FDA grants orphan drug designation to OM-301 for the treatment of patients with AML and MM

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