JBI-802 granted orphan drug designation by the FDA

On January 5, 2023, JBI-802, an oral, potent, selective dual inhibitor of two epigenetic targets of the transcriptional regulator CoREST complex, LSD1 and HDAC6, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with acute myeloid leukemia.¹

This designation is based on several relevant preclinical models, including erythroid leukemia, where JBI-802 demonstrated synergistic antitumor activity and a favorable safety profile.¹

JBI-802 also received orphan drug designation for the treatment of small cell lung cancer.¹ The first-in-human trial evaluating JBI-802 in multiple neuroendocrine tumors is ongoing, with plans to expand into hematologic cancers, including acute myeloid leukemia, essential thrombocythemia, and other myeloproliferative cancers.¹