mebufotenin inhaled (GH001) / GH Research - LARVOL DELTA

Safety and tolerability results from a phase 2b, double-blind trial with an open-label extension of GH001 in treatment-resistant depression (CINP 2026) - P2 | "GH001 administered as an IDR with up to five re-treatments over 6 months was well tolerated in patients with TRD. There were no new safety or tolerability concerns in the 6-month OLE. The overall safety and tolerability profile remained consistent with previously reported trials investigating GH001, supporting its potential as a new treatment option for TRD without compromising patient safety."

Clinical • P2b data • Asthma • CNS Disorders • Depression • Immunology • Major Depressive Disorder • Mood Disorders • Psychiatry • Respiratory Diseases

Rapid antidepressant effects of inhaled GH001 in treatment-resistant depression: results from a phase 2b, double-blind, randomized controlled trial with 6-month follow-up (CINP 2026) - P2 | "GH001 was well tolerated and showed rapid, significant depressive symptom improvements in TRD. Data from the OLE indicate GH001 resulted in sustained remission over 6 months with infrequent treatments."

Clinical • P2b data • CNS Disorders • Depression • Major Depressive Disorder • Migraine • Mood Disorders • Psychiatry

GH001 Efficacy is Independent of Prior Antidepressant Treatment Failures in Treatment-Resistant Depression: A Post Hoc Analysis of a Phase 2b Randomized Controlled Trial. (PubMed, Psychopharmacol Bull) - "Secondary endpoints were not associated with treatment history. The efficacy of GH001 in patients with TRD appears largely independent of number of prior lifetime antidepressant treatments, and further research in patients with extensive treatment histories will be conducted in subsequent development stages."

Journal • P2b data • Retrospective data • CNS Disorders • Depression • Major Depressive Disorder • Mood Disorders • Psychiatry

Inhaled Mebufotenin (GH001) for Adult Patients With Postpartum Depression: A Phase 2a Open-Label Clinical Trial. (PubMed, J Clin Psychiatry) - P2 | " GH001 demonstrated rapid and significant improvements in depressive symptoms and remission of PPD with an acceptable safety profile and parallel improvements across secondary end points. Trial Registration: ClinicalTrials.gov identifier: NCT05804708; EudraCT identifier: 2021-006879-42."

Journal • P2a data • CNS Disorders • Depression • Major Depressive Disorder • Mood Disorders • Pain • Postpartum Depression • Psychiatry

Pharmacokinetics and Safety of GH001 Delivered Via a GH001 Aerosol Delivery System in Healthy Subjects (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: GH Research Ireland Limited

New P1 trial

GH001 vs Placebo in Patients With Treatment-Resistant Depression: A Randomized Clinical Trial. (PubMed, JAMA Psychiatry) - P2 | "In this study, an individualized dosing regimen of inhaled GH001 resulted in significant improvements in depression symptoms relative to placebo and was well tolerated, supporting its potential as a novel, rapid-acting treatment for treatment-resistant depression. ClinicalTrials.gov Identifier: NCT05800860."

Clinical • Journal • CNS Disorders • Depression • Mood Disorders • Psychiatry

GH Research PLC…reported that it will provide an update on the status of its Investigational New Drug Application (IND) for GH001 with the U.S. Food and Drug Administration (FDA) and its global pivotal Phase 3 program in treatment-resistant depression (TRD) on Monday, January 5, 2026, at 7.00 a.m. EST (GlobeNewswire)

IND • Depression

Safety and tolerability results from a phase 2b, double-blind trial with an open-label extension of GH001 in treatment-resistant depression (ECNP 2025) - P2 | "Results from the double-blind part of this trial indicate GH001 was well tolerated in patients with TRD. Further data from the OLE will inform on the safety of GH001 up to 6 months."

