Syndax to Present Revumenib Abstracts at 66th ASH Meeting

Syndax Pharmaceuticals has announced the acceptance of multiple abstracts evaluating revumenib, an oral small molecule menin inhibitor, for oral presentation at the upcoming 66th American Society of Hematology (ASH) Annual Meeting in San Diego, California, from December 7-10, 2024. These presentations will discuss the safety and efficacy of revumenib in the treatment of acute leukemias, either as monotherapy or in combination.

Dr. Neil Gallagher, President and Head of Research and Development at Syndax, expressed the company’s dedication to developing revumenib as a transformative therapy for both adult and pediatric patients with acute leukemias, particularly those with KMT2A rearrangements (KMT2Ar) or mutant nucleophosmin (mNPM1).

The FDA has granted Priority Review for the New Drug Application (NDA) for revumenib, aimed at treating patients with relapsed or refractory (R/R) KMT2A-rearranged acute leukemia. The review is being conducted under the FDA’s Real-Time Oncology Review Program and has a Prescription Drug User Fee Act (PDUFA) action date of December 26, 2024.

Syndax reported the completion of enrollment in the final AUGMENT-101 pivotal trial cohort for patients with R/R mNPM1 acute myeloid leukemia (AML) in March 2024. Topline data from this cohort is anticipated by the fourth quarter of 2024, potentially supporting a supplemental NDA filing in the first half of 2025.

The 66th ASH Annual Meeting will feature results from various clinical trials of revumenib. The AUGMENT-101 Phase 2 trial has shown promising results, with a 64% overall response rate (ORR) in patients with R/R KMT2Ar acute leukemia. The trial’s primary endpoint was achieved with a complete remission (CR) or CR with partial hematological recovery (CRh) rate of 23% among 97 patients in the efficacy population as of February 2024. The CRc rate was 42%, and 61% of CR/CRh responders achieved measurable residual disease (MRD) negativity. Additionally, 34% of patients who achieved ORR proceeded to hematopoietic stem cell transplantation (HSCT), with some resuming revumenib post-HSCT.

The SAVE trial, a Phase 1/2 study, evaluated an all-oral combination regimen of revumenib, venetoclax, and decitabine/cedazuridine in patients with R/R AML. The trial showed an 88% ORR, with 58% achieving CR/CRh and high rates of MRD negativity. The combination was generally well-tolerated, and many patients proceeded to HSCT. The median follow-up was 6.6 months, with a 6-month relapse-free survival rate of 59% and overall survival rate of 74%.

Another trial, INTERCEPT, investigated revumenib as pre-emptive therapy for MRD positive AML. Preliminary data showed positive MRD reduction in patients with mNPM1 mutations, with a safe and manageable safety profile, including reversible Grade 3 QTc prolongation in two patients.

Revumenib is being developed for treating acute leukemias with KMT2A rearrangements and mutant NPM1 AML. It has received Orphan Drug Designation for AML, acute lymphoid leukemia (ALL), and acute leukemias of ambiguous lineage (ALAL) from the U.S. FDA, as well as Fast Track and Breakthrough Therapy Designations.

Syndax Pharmaceuticals is dedicated to advancing its pipeline of innovative cancer therapies, aiming to significantly impact cancer care and improve patient outcomes.