The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for OPN-6602, an oral small-molecule EP300/CBP inhibitor, for patients with multiple myeloma (MM) who have undergone at least one prior therapy.
OPN-6602 targets the EP300 and CBP proteins, which are involved in the proliferation and survival of MM cells, according to Opna Bio, the manufacturer of the drug. The therapy is currently being tested in a phase 1 clinical trial.
Recent data presented at the American Society of Hematology (ASH) Annual Meeting & Exposition in December 2024 highlighted the promising results of OPN-6602 in preclinical studies, showing significant reduction in MM cell growth and survival, thus supporting its potential as a novel treatment option, the company wrote in a press release.
If approved, OPN-6602 could become a pivotal new option for patients with MM, the company reported.
