Syndax Updates Positive Data on Revumenib Trials in Acute Leukemias at EHA 2024

Syndax Pharmaceuticals, a clinical-stage biopharmaceutical company, has announced promising updated results from trials involving their menin inhibitor, revumenib, targeting acute leukemias. The data was presented at the European Hematology Association (EHA) 2024 Congress.

Revumenib, a selective small molecule inhibitor, has shown significant potential in combination therapies for acute myeloid leukemia (AML). The BEAT AML trial, which combined revumenib with venetoclax and azacitidine, yielded a 96% composite complete remission (CRc) rate in newly diagnosed AML patients with mNPM1 or KMT2Ar mutations. Out of the 24 patients evaluated, 92% also achieved minimal residual disease (MRD) negative status. This trial involved patients aged 60 years or older, and the treatment was well-tolerated, showing no new or increased safety concerns. The trial is ongoing, aiming to establish the optimal dose for future studies, with plans to initiate a pivotal trial by the end of 2024.

Parallelly, the AUGMENT-102 trial explored revumenib in combination with fludarabine and cytarabine (FLA) in pediatric patients with relapsed or refractory (R/R) mNPM1, NUP98r, or KMT2Ar AML. The results revealed a 52% CRc rate among 27 patients, with most achieving MRD negative status. The treatment regimen was generally tolerable even in heavily pretreated patients, with no increased frequency of adverse events compared to historical data or revumenib monotherapy. A significant observation was that the higher dose of revumenib (163 mg q12h) was associated with faster remission and fewer adverse events.

In addition to these trials, Syndax presented results from the AUGMENT-101 study at the EHA Congress. This study focused on revumenib as a monotherapy in R/R KMT2Ar acute leukemia. The findings highlighted the efficacy and safety of revumenib, reinforcing its potential as a crucial component in the treatment landscape for acute leukemias.

Revumenib, which targets the menin-KMT2A binding interaction, is being developed to treat various forms of acute leukemia, including those with mNPM1 and KMT2Ar mutations. The drug has received multiple designations from the FDA, including Orphan Drug and Breakthrough Therapy, highlighting its significance and potential impact on treatment paradigms.

Syndax Pharmaceuticals remains dedicated to advancing revumenib across different acute leukemia patient populations. As the company moves closer to potential regulatory approvals for revumenib in the relapsed or refractory setting, it continues to gather and present clinical data to support its use both as a monotherapy and in combination with other treatments.

Overall, the updated data from the BEAT AML and AUGMENT-102 trials underscore revumenib's promising role in enhancing standard care for acute leukemia patients, demonstrating high remission rates and manageable safety profiles in both newly diagnosed and relapsed/refractory settings. Syndax’s ongoing research and forthcoming trials aim to solidify revumenib’s position as a cornerstone therapy for these challenging hematologic malignancies.