Agios Reports Updated Data From AG-348 Trial - Quick Facts

(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO) reported updated data from its pyruvate kinase-R (PKR) activator demonstrating the potential for the first disease-modifying treatment for patients with pyruvate kinase deficiency. DRIVE PK is an ongoing global open-label, Phase 2, safety and efficacy trial evaluating AG-348 in adult, transfusion-independent patients with PK deficiency. The company reported that 48% of all 52 treated patients and 57% of patients with at least 1 missense mutation treated with AG-348 experienced a maximum Hb increase from baseline. A safety analysis conducted for all 52 treated patients as of the data cut-off shows that AG-348 continues to be well tolerated.

Chris Bowden, chief medical officer at Agios, stated: "The rapid and sustained hemoglobin increases shown in DRIVE PK, combined with improvements in hemolysis related parameters, indicate that AG-348 is having a meaningful impact on the biology of PK deficiency. We look forward to advancing this novel investigational therapy into a planned global pivotal program in the first half of 2018."

Separately, Agios announced new efficacy and safety data from the ongoing Phase 1/2 dose-escalation and expansion study evaluating investigational oral IDHIFA (enasidenib) in patients with relapsed or refractory acute myeloid leukemia and an isocitrate dehydrogenase-2 mutation. The data demonstrated an overall response rate of 37 percent, including a complete response rate of 20.1 percent in 214 patients with relapsed or refractory acute myeloid leukemia who received enasidenib at 100 mg daily, which was the recommended starting dose in the expansion phases of the trial. The overall safety profile observed for enasidenib was consistent with previously reported data.

The New Drug Application for IDHIFA is currently under Priority Review with the U.S. FDA for the treatment of patients with relapsed or refractory acute myeloid leukemia with an IDH2 mutation. The NDA has been given a Prescription Drug User Fee Act action date of August 30, 2017.