The FDA Is Reviewing A New Treatment Option For Relapsed/Refractory AML

Syndax Pharmaceuticals recently announced that the FDA will prioritize the review of revumenib for the treatment of adult and pediatric relapsed or refractory KMT2A-rearranged (KMT2Ar) acute leukemia. We will know about the FDA's decision by September 26, 2024.

What Is Revumenib?

Revumenib is a small molecule inhibitor that targets a protein called menin. Menin plays a role in the growth and survival of leukemia cells. By blocking menin, revumenib aims to disrupt the cancerous process and lead to the death of leukemia cells.

Revumenib is primarily being investigated for the treatment of relapsed or refractory (R/R) acute leukemias. This means the leukemia has either returned after initial treatment (relapsed) or hasn't responded well to standard therapies (refractory). Specifically, clinical trials are focusing on:

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

It's considered a "first-in-class" menin inhibitor, meaning it's one of the first drugs developed to target this specific protein in leukemia treatment.

The AUGMENT-101 Trial

The data supporting the approval submission was released in December 2023) and came from a pivotal AUGMENT-101 trial (phase 2) designed to evaluate revumenib's safety, tolerability, and efficacy. This trial studied revumenib, administered orally, to treat relapsed and refractory AML patients who had received several previous treatments with unsuccessful results.

• 63% responded to the treatment.
• 70% of the patients who achieved a complete remission were MRD negative. 
• 39% received a stem cell transplant.
• 50% restarted revumenib for maintenance therapy post-transplant.

The study investigators shared

"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." Ibrahim Aldoss

Some of the most common side effects patients experienced while taking revumenib were diarrhea, vomiting, decreased appetite, fatigue, dysgeusia (which affects one's ability to taste), and anemia. Most side effects were easily controlled, and revumenib was well tolerated. 

Source:

• Syndax Announces FDA Priority Review of NDA for Revumenib for the Treatment of Relapsed/Refractory KMT2Ar Acute Leukemia
• Revumenib Found Effective Against High-Risk KMT2Ar Acute Leukemia

Learn More About Revumenib

• ASH 2023: Revumenib Combined With Decitabine/Cedazuridine And Venetoclax For Relapsed Or Refractory AML
• Revumenib Targets KMT2A And NPM1 Mutations