The FDA recently approved revumenib as the first effective therapy for patients with treatment-resistant leukemia after the drug was found effective in clinical trials led at Winship and other participating sites.
Martha L. Arellano, MD
The U.S. Food and Drug Administration (FDA) on November 15, 2024, approved revumenib—the first of a new class of drugs called menin inhibitors for aggressive, treatment-resistant leukemia. The FDA granted priority review for the revumenib application, designating the drug both a breakthrough and an orphan drug.
The FDA’s approval is based on data from 104 adults and pediatric patients in the multisite combined phase 1/2 AUGMENT-101 trial, sponsored by Syndax Pharmaceuticals. Winship Cancer Institute of Emory University was one of the 22 participating trial sites in five countries. According to the FDA, this approval is based on revumenib’s positive effects in 63% of study participants. The results of the study were published in the Journal of Clinical Oncology.
Revumenib is the first treatment for people whose leukemia has recurred after earlier treatment and carries a molecular change called a KMT2A translocation. These genetic changes are found in children and adults with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and mixed phenotype acute leukemia (MPAL). KMT2A translocation allows a protein called menin to propel cancer growth. Revumenib is the first drug that targets the interaction between menin and other proteins, turning leukemia cells back into normal blood cells. In the trial, revumenib led to high remission rates with a predictable safety profile.
“This was a dramatic contrast to the usually poor prognosis for patients with the KMT2A translocation, among whom less than 10% achieve remission with currently available therapy,” says Martha L. Arellano, MD, principal investigator for the AUGMENT-101 trial at Winship. Arellano is a member of Winship’s Discovery and Developmental Therapeutics Research Program, professor of hematology and medical oncology at Emory University School of Medicine, program director of the Emory Hematology and Medical Oncology Fellowship Program and a Winship physician specialized in treating leukemia and myelodysplastic syndromes.
“This is truly a breakthrough and the only FDA-approved drug for treatment of these extremely aggressive leukemias,” Arellano says. “It offers new hope for patients for whom multiple lines of treatment have failed to place their leukemia into remission.” She adds that by achieving remission, revumenib offers patients the opportunity to pursue stem cell transplantation in the hope of achieving a cure. It is also given as a maintenance therapy after transplantation to try to prevent post-transplant relapse. As revumenib is an oral drug, Arellano says a patient can take it at home with monitoring by their oncologist.
Arellano was assisted in the trial by clinical research coordinators Karin Chappelle and Danielle Alexander. Chappelle, Clinical Research Coordinator III, says the trial required “incredible effort and collaboration.” She notes that Winship was one of the top enrolling trial sites, caring for several patients across all demographics and backgrounds. “A collaborative team of nurses, research and data coordinators, physicians, social workers, APPs, medical interpreters, pharmacists, regulatory personnel and more ensured that these patients were given the opportunity to benefit from revumenib.”
Speaking of the AUGMENT-101 trial and other clinical trials underway at Winship, Arellano says, “Winship is committed to serving our community and to leaving no one behind. In that light, our team worked tirelessly to provide access to this innovative treatment to our patients, regardless of socioeconomic status, race or ethnicity.”
For her part, Danielle Alexander, Clinical Research Coordinator II, says, “Although our study team was challenged by the complexity of the study, decreasing the burden of cancer for patients and their families remained at the forefront.” She adds, “It takes a few to make a difference for all, and it is truly exciting to witness the approval of revumenib.”