Phase 1/2 Study of AG-221 in Subjects With Advanced Hematologic Malignancies With an IDH2 Mutation | Aplastic Anemia and MDS International Foundation

Phase 1/2 Study of AG-221 in Subjects With Advanced Hematologic Malignancies With an IDH2 Mutation

Original Publication Date: 
Thursday, August 30, 2018
Article Source: 
Web Content

This multicenter study, which began in 2013, was led by Dr. Eytan Stein at Memorial Sloan Kettering Cancer Center in New York, NY. Its purpose was to evaluate the safety and efficacy of orally administered AG-221 (Enasidenib) in subjects with advanced Acute Myeloid Leukemia (AML) that harbor an IDH2 mutation. As a result of this phase I/II study, and Celgene’s request for priority review status, the FDA approved of AG-221 in August, 2017 for patients with relapse and/or refractory AML with an IDH2 mutation. During the study, researchers discovered that AG-221 also had a positive effect on MDS patients with an IDH2 mutation. This discovery led to a clinical trial involving AG-221 for the treatment of MDS with an IDH2 mutation, which is currently being led by Dr. Courtney DiNardo and Dr. Guillermo Garcia-Manero at MD Anderson in Houston, TX. Drs. DiNardo and Garcia-Manero were able to secure the assistance of the MDS Clinical Research Consortium and expand the trial to the CRC’s six participating institutions. This allows the research team to collaborate and share resources, and it expedites the study’s timeline. Successful completion of this trial could eventually lead to an FDA approval of AG-221 for patients with MDS who have an IDH2 mutation.