Acute Leukemia: 30% of Participants Achieve Remission with New Drug

As of 2020, more than 474,000 people around the world have leukemia — a cancer of the blood. Of the two main types of leukemia, acute leukemia is the most aggressive causing cells to multiply very rapidly in the blood and blood marrow. 

Although there are treatments for acute leukemia, survival rates depend on a variety of factors including a person’s age and how progressive their disease is.

To add to treatments available for acute leukemia, Syndax Pharmaceuticals, Inc. recently announced Phase 1 results for its Phase 1/2 AUGMENT-101 trial of revumenib in people with nucleophosmin mutant (mNPM1) and KMT2A-rearranged (KMT2Ar) relapsed/refractory (R/R) acute leukemia.

Of the 60 people included in the study, 18 of them experienced complete remission or complete remission with partial recovery of peripheral blood counts (CR/CRh) for a little over nine months after treatment.

The results were also published in a study in the journal Nature.

Nucleophosmin mutant (mNPM1) acute leukemia is a type of acute myeloid leukemia (AML) — a cancer of the blood cells. Although AML can happen at any age, it is most common in people over the age of 45.

The mNPM1 acute leukemia occurs when the nucleophosmin (NPM1) gene in the body becomes mutated. About 30% of all cases of AML are caused by an NPM1 gene mutation.

KMT2A-rearranged (KMT2Ar) relapsed/refractory (R/R) acute leukemia occurs from a mutation of the KMT2A gene. KMT2Ar acute leukemia can present as either AML, acute lymphoblastic leukemia (ALL) or mixed phenotype acute leukemia (MPAL).

Previous research shows KMT2Ar acute leukemia to have high levels of chemotherapy resistance and relapse.

In KMT2Ar acute leukemia, the KMT2A genes make fusion proteins that need to interact with a specific protein called menin in order for cancer to grow. Additionally, mNPM1 acute leukemia is also dependent on specific genes and has been shown to be affected by the menin-KMT2A interaction.

Syndax Pharmaceuticals also recently released another study further confirming the dependency of mNPM1 and KMT2Ar acute leukemias on the menin-KMT2A interaction. And previous research shows that menin inhibitors can help in the treatment of acute leukemia.

For this study, Dr. Stein and his team administered revumenib to 60 people with either mNPM1 or KMT2Ar acute leukemia. All participants had been heavily pretreated with about four prior therapies and 46% of them had previously received a stem cell transplant.

Upon analysis, researchers found 30% — or 18 of the study participants — experienced a CR/CRh with a median duration of CR/CRh response of 9.1 months. And 78% of those 18 participants — or 14 of them — attained measurable residual disease (MRD) negativity.

“Most drugs that are oral targeted therapies don’t lead to MRD negativity, let alone a rate of MRD negativity of nearly 80%. We hope this translates into better outcomes for patients,” Dr. Stein said.

The phase 1 clinical trial also included data from 68 people to evaluate the safety of revumenib. According to researchers, the medication was well-tolerated by study participants and no participants stopped taking the therapy due to treatment-related adverse effects.

“The robust clinical dataset from a heavily pretreated relapsed/refractory patient population demonstrates that revumenib monotherapy was associated with encouraging clinical benefit, including deep molecular remissions and durable responses, with minimal toxicities,” says Dr. Ghayas C. Issa, assistant professor in the Department of Leukemia in the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center.


Issa, G.C., Aldoss, I., DiPersio, J. et al. The menin inhibitor revumenib in KMT2A-rearranged or NPM1-mutant leukaemia. Nature (2023).

Corrie Pelc (March 21, 2023). Acute leukemia: 30% of participants achieve remission with new drug. MedicalNewsToday. Retrieved on March 21, 2023 from 

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