GH Research Announces FDA Lifts Clinical Hold on GH001, Clearing Path for Global Phase 3 Initiation in 2026
January 5, 2026 – Biotechnology, Clinical Trials, Drug Discovery, Other, Pharmaceutical – FDA clearance, GH Research, Investigational New Drug, biopharma, mental health disorders, neurology
- GH001 cleared by FDA for U.S. clinical investigation, enabling U.S. subject enrollment
- Company to seek FDA alignment on global Phase 3 program replicating Phase 2b design Phase 3 initiation targeted for 2026
5 January 2026 — Dublin, Republic of Ireland — GH Research PLC, a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients by developing a practice-changing treatment in depression, today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on its Investigational New Drug Application (IND) for GH001.
This clearance enables U.S. subject enrollment and progresses the company toward alignment of its development across major jurisdictions.
“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,” said Dr. Velichka Valcheva, Chief Executive Officer. “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.”
GH001 Phase 2b Profile Highlights from the Previously Reported Phase 2b trial (GH001-TRD-201):
- Primary endpoint met: -15.5 point placebo-adjusted MADRS reduction on Day 8 (p<0.0001)
- Ultra-rapid remission (57.5% of patients on Day 8); 73% remission at 6 months with infrequent dosing (~4 treatments on average)
- Short psychoactive experience (median of ~11 minutes)
- No required psychotherapy
- 99% of patients discharge-ready within 1 hour of dosing
- Favorable safety: well-tolerated, no serious treatment-related adverse events
- Recent presentations at scientific conferences reinforce GH001’s potential for integration into existing interventional psychiatry practices through its convenient administration profile.
Michael E. Thase, MD, Professor of Psychiatry, Perelman School of Medicine at the University of Pennsylvania, said, “The large and rapid antidepressant effect observed with GH001 in the Phase 2b trial, combined with sustained remission through infrequent, short clinic visits, has the potential to be practice-changing for patients with treatment-resistant depression.”
About GH Research PLC
GH Research PLC is a clinical-stage biopharmaceutical company dedicated to transforming the lives of patients by developing a practice-changing treatment in depression. GH Research PLC’s initial focus is on developing its novel and proprietary mebufotenin therapies for the treatment of patients with treatment-resistant depression (TRD). Visit: www.ghres.com.
