FDA Grants Orphan Drug Designation to OM-301 for Multiple Myeloma


The FDA has now granted OM-301 2 orphan drug designations for the treatment of patients with multiple myeloma and acute myeloid leukemia.

OM-301 has received an orphan drug designation from the FDA for the treatment of patients with multiple myeloma, according to Oncolyze.1

OM-301 is an investigational drug candidate being evaluated for the treatment of several hematologic and solid cancer indications. The fusion peptide binds to HDM2 which is located on the surface of cancer cells and induces cell death while sparing normal and healthy cells.2 Once one of the ends of OM-301 binds to HDM2 on the cell surface, the other end attaches to the external surface of the cell membrane.

The company envisions delivering the agent to patients via intravenous infusion, once daily, for several days, but this will be further evaluated in upcoming clinical trials.

In preclinical studies of OM-301, the agent has demonstrated efficacy against multiple myeloma and was effective against 8 multiple myeloma cell lines, including p53-mutated and null cell lines.1 Survival with the agent was also prolonged in an in-vivo, proof-of-concept study.

Findings from a preclinical study also showed that although OM-301 was designed for p53-selective cells, the agent may instead interact with BCL-2 to induce mitochondrial dysfunction and cell death, irrespective of TP53 status.3

When given at an intraperitoneal dose of 20 mg/kg/body weight 2 times a day, OM-301 led to a significant reduction in tumor size with respect to vehicle control (n = 7), showing the stability of OM-301 without any loss of its activity (P < .0001). OM-301 also significantly increased survival in the study (P < .0001).

These data suggest that OM-301 may be a novel and effective therapeutic option for the treatment of patients with multiple myeloma.

Additionally in January 2022, the FDA also granted orphan drug designation to OM-301 for the treatment of patients with acute myeloid leukemia (AML) as preclinical models demonstrated the efficacy of OM-301. In human AML that had been transplanted into mice, OM-301 was found to have efficacy.2

The agent continues to be investigated for patients with AML and multiple myeloma, as well as for patients with solid tumors.

Now, Oncolyze is advancing OM-301 through Good Manufacturing Practice–compliant toxicology. The company anticipates they will open an investigational new drug application with the FDA before launching the first phase 1/2 clinical trial of OM-301 in 2023.

  1. Oncolyze announces FDA orphan drug designation for OM-301 for the treatment of multiple myeloma. News release. Onoclyze. April 11, 2023. Accessed April 14, 2023. https://bit.ly/43AboaG
  2. Pipeline. Oncolyze. Accessed April 14, 2023. https://oncolyze.com/pipeline
  3. Nigam L, Zhu Y, Troadec E, et al. OM301, a synthetic polypeptide containing the p53TA (Transactivation) domain, impairs mitochondrial activity and survival of myeloma cells. Blood. 2021;138(suppl 1):2661. doi:10.1182/blood-2021-151506
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