Benzinga | Oct 26, 2020 07:14AM EDT

Forma Therapeutics Announces Top-Line Olutasidenib Data From Planned Interim Analysis Of Registrational Phase 2 Clinical Trial In Acute Myeloid Leukemia

FT2102-HEM-101 clinical trial data demonstrate a 30% CR and 3% CRh rate with olutasidenib monotherapy in 123 relapsed or refractory IDH1m AML patients

While median duration of CR/CRh has not been reached, sensitivity analysis indicates the median duration of CR/CRh to be 13.8 months

Favorable tolerability profile consistent with Phase 1 study results

Data will be submitted for discussion at an upcoming medical meeting

Forma Therapeutics Holdings, Inc. (NASDAQ:FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced positive top-line data from a planned interim analysis of a registrational Phase 2 clinical trial of olutasidenib, Forma's selective inhibitor for hematological malignancy cancers with mutations in isocitrate dehydrogenase 1 (IDH1m). Olutasidenib demonstrated a favorable tolerability profile as a monotherapy in patients with IDH1m relapsed/refractory acute myeloid leukemia (R/R AML), and achieved a composite complete remission (CR+CRh, or complete remission plus complete remission with partial hematologic recovery) rate of 33.3% (30% CR and 3% CRh), the primary efficacy endpoint. While a median duration of CR/CRh has not been reached, a sensitivity analysis (with a hematopoietic stem cell transplant or HCST as the end of a response) indicates the median duration of CR/CRh to be 13.8 months.

Safety results are consistent with previously reported Phase 1 clinical trial results1,2. The most common adverse events (AEs) observed were nausea, constipation, increased white blood cell count, decreased red blood cell count, fever, febrile neutropenia and fatigue.

"We are pleased to announce these compelling top-line data," said Patrick Kelly, MD, chief medical officer of Forma Therapeutics. "The safety profile and the duration of the response we're seeing supports the potential for olutasidenib to become a leading therapy for R/R IDH1m AML patients. While the multi-cohort Phase 2 trial is ongoing, this specific cohort was designed to serve as a pivotal study; these efficacy data support an early stop in enrollment in favor of moving the program forward."

Additional analyses and other outcome measures will be presented at an upcoming medical meeting.

Study Design

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, FT2102-HEM-101, was an open-label, dose-escalation and expansion study of olutasidenib alone and in combination with azacitidine (AZA). The pivotal Phase 2 study is an open-label, fixed-dose study of olutasidenib as a monotherapy in IDH1m AML patients. The Phase 2 study includes other cohorts of olutasidenib in combination with AZA in IDH1m AML/MDS populations. The primary efficacy-evaluable population of the pivotal phase 2 study is comprised of 123 R/R AML patients, who received olutasidenib 150 mg BID at least six months prior to the interim analysis cutoff date of June 18, 2020. The primary endpoint is a composite of a complete remission (CR) plus a complete remission with partial hematological recovery (CRh), defined as less than 5% blasts in the bone marrow, no evidence of disease and partial recovery of peripheral blood counts (platelets >50,000/microliter and ANC >500/microliter).