Revumenib

FDA approves revumenib for relapsed or refractory acute leukemia with a KMT2A translocation, showing a 21.2% CR+CRh rate and median duration of 6.4 months. 14% of transfusion-dependent patients achieved independence, and 48% of baseline-independent patients remained so. Common toxicities include hemorrhage, nausea, and infection.

FDA approves Revuforj (revumenib) as the first menin inhibitor for relapsed or refractory acute leukemia with a KMT2A translocation in adult and pediatric patients. Efficacy based on a 21% complete remission rate in a Phase 1/2 trial. Safety concerns include differentiation syndrome and QTc interval prolongation.

Syndax Pharmaceuticals' Phase II trial of revumenib met primary endpoint in relapsed or refractory AML, but stock dropped 25.6%. The AUGMENT-101 trial showed 23% CR/CRh, 4.7 months median duration, and 64% MRD-negative patients. Safety issues included QTc prolongation and differentiation syndrome. Revumenib targets menin-MLL interaction, competing with Kura Oncology's ziftomenib. Revumenib projected to generate $634m by 2030.

Syndax Pharmaceuticals disclosed positive top-line results from the phase II Augment-101 study for revumenib in AML, but shares dropped 26%.

Syndax Pharmaceuticals announced positive Phase 2 trial results for revumenib in treating relapsed or refractory mutant NPM1 acute myeloid leukemia, with a 23% complete remission rate and a 47% overall response rate in heavily pre-treated patients. The company plans to file a supplemental New Drug Application in 2025 and present further data at the American Society of Hematology Annual Meeting in December 2024.

Syndax announces positive Phase 2 AUGMENT-101 trial results for revumenib in relapsed or refractory mutant NPM1 acute myeloid leukemia (AML), achieving a 23% complete remission rate. The drug, an oral menin inhibitor, shows promise in heavily pretreated patients, with 47% overall response rate and 64% minimal residual disease negativity. Upcoming milestones include FDA approval for R/R KMT2A-rearranged acute leukemias, data presentation at ASH 2024, and initiation of a pivotal combination trial.

Syndax Pharmaceuticals reports Q3 2024 financial results, highlights clinical data for revumenib and Niktimvo™ at ASH Annual Meeting, and anticipates FDA approval and U.S. launch of revumenib for R/R KMT2Ar acute leukemia by December 26, 2024. The company expects topline data from AUGMENT-101 trial in 4Q24, potential sNDA filing in 1H25, and a $350 million royalty funding agreement for Niktimvo to fund through profitability.

Syndax Pharmaceuticals and Royalty Pharma announce a $350 million synthetic royalty funding agreement for Niktimvo™ (axatilimab-csfr), expected to fund Syndax through profitability with proforma cash approaching $800 million as of June 30. The agreement involves a 13.8% royalty on U.S. net sales of Niktimvo, with royalty payments ceasing upon reaching 2.35x the upfront payment. Niktimvo is a first-in-class anti-CSF-1R antibody approved for chronic graft-versus-host disease (GVHD) in the U.S., co-commercialized by Syndax and Incyte, with a planned U.S. launch by early Q1 2025.

Royalty Pharma plc and Syndax Pharmaceuticals announce a $350 million synthetic royalty funding agreement for Niktimvo (axatilimab-csfr), a first-in-class treatment for chronic graft-versus-host disease (GVHD). The agreement includes a 13.8% royalty on U.S. net sales of Niktimvo, with payments ceasing upon reaching a 2.35x multiple. The funds are expected to support Syndax through profitability and the launch of Niktimvo in the U.S. by early 2025.