WEBULL

FDA Lifts Hold On GH Research's Depression Drug Trial After Two Years

Benzinga · 5d ago

The U.S. Food and Drug Administration (FDA) on Monday lifted the clinical hold on GH Research Plc’s (NASDAQ:GHRS) Investigational New Drug Application (IND) for GH001.

“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.”

In February 2025, GH Research said its Phase 2b trial with GH001, an inhalable bufotenin product candidate for treatment-resistant depression, met its primary endpoint.

GH001 led to a significant reduction from baseline of -15.2 points in Montgomery-Åsberg Depression Rating Scale (MADRS) total score on Day 8, compared with +0.3 points in the placebo group (difference of -15.5 points, p<0.0001).

Also Read: Depression-Focused GH Research Shares Could See Significant Upside: Analyst

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.”

In September 2023, the FDA placed a clinical hold on the GH001 IND due to insufficient information to assess risks to human subjects.

Competitive Landscape

In January 2025, the FDA approved Johnson & Johnson's (NYSE:JNJ) supplemental New Drug Application for Spravato (esketamine) CIII nasal spray, as the first and only monotherapy for adults living with major depressive disorder who have had an inadequate response to at least two oral antidepressants.

In June 2025, Alto Neuroscience Inc. (NASDAQ: ANRO) purchased Chase Therapeutics Corporation‘s portfolio of dopamine agonist drug combinations for treatment-resistant depression for over $100 million.

In September 2025, Atai Life Sciences (NASDAQ:ATAI) and Beckley Psytech Limited released data from a proof-of-concept study investigating a two-dose induction regimen of BPL-003 (intranasal mebufotenin benzoate) in patients with treatment-resistant depression.

GHRS Price Action: GH Research shares were up 34.52% at $17.86 at the time of publication on Monday, according to Benzinga Pro data.

Read Next:

Photo via Shutterstock