Syndax’s Remarkable Leap: Leukemia Trial Halted Prematurely as Drug Triumphs, Meeting Primary Endpoint

Syndax Pharmaceuticals has unveiled encouraging topline data from its pooled analysis of the AUGMENT-101 trial, which assessed its menin inhibitor, revumenib, in adult and pediatric patients dealing with relapsed/refractory KMT2A-rearranged acute myeloid leukemia and acute lymphoid leukemia.

The Phase I/II AUGMENT-101 study demonstrated clinically significant outcomes, meeting its primary endpoint during the protocol-defined interim analysis stage. Among the 57 patients in the pooled KMT2Ar acute leukemia group, revumenib achieved a complete remission (CR) or CR with partial hematological recovery (CRh) rate of 23%.

Furthermore, the overall response rate reached 63% among efficacy-evaluable patients, with a median duration of complete remissions lasting 9.1 months.

Despite these positive trial results, Syndax’s share price experienced a 9% decline on the announcement day. This drop may be attributed to comparisons with a rival drug candidate from Kura Oncology, ziftomenib, which reported a 30% complete response rate in AML patients with the mNPM1 mutation.

Nevertheless, Syndax made the decision to halt the pivotal trial to support accrual in the KMT2Ar cohorts, following a recommendation from an Independent Data Monitoring Committee. The company plans to submit a New Drug Application for revumenib as a treatment for relapsed/refractory KMT2Ar acute leukemia to the FDA by the end of 2023.

Ibrahim Aldoss, the principal investigator of the AUGMENT-101 trial, emphasized the critical need for new therapies to address relapsed/refractory KMT2Ar acute leukemias, given the lack of approved treatments for this patient population, where the expected response rate to standard of care treatment is less than 10%, and survival expectations are less than three months.

Syndax’s CEO, Michael Metzger, expressed confidence in revumenib’s potential as a practice-changing clinical agent, emphasizing its utility and potential as a first- and best-in-class treatment.

The drug was well-tolerated, consistent with previous data, and adverse events leading to dose reductions were minimal. Although some patients experienced differential syndrome (DS) of grade 3 or 4, no patients discontinued treatment due to DS.

Syndax also reported smooth progress in enrolling patients in the mNPM1 cohort of AUGMENT-101, and the company is financially secure, with funding through the second half of 2025, including support for key milestones and product launches planned for 2024.

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