Company: GHRS
Filing Date: 2025-07-29
Form Type: 20-F/A
Source: 0001140361-25-027850
Chunk: 173

Company: GH Research PLC
Filing Date: 2025-07-29
Form: 20-F/A
Chunk 173
---
 of GH001 administered via a proprietary aerosol delivery device in healthy subjects. Trial Design This is an open-label, Phase 1 clinical trial in healthy subjects that will include single- and multiple-dose parts. Following a screening period of up to 4 weeks prior to baseline, one single dose (Part 1), an IDR (Part 2), or two single doses (Part 3) of GH001 will be administered. In Part 1 and Part 2, GH001 will be administered via a proprietary aerosol delivery device on Day 1. In Part 2, the IDR consists of up to three increasing doses of GH001 given at approximately 1-hour intervals. The primary endpoint of this trial is to assess the pharmacokinetic profile of mebufotenin and bufotenine, a metabolite of mebufotenin, and safety and tolerability. Participants are monitored on the dosing day, with additional follow-up visits on Day 8 and Day 31. Regulatory Interactions Following a type C meeting with the FDA in May 2023, in August 2023 we submitted an IND for GH001, delivered with our proprietary aerosol delivery device, to the FDA. The IND-opening study was proposed to be a Phase 1 clinical pharmacology trial in healthy volunteers (GH001-HV-106), designed to support bridging to the clinical data generated with the third-party device we currently use in our clinical trials. As previously announced, i n September 2023, at the end of the 30-day statutory IND review period, the FDA advised us that it had placed our IND on clinical hold, and in October 2023, with a formal clinical hold letter, the FDA requested that we provide (i) an inhalation toxicology study in a non-rodent species and an additional inhalation toxicology study in rats, related to respiratory tract histology findings from a previously completed inhalation toxicology study in rats, (ii) additional device design verification information and (iii) updates to our investigator brochure. 104 Table of Contents In February 2024, we requested a meeting with the FDA to discuss specific aspects of their device feedback and this meeting was executed by way of written response. On January 10, 2025, we announced the completion of the requested inhalation toxicology studies and we are now preparing to engage with the FDA in advance of providing a complete response to the clinical hold, which we plan to submit in mid-2025. Indication Expansion Opportunities for GH