Syndax Pharmaceuticals announced positive topline data from the protocol-defined pooled analysis of the pivotal AUGMENT-101 trial of revumenib, a first-in-class menin inhibitor, in adult and pediatric patients with relapsed/refractory (R/R) KMT2A-rearranged (KMT2Ar) acute myeloid leukemia (AML) and acute lymphoid leukemia (ALL). Syndax continues to expect to submit an NDA for revumenib for the treatment of R/R KMT2Ar acute leukemia to the U.S. Food and Drug Administration (FDA) by year-end. Syndax is funded into the second half of 2025, including through multiple key milestones and both product launches in 2024, which will put on a clear path to realize the full blockbuster potential of revumenib and axatilimab.

There are no approved treatments for this population, where currently the expected response rate to standard of care treatment is less than 10%, and the expectation for survival is less than three months.2 This pivotal dataset of revumenib monotherapy in heavily pretreated R/R patients is very compelling in that it demonstrates significant clinical benefit that includes deep molecular remissions and is well tolerated. In addition to the clinical trials that Syndax is conducting, the Company is working with cooperative groups and leading investigators to further expand on the potential clinical benefit of revumenib. Syndax expects to achieve the following revumenib milestones by year-end: Present additional AUGMENT-101 data in R/R KMT2 Ar acute leukemia patients at an upcoming medical meeting.

Present preliminary data illustrating revumenib's promising profile when combined with standard-of-care chemotherapy and venetoclax regimens. Revumenib was granted Orphan Drug Designation by the FDA and European Commission for the treatment of patients with AML, and Fast Track designation by the FDA for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement. Following the receipt of Breakthrough Therapy designation from the FDA for revumenib for thetreatment of R/R acute leukemia harboring a KMT2 A rearrangement, regardless of age or tumor type, and based on discussions with the FDA, the Company decided to pool data from the AUGMENT-101 cohorts enrolling R/R KMT2A AML and R/R KMT2AR ALL.