On Tuesday, Syndax Pharmaceuticals Inc ( SNDX ) revealed topline results from the relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML) cohort in the pivotal Phase 2 portion of the AUGMENT-101 trial of revumenib.

The cohort met the primary endpoint with a complete remission (CR) plus CR with a partial hematological recovery (CRh) rate of 23% among the efficacy evaluable adults.

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Among the patients who achieved CR/CRh, 12 had a CR, and three had a CRh.

At the data cutoff, the observed median duration of CR/CRh responses was 4.7 months, with three patients remaining in response.

Minimal residual disease (MRD) status was assessed in 14 of 15 patients who achieved CR/CRh, 64% (9/14) of whom were MRD negative. The overall response rate (ORR) was 47%.

17% of patients who achieved an overall response underwent hematopoietic stem cell transplant (HSCT) following treatment with revumenib, with three resuming revumenib therapy post-transplant.

The safety population included 84 adult and pediatric patients with R/R mNPM1 AML in the Phase 2 portion of the AUGMENT-101 trial.

The safety profile observed with revumenib in this population was consistent with previously reported data.

Treatment-related adverse events (TRAEs) leading to treatment discontinuations were 5%

The company is conducting several trials of revumenib across the treatment landscape in mNPM1 and KMT2A-rearranged (KMT2Ar) acute leukemias.

Syndax expects to achieve the following upcoming revumenib milestones:

Receive FDA approval for R/R KMT2Ar acute leukemias in the fourth quarter of 2024.

Initiate a pivotal combination trial with venetoclax/azacitidine in newly diagnosed mNPM1 AML or KMT2Ar acute leukemias by year-end 2024.

Publish pivotal AUGMENT-101 results in R/R mNPM1 AML patients and present results at a medical conference in the first half of 2025.

Submit a supplemental NDA for R/R mNPM1 AML in the first half of 2025.

Price Action: SNDX stock is down 22.1% at $16.96 at last check Tuesday.

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