Clinical • P2b data • CNS Disorders • Depression • Major Depressive Disorder • Mood Disorders • Psychiatry • Suicidal Ideation

Long-term data for GH001 in patients with treatment-resistant depression (ECNP 2025) - "Honoraria – Angelini, GH Research, Janssen, and Novartis. Advisory boards – Douglas Pharmaceuticals, GH Research, Janssen, MSD, and Novartis (relationships reported within the last three years)"

Clinical • CNS Disorders • Depression • Migraine • Mood Disorders • Psychiatry

Psychoactive effects of GH001 in patients with treatment-resistant depression: results from a phase 2b, double-blind, randomised controlled trial (ECNP 2025) - P2 | "Administration of GH001 elicited pronounced PsE, as assessed by the PES, with 94.6% of participants who complied with dose escalation achieving a PE in this trial of patients with TRD. These results demonstrate the utility of the PES in guiding IDR dose escalation to maximise the therapeutic effects of GH001. PD effects of GH001 will be further evaluated in the open-label extension, which allows for up to five re-treatments with GH001 over a 6-month period."

Clinical • P2b data • CNS Disorders • Depression • Mental Retardation • Mood Disorders • Psychiatry

Evaluation of the peak experience scale as a rapid assessment tool for the strength of a psychoactive experience with 5-MeO-DMT. (PubMed, Front Psychol) - "Data were gathered from three separate studies in which a 5-MeO-DMT formulation (GH001) was administered via pulmonary inhalation to healthy volunteers and patients with treatment resistant depression (N = 84) as either single doses (0 [placebo], 2, 6, 12, 18 mg) or an incremental individualized dosing regimen (IDR)...The PES is concluded to be an effective tool to rapidly assess the strength of the psychedelic experience with 5-MeO-DMT. The PES could prove useful to gain fast insight into the strength of a psychedelic dose in individual patients and potentially guide dose and re-dose selection of rapid-acting psychedelics."

Journal • CNS Disorders • Depression • Psychiatry

A Trial of GH001 in Patients With Treatment-resistant Depression (clinicaltrials.gov) - P2 | N=81 | Completed | Sponsor: GH Research Ireland Limited | Active, not recruiting ➔ Completed

Trial completion • CNS Disorders • Depression • Mood Disorders • Psychiatry

GH Research Reports Full Year 2024 Financial Results and Provides Business Updates (GlobeNewswire) - "Phase 2b clinical trial of GH001 in patients with treatment-resistant depression on track for completion of last patient visit in the open-label extension in Q1 2025...'We are preparing to engage with the FDA in advance of providing a full response to the IND hold which we are on track to submit in mid-2025.'"

IND • Trial status • CNS Disorders • Depression

Phase 2 Clinical Trial of GH001 in Bipolar II Disorder (clinicaltrials.gov) - P2 | N=6 | Terminated | Sponsor: GH Research Ireland Limited | N=15 ➔ 6 | Trial completion date: Dec 2023 ➔ Dec 2024 | Recruiting ➔ Terminated | Trial primary completion date: Dec 2023 ➔ Nov 2024; Recruitment of this patient population was more challenging than anticipated. The sponsor concluded that a sufficient amount of patients have completed the trial to demonstrate proof of concept in this patient population.

Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Bipolar Disorder • CNS Disorders • Depression • Psychiatry

Phase 2 Clinical Trial of GH001 in Postpartum Depression (clinicaltrials.gov) - P2 | N=10 | Terminated | Sponsor: GH Research Ireland Limited | N=15 ➔ 10 | Recruiting ➔ Terminated; Recruitment of this patient population was more challenging than anticipated. The sponsor concluded that a sufficient amount of patients have completed the trial to demonstrate proof of concept in this patient population.

Enrollment change • Trial termination • CNS Disorders • Depression • Mood Disorders • Postpartum Depression • Psychiatry

GH Research Announces Primary Endpoint Met in Phase 2b Trial with GH001 in TRD Demonstrating -15.5 Point Placebo-adjusted MADRS Reduction (GlobeNewswire) - P2b | N=80 | NCT05800860 | Sponsor: GH Research Ireland Limited | "Primary endpoint met, GH001 led to an ultra-rapid anti-depressant effect with a significant placebo-adjusted MADRS reduction from baseline of -15.5 on Day 8 (p<0.0001); The majority of the patients treated with GH001 achieved remission with a 57.5% remission rate on Day 8 compared with 0% in the placebo group (p<0.0001); All other secondary endpoints were met with clinically and statistically significant improvements on Day 8, compared with placebo; During the double-blind part, GH001 was well tolerated and no serious adverse events (SAE) were reported. There was no evidence of treatment-emergent suicidal ideation or behavior. As of January 22, 2025, no SAEs have been reported throughout the open label extension (OLE)...Patients who had remission on Day 8 after their first active treatment had a 91.7% remission rate at 6 months..."

