OPN-6602 is an oral small molecule inhibitor of E1A-binding protein (EP300) and CREB-binding protein (CBP) in development by Opna Bio. The biopharmaceutical company announced in a press release that the agent has received Orphan Drug designation (ODD) for multiple myeloma from the FDA.1
“We are pleased to have received ODD for OPN-6602 for the treatment of multiple myeloma, a further validation of the drug’s therapeutic potential in patients with this disease who have limited treatment options once they have relapsed,” commented Opna Bio cofounder and chief scientific officer Gideon Bollag, PhD.
This drug is currently under evaluation in a first-in-human phase I trial in patients with relapsed or refractory multiple myeloma in progress at several sites across the US. Opna Bio has sponsored this trial and expects to finish its single agent, dose-escalation phase in 2026.
In December 2024, Opna Bio presented multiple myeloma mouse xenograft model data on OPN-6602 at the American Society of Hematology Annual Meeting in San Diego, California. In a press release, the company reported that OPN-6602 monotherapy produced 71% tumor suppression and resulted in 100% tumor regression when used in combination with dexamethasone, pomalidomide, or mezigdomide. The agent also showed potential to defeat resistance mechanisms of disease to standard-of-care regimens along with reduced toxicity.2
Beyond the phase I trial currently underway, Opna Bio plans to develop OPN-6602 in combination with other standard-of-care agents for treatment of multiple myeloma.1
References
- Opna Bio receives orphan drug designation for OPN-6602, an oral EP300/CBP bromodomain inhibitor, for multiple myeloma. News release. Opna Bio. February 12, 2025. Accessed February 19, 2025.
- Preclinical data with OPN-6602 in multiple myeloma show 100% tumor regression in combination treatment studies. News release. Opna Bio. December 9, 2024. Accessed February 19, 2025
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