Slide 1: GH Research R&D Update July 2025
GH Research R&D Update July 2025
Slide 2: Disclaimer and forward-looking statements
This presentation has been prepared by GH Research PLC (“GH Research”). Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by the presenter or GH Research or any director, employee, agent, or adviser of GH Research. This presentation doe
Slide 3: Summary of GH Research R&D Updates
Summary of GH Research R&D Updates 1 2 3 On Track to Commence TRD Pivotal Program in 2026 Strong and Consistent Final Data from GH Mebufotenin in TRD Best in Class, Best in Molecule, Best in Therapeutic Category GH001 & GH002 Formulations: Progress Update Note: To-date, no head-to-head comparisons o
Slide 4: GH001 Update: IND Hold Response from the FDA
GH001 Update IND Hold Response from the FDA received. Only one hold topic remaining - the FDA requested that we either provide additional data or further justification related to the respiratory tract histology findings in rats. We strongly believe, based on scientific evidence, that the respiratory
Slide 5: Phase 2b Trial with an Open-Label Extension in TRD
Phase 2b Trial with an Open-Label Extension in Patients with Treatment-Resistant DepressionFull Analysis Set // Efficacy Clinicaltrials.gov ID: NCT05800860 1 2 3 On Track to Commence TRD Pivotal Program in 2026 Strong and Consistent Final Data from GH Mebufotenin in TRD Best in Class, Best in Molecu
Slide 6: Primary Endpoint: Mean MADRS Reduction of -15.5 on Day 8 vs Placebo
Primary Endpoint: GH001 Led to Mean MADRS Reduction from Baseline of -15.5 on Day 8a vs Placebo (P<0.0001) a. FDA Guidance notes that efficacy with rapid-acting antidepressants generally should be demonstrated within 1 week, supporting a primary efficacy endpoint within this timeframe.1 Abbreviation
Slide 7: 73% Remission Rate at 6 Months in OLE Completers
Double-blind n=40 patients who received GH001 OLE n=63 OLE completersa Day 8 Month 6b 73% Remission Rate at 6 Months in OLE Completers a. 63 patients who received active drug and completed the 6-month OLE per protocol (18 patients who terminated early are excluded), in DB one early termination (n=1)
Slide 8: Remission on Day 8 / Remission at Months
Remission on Day 8 / Remission at 6 Months a. Remission defined as a MADRS total score ≤10 b. ‘6 Months’ or ‘Month 6’ (end of trial) was at approximately 6 months post-study start (mean 168 days from Day 1 of double-blind period) c. Patients who completed the 6-month open-label extension follow-up p
Slide 9: 83% Fewer Treatment Visits with GH001 than with Spravato
GH001 Based on ICER estimate Treatment visits in 6 months 4 visitsa 23 visitsc 73.0% remission at 6 monthsb 83% Fewer Treatment Visits with GH001 than with Spravato Note: To-date, no head-to-head comparisons of any other products to any of our product candidates in any clinical trial have been compl
Slide 10: Patients Discharge Ready Within 1 Hour of Dose at 99% of Visits
Patients Discharge Ready Within 1 Hour of Dose at 99% of Visits (DB & OLE) Abbreviations: DB = Double-blind; OLE = Open-label extension; h = Hour 10 In total, only 2 patients across 3 visits not discharge ready at 1h Data from >250 GH001 treatment visits and 81 patients NEW DATA % of GH001 Treatment
Slide 11: Safety Data: Duration of Treatment Emergent Adverse Events
a. 3 events resolved within 24 hours: 2 events of fatigue and 1 of nausea; b. 3 events resolved within 72 hours: 1 of headache, 1 of memory impairment and 1 of tearfulness; c. 1 event (headache) lasted for longer than 72 hours TEAEs were classified according to the Medical Dictionary of Regulatory A
Slide 12: Treatment-Emergent Adverse Events of Special Interest
Treatment-Emergent Adverse Events of Special Interest (AESI) (Double-Blind Part) Abbreviations: AESI = Adverse Event of Special Interest; TEAE = Treatment-emergent adverse event. 12 NEW DATA GH001 (n=40) Placebo (n=41) Primary System Organ Class Preferred Term Patients n (%) Events n Patients n (%)
Slide 13: Topline Safety Data - 6 Months Open Label Extension
Abbreviations: SAE = Serious adverse event; TEAE = Treatment-emergent adverse event. No treatment-related SAEs during the 6-month duration of the trial No TEAEs of suicidal intent or suicidal behaviour occurred throughout the 6-month duration of the trial Lower rates of suicidal ideation were observ
Slide 14: Shortest Psychoactive Experience Amongst Therapeutic Class for TRD
Shortest psychoactive experience amongst therapeutic class for TRD, supports: Reduced requirement for clinic staff in the dosing room in commercial set up Shorter to discharge readiness Rapidly-resolving TEAEs GH001 2h 3h 4h 5h 6h Median Duration of the Psychoactive Experience of 11 minutes (DB & OL
Slide 15: Potential for GH Mebufotenin in TRD
Potential for GH Mebufotenin in TRD Best in Molecule Fast in Fast out (FIFO) formulations shown to be better than slower release formulations (intranasal) 15,5 vs 5 pbo corrected No psychotherapy needed Minimal Aes , minimal psychoactive phase Best in Class GH formulations shown far better efficacy
Slide 16: Summary of GH Research R&D Updates
Summary of GH Research R&D Updates 1 2 3 On Track to Commence TRD Pivotal Program in 2026 Strong and Consistent Final Data from GH Mebufotenin in TRD Best in Class, Best in Molecule, Best in Therapeutic Category GH001 & GH002 Formulations: Progress Update
Slide 17: Two 'Fast-in-Fast-Out' Mebufotenin Formulations
Two ‘Fast-in-Fast-Out’ Mebufotenin Formulations GH001 (Inhaled Mebufotenin) T1/2 =y cmax = z, psychoactive period = 10-15m Regulatory status: UK MHRA approval and cross over study completed with new device US: IND resubmitted. Received response letter clearing 3 of 4 issues. Call for more data and a
Slide 18: Summary of GH Research R&D Updates
Summary of GH Research R&D Updates 1 2 3 On Track to Commence TRD Pivotal Program in 2026 Strong and Consistent Final Data from GH Mebufotenin in TRD Best in Class, Best in Molecule, Best in Therapeutic Category GH001 & GH002 Formulations: Progress Update
Slide 19: On Track to Commence TRD Pivotal Program in 2026
On Track to Commence TRD Pivotal Program in 2026 Abbreviations: TRD = Treatment-Resistant Depression; RoW = Rest of World; CRO = Contract Research Organisation; HTA = Health Technology Assessment Body; EoP2 = End of Phase 2 Pivotal Program Design building on Ph2b Results Site selection in US, EU and
Slide 20: Summary of GH Research R&D Updates
Summary of GH Research R&D Updates 1 2 3 On Track to Commence TRD Pivotal Program in 2026 Strong and Consistent Final Data from GH Mebufotenin in TRD Best in Class, Best in Molecule, Best in Therapeutic Category GH001 & GH002 Formulations: 2 PK Equivalent Solutions 4 $315.3 million cash and investme