Revumenib combo shows promising results in AML clinical trial

Published 12/06/2025, 17:06
Revumenib combo shows promising results in AML clinical trial

MILAN - Syndax Pharmaceuticals (NASDAQ:SNDX), a biopharmaceutical company with a market capitalization of $903 million and currently trading at $10.43, reported positive results from its BEAT AML trial evaluating revumenib in combination with venetoclax and azacitidine in newly diagnosed mutant NPM1 and KMT2A-rearranged acute myeloid leukemia patients, according to a company press release. According to InvestingPro analysis, the company maintains a strong financial position with more cash than debt on its balance sheet.

The trial data, published in the Journal of Clinical Oncology and presented at the European Hematology Association Annual Congress in Milan, showed a 67% complete remission rate and 88% overall response rate among the 43 enrolled patients. All responders with measurable residual disease assessment (37 patients) achieved MRD negativity. While the clinical results are promising, InvestingPro data shows the company is not yet profitable, with analysts expecting continued losses this year. However, five analysts have recently revised their earnings expectations upward for the upcoming period.

The study population included patients 60 years and older with a median age of 70, with 79% having mNPM1 AML and 21% having KMT2Ar AML. The combination therapy was generally well-tolerated at both tested dose levels.

Common non-hematologic treatment-emergent adverse events included nausea (60%), constipation (53%), QTc prolongation (44%), hypokalemia (44%), and vomiting (42%). Grade 3 or higher non-hematologic adverse events were reported as rare.

In an early survival analysis with median follow-up of 6.9 months, median overall survival was 15.5 months. Subset analysis showed a composite complete remission rate of 77% and median overall survival of 15.5 months in mNPM1 patients with intermediate risk, while KMT2Ar patients demonstrated an 89% composite complete remission rate and 18.0 months median overall survival.

The company noted that enrollment is underway in the pivotal Phase 3 EVOLVE-2 trial, which is evaluating revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 AML patients unfit for intensive chemotherapy.

Revumenib (brand name Revuforj) is an oral, selective menin inhibitor that received FDA approval for treating relapsed or refractory acute leukemia with KMT2A translocation in adult and pediatric patients one year and older. With a current ratio of 5.8 and liquid assets exceeding short-term obligations, the company appears well-positioned to continue its development programs. Discover more insights about SNDX and access comprehensive analysis with a InvestingPro subscription, which includes exclusive ProTips and detailed financial metrics for over 1,400 US stocks.

In other recent news, Syndax Pharmaceuticals reported a better-than-expected first-quarter performance for 2025, with revenue reaching $20.04 million, surpassing the anticipated $14.84 million. This strong financial showing was largely driven by the successful launch of their product Revuforj, which exceeded expectations by generating $20 million in sales. The company’s earnings per share also beat forecasts, coming in at -0.98 compared to the expected -1.24. In addition, Syndax’s menin inhibitor Revumenib achieved a 26% complete remission rate in a study involving patients with relapsed or refractory mutant NPM1 acute myeloid leukemia. The study demonstrated a median overall survival of 23.3 months among responding patients. Furthermore, Syndax has submitted a supplemental New Drug Application to the FDA for Revumenib, seeking approval for use in mutant NPM1 AML. Analyst firms have responded positively to these developments, with Scotiabank raising its stock price target for Syndax to $17 and BofA Securities increasing it to $26, citing strong sales and market potential for Revuforj. These recent achievements underscore the company’s progress and potential in the pharmaceutical market.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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