GH Research announced that the FDA has lifted a clinical hold on the company’s IND for GH001 inhaled mebufotenin (5-MeO-DMT) for the treatment of treatment-resistant depression and that initiation of a Phase 3 trial is expected this year. The agency placed the clinical hold in 2023, citing “Insufficient information to assess risks to human subjects,” and the company announced submission of its response in June 2025.
In January 2025, GH Research said that it had completed toxicology studies necessary for its response to the hold and announced results from two Phase 2a trials. The following month, the company announced that a Phase 2b trial of GH001 in patients with treatment-resistant depression that was conducted in Europe had met its primary endpoint.
GH Research CEO Velichka Valcheva commented, “The FDA clearance is a major milestone and positions us to advance GH001 as a potential ultra-rapid and durable treatment option for TRD patients. We continue to expect initiation of our global pivotal program in 2026. We look forward to meeting with the FDA to align on the design for the pivotal Phase 3 program.”
Read the GH Research press release
