Latest Updates of Revumenib for Acute Leukemia

Revumenib (Revufori) is a new medication recently approved for relapsed/refractory acute leukemia patients with the KMT2A translocation. Including both adult and pediatric patients one year and older.

However, there is still ongoing research with revumenib, which we will review in this article. These updates were presented at the 66th annual ASH conferences, where we interviewed lead researchers to bring you all the details you need to know! 

To learn more about other menin inhibitors, you can read our previous article: Menin Inhibitors: A New Treatment Option for Some Leukemia Patients

How Revumenib Works

Revumenib is a potent, oral, selective inhibitor of menin (a tumor suppressor protein). It prevents menin from binding to the KMT2A gene. 

Revumenib in an All-Oral Treatment Combination for Children Over 12 and Adults 

We’ll first review the phase I/II study of revumenib with decitabine/cedazuridine and venetoclax (the SAVE study) in relapsed/refractory AML (NCT05360160). 

This trial allowed patients older than 12 years old to participate, but the study is still ongoing. The median age was 35 years (range, 12-79 years), including 5 children. Patients received ASTX727 (decitabine/cedazuridine): 35 mg/100 mg PO daily on days 1-5, and venetoclax: 400 mg PO daily on days 1-14. Revumenib doses varied between patients to find the one that works best. 

The Study Results

• The overall response rate was 88%, with 74% achieving MRD negativity  
• Twelve patients were able to undergo stem cell transplant 
• Six months after starting treatment with revumenib, 59% were in remission, and 74% were alive 

Most common side effects included alterations in the electrocardiogram (QT prolongation in 58% of patients), liver enzyme elevation, and low white blood cell counts with fever (febrile neutropenia). 

These promising results support further investigation in newly diagnosed patients.

An Ongoing Study for Relapsed/Refractory Acute Leukemias 

The AUGMENT-101 Phase 2 study is an ongoing trial that evaluates revumenib, a selective menin inhibitor, as a treatment for relapsed or refractory acute leukemias with KMT2A rearrangements (KMT2Ar). The study represents the largest evaluation of menin inhibition in this patient population to date.

A total of 116 patients received at least one dose of revumenib; 24% of patients were pediatric. Among 97 evaluable patients, the study demonstrated promising response rates and MRD negativity:

• The overall response rate was 64% 
• 23% of patients achieved complete remission that lasted for at least 2 months, and for the majority, more than 6 months
• 58% of all responders achieved MRD negativity 
• Of those responding to the treatment, 34% of patients could receive a stem cell transplant after therapy, and nine patients resumed revumenib treatment as maintenance

The most common severe side effects were febrile neutropenia, anemia, and low platelet counts. Investigators are also closely monitoring patients who can experience differentiation syndrome (15%) and alterations in their heartbeat (13%). 

Final Thoughts

The latest research on revumenib highlights significant progress for relapsed/refractory acute leukemia with KMT2A rearrangements. Offering the potential for patients to undergo curative treatments like stem cell transplantation. However, research is ongoing, and continued patient participation in trials is essential to compare revumenib to other standard treatments and study long-term outcomes.

Your participation in clinical trials might not only offer you access to cutting-edge treatments but also contribute to improving care for future patients. That’s why HealthTree helps you every step of the way with the multiple free resources Cure Hub Registry offers, like exploring personalized clinical trial options, tracking labs and past treatments, and so much more! 

Join Cure Hub Now

Empower yourself! Both patients and caregivers can take charge of their treatment and care by staying informed. Click here to read more exciting Leukemia news!