Syndax’s revumenib shows 26% complete remission rate in AML study

MILAN - Syndax Pharmaceuticals (NASDAQ:SNDX), a clinical-stage biopharmaceutical company with a market capitalization of $938 million and strong liquidity position, reported Thursday that its menin inhibitor Revuforj® (revumenib) achieved a 26% complete remission rate in patients with relapsed or refractory mutant NPM1 acute myeloid leukemia (AML), according to new data from its pivotal AUGMENT-101 trial. According to InvestingPro data, the company maintains a healthy current ratio of 5.8, indicating robust short-term financial stability.

The study evaluated 77 efficacy-evaluable patients with mutant NPM1 AML, showing an overall response rate of 48%. The median duration of complete remission was 4.7 months, with 63% of responding patients achieving minimal residual disease negativity. Five analysts have recently revised their earnings expectations upward, with InvestingPro analysis suggesting the stock is currently undervalued based on its Fair Value assessment.

Among patients who achieved any response, median overall survival reached 23.3 months, compared to 4.8 months in the overall study population.

The data demonstrated consistent responses regardless of prior treatments, with complete remission rates of 45% in patients without prior venetoclax exposure and 19% in those previously treated with the drug.

In a separate Phase 1 portion of the trial, revumenib showed a 60% overall response rate in five patients with NUP98-rearranged AML, another genetic alteration associated with HOX gene upregulation.

The safety profile remained consistent with previous reports, with 4.8% of patients discontinuing treatment due to treatment-related adverse events. The most common serious adverse reactions included infection (24%), febrile neutropenia (19%), and bacterial infection (17%).

Revuforj® received FDA approval in November 2024 for relapsed or refractory acute leukemia with KMT2A translocations. Syndax submitted a supplemental New Drug Application in April 2025 seeking approval for revumenib in mutant NPM1 AML based on these findings.

The data were presented at the European Hematology Association Annual Congress in Milan, according to the company’s press release statement. While the company reported revenue of $43.7 million in the last twelve months, analysts project sales growth for the current year. Discover more insights about SNDX and 1,400+ other stocks through comprehensive InvestingPro Research Reports, which transform complex financial data into actionable intelligence.

In other recent news, Syndax Pharmaceuticals reported a strong first quarter with earnings per share (EPS) of -0.98, surpassing the forecasted -1.24, and revenue of $20.04 million, exceeding the anticipated $14.84 million. The company credited robust sales from its recently launched products, particularly Revuforj, which generated $20 million in sales compared to the consensus estimate of $12 million. BofA Securities responded to this positive performance by raising the price target for Syndax to $26.00, maintaining a Buy rating. Similarly, Scotiabank increased its price target to $17.00, citing strong sales of Revuforj and a successful market launch.

Syndax’s management expressed confidence in the company’s strategic initiatives, noting the potential for further growth driven by Revuforj and Niktimvo. The completion of a supplemental New Drug Application (sNDA) filing under the FDA’s Real-Time Oncology Review program for the NPM-1 mutant treatment segment was highlighted as a significant development. Analysts from BofA Securities emphasized that the company’s current stock price might not fully reflect its near-term opportunities, projecting 2026 revenue at $252 million compared to the consensus estimate of $168 million.

The company’s financial position remains strong, with $602.1 million in cash and equivalents as of March 31, 2025. The initial sales figures of Revuforj and Niktimvo are expected to alleviate some investor concerns as the company navigates the second half of the year. Syndax’s strategic focus on product launches and market expansions aims to achieve profitability, supported by positive feedback from prescribers and payers.

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