ASH Roundup: Immunotherapy Stars, Gene Therapy Leaps Ahead

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raise about $188 million in a follow-on offering on Wednesday.

The so-called PD-1 checkpoint inhibitors, meanwhile, proved they just might have a place in treatment regimens for various lymphomas—just not multiple myeloma, a disappointment to some of the experts in attendance.

We also outlined the uphill battle of Sunesis Pharmaceuticals (NASDAQ: SNSS) to gain approval for its leukemia drug, vosaroxin. The drug failed a Phase 3 trial earlier this year; Sunesis contends that certain subgroups of patients in that study had a statistically significant benefit, and will head to the FDA to file for approval of the drug in the first quarter of 2015.

But there was much more that we couldn’t get to from companies around the Xconomy network, from Boston to San Francisco. So we’ve decided to round up some of those tidbits below.

—Bluebird wasn’t the only company to report encouraging beta-thalassemia data at ASH. Shares of Cambridge-based Acceleron Pharma (NASDAQ: XLRN) climbed more than 13 percent Monday after positive results from mid-stage trials of two drugs, sotatercept and luspatercept (formerly ACE-536), for beta-thalassemia and myelodysplastic syndrome (a disease of the bone marrow). Both drugs helped increase patients’ hemoglobin levels and reduce some patients’ transfusion burdens. We profiled Acceleron’s efforts, and its prospective battle with Bluebird, back in July.

—Cambridge, MA-based Alnylam Pharmaceuticals (NASDAQ: ALNY) saw its shares rise five percent following two ASH releases. One highlighted interim Phase 1 data from ALN-AT3, a subcutaneous RNA interference drug for hemophilia and other rare bleeding disorders. Full data from the trial is due in mid-2015. The company also filed an application with U.K. regulators to begin a Phase 1/2 trial for ALN-CC5 in paroxysmal nocturnal hemoglobinuria, the ultra-rare disease treated by eculizumab (Soliris) from Alexion Pharmaceuticals (NASDAQ: ALXN). Assuming Alnylam gets clearance, initial data are expected next year.

Amgen (NASDAQ: AMGN) followed up last week’s FDA approval of the antibody blinatumumab (Blincyto) by releasing long-term survival data for adult patients with acute lymphoblastic leukemia. Of 36 treated, 10 have survived nearly 30 months, and seven of them were relapse-free. Amgen could also report that for multiple myeloma, carfilzomib (Kyprolis) in combination with two other drugs delivered a median progression-free survival of 26.3 months, which bests the previous top figure of 19 months, according to the Mayo Clinic investigator who presented the Phase 3 results.

—One of ASH’s biggest winners: Summit, NJ-based Celgene (NASDAQ: CELG). Several of its partners and equity investments—Agios, Acceleron, and Bluebird—rolled out positive data and saw shares climb. And the so-called PD-1 checkpoint inhibitors disappointed in multiple myeloma, kneecapping a potential threat to Celgene’s franchise.

—French firm Cellectis—which is partnered with New York-based Pfizer (NYSE: PFE)—said it plans to start human trials with its CAR-T immunotherapy platform that does not require a patient’s own T cells as the basis for the treatment. The firm says it has developed an … Next Page »

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