Oct. 7 Quick Takes: label expansion for Alnylam’s Oxlumo

FDA extended the label of Oxlumo lumasiran from Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY) to more advanced patients with primary hyperoxaluria type 1 (PH1), an ultra-rare disease that leads to kidney stones and kidney failure. The siRNA therapy, which lowers urinary and plasma oxalate levels, was first approved for PH1 in 2020, making it the first targeted therapeutic for the condition.

Five years after the first autologous CAR T cell therapy was approved, an off-the-shelf allogeneic CAR T program, ALLO-501A, is moving into a Phase II trial that Allogene Therapeutics Inc. (NASDAQ:ALLO)
expects to support regulatory approval in the U.S. ALPHA2 is a single-arm study testing the CD19-directed CAR T therapy in relapsed/refractory large B-cell lymphoma (LBCL) patients. The primary endpoint is objective response rate (ORR). In prior trials, ALLO-501A showed slightly lower overall ORR than autologous CAR T cells in LBCL. In June, competitor Precision Biosciences Inc. (NASDAQ:DTIL) reported a 100% overall survival rate in 11 evaluable patients with  diffuse large B cell lymphoma treated with CD19-directed allogeneic CAR T PBCAR0191 in a Phase I/II trial. Additional data from ALLO-501A are expected at the American Society of Hematology meeting in December...