FDA Grants Priority Review for Revumenib to Treat Relapsed or Refractory AML

Syndax Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) granted revumenib a Priority Review for treating adults and children with acute myeloid leukemia that has come back or had unsuccessful previous treatments. The FDA is expected to make a decision by September 26, 2024.

What Is Revumenib?

Revumenib is considered a "first-in-class" menin inhibitor, making it one of the first drugs designed to target this specific protein in leukemia treatment.

Menin helps leukemia cells grow and reply, particularly in cases where the KMT2A gene is affected. Revumenib disrupts these processes, leading to the death of leukemia cells.

Revumenib is currently being investigated as a potential treatment for leukemias that have either returned after initial treatment (relapsed) or have not responded to standard therapies (refractory). This medication is being studied for:

• KMT2A-rearranged (KMT2Ar) acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML)
• NPM1-mutant (NPM1m) acute myeloid leukemia

The AUGMENT-101 Trial

The FDA decided to do a Priority Review based on the data from the phase 2 AUGMENT-101 ongoing trial, which was key for assessing the safety, tolerability, and efficacy of revumenib. The trial involved 57 patients with relapsed and refractory AML who had multiple unsuccessful treatments.

What are the Key Findings from the AUGMENT-101 Trial?

• 63% of patients responded to revumenib treatment.
• 44% achieved complete remission.
• 50% of those who received a transplant restarted revumenib as maintenance therapy post-transplant. 

"Any time you have relapsed or refractory acute leukemia, the only cure is a transplant, but to do that, you have to have a response. We observed encouraging, durable, and meaningful responses, and many of these patients were able to proceed successfully to transplant. We have not seen this level of activity with any other available treatment in this advanced disease setting." -Dr. Ibrahim Aldoss, a leading investigator in the study.

After revumenib, 39% of patients could undergo a stem cell transplant.

What are Revumenib’s Side Effects?

Most of these side effects were manageable, and overall, revumenib was well tolerated by patients. But the most common side effects included:

• Gastrointestinal: diarrhea, vomiting, decreased appetite, change in taste
• Others: fatigue and anemia. 

Upcoming FDA Decision and Ongoing Research with Revumenib

In conclusion, the potential approval of revumenib offers new alternatives for patients with relapsed or refractory KMT2A-rearranged acute leukemia. While the journey with leukemia can be challenging, advances like this bring us closer to more effective treatments. If approved, revumenib could provide a new option for those who have exhausted other therapies, offering a chance at remission and, for some, the possibility of moving forward to a life-saving transplant. 

Revumenib is being actively studied in clinical trials such as the AUGMENT-101 trial, where a single agent is tested in relapsed/refractory disease but also combined with chemotherapy in newly diagnosed acute leukemia.

Remember, you are not alone in this journey; there are always new developments and research efforts to find a cure.

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Sources: 

• A Study of Revumenib in R/​R Leukemias Including Those With an MLL/​KMT2A Gene Rearrangement or NPM1 Mutation (AUGMENT-101)
• Syndax Presents Updated Positive Data from BEAT AML and AUGMENT-102 Phase 1/2 Combination Trials of Revumenib in Patients with Acute Leukemias at EHA 2024 Congress
• Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101)