GH Research (NASDAQ:GHRS) shares surged in early trading Monday after the U.S. Food and Drug Administration lifted a long-standing clinical hold on its Investigational New Drug (IND) application for GH001.
The decision paves the way for the Dublin-based biotech to begin U.S. patient enrollment and initiate a global Phase 3 program for treatment-resistant depression (TRD) as early as 2026.
The clearance removes a major regulatory hurdle for GH Research’s lead candidate, an inhalable formulation of mebufotenin (5-MeO-DMT).
The company now plans to consult with the FDA to align on a Phase 3 trial design that replicates the success of its earlier Phase 2b study, which demonstrated "ultra-rapid" results for patients who had failed multiple prior therapies.
Data from that Phase 2b trial remains a cornerstone of the company’s valuation.
The treatment achieved a -15.5 point placebo-adjusted reduction in the Montgomery-Åsberg Depression Rating Scale (MADRS) by Day 8.
Perhaps more striking for clinicians is the durability and speed of the drug: 57.5% of patients reached remission within a week, and 73% remained in remission at six months following an average of only four treatments.
Unlike existing psychedelic-based therapies that require hours of supervision, GH001 features a median psychoactive duration of just 11 minutes, allowing most patients to be discharged within an hour.