Syndax Pharma Surges on FDA Approval for First-in-Class Leukemia Drug
The U.S. regulator greenlights Revuforj for a common, hard-to-treat form of Acute Myeloid Leukemia, marking a pivotal commercial milestone for the company.
Syndax Pharmaceuticals Inc. (NASDAQ: SNDX) shares climbed over 3% in Friday trading after the company announced it had secured U.S. Food and Drug Administration (FDA) approval for Revuforj, its targeted therapy for a specific, high-need group of acute myeloid leukemia (AML) patients.
The approval marks a critical turning point for the $1.26 billion biotechnology firm, greenlighting the first and only treatment specifically for adult and pediatric patients with relapsed or refractory (R/R) AML characterized by a nucleophosmin 1 (NPM1) mutation. This genetic alteration is one of the most common in adult AML, found in approximately 30% of cases, and has historically lacked targeted therapeutic options, representing a significant unmet medical need.
"We are thrilled to have secured a second indication for Revuforj, making it the first and only menin inhibitor that is FDA-approved for multiple acute leukemia subtypes in both adults and children," said Michael A. Metzger, Chief Executive Officer of Syndax, in a company press release. "The breadth of the indicated patient population highlights the compelling and consistent efficacy and tolerability of Revuforj."
Clinical Backing and Market Potential
The FDA’s decision was based on positive results from the AUGMENT-101 Phase 2 trial, which evaluated the drug’s efficacy in a heavily pre-treated patient population. The study demonstrated a complete remission (CR) plus complete remission with partial hematological recovery (CRh) rate of 23% among the 65 patients evaluated. According to data presented at the 2025 European Hematology Association Congress, the median time for patients to achieve a response was 2.8 months.
Revuforj, known scientifically as revumenib, is a potent, oral, small molecule inhibitor of the menin-KMT2A protein interaction, a key pathway that drives cancer growth in certain types of leukemia. The approval expands upon Revuforj's earlier clearance for treating leukemia with KMT2A rearrangements, solidifying its position as a cornerstone asset for Syndax.
For investors, the approval is a de-risking event that transitions a key pipeline asset into a commercial product, unlocking a new revenue stream. Prior to the announcement, Wall Street analysts held a consensus 'Strong Buy' rating on the stock, with an average price target of $37.69, suggesting significant upside from its current price of around $15.22.
Commercial Strategy and Competitive Field
Syndax confirmed that Revuforj will be made available immediately in the United States through its established network of specialty distributors and pharmacies. The company has also launched SyndAccess, a patient support program designed to assist with access and affordability.
The drug's first-in-class status for NPM1-mutated AML gives it a crucial head start. While the field of AML treatment is competitive, the lack of targeted therapies for this specific mutation provides a clear market opportunity. However, other companies are developing similar menin inhibitors, such as ziftomenib, which could introduce competition in the future.
Metzger expressed confidence in the company's commercial readiness, stating, "Our launch into this second population will greatly benefit from physicians’ already strong familiarity with Revuforj and positive experience treating well over 1,000 patients in clinical trials and nearly one year of commercial use." The company's ability to successfully commercialize the drug and achieve meaningful market penetration will be a key focus for investors in the coming quarters.