Agios reports additional Phase I data for ivosidenib in AML

Agios Pharmaceuticals Inc. (NASDAQ:AGIO) reported data from 125 patients with isocitrate dehydrogenase 1 (IDH1) mutation-positive acute myelogenous leukemia (AML) in a Phase I trial showing that oral ivosidenib (AG-120) led to 27 complete responses and 11 complete responses with partial hematologic recovery, defined as <5% of blasts in the bone marrow, no evidence of disease and partial recovery of peripheral blood counts. Median duration of response was 6.5 months and median overall survival (OS) was 8.8 months.

In 34 untreated AML patients, AG-120 led to an overall response rate (ORR) of 55.9% and a complete response rate of 20.6%. Median duration of response was 9.2 months. In 12 patients with myelodysplastic syndrome (MDS), AG-120 led to an ORR of 91.7% and a complete response rate of 41.7%. Data were presented at the American Society of Hematology meeting in Atlanta in December. Agios submitted an NDA to FDA later that month for ivosidenib to treat relapsed or refractory AML in patients with an IDH1 mutation...