a “nonsense mutation.” PTC’s shares closed at $16.55 apiece after its first trading day, an increase of about 10 percent.

—Speaking of Duchenne Muscular Dystrophy, Cambridge, MA-based Sarepta Therapeutics (NASDAQ: SRPT) showed that the results from the mid-stage study of its DMD drug, eteplirsen, continue to hold up after 84 weeks while showing no significant side effects. Xconomy’s National Biotech Editor, Luke Timmerman, recently chronicled Sarepta’s meteoric rise over the past year. Its stock trades at about $39 per share; it was worth around $4 per share in June 2012.

—Cambridge, MA-based OvaScience (NASDAQ: OVAS) might release its first product, Augment, next year, but the company’s biggest value driver is its next-generation form of in vitro fertilization, named OvaTure. OvaScience’s plan is to isolate immature egg cells known as egg precursor cells, mature them into young, healthy eggs, and show that they can be fertilized. If they can, OvaScience would be able to change the current treatment paradigm of IVF, and debunk the idea that a woman is born with a fixed number of eggs.

—Shares of Cranbury, NJ-based Amicus Therapeutics (NASDAQ: FOLD) tumbled more than 20 percent on Monday as the company indicated plans to delay filing a new drug application with the FDA for migalastat, its experimental oral drug for Fabry Disease. Amicus didn’t think it could get migalastat approved in the U.S. on the data it already has from its late-stage clinical trial, so it’s “trading time for certainty,” CEO John Crowley told me. Crowley also says the company is exploring potential partnerships to raise cash.

—Chelsea, MA-based Civitas Therapeutics, a spinout of Alkermes (NASDAQ: ALKS), landed its second grant from the Michael J. Fox Foundation since 2011. The new $1 million grant will help support a second mid-stage study for CVT-301, an inhalable version of levodopa, or L-dopa pills, a decades-old treatment for Parkinson’s Disease. Civitas CEO Glenn Batchelder says that the company plans to put a Series B round together to help fund the study as well; Civitas raised a $25 million Series A in 2011.

—Cambridge, MA-based Idenix Pharmaceuticals’ (NASDAQ: IDIX) shares plummeted late Thursday as the FDA requested more information about the safety profile of its preclinical hepatitis C uridine nucleotide prodrug, IDX20963, delaying the company’s plans to begin testing it in clinical trials. The FDA slapped clinical holds on two of Idenix’s other hepatitis C drugs, IDX184 and IDX19368, last year after a similar drug being tested by Bristol-Myers Squibb exhibited severe safety problems in clinical trials. Idenix quit developing the two drugs altogether. Idenix’s shares fell more than 25 percent in after-hours trading on Thursday.

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

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