P2b data • CNS Disorders • Depression

GH Research to Provide Update on Phase 2b Trial with GH001 in TRD (GlobeNewswire) - "GH Research PLC...announced that it will host a conference call and live webcast on Monday, February 3, 2025, at 8.00 a.m. EST to provide an update on data from the randomized, double-blind, placebo-controlled Phase 2b trial of GH001 in treatment-resistant depression (TRD)."

P2b data • Depression • Major Depressive Disorder

A Trial of GH001 in Patients with Treatment-resistant Depression (clinicaltrials.gov) - P2 | N=80 | Active, not recruiting | Sponsor: GH Research Ireland Limited | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2024 ➔ Mar 2025 | Trial primary completion date: Jun 2024 ➔ Oct 2024

Enrollment closed • Trial completion date • Trial primary completion date • CNS Disorders • Depression • Mood Disorders • Psychiatry

Pharmacokinetics of GH001 Delivered Via a Proprietary Aerosol Delivery Device in Healthy Subjects (clinicaltrials.gov) - P1 | N=52 | Recruiting | Sponsor: GH Research Ireland Limited | Not yet recruiting ➔ Recruiting

Enrollment open

Pharmacokinetics of GH001 Delivered Via a Proprietary Aerosol Delivery Device in Healthy Subjects (clinicaltrials.gov) - P1 | N=52 | Not yet recruiting | Sponsor: GH Research Ireland Limited

New P1 trial

Phase 2 Clinical Trial of GH001 in Postpartum Depression (clinicaltrials.gov) - P2 | N=15 | Recruiting | Sponsor: GH Research Ireland Limited | Trial completion date: Dec 2023 ➔ Sep 2024 | Trial primary completion date: Dec 2023 ➔ Sep 2024

Trial completion date • Trial primary completion date • CNS Disorders • Depression • Mood Disorders • Postpartum Depression • Psychiatry

A Trial of GH001 in Patients With Treatment-resistant Depression (clinicaltrials.gov) - P2 | N=80 | Recruiting | Sponsor: GH Research Ireland Limited

Trial completion date • Trial primary completion date • CNS Disorders • Depression • Mood Disorders • Psychiatry

A Trial of GH001 in Patients With Treatment-resistant Depression (clinicaltrials.gov) - P2 | N=80 | Recruiting | Sponsor: GH Research Ireland Limited | Phase classification: P2b ➔ P2

Phase classification • CNS Disorders • Depression • Mood Disorders • Psychiatry

GH Research Reports Second Quarter 2023 Financial Results and Provides Business Updates (GlobeNewswire) - "As previously announced, we expect to submit an IND with the U.S. FDA for GH001 in TRD, delivered with our proprietary aerosol delivery device, in the third quarter of 2023. Pending clearance by the FDA, we expect to initiate a Phase 1 clinical pharmacology trial of GH001 delivered with our proprietary aerosol delivery device in healthy volunteers (GH001-HV-106) in the fourth quarter of 2023....Our Phase 2a proof-of-concept clinical trial of GH001 in postpartum depression (GH001-PPD-203) is ongoing and, as previously announced, is expected to be completed in the fourth quarter of 2023. Our Phase 2a proof-of-concept clinical trial of GH001 in bipolar II disorder with a current depressive episode (GH001-BD-202) is recruiting slower than previously projected and we now expect this trial to be completed in the first quarter of 2024."

IND • New P1 trial • Trial completion date • Bipolar Disorder • CNS Disorders • Depression • Mood Disorders

A phase 1/2 trial to assess safety and efficacy of a vaporized 5-methoxy-N,N-dimethyltryptamine formulation (GH001) in patients with treatment-resistant depression. (PubMed, Front Psychiatry) - P1/2 | "Administration of GH001 to a cohort of 16 patients with TRD was well tolerated and provided potent and ultra-rapid antidepressant effects. Individualized dosing with up to three doses of GH001 on a single day was superior to single dose administration.Clinical Trial registration: Clinicaltrials.gov Identifier NCT04698603."

Journal • P1/2 data • CNS Disorders • Depression • Mood Disorders • Psychiatry