GH Research Advances Clinical Trials for GH001 in the Fight Against Treatment-Resistant Depression

DUBLIN, Ireland, Sept. 29, 2023 -- GH Research PLC GHRS, a clinical-stage biopharmaceutical company, today offered fresh updates and insights regarding its ongoing clinical development program for GH001, an investigational inhalable mebufotenin (5-MeO-DMT) therapy being studied for its potential in addressing treatment-resistant depression (TRD).

Strategic Clinical Development

Building on the momentum gained from a successful Phase 1/2 trial in TRD patients, GH Research is currently progressing with a multi-center, randomized, double-blind, placebo-controlled Phase 2b study in Europe for GH001 GH001-TRD-201. This trial involves an inhalation device sourced externally. Meanwhile, the company has filed an Investigational New Drug Application (IND) with the U.S. Food and Drug Administration (FDA) to initiate a Phase 1 clinical pharmacology trial for GH001 using a proprietary aerosol delivery device GH001-HV-106. This step, contingent on FDA approval, aims to allow a transition to a global Phase 3 pivotal program leveraging the proprietary device, subject to data confirmation and regulatory clearances.

Progress in the European Phase 2b Clinical Trial

The ongoing Phase 2b study, authorized in seven European nations, is designed to enroll about 80 participants across nearly 20 sites. Its principal goal is to evaluate the effectiveness of GH001's single-day individualized dosing regimen (IDR) compared to a placebo, as measured by the change in the Montgomery-Åsberg Depression Rating Scale (MADRS) after a week-long double-blind phase. Following this, a 6-month open-label phase allows for the continuation of treatment using the GH001 IDR as needed, depending on patients' clinical conditions. With a large proportion of the sites already operational and more initiations planned, GH Research anticipates concluding the double-blind phase by the third quarter of 2024, with comprehensive data expected by the end of that year's third or fourth quarter.

IND Submission Update for GH001

The recent IND submission for GH001 to the FDA resulted in a clinical hold due to inadequate information to evaluate risks. The FDA has committed to providing detailed feedback within 30 days, and GH Research looks forward to addressing any concerns to progress with its trial. Further updates will be shared in the next earnings release scheduled for November 2023.

Company Overview

At GH Research PLC, the mission to revolutionize the therapeutic landscape for psychological and neurological conditions is driven by novel mebufotenin (5-MeO-DMT) therapies. The company is currently centering its efforts on individuals with treatment-resistant depression.

About GH001

Leading the way in GH Research's product pipeline, GH001 has shown promise following two Phase 1 trials in healthy volunteers and a Phase 1/2 trial in TRD patients. With remarkable signs of ultra-rapid remission in 87.5% of the TRD patients treated with GH001 during the Phase 2 trial, the product candidate has high potential for transforming current TRD treatment methods. Besides the ongoing Phase 2b trial, GH001 is under investigation in two Phase 2a proof-of-concept studies that focus on bipolar II disorder and postpartum depression.

Advancements in GH002 and GH003

The development of GH002, mebufotenin (5-MeO-DMT) for intravenous use, is in Phase 1 clinical trials. GH003, another mebufotenin (5-MeO-DMT) formulation for intranasal administration, is in its preclinical stage. GH Research anticipates further developments of GH002 and GH003 for select populations and specific scenarios within mental health and neurology.

Forward-Looking Statements: This announcement contains projections about GH Research's operational and financial outlooks, strategy, product candidates, regulatory submissions, discussions, and potential outcomes, among other information. While these are based on current management beliefs, actual future events may differ significantly as they are subject to risks, uncertainties, and various factors.

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