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FDA grants breakthrough therapy designation to enzomenib for R/R KMT2A-rearranged acute leukemia
On October 26, 2026, the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to enzomenib, an oral menin inhibitor, for the treatment of patients with relapsed/refractory (R/R) acute leukemia with KMT2A rearrangement (KMT2Ar).1
Enzomenib is currently being evaluated in the ongoing phase I/II Horizen-1 trial (NCT04988555) as monotherapy for the treatment of R/R acute leukemias, including acute lymphoblastic leukemia (ALL) with KMT2Ar.1,2 Results from the Horizen-1 trial demonstrated an overall response rate (ORR) of 73.3%, with a composite complete response (CRc) rate of 60% among patients with acute leukemia with KMT2Ar at the recommended phase II dose.3 The most common adverse events included nausea (39.7%), vomiting (27.6%), and febrile neutropenia (27.6%); no dose-limiting toxicities were reported.3
References
- Business Wire. Sumitomo Pharma America announces enzomenib (DSP-5336) receives FDA breakthrough therapy designation for treatment of R/R KMT2A-rearranged acute leukemia. https://www.businesswire.com/news/home/20261026951054/en/Sumitomo-Pharma-America-Announces-Enzomenib-DSP-5336-Receives-FDA-Breakthrough-Therapy-Designation-for-Treatment-of-RR-KMT2A-Rearranged-Acute-Leukemia. Published Oct 26, 2026. Accessed Oct 27, 2026.
- ClinicalTrials.gov. A phase 1/2 study of enzomenib (DSP-5336) in patients with acute leukemia (Horizen-1) https://clinicaltrials.gov/study/NCT04988555. Updated Mar, 2026. Accessed Oct 27, 2026.
- Daver N. Monotherapy update from phase 1 portion in phase1/2 trial of the menin-MLL inhibitor enzomenib (DSP-5336) in patients with relapsed or refractory acute leukemia. Oral Abstract #763. Presented at: 67th American Society of Hematology Annual Meeting and Exposition; Dec 6–9, 2025; Orlando, US.
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This content is accurate as of the date of publication and is not updated retrospectively. Please refer to the latest available evidence, guidelines, and relevant regulatory information when making clinical decisions.
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