its experimental gene therapy for Duchenne muscular dystrophy, which has shown promise in an early study. The FDA temporarily halted the study in July due to a manufacturing issue.
—The FDA approved duvelisib (Copiktra), from Verastem (NASDAQ: [[ticker:VSTM]]), for people with chronic lymphocytic leukemia, small lymphocytic leukemia, or follicular lymphoma who have failed at least two treatments. Duvelisib, which Verastem bought from Infinity Pharmaceuticals in 2016, is now the company’s first marketed product. It has a monthly list price of $11,800, which comes to $141,600 per patient, per year.
—The agency also cleared dacomitinib (Vizimpro), from Pfizer (NASDAQ: [[ticker:PFE]]), for advanced lung cancer patients whose tumors have one of two specific genetic alterations.
—Merck ended a policy mandating that its CEO retire at age 65, and announced current top executive Ken Frazier has agreed to stay aboard until at least December 2019, when he turns 65.
—Epizyme (NASDAQ: [[ticker:EPZM]]) said that the FDA has allowed the resumption of trials involving its top experimental drug tazemetostat. The green light comes five months after a new case of cancer in a patient prompted a partial hold on all tazemetostat trials. Epizyme enjoyed a brief stock bump, but shares remain well below their 52-week high.
—Lyra Therapeutics, a maker of ear, nose, and throat medicines, has raised $29.5 million to fund Phase 2 testing of its lead drug.
—Janssen Biotech terminated a four-year alliance with Geron (NASDAQ: [[ticker:GERN]]) and returned to the biotech full rights to the experimental blood disorder drug imetelstat. Geron’s stock price plummeted 68 percent on the news.
DATA DUMPS
—Alnylam Pharmaceuticals (NASDAQ: [[ticker:ALNY]]) could file for accelerated approval of its second RNA interference medicine, givosiran, by the end of the year. Interim results from a Phase 3 study showed the drug had an impact on people with acute hepatic porphyria, a potentially deadly genetic disease.
—Alexion Pharmaceuticals (NASDAQ: [[ticker:ALXN]]) is preparing to file for approval of its drug eculizumab (Soliris) after positive Phase 3 data in neuromyelitis optica spectrum disorder, a rare disease affecting the central nervous system. The blockbuster drug is already approved to treat two rare blood disorders.
—TG Therapeutics (NASDAQ: [[ticker:TGTX]]) delayed the release of Phase 3 data for a combination of drugs for the chronic lymphocytic leukemia and changed the goals of the study. TG did not say that the study failed. Instead, it blamed the delay on not having enough long-term information about the patients who responded to the drug. Investors weren’t buying it, sending shares down more than 40 percent this week. Here’s more from STAT.
—Shares of Lexington, MA-based Aldeyra Therapeutics (NASDAQ: [[ticker:ALDX]]) surged 35 percent after the company reported encouraging results from a mid-stage study of an anti-inflammatory drug, reproxalap, in dry eye disease. A Phase 3 study should begin next year.
—The New England Journal of Medicine published interim results from a 3,573-patient Phase 2 study of M72/AS01e, an experimental vaccine from GlaxoSmithKline (NYSE: [[ticker:GSK]]) for tuberculosis. The vaccine may be able to reduce the incidence of tuberculosis in adults who don’t have HIV infections, but the results will have to be confirmed with further testing.
WHEELING & DEALING
—Alexion paid $400 million in cash up front to acquire Syntimmune, a New York and Waltham, MA, startup developing a drug for rare autoimmune diseases.
—Women’s health company Hologic (NASDAQ: [[ticker:HOLX]]) bought Focal Therapeutics for $125 million cash. Privately held Focal has commercialized a marker used in breast-conserving surgery.
—Roche bought Tusk Therapeutics, a U.K.-based developer of cancer immunotherapy drugs focused on immune cells known as regulatory T cells, for about $81 million up front.
—Eli Lilly (NYSE: [[ticker:LLY]]) paid Chugai $50 million up front for an oral diabetes drug on the verge of its first human tests. The drug gives the Indianapolis pharmaceutical giant a new asset to challenge Novo Nordisk (NYSE: [[ticker:NVO]]) in the race to bring a pill version of the injectable GLP-1 diabetes drugs that help control patients’ blood sugar.
—Cerecor (NASDAQ: [[ticker:CERC]]) is paying approximately $27 million in stock to buy Ichorion Therapeutics, a developer of drugs for rare metabolic diseases.
EVENT NEWS
—Don’t miss “Boston’s Life Science Disruptors” on Oct. 17 at the Koch Institute for Integrative Cancer Research. This year we’ll be featuring behind-the-scenes looks at startups aiming to target RNA with drugs, take CRISPR gene editing to the next level, and study the genetic activity of single cells. You can register here.
Frank Vinluan and Alex Lash contributed to this report.
