GH Research Achieves Breakthrough with Ultra-Rapid Antidepressant GH001 in Phase 2b Trial

GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company, announced promising results from its Phase 2b clinical trial of GH001, an inhalable mebufotenin product candidate, for patients with treatment-resistant depression (TRD). The randomized, double-blind, placebo-controlled study demonstrated that GH001 achieved its primary endpoint, delivering an ultra-rapid antidepressant effect.

Patients treated with GH001 experienced a significant reduction in depressive symptoms, with a placebo-adjusted decrease of -15.5 points on the Montgomery-Åsberg Depression Rating Scale (MADRS) by Day 8 (p<0.0001). Remarkably, 57.5% of patients achieved remission by Day 8 compared to none in the placebo group.

All secondary endpoints were met, with GH001 showing statistically significant improvements in the Clinical Global Impression-Severity (CGI-S) scale, Hamilton Anxiety Rating Scale (HAM-A), and Quality of Life Enjoyment and Satisfaction Questionnaire (Q-LES-Q-SF) on Day 8.

Safety data from the double-blind phase indicated GH001 was well tolerated, with no serious adverse events (SAEs) reported. There was no evidence of treatment-emergent suicidal ideation or behavior, and 97.4% of patients were discharge-ready within one hour of the last dose.

As of January 22, 2025, in the ongoing open-label extension (OLE) phase, 77.8% of completers remained in remission at the six-month mark, with most requiring only 1-4 treatments over this period. No SAEs have been reported in the OLE to date.

“A novel treatment with such a large and rapid effect, particularly one that may require only infrequent, short clinic visits, has the potential to be a practice-changing treatment,” said Professor Michael E. Thase, MD, from the University of Pennsylvania.

GH Research’s CEO, Dr. Villy Valcheva, highlighted the significance of these results, stating, “Today, as we share our unprecedented positive Phase 2b data, we celebrate a significant milestone in our journey to interventional psychiatry and pave the way for our future commercial success with GH001 in treatment-resistant depression.”

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