The FDA has approved Syndax Pharmaceuticals' Revuforj (revumenib) for the treatment of patients aged one year and older with relapsed or refractory acute leukemia harboring KMT2A translocations. This marks the first regulatory approval for a menin inhibitor and offers a new therapeutic option for this aggressive form of leukemia.
The approval was granted based on data from the Phase 1/2 AUGMENT-101 trial, which evaluated the efficacy and safety of Revuforj in patients with relapsed or refractory KMT2A-rearranged leukemia. The study demonstrated that treatment with Revuforj resulted in a 21% complete remission (CR) or complete remission with partial hematological recovery (CRh).
"Revuforj is a major breakthrough for patients with this type of leukemia and represents a substantial improvement over what has been historically observed in these patients with previously available therapies," said Ghayas Issa, associate professor of leukemia at the University of Texas MD Anderson Cancer Center.
The FDA had extended its review period by three months to allow for a more thorough evaluation of the data submitted by Syndax but ultimately made its decision a month ahead of the revised deadline. Syndax plans to launch Revuforj before the end of this month.
Competitive Landscape
Syndax's primary competitor in the menin inhibitor space is Kura Oncology, which is developing ziftomenib. Kura Oncology is expected to release results from its registrational KOMET-001 trial of ziftomenib in relapsed or refractory NPM1-mutant AML early next year. Data from a phase 1a dose-escalation study of ziftomenib in NPM1-mutated and KMT2A-rearranged AML is due to be presented at this year's ASH congress.
Further Research
Syndax is also preparing to present updated results from the AUGMENT-101 trial and data from the Phase 1/2 SAVE trial, which is evaluating Revuforj in combination with chemotherapy and venetoclax in KMT2A-rearranged, NPM1-mutated, or NUP98-rearranged AML, at an upcoming conference.
