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

The FDA has granted an orphan drug designation to OM-301 for the treatment of patients with multiple myeloma.

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

OM-301 is an investigational fusion peptide that binds to HDM2 on the surface of cancer cells before selectively creating pores in those cells to induce cell death.2,3 The HDM2-binding element is derived from the HDM2-binding component of the p53 protein. After one end of OM-301 binds to HDM2 on the cell surface, the other end is designed to burrow into the cell membrane.

Preclinical research has demonstrated that OM-301 is effective against multiple myeloma.1 OM-301 was effective against eight multiple myeloma cell lines, including p53-mutated and null cell lines. The agent also prolonged survival 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.4 These findings suggested that OM-301 may be a novel and effective therapeutic option for the treatment of patients with multiple myeloma.

Previously, OM-301 received an orphan drug designation from the regulatory agency for the treatment of patients with acute myeloid leukemia (AML).

In human AML that had been transplanted into mice, OM-301 was found to have efficacy. At 4, 8, and 12 weeks, mice treated with OM-301 had significantly lower percentages of CD45-positive cells in the peripheral blood compared with vehicle-treated mice.3

Additionally, OM-301 led to a near doubling of survival in human AML–transplanted mice (P < .0001). At day 100, all secondary transplant mice treated with OM-301 were alive, compared with approximately 10% of vehicle-treated mice. No vehicle-treated mice survived past 110 days, and 50% of OM-301–treated mice survived past 150 days.

Oncolyze is currently advancing OM-301 through Good Manufacturing Practice–compliant toxicology and expects to open an investigational new drug application with the FDA before launching the first phase 1/2 clinical trial of the agent in 2023.2

References

  1. Oncolyze announces FDA orphan drug designation for OM-301 for the treatment of multiple myeloma. News release. Onoclyze. April 11, 2023. Accessed April 13, 2023. https://www.newswire.com/news/oncolyze-announces-fda-orphan-drug-designation-for-om-301-for-the-22007391
  2. Pipeline. Oncolyze. Accessed April 13, 2023. https://oncolyze.com/pipeline
  3. Science. Oncolyze. Accessed April 13, 2023. https://oncolyze.com/science
  4. 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