Mebufotenin

Mebufotenin is the compound at the center of GH Research’s GH001 program for treatment-resistant depression. The direct-source record is useful but not perfectly tidy: the JAMA Psychiatry abstract describes GH001 as a synthetic formulation of inhaled mebufotenin, while older shorthand around the program often used 5-MeO-DMT. This page keeps those layers visible instead of treating the compound name and the company asset as interchangeable. That distinction matters because the underlying phase 2b signal is strong enough that readers need a clean read of what is actually being studied, not just a pile of blurred labels.

What the evidence package says

The strongest evidence checkpoint is the peer-reviewed randomized phase 2b paper published on Mar. 25, 2026. According to the PubMed record, the placebo-controlled portion used an individualized single-day inhalation regimen of up to three escalating doses and reported 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.

Alias versus asset naming

For SCM’s v1 handling, mebufotenin and 5-MeO-DMT belong to the alias/synonym layer of the compound record. GH001 does not. GH001 is the program/formulation/company-asset layer that GH Research is moving through clinical development. Keeping that distinction explicit prevents the wiki from collapsing a compound, a formulation, and a company program into one vague label.

Why this dossier matters

This dossier matters because the naming issue sits on top of a genuinely interesting clinical signal. The randomized phase 2b paper reports a large day 8 MADRS difference and remission in 23 of 40 patients on GH001 versus 0 of 41 on placebo, which is why this compound/program cluster is drawing real attention. Readers need both pieces at once: why the results are interesting on the merits and why the compound and program labels still should not be collapsed into one vague term.