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

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: [[ticker: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: [[ticker:ALNY]]) and its partner The Medicines Co. (NASDAQ: [[ticker: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

Author: Ben Fidler

Ben is former Xconomy Deputy Editor, Biotechnology. He is a seasoned business journalist that comes to Xconomy after a nine-year stint at The Deal, where he covered corporate transactions in industries ranging from biotech to auto parts and gaming. Most recently, Ben was The Deal’s senior healthcare writer, focusing on acquisitions, venture financings, IPOs, partnerships and industry trends in the pharmaceutical, biotech, diagnostics and med tech spaces. Ben wrote features on creative biotech financing models, analyses of middle market and large cap buyouts, spin-offs and restructurings, and enterprise pieces on legal issues such as pay-for-delay agreements and the Affordable Care Act. Before switching to the healthcare beat, Ben was The Deal's senior bankruptcy reporter, covering the restructurings of the Texas Rangers, Phoenix Coyotes, GM, Delphi, Trump Entertainment Resorts and Blockbuster, among others. Ben has a bachelor’s degree in English from Binghamton University.