Syndax Pharmaceuticals Expands Revuforj Access and Reports Strong Commercial Performance

Syndax Pharmaceuticals and the World Orphan Drug Alliance announced a collaboration to expand access to Revuforj (revumenib) through a Managed Access Program for eligible patients outside the U.S., in markets where local regulations and funding allow use of novel medicines. The company reported strong commercial performance at the Guggenheim biotech conference on February 15, with the Chief Executive Officer stating that Syndax started 2026 on a strong footing based on the performance of two key marketed products.

The company generated $125 million in annual net sales for Revuforj and $152 million for Niktimvo in the first 11 months, with both products showing quarter-over-quarter sales growth. Revuforj's revenue growth is driven by continued penetration in its labeled indication for KMT2A-rearranged acute myeloid leukemia.

Revuforj, an oral first-in-class menin inhibitor, is FDA approved in the U.S. for relapsed or refractory acute leukemia with a KMT2A gene translocation in adult and pediatric patients one year and older, and for relapsed or refractory AML with a susceptible NPM1 mutation in adult and pediatric patients one year and older who have no satisfactory alternative treatment options.

The company is targeting a significant opportunity with Niktimvo in chronic GVHD, with approximately 6,500 patients that could expand to about 17,000. The company could generate up to $1 billion in third-line plus sales. Syndax has a strategic collaboration with Incyte to co-promote the product.

Syndax highlighted Revuforj data at the 67th American Society of Hematology Annual Meeting, including safety details. Differentiation syndrome occurred in 25% of 241 patients at the recommended dose, and QTc interval prolongation was reported in 36% of patients in clinical trials.

Niktimvo (axatilimab-csfr) is an FDA-approved monoclonal antibody targeting the CSF-1 receptor, with several clinical trials underway across the treatment continuum. On December 18, 2025, the Board approved bylaw amendments stating that, from that date, new shares of capital stock are issued only in uncertificated form, while existing share certificates remain valid until surrendered to the company.

Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company focused on developing and commercializing innovative, first-in-class, targeted therapies for cancer, particularly acute leukemia and chronic graft-versus-host disease.