Revumenib

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.

On November 15, 2024, the FDA approved revumenib for treating relapsed or refractory acute leukemia with KMT2A translocation in patients aged one year and older. Efficacy showed a 21.2% CR+CRh rate, with a median duration of 6.4 months. Common adverse reactions included hemorrhage and nausea. Revumenib's availability is through an expanded access program for patients under 40 kg.

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.