Agios Pharmaceuticals Inc. said patients with a type of blood cancer continue to respond when treated with Tibsovo alone.
In a phase 1 study, patients with acute myeloid leukemia who did not respond to prior treatment or whose cancer has returned after previously being in remission, and have an isocitrate dehydrogenase-1, or IDH1, mutation, showed a combined complete remission and partial recovery of peripheral blood counts — the main goal of the study — at a rate of 31.8%.
Of the 179 patients in the trial, 41.9% showed an overall response rate, or some reduction in the cancer.
IDH1-mutation clearance, or the absence of the IDH1 mutation, was observed in 23% of patients who achieved complete remission and complete remission with partial hematologic recovery. Preliminary data suggest that acute myeloid leukemia, or AML, patients with IDH- mutation clearance in bone marrow mononuclear cells who have achieved complete remission and complete remission with partial hematologic recovery live longer than those without IDH1-mutation clearance.
The U.S. Food and Drug Administration granted Tibsovo, also known as ivosidenib, priority review, and it will announce its decision on whether the drug is approved by Aug. 21.
AML, a cancer of blood and bone marrow characterized by rapid disease progression, is the most common acute leukemia affecting adults. The vast majority of patients do not respond to chemotherapy and progress to relapsed/refractory AML. The five-year survival rate for AML is about 27%. IDH1 mutations are present in about 6% to 10% of AML cases.
The 2018 American Society of Clinical Oncology meeting is expected to bring together more than 32,000 professionals from all over the world, with more than 2,500 study abstracts to be presented on site and an additional 3,350 abstracts to be published online.
