Syndax Pharmaceuticals announced positive results from its Phase 2 AUGMENT-101 trial evaluating revumenib for the treatment of relapsed or refractory mutant NPM1 (mNPM1) acute myeloid leukemia (AML). The trial met its primary endpoint, demonstrating a complete remission (CR) and CR with partial hematological recovery (CRh) rate of 23% among evaluable adult patients.
Key Findings from AUGMENT-101
The AUGMENT-101 trial included 64 adult patients with a median age of 65 who had received multiple prior lines of therapy. Notably, 75% of the participants had prior exposure to venetoclax, a common AML treatment. In this heavily pre-treated population, revumenib achieved a 47% overall response rate. The safety profile was also favorable, with only 5% of patients discontinuing treatment due to treatment-related adverse events. The most common severe adverse events included QTc prolongation, anemia, febrile neutropenia, differentiation syndrome, and decreased platelet count.
Clinical Significance
Dr. Eytan M. Stein from Memorial Sloan Kettering Cancer Center emphasized the importance of these findings for patients with limited treatment options and poor prognosis, particularly those who have failed venetoclax treatment. The results suggest that revumenib could offer a valuable new therapeutic option for this challenging patient population.
Future Plans
Syndax Pharmaceuticals is preparing to submit a supplemental New Drug Application (sNDA) to the FDA in the first half of 2025. The company also plans to present further data at the American Society of Hematology Annual Meeting in December 2024 and initiate additional clinical trials to explore the clinical benefits of revumenib in various acute leukemias.
Financial and Strategic Context
In addition to the clinical progress of revumenib, Syndax Pharmaceuticals has also made significant financial strides. A $350 million royalty agreement with Royalty Pharma for Niktimvo is expected to strengthen the company's financial position and support the commercialization of both Niktimvo and revumenib. Syndax reported $399.6 million in cash as of September 30, with operating expenses for Q3 standing at $102.1 million. The company is preparing for the commercial launch of Niktimvo in early Q1 2025, targeting a market worth $1.5 billion to $2 billion for third-line cGVHD treatment.
