(RTTNews) – Agios Pharmaceuticals, Inc. (AGIO) reported updated information from a pyruvate kinase-R (PKR) activator demonstrating a intensity for a initial disease-modifying diagnosis for patients with pyruvate kinase deficiency. DRIVE PK is an ongoing tellurian open-label, Phase 2, reserve and efficiency hearing evaluating AG-348 in adult, transfusion-independent patients with PK deficiency. The association reported that 48% of all 52 treated patients and 57% of patients with during slightest 1 missense turn treated with AG-348 gifted a limit Hb boost from baseline. A reserve research conducted for all 52 treated patients as of a information cut-off shows that AG-348 continues to be good tolerated.
Chris Bowden, arch medical officer during Agios, stated: “The fast and postulated hemoglobin increases shown in DRIVE PK, total with improvements in hemolysis associated parameters, prove that AG-348 is carrying a suggestive impact on a biology of PK deficiency. We demeanour brazen to advancing this novel investigational therapy into a designed tellurian pivotal module in a initial half of 2018.”
Separately, Agios announced new efficiency and reserve information from a ongoing Phase 1/2 dose-escalation and enlargement investigate evaluating investigational verbal IDHIFA (enasidenib) in patients with relapsed or adverse strident myeloid leukemia and an isocitrate dehydrogenase-2 mutation. The information demonstrated an altogether response rate of 37 percent, including a finish response rate of 20.1 percent in 214 patients with relapsed or adverse strident myeloid leukemia who perceived enasidenib during 100 mg daily, that was a endorsed starting sip in a enlargement phases of a trial. The altogether reserve form celebrated for enasidenib was unchanging with formerly reported data.
The New Drug Application for IDHIFA is now underneath Priority Review with a U.S. FDA for a diagnosis of patients with relapsed or adverse strident myeloid leukemia with an IDH2 mutation. The NDA has been given a Prescription Drug User Fee Act movement date of Aug 30, 2017.