Forma Therapeutics Holdings, Inc. announced that topline interim data from its Phase 2 trial of olutasidenib in relapsed/refractory acute myeloid leukemia (R/R AML) will be presented at the upcoming 2021 American Society of Clinical Oncology (ASCO) Annual Meeting taking place June 4-8. Olutasidenib, Forma’s selective inhibitor for cancers with mutations in isocitrate dehydrogenase 1 (IDH1m), demonstrated positive efficacy and a favorable tolerability profile as a monotherapy in patients with IDH1m R/R AML, achieving the primary endpoint of a composite complete remission (CR) or CR plus CR with partial hematologic recovery (CRh) rate of 33.3% (30% CR and 3% CRh). The presentation is based on an interim analysis from a pivotal trial arm evaluating continuous treatment with 150 mg twice daily of oral olutasidenib. The data indicate the duration of CR/CRh for people on treatment was 13.8 months. Among patients with a complete remission (CR) who were transfusion-dependent at baseline, 56-day transfusion independence was achieved in 100% of patients as measured by platelets and 83% as measured by red blood cells. The Phase 1/2 study is a multicenter, open-label, multi-cohort evaluation of the safety, efficacy and pharmacokinetics/pharmacodynamics (PK/PD) of olutasidenib for patients with AML or myelodysplastic syndrome (MDS) with an IDH1 mutation. Phase 1 of the trial, 2102-HEM-101, was an open-label, dose-escalation and expansion study of olutasidenib alone and in combination with azacitidine (AZA). The Phase 2 portion was an open-label, fixed-dose study of olutasidenib as a monotherapy and in combination with AZA in multiple IDH1m AML/MDS populations. The primary efficacy evaluable population is comprised of 123 R/R AML patients enrolled in Cohort 1, who received 150 mg of olutasidenib BID at least six months prior to the interim analysis cutoff date of June 18, 2020. The primary endpoint of the Phase 2 pivotal study is a complete remission (CR) plus a complete remission with partial hematological recovery (CRh) that is defined as 50,000/microliter and ANC >500/microliter). Key Study Findings: Efficacy (n=123): Olutasidenib induced a durable CR/CRh rate of 33.3% (95% CI 25.1, 42.2), which is the primary endpoint of the study: The CR rate was 30.0% (37 of 123 patients) and the CRh rate was 3.0% (4 of 123 patients). While the median duration of response was not yet reached, in a sensitivity analysis with hematopoietic stem cell transplant considered as the end of a response, the median duration was 13.8 months. The median duration of overall response was 11.7 months. The median overall survival (OS) was 10.5 months. The median OS for non-CR/CRh responders was 15.0 months. A median OS has not yet been reached for the CR/CRh population, with an 18-month survival estimate of 87.0%. Transfusion independence was achieved in all response groups at 56 days, particularly in those achieving CR, with 100% independence for platelet transfusions and 83.0% independence for red blood cells. Safety (n=153). Olutasidenib was well-tolerated, with adverse events (AEs) consistent with the late stage disease and the heavily pre-treated population. A safety analysis for all 153 patients enrolled in the Phase 2 Cohort 1 found the most common grade 3/4 (= 20% or = 10%) treatment-emergent adverse events (TEAEs) were febrile neutropenia (20%), anemia (19%), thrombocytopenia (16%), neutropenia (13%), leukocytosis (9%) and fatigue (forward-looking statements contained in this press release, including, without limitation, those risks and uncertainties related ability to execute on strategy; the finalization of Phase 2 study in R/R AML and final audit and quality controlled verification of interim data and related analyses; positive results from interim data analyses may not be predictive of final results; risks related to planned regulatory submissions and developments; and other risks identified in SEC filings, including those risks discussed under the heading 'Risk Factors' in Quarterly Report on Form 10-Q for the quarter ended March 31, 2021, as well as other risks detailed in subsequent filings with the SEC.