Project Grant R01FD007548

The University of California, Los Angeles (UCLA) was awarded a $849,648 Project Grant from the Food and Drug Administration (FDA) Research program (CFDA 93.103) to conduct a Phase 2 clinical trial on the safety and efficacy of the dual PI3K + HDAC inhibitor drug fimepinostat (CUDC-907) for the treatment of patients with Cushing's disease. The study, which began on September 5, 2024, will assess fimepinostat's ability to suppress pituitary tumors and normalize cortisol levels in 20 subjects over a 4-week period. Key outcome measures include changes in 24-hour urinary free cortisol, plasma ACTH, and clinical signs/symptoms of hypercortisolism. UCLA will serve as the sole prime recipient for this grant, with the study taking place at its Los Angeles facilities. The findings from this pilot trial will inform the design of larger, longer-term clinical studies on fimepinostat as a potential therapy for the rare and life-threatening Cushing's disease.