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 partnered off had he known some things he knows now about the potential of subcutaneous RNAi drugs. Shares of Medicines Co., meanwhile, are up about 20 percent this week.

—Cambridge-based Sage Therapeutics (NASDAQ: SAGE) said that it’s seen enough positive signs from a small, exploratory study in patients with essential tremor to move a program into Phase 2 testing. The catch: it won’t be SAGE-547, the drug Sage used in the trial. CEO Jeff Jonas explained why; Sage is using its lead drug as a “probe” in tiny exploratory studies in various diseases, to see if follow-on drugs that work in the same way might have a chance to succeed.

—Waltham, MA-based Tesaro (NASDAQ: TSRO) has officially made the transition to a commercial-stage company, having won FDA approval of rolapitant (Varubi), an anti-nausea drug for cancer patients. Now the question is just how well rolapitant will sell in a competitive market with a number of already approved drugs, among them Merck’s aprepitant (Emend). Tesaro hasn’t yet disclosed the price of rolapitant.

—Cambridge-based Ariad Pharmaceuticals (NASDAQ: ARIA) became a case study in how M&A rumors affect stock prices. Ariad shares soared 42 percent on a Bloomberg report that Baxalta was weighing a buyout, and then plummeted back to similar levels after a follow-up story dispelled those rumors claiming talks had broken off.

—The FDA granted a priority review to New York-based Intercept Pharmaceuticals’s (NASDAQ: ICPT) experimental drug obeticholic acid as a treatment for primary biliary cirrhosis. The agency will decide whether to approve the drug by Feb. 29.

—The agency this week also bestowed fast track and qualified infectious disease product designations to Tetraphase Pharmaceuticals’s (NASDAQ: TTPH) intravenous formulation of TP-271, a broad spectrum antibiotic for a variety of respiratory bacterial infections. These designations are meant to speed up the FDA’s review of the drug.

—Amesbury, MA-based Ivenix, a medtech company with a next-gen infusion pump, got $42 million in venture funding led by WuXi Healthcare Ventures. The company’s pump delivery system delivers drugs at a steady, consistent rate and doesn’t require adjustment from nurses.

—Google, Paris-based drug giant Sanofi, and Boston-based Joslin Diabetes Center are teaming up in a bid to improve care and outcomes for people with Type 1 and Type 2 diabetes. The companies plan to work together to develop new tools to monitor and intervene with the disease.

David Holley contributed to this report

—Photo of Boston Skyline from Malone Park in Chelsea. Photo by Flickr user Bill Damon, used under Creative Commons license.