Breakthrough Success: AUGMENT-101 Trial Hits Primary Endpoint with Consistent CR/CRh Rates Across Adult and Pediatric KMT2Ar AML and ALL Cohorts

According to the data presented at the ASH 2023 conference, as of the July 2023 data cutoff, the pivotal trial AUGMENT-101 included 94 patients with acute leukemia within the KMT2Ar cohorts. Among them, 57 individuals received central confirmation of their KMT2Ar status, had adequate follow-up, and were part of the population evaluated for efficacy.  Among the patients evaluated for efficacy, a notable 63% demonstrated an overall response, while 44% achieved a composite response (CRc). Minimal residual disease (MRD) status was examined in 22 out of the 25 patients who attained a CRc, revealing that 68% were MRD negative. Favorable responses were observed across all major subgroups, including those with varying numbers of prior treatments and prior stem cell transplant history. Out of the individuals with an overall response, 39% underwent hematopoietic stem cell transplant (HSCT), with eight of them not achieving a complete response (CR) or CR with partial hematologic recovery (CRh) before the transplant. Furthermore, the median overall survival was 8 months at the time of data cutoff.

In terms of safety analysis, revumenib demonstrated good tolerability, with a consistent and well-maintained safety profile. Among the 94 patients, Grade 3 differentiation syndrome (DS) occurred in 15%, while Grade 4 DS was observed in 1% of patients, and there were no Grade 5 DS. Additionally, Grade 3 QTc prolongation was noted in 14%, and no Grade 4 or 5 events were reported. Importantly, there were no trial discontinuations attributable to DS, cytopenias, or QTc prolongation.

KOL Insights

“I am pleased that this pivotal dataset of revumenib as a monotherapy in heavily pretreated R/R patients continues to support its profile as a potential best- and first-in-class therapy, Of particular note, the data presented today demonstrate rapid responses with revumenib, with a median time to CR/CRh of 1.9 months, which is particularly impressive in this patient population. Responses were also observed across all major subgroups, with a similar CR/CRh rate across adult and pediatric patients, which speaks to the wide clinical utility of revumenib across this underserved patient population.” –Expert Opinion.

Conclusion

The interim analysis of the combined KMT2Ar Acute Myeloid Leukemia Market and Acute Lymphocytic Leukemia cohorts in the pivotal AUGMENT-101 trial successfully achieved its primary endpoint, demonstrating a consistent CR/CRh rate among both adult and pediatric patients.

Refer to the Related Reports for More In-depth Insights –