East Coast Biotech Roundup: Bluebird, Moderna, Navitor, & More

6/20/14Follow @benthefidler

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use T-cells from a single donor in multiple patients. Like the rest of the CAR-T field, these efforts are very early—Cellectis doesn’t have a prospective CAR-T therapy in clinical trials as of yet.

—Summit, NJ-based Celgene (NASDAQ: CELG) and Patrick Soon-Shiong have cut another deal. Just a few months after teaming with Shiong’s Los Angeles-based NantBioscience to develop nanoparticle albumin-bound cancer drugs and putting $75 million into the company, Celgene has poured $25 million into another Shiong entity, NantHealth, a healthcare cloud-based IT company developing an operating system to help with cancer diagnosis and treatment. Celgene and Shiong first teamed up when the big cancer drugmaker bought Shiong’s Abraxis Bioscience for $2.9 billion in 2010. It got protein-bound paclitaxel (Abraxane) out of the deal.

—Celgene also showed more support for Agios Pharmaceuticals (NASDAQ: AGIO), exercising an option to grab worldwide rights to the Cambridge-based company’s blood cancer drug, AG-221. Agios is still eligible to receive up to $120 million in milestone payments and royalties on sales of AG-221, and has an option to help commercialize the drug in the U.S. Agios also provided updated data from an ongoing study of AG-221. I covered the initial results from that study in April.

—Boston-based Vertex Pharmaceuticals (NASDAQ: VRTX) continued to sharpen its focus on cystic fibrosis this week, licensing a prospective influenza drug called VX-787 to Johnson & Johnson (NYSE: JNJ) subsidiary Janssen Pharmaceuticals. Vertex received a $30 million cash payment up front, and is eligible to receive additional unspecified milestone and royalty payments. Vertex wrapped up a Phase 2a study of VX-787 last year.

—Sanofi and New York-based Regeneron Pharmaceuticals (NASDAQ: REGN) provided updated data from one of the six Phase 3 studies of their rheumatoid arthritis antibody drug prospect, sarilumab, at the European League Against Rheumatism’s annual meeting in France. The two companies released initial data from the study last fall, but added new numbers this week showing that the response rates seen in rheumatoid arthritis patients have largely held up over 52 weeks of treatment. Sarilumab is one of several drugs Sanofi and Regeneron are working on together, including dupilumab, a prospect for allergic asthma, and cholesterol-lowering drug alirocumab.

—Fresh off raising a massive $200 million private financing round, Hayward, CA- and Boston-based Intarcia Therapeutics presented the first data from a 60-patient Phase 3 study of ITCA 650, its drug/device combination therapy for type 2 diabetes, at the American Diabetes Association’s annual meeting in San Francisco. Intarcia said after six months, ITCA 650 helped lower the blood sugar levels of 78 percent of the patients in the study by at least 2 percent, 50 percent of them by 3 percent, and 22 percent of them by at least 4 percent. Intarcia expects to report full results from the study in September.

—Watertown, MA-based Selecta Biosciences made a flurry of moves this week. The startup nabbed about $9 million in grants from the Bill & Melinda Gates Foundation and the National Institutes of Health, cut a deal with China’s 3SBio to make a souped-up version of its gout drug, pegsiticase, and revealed new programs that it’s developing for hemophilia A, life-threatening allergies, and type-1 diabetes. Selecta came out of the lab of MIT professor Bob Langer a few years ago with a plan to develop polymer nanoparticle vaccines that are supposed to selectively target a subset of white blood cells, and thus elicit more powerful immune responses than traditional vaccines while limiting side effects.

—Shares of Monmouth Junction, NJ-based Insmed (NASDAQ: INSM) soared more than 40 percent after the FDA unexpectedly granted a “breakthrough therapy” designation to the company’s inhalable antibiotic, liposomal amikicin (Arikayce), as a treatment for nontuberculosis mycobacterial lung infections. The FDA decision, based on the Phase 2 results Insmed revealed earlier this year, means the drug will get a speedier review from the agency than it otherwise would have. Insmed’s drug prospect has seen its share of ups and downs over the years. It faced a clinical hold in 2011, and actually failed to meet its main goal in the Phase 2 study that led to this week’s FDA decision (Insmed said at the time that the drug succeeded by a different, secondary measure that it hadn’t expected to hit, yet found more significant).

—Cambridge-based BIND Therapeutics (NASDAQ: BIND) added another name to the list of partners tapping into its nanoparticle technology to develop drugs—this time, Roche, which will work with the company to create nanodrugs in therapeutic areas outside of oncology. Financial terms of the deal weren’t disclosed. Bind already has deals in place with Pfizer, AstraZeneca, and Amgen.

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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