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Syndax Announces U.S. FDA Breakthrough Therapy Designation Granted for Revumenib for the Treatment of Adult and Pediatric Patients with Relapsed or Refractory KMT2A- Rearranged (MLLr) Acute Leukemia
-- Revumenib is the first and only investigational treatment for R/R KMT2Ar acute leukemia to receive Breakthrough Therapy Designation -- -- Designation is
About this update from Syndax Pharmaceuticals, Inc.
[{"type":"text","content":"-- Revumenib is the first and only investigational treatment for R/R KMT2Ar acute leukemia to receive Breakthrough Therapy Designation --\n-- Designation is based on Phase 1 data from the AUGMENT-101 trial that showed a 27% CR/CRh rate in KMT2A patients treated at RP2D --\n-- Company remains on track to submit an NDA for revumenib by the end of 2023 with the potential for an expedited approval with a broad indication --\nWALTHAM, Mass., Dec. 5, 2022 /PRNewswire/ -- Syndax Pharmaceuticals, Inc. (Nasdaq: SNDX), a clinical-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for revumenib for the treatment of adult and pediatric patients with relapsed or refractory (R/R) acute leukemia harboring a KMT2A rearrangement (KMT2Ar). Revumenib is the Company's highly selective, oral menin inhibitor.\n\"The Breakthrough Therapy Designation underscores revumenib's potential as a first- and best-in-class therapy to meaningfully change the treatment paradigm for patients with R/R KMT2Ar acute leukemia, whether it presents clinically as acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL), in adults or children,\" said Michael A. Metzger, Chief Executive Officer. \"Revumenib has the potential, if approved, to be the first drug to address the significant unmet need in KMT2Ar leukemia believed to occur in up to 10% of all acute leukemias, including in approximately 80% of infant acute leukemias. Syndax is committed to bringing revumenib to these patients as quickly as possible and we look forward to working collaboratively with the FDA to expedite a potential approval of revumenib.\"\nThe BTD is supported by Phase 1 data from the AUGMENT-101 trial. Ten of 37 patients (27%) with age and phenotype agnostic KMT2Ar acute leukemia treated at doses meeting the protocol defined criteria for the recommended Phase 2 dose (RP2D) and evaluable for efficacy as of the March 2022 data cutoff achieved a complete remission as measured by a CR/CRh. Included in this analysis were patients treated in Arm A (226 and 276 mg q12 hours not receiving a strong CYP3A4 inhibitor) and Arm B (113 and 163 mg q12 hours receiving a strong CYP3A4 inhibitor).\nAs previously announced, additional data from the Phase 1 po...