A phase 1 clinical trial to evaluate a treatment for patients with relapsed or refractory acute leukemias — including those with MLL1/KMT2A gene rearrangements or NPM1 mutations — is now underway in multiple centers across the United States.

The research, developed by Biomea Fusion, Inc., is intended to examine BMF-219, an irreversible covalent menin inhibitor — meaning it blocks menin, a protein that plays a role in tumor signaling pathways in genetically defined leukemias.

Acute myeloid leukemia (AML; the most common form of acute leukemia) is the leading type of leukemia-related death in the U.S. and Europe. Around 20,000 people are diagnosed in the U.S. each year, with just a 29% five-year survival rate, according to the National Cancer Institute. Patients with relapsed or refractory AML have an average overall survival of just three to nine months. Additionally, researchers estimate that around 45% of all patients with AML have menin-dependent genetic drivers.

Acute lymphocytic leukemia (ALL), on the other hand, is less common. According to the National Cancer Institute, there are around 6,000 new cases in the U.S. annually. The five-year survival rate (70%) is also more hopeful, and approximately 10-15% of adult patients with ALL and 60-70% of pediatric patients with ALL have MLL1/KMT2A gene rearrangements.

“Just over four years ago, we took the concept of designing a small molecule that targets menin and brought forward BMF-219 to the clinic to significantly improve the lives of patients,” said Thomas Butler, Biomea’s Chief Executive Officer and Chairman of the Board, in a news release.

The trial goals are to assess the safety, tolerability and pharmacokinetics/pharmacodynamics — or how the body affects the drug and vice versa — of BMF-219, which researchers plan to give patients once daily at 100 mg in the form of an oral pill for continuous 28-day cycles. Menin inhibition is expected to have a therapeutic benefit for patients in the subpopulations the trial is focused on.

“BMF-219 is just our first asset graduating now into clinical development. Our team has evolved significantly and is set up today not only to explore the full potential of BMF-219 and the inhibition of the menin pathway but also to advance several other programs into the clinic,” said Ramses Erdtmann, president of Biomea Fusion.

The trial is expected to be complete in June 2023, with 100 patient participants. The plan is to begin with a dose-escalation/dose-expansion study, followed by a recommended dose phase, with multiple study groups. The different patient groups are expected to include individuals with acute leukemia, diffuse large B-cell lymphoma or multiple myeloma.

Patients are currently being recruited at The University of Texas MD Anderson Cancer Center in Houston. Recruitment is also expected to open in California, Florida, Ohio and Tennessee at various centers.

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