Imara Inc. reported a mixed bag of phase IIa study data for its lead candidate, IMR-687, for treating sickle cell disease in adults, bringing an end to a study that the company found cumbersome and that eventually stung its stock.
Syros Pharmaceuticals Inc. and Global Blood Therapeutics Inc. (GBT) agreed to a discovery, development and commercialization deal to treat sickle cell disease (SCD) and beta-thalassemia. GBT, of South San Francisco, Calif., will pay Syros $20 million up front and fund up to $40 million in preclinical research for at least three years, with the goal of identifying targets and discovering drugs to induce fetal hemoglobin.
Protagonist Therapeutics Inc. stock (NASDAQ:PTGX) nosedived 47% on Tuesday after the company released preliminary results from its phase II open-label study of PTG-300, an injectable hepcidin mimetic to treat patients with transfusion-dependent beta-thalassemia.
DUBLIN – Crispr Therapeutics AG has delivered what appears, so far at least, to be a safe, functional cure for the first patient enrolled in each of its phase I/II trials of lead CRISPR/Cas9 gene editing therapy CTX-001, in beta-thalassemia and in sickle cell disease.
Nearly a month ahead of the PDUFA date, red blood cell maturation drug luspatercept cleared the FDA for treating anemia in adults with beta-thalassemia who require regular red blood cell (RBC) transfusions. Branded Reblozyl, the drug, developed in a collaboration between Celgene Corp. and Acceleron Pharma Inc., is expected to be available in one week following approval.
