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On April 11, 2023, it was announced that OM-301, an investigational drug, was granted orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of patients with multiple myeloma (MM).1,2 This follows its previous orphan drug designation received for the treatment of patients with acute myeloid leukemia (AML) on January 5, 2022.3
OM-301 is a fusion peptide that binds to HDM2 (human double minute-binding protein-2), a protein overexpressed on the cell surface of several types of hematologic malignancies and solid tumors. The attachment of OM-301 to the cancer cell surface leads to a membrane tension that creates pores in those cells and causes cell lysis.1,2,4
OM-301 has shown strong anti-MM activity in preclinical models. In vitro, it has demonstrated cytotoxic effects against multiple myeloma cells lines, including TP53-mutated/null cell lines (L363, RPMI-8226, U266, JJN3, and KMS11), with minimal cytotoxic effects against human peripheral blood mononuclear cells, and has also significantly reduced the tumor volume and prolonged survival within in vivo models.5 In a recent human AML transplanted mouse model, OM-301 demonstrated high efficacy.2 Based on these data, OM-301 presents a novel and effective treatment option for both AML and MM.
The proposed administration for OM-301 is a once daily intravenous infusion given on specified days, which will be determined in upcoming trials. The first phase I/II clinical trial of OM-301 is set to begin in 2023.2
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