5-MEO-DMT developer GH Research has provided an update on its main clinical programs. The company is notoriously short on communications outside of the obligatory company updates, so investors are always keen on getting a glimpse of GH Research’s latest announcements.

Main points include:

• Currently recruiting for a European Phase 2b trial of GH001 in patients with treatment-resistant depression (TRD). Top-line data available in the third or the fourth quarter of 2024.
• Submitted an Investigational New Drug Application (IND) for GH001 with the FDA to initiate a Phase 1 trial with GH001 administered using proprietary aerosol delivery device (GH001-HV-106).
• The FDA has placed the requested IND on clinical hold due to “Insufficient information to assess risks to human subjects”. GH Research is awaiting more details and plans to provide an update at the next earnings release in November 2023.

See the full press release below

GH Research Provides Updates and Guidance on its Clinical Development Program for GH001 in Treatment-Resistant Depression

DUBLIN, Ireland, Sept. 29, 2023 (GLOBE NEWSWIRE) — GH Research PLC (Nasdaq: GHRS), a clinical-stage biopharmaceutical company dedicated to transforming the treatment of psychiatric and neurological disorders, today provided updates and guidance on its clinical development program for GH001, its proprietary inhalable mebufotenin (5-MeO-DMT) product candidate, in treatment-resistant depression (TRD).

Development Strategy

As previously announced, following successful completion of our Phase 1/2 trial of GH001 in patients with TRD (GH001-TRD-102), we are recruiting for a European multi-center, randomized, double-blind, placebo-controlled Phase 2b trial of GH001 in patients with TRD (GH001-TRD-201), where GH001 is administered using an externally-sourced inhalation device. Separately, we submitted, in the third quarter of 2023, an Investigational New Drug Application (IND) for GH001 with the U.S. Food and Drug Administration (FDA), with the purpose to initiate a Phase 1 healthy volunteer clinical pharmacology trial, where GH001 is administered using our proprietary aerosol delivery device (GH001-HV-106). The trial, subject to regulatory clearance from the FDA, is designed to support bridging to the clinical data generated with the externally-sourced inhalation device we currently use in our European Phase 2b trial such that we can initiate, subject to data and regulatory clearance, a subsequent global Phase 3 pivotal program using our proprietary device.

Update and Guidance on European Phase 2b Clinical Trial of GH001 in Patients with TRD

Our multi-center, randomized, double-blind, placebo-controlled Phase 2b trial of GH001 in patients with TRD (GH001-TRD-201) is now approved in seven European countries and is expected to recruit approximately 80 patients across approximately 20 sites. The primary objective of the trial is to determine the efficacy of our single-day individualized dosing regimen (IDR) of GH001 compared with placebo in improving depressive symptoms as assessed by the mean change from baseline in Montgomery-Åsberg Depression Rating Scale (MADRS) at the end of the 7-day double-blind phase. The double-blind phase is followed by a 6-month open-label extension phase where all patients can receive treatment with the GH001 IDR as-needed, based on the patient’s clinical status. With 14 sites initiated and additional site initiations scheduled, we are now in a position to provide guidance on completion of the double-blind phase of this trial, which is expected to occur in the third quarter of 2024, with top-line data available in the third or the fourth quarter of 2024.

Update on IND for GH001

We recently submitted an IND for GH001 with the FDA. At the end of the 30-day review period, the FDA advised that it had placed our IND on clinical hold due to “21 CFR 312.42(b)(1)(iv): Insufficient information to assess risks to human subjects”. The FDA indicated that a letter with additional details will be issued within 30 days. We are awaiting that follow-up letter from the FDA and look forward to working with them to resolve any outstanding concerns. We plan to provide an update in our next earnings release in November 2023.