Companies
GH Research
Summary
GH Research is a biotech company developing GH001, described in the peer-reviewed literature as a synthetic formulation of inhaled mebufotenin, for treatment-resistant depression. In early 2026 the company said FDA lifted the GH001 clinical hold, and the program’s phase 2b results were then published in JAMA Psychiatry.
Why it matters
GH Research is one of the clearer tests of whether a short-duration inhaled psychedelic program can move from strong phase 2b data into a real pivotal path. The record also needs careful reading because company, compound, and program naming can blur together if the dossier is too loose.
Research notes
Context, reporting, and structured background for this dossier.
GH Research is a biotech company focused on GH001 for treatment-resistant depression. In the peer-reviewed literature, GH001 is described as a synthetic formulation of inhaled mebufotenin. The company’s most important 2026 checkpoints were procedural and evidentiary: on Jan. 5 it said FDA lifted the clinical hold on the GH001 IND, and on Mar. 25 the program’s phase 2b results appeared in JAMA Psychiatry.
Lead program and evidence
The randomized phase 2b paper remains the strongest anchor for the program. According to PubMed, the placebo-controlled portion enrolled adults with treatment-resistant depression at 16 European sites and used up to three escalating inhaled doses on a single day. The paper reports a least-squares mean difference of -15.5 points on MADRS at day 8 for GH001 versus placebo, with remission in 23 of 40 patients on GH001 versus 0 of 41 on placebo during the blinded period.
Why the source language still needs care
GH Research’s releases are useful, but they are not the same thing as an FDA memo or a neutral trial summary. The cleanest defensible company-level read is narrower: the U.S. regulatory hold is no longer blocking the IND, the central phase 2b package is peer-reviewed, and Phase 3 remains forward-looking company guidance until a pivotal program is actually underway.
This dossier also keeps the company, compound, and product-candidate layers separate on purpose. Mebufotenin and 5-MeO-DMT belong to the alias/synonym layer of the compound record, while GH001 is the program/formulation that GH Research is trying to move through development.
Citations and source links
Source material used to ground or extend this dossier.
- [1] Official site
Related reporting
Recent SCM reporting that overlaps this dossier’s company, program, or governance record.
- GH001 After the Clinical Hold Lift: What Actually Changed
GH001's FDA clinical hold is over, and the program now has a peer-reviewed phase 2b paper in JAMA Psychiatry. That is real progress, but it is not the same thing as pivotal proof or an FDA endorsement of efficacy.
- 5-MeO-DMT, Mebufotenin, and GH001: What Those Names Actually Mean
GH001 is the clinical-program name. Mebufotenin and 5-MeO-DMT are the compound-name layer around it. Keeping those labels separate makes the phase 2b signal easier to read: promising, source-backed, and still not the same as pivotal proof.
- GH001 Is Not BPL-003
GH Research and Beckley Psytech are both building clinical programs around the 5-MeO-DMT / mebufotenin layer. The useful map keeps the assets separate: GH001 is an inhaled mebufotenin program; BPL-003 is an intranasal 5-MeO-DMT benzoate program.
Related entities
Other dossiers that help connect this page to the wider reporting record.
- Mebufotenin
Mebufotenin is the compound at the center of GH Research’s GH001 program for treatment-resistant depression. The naming needs care, but this dossier matters for more than nomenclature: the peer-reviewed phase 2b paper reports a large day 8 signal and remission in 23 of 40 patients on GH001 versus 0 of 41 on placebo.
- Treatment-Resistant Depression (TRD)
- FDA
The U.S. Food and Drug Administration is the gatekeeper for psychedelic drug development in the United States, from IND clearance through advisory committee review and any eventual approval or rejection.