Feb. 17 Quick Takes: Agios wins FDA approval in rare anemia

More than a year after selling off its cancer business, Agios Pharmaceuticals Inc. (NASDAQ:AGIO) has gained its first non-oncological FDA approval. The agency authorized Pyrukynd mitapivat to treat hemolytic anemia associated with pyruvate kinase deficiency, for which the PKR activator was under priority review. Pyrukynd had met the primary endpoints in two Phase III trials for the indication. CEO Jacqualyn Fouse said last year Agios believes Pyrukynd “easily has blockbuster potential across a range of hemolytic anemias”; the biotech hopes to add approvals for sickle cell disease and thalassemias by 2026.

Arvinas Inc. (NASDAQ:ARVN) said it intends to initiate accelerated approval discussions with FDA and a pivotal trial by year-end for bavdegalutamide (ARV-110), a proteolytic targeting chimera (PROTAC) androgen receptor degrader, in a molecularly defined subset of metastatic castration-resistant prostate cancer (mCRPC). Phase I and interim Phase II data showed the bavdegalutamide had a PSA50 rate of 46% in 28 patients with androgen receptor T878X/H875Y tumor mutations, and the company reported 2 durable RECIST partial responses out of seven RECIST-evaluable patients with the mutations. The company also said PSA reduction and RECIST signals were observed across all patient subgroups regardless of mutation status, but did not provide details about patient subgroups without T878X/H875Y mutations...