Syndax Pharmaceuticals (NASDAQ:SNDX) executives highlighted a “transformational year” in 2025 during the company’s fourth-quarter earnings call, pointing to momentum from two commercial launches, expanded labeling for its menin inhibitor Revuforj, and a growing development pipeline aimed at moving products into earlier lines of therapy and new indications.
Commercial performance: Revuforj growth accelerates after label expansion
Management said Revuforj generated $124.8 million in net revenue in 2025, its first full year on the market. Fourth-quarter Revuforj net revenue was $44.2 million, up 38% from the prior quarter, with total prescriptions increasing about 35% quarter-over-quarter.
Closter said new patient starts increased about 20% in the fourth quarter, bringing the total to approximately 1,050 patients treated commercially since launch. He added that, with most of those patients in KMT2A, the company is approaching 50% penetration of the KMT2A incident population in the first year.
Account adoption also increased, with more than 80% of Tier 1 and Tier 2 accounts “activated” in the fourth quarter, up from 70% in the third quarter, alongside broader uptake across academic and community settings.
NPM1 ramp and reimbursement progress
Executives emphasized that the NPM1 opportunity expands Revuforj’s addressable population, with Closter saying the second indication “triples” the annual addressable patient population to about 6,500 patients. He noted that formulary coverage for NPM1 was “complete at 97% of all lives covered” just four months after approval.
On patient mix, management said the latest available information suggested NPM1 patients represented about 20% of new starts exiting the third quarter, with early indicators pointing to at least 30% of new starts in the fourth quarter. Closter said the company expects NPM1 to grow meaningfully and suggested it could move toward a “50/50” split versus KMT2A given the larger NPM1 population.
Asked about competitive impact, Metzger said the company did not “feel much of an impact” on prescribing and reiterated expectations to “dominate this space,” citing Revuforj’s product profile and execution.
Revuforj duration and post-transplant maintenance trends
Syndax described several evolving treatment dynamics that it expects will lengthen time on therapy. In 2025, average Revuforj duration of therapy was in the four- to six-month range, but Closter said the company expects that to extend to six to 12 months in the second year of launch as treatment patterns mature.
Management discussed post-transplant patterns in KMT2A patients in response to multiple analyst questions:
- Closter estimated about one-third of KMT2A patients treated with Revuforj proceed to stem cell transplant and then pause therapy for about three to four months for engraftment.
- He said claims data suggested 40% to 45% of those patients have restarted Revuforj post-transplant, up from 35% to 40% previously.
- Metzger said physicians “want to put all their patients back on maintenance” and suggested steady-state restart rates could be in the 70% to 80% range, while acknowledging not all patients will be able to resume treatment.
Executives also noted increasing use in combinations. Claims data showed about 40% of Revuforj usage in combination with other therapies, up from 33% in the third quarter, even though the product is approved and promoted as a monotherapy. In Q&A, management said combination use is often with venetoclax and azacitidine (ven/aza), and also with chemotherapy regimens.
Niktimvo: strong launch metrics and persistency
Syndax also reported continued growth for Niktimvo (axatilimab) in chronic graft-versus-host disease (GVHD). Fourth-quarter net revenue was $56 million, up 22% from the prior quarter, and 2025 net revenue was $151.6 million (from the start of launch through year-end).
Closter said that from launch through the end of the fourth quarter, approximately 13,500 infusions were administered to more than 1,400 patients. Management highlighted persistency, stating that about 60% to 70% of patients who started therapy in the first quarter of 2025 remained on treatment at month 10. The company also said 90% of U.S. bone marrow transplant centers have prescribed Niktimvo, with all centers placing repeat orders year to date.
On financial contribution, the company’s 50% share of Niktimvo product contribution totaled $42.4 million in collaboration revenue to Syndax in 2025, after deducting cost of sales and commercial expenses. CFO Keith Goldan said the company expects the Niktimvo margin contribution to be in the 25% to 30% range in the near term, increasing longer term as sales grow while much of the expense base stays fixed.
Pipeline and upcoming clinical milestones: frontline menin trials and IPF readout
Chief Medical Officer Nick Botwood said global enrollment is underway in pivotal frontline trials for revumenib (Revuforj), including:
- EVOLVE-2, a phase III trial in newly diagnosed NPM1 or KMT2A patients unfit or eligible for less intensive therapy, evaluating revumenib plus ven/aza, with dual primary endpoints of complete remission and overall survival (based on the NPM1 population).
- REVEAL, a phase III trial in fit NPM1 patients combining revumenib with intensive chemotherapy, with dual primary endpoints of MRD-negative complete remission and event-free survival.
- RAVEN, an innovative phase II study in fit newly diagnosed KMT2A patients evaluating revumenib with ven/aza as a potentially less toxic alternative to intensive chemotherapy, conducted in collaboration with leading research coalitions.
Botwood also reviewed real-world and post-transplant maintenance efforts discussed at the prior year’s ASH meeting, including reports from MD Anderson (10 pediatric post-transplant maintenance patients) and Moffitt Cancer Center (a real-world series reporting overall response and MRD-negativity rates among relapsed/refractory patients treated primarily with revumenib in combinations).
For axatilimab beyond chronic GVHD, Botwood said Syndax completed enrollment in MAXPIRe, a phase II randomized, double-blind, placebo-controlled trial in approximately 135 patients with idiopathic pulmonary fibrosis (IPF), with top-line data expected in the fourth quarter of 2026. The primary endpoint is the annualized rate of decline in forced vital capacity (FVC) measured at 26 weeks. In Q&A, Botwood said the company would look for both statistical significance and clinical relevance versus historical controls when evaluating proof-of-concept, and indicated that a planned phase III would use a standard 52-week endpoint.
Goldan said total 2025 revenue was $172.4 million and that the company ended 2025 with $394 million in cash, equivalents, and marketable securities. For 2026, Syndax reiterated guidance that total R&D plus SG&A expenses will be approximately $400 million, excluding an estimated $50 million in non-cash stock compensation, and said expenses are expected to be relatively flat quarter to quarter. With growing revenue and stable expenses, Goldan said Syndax expects to reach profitability without raising additional capital.
About Syndax Pharmaceuticals (NASDAQ:SNDX)
Syndax Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for the treatment of cancer. Headquartered in Waltham, Massachusetts, the company focuses on small-molecule inhibitors that target key epigenetic and protein interaction pathways. Syndax’s research platform aims to enhance the effectiveness of existing therapies and address high unmet medical needs in oncology.
The company’s lead investigational candidate, entinostat, is a selective class I histone deacetylase (HDAC) inhibitor being evaluated for multiple solid tumor and hematologic indications.
