Gh Research Plc · NASDAQ:GHRS
Gh Research — Investor Presentation (January 2023)
30 slides from Gh Research's investor presentation, Jan 9, 2023
Slide 1: Corporate Presentation
1 Corporate Presentation GH Research PLC (NASDAQ: GHRS) GH Research January 2023 2023© GH Research PLC
Slide 2: Disclaimer Regarding Forward-Looking Statements
Disclaimer Regarding Forward - Looking Statements This presentation has been prepared by GH Research PLC (“GH Research”) for informational purposes only and not for any other purpose. Nothing contained in this presentation is, or should be construed as, a recommendation, promise or representation by
Slide 3: Seeking Ultra-Rapid, Durable Remissions in Depression
Seeking Ultra - Rapid, Durable Remissions in Depression 3 2023© GH Research PLC
Slide 4: Stage of Development
Stage of Development PROGRAMS INDICATION PRECLINICAL PHASE 1 PHASE 2a PHASE 2b PHASE 3 CURRENT STATUS GH001 Mebufotenin (5 - MeO - DMT) for inhalation administration Treatment - Resistant Depression (TRD) Phase 2b CTAs submitted (GH001 - TRD - 201) Bipolar II Disorder * (BDII) Phase 2a POC trial ini
Slide 5: The Problem for Patients with Depression
0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 0 2 4 6 8 10 12 14 Cumulative Remission Rate (% Patients) Week ... Remission Rates in TRD < 15% Established Therapies are Slow - Acting 37% 31% 14% 13% 56% 62% 67% 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 1 2
Slide 6: Large and Open Depression Market in the EU and US
Large and Open Depression Market in the EU and US 6 First Line MDD Second Line MDD Treatment - Resistant Depression (TRD) Patients cycle through ineffective therapies for TRD • Diagnosed: ~ 48M • Treated (pharmacotherapy ± psychotherapy): ~ 24M • Non - response to first line: ~ 13M • Non - response
Slide 7: Mebufotenin (5-MeO-DMT) and GH001
• Mebufotenin ( 5 - Methoxy - N,N - Dimethyltryptamine, 5 - MeO - DMT) • Naturally - occurring psychoactive substance from tryptamine class • Highly potent agonist on 5 - HT1A and 5 - HT2A receptors • High propensity to induce peak experiences (PE), which may be a surrogate marker for therapeutic ef
Slide 8: GH001 Individualized Dosing Regimen (IDR)
GH001 Single Dose: Inter - Person Variability GH001 Individualized Dosing Regimen (IDR): Maximization of Ultra - Rapid and Durable Remissions MADRS score MADRS score MADRS score MADRS score Dose 1 Dose 1 Dose 2 Dose 3 Dose 2 Dose 1 No remission Remission Remission Remission Remission Hypothetical Pa
Slide 9: Phase 1 Trial in Healthy Volunteers GH001-HV-101 (Completed)
Phase 1 Trial in Healthy Volunteers GH001 - HV - 101 (Completed) 9 Clinicaltrials.gov ID: NCT04640831 2023© GH Research PLC
Slide 10: Design of Phase 1 Trial in Healthy Volunteers
GH001 Administration Day 1 Day 7 GH001 2 mg (n=4) GH001 6 mg (n=6) GH001 12 mg (n=4) GH001 18 mg (n=4) HV (n=18) Part A (Single Dose) Part B (IDR) Primary Endpoint: • Safety until day 7 • Peak Experience Scale (PE Scale) 1 HV (n=4) Primary Endpoint: • Safety until day 7 • Peak Experience Scale (PE S
Slide 11: Part A and Part B – Safety
