Between a battery of snowstorms, there was a parade this past week in Boston, as New Englanders lined the streets to celebrate Pete Carroll’s horrific goal-line call—I mean, a Patriots Super Bowl victory (congrats, Pats fans). In biotech, the theme of the week was volatility. Indices like the Nasdaq Biotechnology Index (NASDAQ: NBI) vastly underperformed the greater market. FDA commissioner Margaret Hamburg announced she’ll be stepping down in March, amid speculation that the newly hired Duke University cardiologist Robert Califf will fill her shoes. The agency granted a “breakthrough therapy” designation to one company, and stripped it from another. We’ve got plenty more headlines below. Let’s get right to it.
—WaVe Life Sciences—a company formed out of the merger of two small companies, Boston-based Ontorii and Japan’s Chiralgen—raised $18 million this past week from RA Capital Management, Kagoshima Shinsangyo Sosei Investment, and SNBL. The company aims to use a specific kind of chemistry work to make RNA-based drugs that are more potent than those synthesized by existing methods. I spoke to CEO Paul Bolno about the effort.
—Cambridge, MA-based Lysosomal Therapeutics hauled in a $20 million Series A round from a group of investors including Atlas Venture and the venture arms of Eli Lilly, Sanofi, and Roche. LTI is developing a drug for Parkinson’s disease that is meat to exploit a link between neurodegeneration and a family of rare genetic disorders like Gaucher.
—One of the ways venture investors can hedge their bets is through “build to buy” deals; startups created with a potential buyer already in place. Cambridge, MA-based Annovation Biopharma is the latest example, and this week it rewarded its backers when it was sold to The Medicines Co. (NASDAQ: MDCO) for potentially more than $60 million. Medicines will now move Annovation’s lead drug candidate—an experimental anesthetic called ABP-700—into further testing. The Parsippany, NJ-based company opted to buy Annovation after positive signs in Phase 1 studies.
—The FDA awarded a breakthrough therapy designation to Cambridge-based Bluebird Bio’s (NASDAQ: BLUE) LentiGlobin, a prospective gene therapy for beta-thalassemia. Drugs winning the designation get a speedier review from the agency than they would otherwise. In the past year, for instance, both Bristol-Myers Squibb’s nivolumab (Opdivo) and Merck’s pembrolizumab (Keytruda)—cancer immunotherapy drugs—were approved months earlier than they would have because of breakthrough status.
—New York-based Pfizer (NYSE: PFE) also benefited from the FDA’s breakthrough tag, winning approval of its highly anticipated breast cancer drug, palbociclib (Ibrance), two months before the company expected a decision. Palbociclib will reportedly cost $9,850 per month, or $118,200 per year of treatment.
—The FDA showed that in certain cases it wouldn’t hesitate to strip the breakthrough designation. The agency rescinded the breakthrough status it had granted to a regimen of two drugs—grazoprevir and elbasvir—Merck has been developing for hepatitis C, citing the availability of other recently approved treatments from Gilead Sciences (NASDAQ: GILD) and AbbVie (NYSE: ABBV).
—This past week, our old colleague Luke Timmerman started his own subscription-based biotech news service, “The Timmerman Report.” One of his first reports was that Cambridge-based Ember Therapeutics, a Third Rock Ventures-backed startup aiming to develop obesity drugs, has been shut down.
—Biogen Idec (NASDAQ: BIIB) may be leading the most advanced, high-profile drug program designed to reverse the effects of multiple sclerosis. But Ardsley, NY-based Acorda Therapeutics (NASDAQ: ACOR) is joining the fray as well. Acorda this past week reported results from a Phase 1 study of rHIgM22, a drug it hopes can help “remyelinate,” or undo the nerve damage that characterizes MS. The drug proved to be safe in a small group of healthy volunteers, which is enough for Acorda to take it into further testing. Whether the drug actually helps MS patients won’t be known for some time.
—Xconomy announced two biotech events this week. For you Big Apple folks, we’re holding our latest installment of “New York’s Life Science Disruptors” on March 12. Our annual spring Boston event, “What’s Hot in Boston Biotech,” is set for April 8. We’ll have more to come on both of these in the next few weeks.
—Lexington, MA-based Synta Pharmaceuticals (NASDAQ: SNTA) plans to slash 20 percent of its workforce, its second round of job cuts since the beginning of 2014. The company has shifted most of its resources to two programs: ganetespib, in Phase 3 for lung cancer and acute myeloid leukemia and Phase 2 for breast and ovarian cancer; and a preclinical cancer drug called STA- 12-8666 that it hopes to begin testing in humans next year.
—Sarissa Capital Management, the firm run by activist investor Alex Denner, has disclosed a 5.8 percent stake in Cambridge-based Aegerion Pharmaceuticals (NASDAQ: AEG), along with intentions to “engage in discussions” with the struggling company. Sarissa believes Aegerion’s shares are undervalued; they’re worth less than a third of what they were over a year ago.
Photo courtesy of flickr user Drew McKenzie via Creative Commons