GH Research PLC (Nasdaq: GHRS) announced in June 2025 that it submitted its complete response to the U.S. Food and Drug Administration (FDA) regarding the previously announced clinical hold of its Investigational New Drug Application (IND) for GH001, a novel treatment for depression. The Dublin-based clinical-stage biopharmaceutical company delivered the response ahead of schedule, addressing the agency's regulatory concerns.
"We are thrilled to have submitted our response to the FDA ahead of schedule, addressing their clear requests with comprehensive data and completed toxicology studies. We remain fully committed to working closely with the agency to bring GH001 to patients," said Dr. Velichka Valcheva, Chief Executive Officer of GH Research.
Novel Inhalation Approach for Depression Treatment
GH001 represents the company's lead product candidate, formulated for mebufotenin administration via a proprietary inhalation approach. This innovative delivery method distinguishes the treatment from conventional depression therapies currently available in the market.
The company is focused on developing what it describes as a practice-changing treatment specifically for patients suffering from depression, with particular emphasis on treatment-resistant depression (TRD).
Phase 2b Trial Demonstrates Significant Efficacy
The regulatory submission follows positive results from GH Research's Phase 2b GH001-TRD-201 trial, which demonstrated statistically significant clinical activity. In this study, GH001 met its primary endpoint with a Montgomery-Åsberg Depression Rating Scale (MADRS) reduction from baseline of -15.5 points compared with placebo on Day 8, achieving statistical significance with p<0.0001.
Based on these observed clinical results, GH Research believes that GH001 has the potential to fundamentally change how treatment-resistant depression is managed in clinical practice today.
Regulatory Path Forward
The submission of the complete IND hold response represents a critical milestone in GH001's regulatory pathway. The company has provided the FDA with comprehensive data and completed toxicology studies to address the agency's previous concerns that led to the clinical hold.
GH Research's ability to submit the response ahead of the anticipated timeline demonstrates the company's preparedness and commitment to advancing the development program. The company now awaits the FDA's review of the submitted materials to determine the next steps for GH001's clinical development program.