Study Safety Group review • No SAEs • All ADRs mild, except two moderate (*) • All ADRs resolved spontaneously • Inhalation well - tolerated • No noteworthy changes in vital parameters, except for temporary, non - clinically relevant increase in heart rate and blood pressure shortly after administra
Slide 12: Part A – Peak Experience Dose-Effects and Inter-Person Variability
0 10 20 30 40 50 60 70 80 90 100 P4 P1 P2 P3 P9 P8 P10 P7 P5 P6 P13 P11 P14 P12 P15 P18 P17 P16 2 mg 6 mg 12 mg 18 mg Average for dose group PE Scale PE Threshold Part A – Peak Experience (PE) Dose - Effects and Inter - Person Variability 12 PE, Peak E xperience 2023© GH Research PLC
Slide 13: Part B – Effect of Intraday Individualized Dosing Regimen
0 10 20 30 40 50 60 70 80 90 100 6 mg 6 mg 12 mg 6 mg 12 mg 6 mg 12 mg 18 mg P21 P19 P22 P20 PE Scale PE Threshold Part B – Peak Experience (PE) Effect of Intraday Individualized Dosing Regimen (IDR) 13 PE, Peak Experience 2023© GH Research PLC
Slide 14: Phase 1/2 Trial in Treatment-Resistant Depression GH001-TRD-102 (Completed)
Phase 1/2 Trial in Treatment - Resistant Depression GH001 - TRD - 102 (Completed) 14 Clinicaltrials.gov ID: NCT04698603 2023© GH Research PLC
Slide 15: Design of Phase 1/2 Trial in TRD
Key Assessments MADRS H2 PE Scale Safety MADRS D1 Cognitive function Safety MADRS D7 Cognitive function Safety GH001 Administration Day 1 Day 7 Design of Phase 1/2 Trial in TRD (GH001 - TRD - 102) 15 1 D efined as inadequate response to at least two adequate courses of pharmacological therapy or one
Slide 16: Phase 1 and Phase 2 – Safety
Phase 1 (Single Dose) and Phase 2 (IDR) – Safety 16 2023© GH Research PLC Study Safety Group review • No SAEs • All ADRs mild, except three moderate* • All ADRs resolved spontaneously • Inhalation well - tolerated • No noteworthy changes in vital parameters, except for temporary, non - clinically re
Slide 17: Phase 1 (Single Dose) – Efficacy (MADRS)
50% 25% 50% 50% 100% 100% 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% 12 mg (n=4) 18 mg (n=4) MADRS remission MADRS response MADRS any improvement MADRS Remission / Response / Improvement Rate Day 7 Phase 1 (Single Dose) – Efficacy (MADRS) 17 PE, Peak Experience; MADRS, Montgomery – Åsberg Depressio
Slide 18: Phase 2 (IDR) – Efficacy (MADRS)
87.5% 87.5% 100% 0% 10% 20% 30% 40% 50% 60% 70% 80% 90% 100% Individualized Dosing Regimen MADRS remission MADRS response MADRS any improvement Phase 2 (IDR) – Efficacy (MADRS) 18 2023© GH Research PLC MADRS Remission / Response / Improvement Rate Day 7 • Primary endpoint met: 7 of 8 patients (87.5%
Slide 19: Phase 2 (IDR) – Efficacy (MADRS Change from Baseline)
- 19.3 - 27.1 - 24.4 -30 -25 -20 -15 -10 -5 0 MADRS Mean Change From Baseline Mean (n=8) Phase 2 (IDR) – Efficacy (MADRS Change from Baseline) 2023© GH Research PLC GH001 19 p=0.0018 p<0.0001 p<0.0001 • Primary endpoint met: 7 of 8 patients (87.5%) had a MADRS remission at day 7, p<0.0001 • 7 of 7 r
Slide 20: MADRS and PE – Observed Improved Outcome in Phase 2 vs Phase 1
MADRS and PE – Observed Improved Outcome in Phase 2 (IDR) vs Phase 1 (Single Dose) Phase 2 (IDR) Phase 1 (Single Dose) 12 mg Phase 1 (Single Dose) 18 mg MADRS Remission Rate Day 7 87.5% (7 of 8) 50% (2 of 4) 25% (1 of 4) Mean MADRS Change Day 7 - 24.4 ( - 76%) - 21.0 ( - 65%) - 12.5 ( - 40%) Rate of
