The U.S. Food and Drug Administration removed the clinical hold on GH Research PLC’s investigational new drug application for its inhalable 5‑MeO‑DMT candidate, GH001, on January 5, 2026. The clearance eliminates the last regulatory obstacle that had stalled U.S. development of the drug and allows the company to enroll U.S. subjects and align its pivotal Phase 3 program across major jurisdictions.
GH001 is an inhaled formulation of 5‑MeO‑DMT that delivers an ultra‑rapid antidepressant effect with a short psychoactive experience. In a Phase 2b trial, the drug produced a placebo‑adjusted reduction of 15.5 points on the Montgomery‑Åsberg Depression Rating Scale by Day 8, a 57.5 % remission rate at Day 8, and sustained remission of 73 % at six months. No serious treatment‑related adverse events were reported, and the median duration of the psychoactive experience was approximately 11 minutes.
The company’s Phase 3 program will replicate the Phase 2b design, enrolling 1,200–1,500 patients across the U.S., Europe, and Japan, with a primary endpoint of sustained remission at 12 weeks. Management reiterated its plan to launch the global pivotal trial in 2026, emphasizing that the FDA clearance removes a key risk that could have delayed the program and increased cash burn. CEO Dr. Velichka Valcheva said the lift “positions us to advance GH001 as a potential ultra‑rapid and durable treatment option for TRD patients.”
Financially, GH Research reported a net loss of $14.0 million in Q3 2025, up from $12.1 million in Q3 2024, while maintaining a strong cash position of $293.9 million as of September 30, 2025. The company remains pre‑revenue, with no revenue reported in Q3 2025, and its burn rate is expected to support operations for 30–36 months at current spending levels.
The treatment‑resistant depression market is estimated to be worth $10–12 billion annually in the U.S. alone. GH001’s rapid onset and durability could position it as a plug‑and‑play alternative to existing interventional psychiatry treatments such as J&J’s Spravato. Analysts note that the drug’s short psychoactive window and inhaled delivery could improve patient adherence and reduce the need for specialized clinical settings.
Investors reacted favorably to the FDA clearance, citing the removal of a regulatory hurdle and the robust Phase 2b data as key drivers of confidence in GH001’s commercial potential.
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