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East Coast Biotech Roundup: X4, Rgenix, Alnylam, Intellia & More

Xconomy Boston — 

What summer doldrums? It’s still not even Labor Day, yet the deals are already coming fast and furious. So fire up the grill and read on for some Series A rounds, crossover investments, data readouts, option-to-buy deals and more.

—As Xconomy reported this week, a stealthy Cambridge, MA-based startup called X4 Pharmaceuticals raised a $37.5 million Series A round. X4 is staffed, in part, by a group of former Genzyme executives, and former Genzyme CEO Henri Termeer is one of its backers. I spoke with CEO Paula Ragan about the company’s plans, which involve going after the same molecular target as an approved Genzyme drug, plerixafor (Mozobil)—but in a completely different way.

—I’ve written extensively about the problems New York City has had birthing and keeping its own biotechs. One of the startups fighting against that trend is a young company out of Rockefeller University called Rgenix. The company, formed in part by a group of scientist-brothers working at Columbia University, Rockefeller, and Memorial Sloan Kettering Cancer Center, recently raised $8 million. Two co-founders, Masoud Tavazoie and Shahram Seyedin-Noor, told me what the company intends to do with a drug discovery engine that, they say, could potentially unearth some novel drug targets for cancer.

—After more than a decade of ups and downs, Cranbury, NJ-based Amicus Therapeutics (NASDAQ: FOLD) is on the doorstep of its first drug approval. This week, it went the M&A route to add another to its portfolio. Amicus paid $229 million up front for Durham, NC-based Scioderm and its experimental drug for a rare, debilitating skin disease called epidermolysis bullosa. I spoke with CEO John Crowley about the deal, which could have an additional benefit, if Scioderm’s drug succeeds in Phase 3 testing and wins FDA approval—a priority review voucher.

—Amicus wasn’t the only one buying biotechs this week. Bristol-Myers Squibb paid $150 million for an option to acquire Lexington, MA-based Promedior if it likes what it sees from mid-stage studies of the startup’s fibrosis drug, PRM-151. Should Bristol exercise the option and buy Promedior—no guarantee, of course—it could shell out a total of $1.25 billion in total payments. CEO Suzanne Bruhn gave me some more details on the thinking behind the deal as well.

—Another week, another crossover round, as Cambridge-based CRISPR-Cas9 gene-editing startup Intellia Therapeutics nabbed a $70 million Series B financing from firms including OrbiMed and Fidelity. The round comes just a few weeks after rival Editas Medicine raised a $120 million round of its own. Alex Lash has more on the financing, and the state of the fast-moving CRISPR-Cas9 world.

—Cambridge-based Alnylam Pharmaceuticals (NASDAQ: ALNY) and its partner The Medicines Co. (NASDAQ: MDCO) disclosed the first clinical results for a cholesterol-lowering RNA interference drug they’re co-developing, and the numbers indicated that—at least so far—it might pose a threat down the road to the newly approved PCSK9-blocking drugs from Amgen and Regeneron Pharmaceuticals/Sanofi. In a small trial, ALN-PCS lowered patients’ LDL, or bad cholesterol levels by an average of 44 percent after 140 days. Here’s more on the study from Forbes and FierceBiotech. The ALN-PCS program was one that CEO John Maraganore told me in January he wouldn’t have … Next Page »

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