Slide 21: Phase 1 Clinical Pharmacology Trial in Healthy Volunteers GH001-HV-103 (Completed)
Phase 1 Clinical Pharmacology Trial in Healthy Volunteers GH001 - HV - 103 (Completed) 21 2023© GH Research PLC Clinicaltrials.gov ID: NCT05163691
Slide 22: Design of Phase 1 Clinical Pharmacology Trial in Healthy Volunteers
GH001 Administration Day 7 GH001 6 mg (n=8+2 placebo) GH001 12 mg (n=8+2 placebo) GH001 18 mg (n=8+2 placebo) HV (n=30) Single - Dose Part IDR Part HV (n=16) GH001 IDR 6, 12, 18 mg to achieve PE (up to 3 doses, 1h interval , n=8) Key Assessments Safety Pharmacokinetics PE Scale Cognitive function Sa
Slide 23: Single Dose and IDR – Safety and Further Results
Safety Review • No SAEs • All ADRs mild • All ADRs resolved spontaneously • Inhalation well - tolerated • No noteworthy changes in vital parameters, except for temporary, non - clinically relevant increase in heart rate and blood pressure shortly after administration of GH001 • No clinically relevan
Slide 24: Phase 2b Trial in Treatment-Resistant Depression GH001-TRD-201
Phase 2b Trial in Treatment - Resistant Depression GH001 - TRD - 201 (Initiation Expected Q1 2023) 24 2023© GH Research PLC EudraCT Number : 2022 - 000574 - 26
Slide 25: Design of Phase 2b Trial in TRD
Design of Phase 2b Trial in TRD (GH001 - TRD - 201) 2023© GH Research PLC 25 GH001 IDR Placebo IDR n=80 Randomization 1:1 Up to 5 GH001 IDR s may be administered during the OLE pro re nata (PRN), based on specific re - treatment criteria D0 MADRS Primary Endpoint ΔMADRS (per FDA guidance for rapid -
Slide 26: Three-Layer Protection Strategy
LAYER 1: REGULATORY EXCLUSIVITY FDA: 5 years (+2.5 years paragraph IV stay) EMA: 10 years (+1 year for new indication) LAYER 3: TECHNICAL Complex bioequivalence for systemically - acting inhalation/intranasal products with high intra - and inter - subject variability LAYER 2: PATENTS 11 patent famil
Slide 27: Board of Directors & Management
Board of Directors & Management 27 Florian Schönharting Michael Forer MSc Chairman of the Board, Co - founder BA, LLB Vice - Chairman of the Board 2023© GH Research PLC Dermot Hanley Duncan Moore BSC, MBA Board Member MPhil, PhD Board Member Theis Terwey PD Dr. med. CEO, Co - founder Julie Ryan ACA,
Slide 28: Scientific Advisors
Scientific Advisors 28 Michael Thase M.D. Professor of Psychiatry, Perelman School of Medicine University of Pennsylvania Madhukar Trivedi M.D. Professor of Psychiatry, UT Southwestern Medical Center Mark Zimmerman M.D. Professor of Psychiatry and Human Behavior, Brown University Eduard Vieta Prof.
Slide 29: Anticipated Milestones and Financial Overview
Anticipated Milestones and Financial Overview • GH001 • Initiate multi - center, randomized, double - blind, placebo - controlled Phase 2b trial in TRD in Q1 2023 • Submit U.S. IND for GH001 with proprietary aerosol delivery device in Q3 2023 • Complete proof - of - concept Phase 2a trials in BDII a
Slide 30: Seeking Ultra-Rapid, Durable Remissions in Depression
Seeking Ultra - Rapid, Durable Remissions in Depression 30 2023© GH Research PLC