Slide 1: A Phase 1/2 Trial of GH001, a Vaporized 5-MeO-DMT Formulation, in Patients with TRD
A Phase 1/2 Trial of GH001, a Vaporized 5-Methoxy-N,N-Dimethyltryptamine Formulation, inPatients with Treatment-Resistant Depression (TRD) 1 Clinicaltrials.gov ID NCT04698603 GH001-TRD-102 ICPR 2022 Johannes Reckweg1, Cees van Leeuwen1, Cécile Henquet2, Thérèse van Amelsvoort2, Natasha Mason1, Ricca
Slide 2: Disclosures
Grants The study was funded by GH Research Advisory Board/Consultant Johannes Reckweg and Jan Ramaekers work as consultants for GH Research 2 GH001-TRD-102 ICPR 2022 Disclosures
Slide 3: The Problem for Patients with Depression
... Remission Rates in TRD < 15% Established Therapies are Slow-Acting ~33% no remission despite 4 treatment steps Adapted from Trivedi et al., Am J Psychiatry 2006 and Rush et al., Am J Psychiatry 2006 Average time to remission is ~6 weeks (STAR*D study, Remission Rate Over Time, Treatment Step 1 =
Slide 4: 5-MeO-DMT and GH001
5-MeO-DMT (5-Methoxy-N,N-Dimethyltryptamine) Naturally-occurring psychoactive substance from tryptamine class Highly potent agonist on 5-HT1A and 5-HT2A receptors Psychoactive effects with ultra-rapid onset (within seconds) and short duration (5 to 30 min) GH001 (5-MeO-DMT administration via a propr
Slide 5: 5-MeO-DMT and Peak Experiences
5-MeO-DMT and Peak Experiences High propensity to induce peak experiences (PE) Feelings of ego dissolution Experience of unity or oneness Profound and meaningful May be a surrogate marker for therapeutic effects Assessed through proprietary Peak Experience Scale Three visual analogue scales (0 – 100
Slide 6: Phase 1 Trial in Healthy Volunteers (GH001-HV-101, n=22)
Phase 1 Trial in Healthy Volunteers (GH001-HV-101, n=22) GH001 single doses of 2 mg, 6 mg, 12 mg, 18 mg and GH001 IDR (6, 12, 18 mg intra-subject dose escalation) No SAEs, all ADRs mild (except two moderate), all ADRs resolved spontaneously, inhalation well-tolerated GH001 single dose with psychoact
Slide 7: GH001 Individualized Dosing Regimen (IDR): More Chances for Remission
GH001 Single Dose: GH001 Individualized Dosing Regimen (IDR): More Chances for Remission 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 Patient 1 Hypothetical Patient 2 Hypothetical Patient 3
Slide 8: Phase 1/2 Trial in TRD (GH001-TRD-102, n=16)
Key Assessments MADRS 2-hrs PE Scale Safety MADRS 1-day Cognitive function Safety MADRS 7-day Cognitive function Safety GH001 Administration Day 1 Day 7 Phase 1/2 Trial in TRD (GH001-TRD-102, n=16) 8 PE, Peak Experience; MADRS, Montgomery-Åsberg Depression Rating Scale;IDR, Individualized Dosing Reg
Slide 9: Phase 1 (Single Dose) – Efficacy (MADRS)
Phase 1 (Single Dose) – Efficacy (MADRS) 9 2 of 4 patients in the 12 mg group and 1 of 4 patients in the 18 mg group had a MADRS remission at day 7 2 of 4 patients in the 12 mg group had a PE and both had a MADRS remission at day 7, 0 of 4 patients in the 18 mg group had a PE PE, Peak Experience; MA
Slide 10: Phase 2 (IDR) – Efficacy (MADRS)
Phase 2 (IDR) – Efficacy (MADRS) 10 PE, Peak Experience; MADRS, Montgomery-Åsberg Depression Rating Scale; MADRS remission, MADRS of ≤10; MADRS response, Reduction of ≥50% from baseline in MADRS; S, Screening; D0-B, Day 0 Baseline; D0-H2, Day 0 2 hours. Primary endpoint met: 7 of 8 patients (87.5%)
Slide 11: Phase 1 (Single Dose) and Phase 2 (IDR) – Safety
Phase 1 (Single Dose) and Phase 2 (IDR) – Safety 11 Adverse Drug Reaction, or ADR, an adverse event with a relationship code to the investigational product of definite, probable, or possible, or where code is missingIDR, Individualized Dosing Regimen; C-SSRS, Columbia-Suicide Severity Rating Scale A
Slide 12: Conclusions
12 GH001 allows rapid and individualized dosing optimization A single dosing day with GH001 IDR achieved a rapid (within 24 hours) and sustained full remission (7 days) of symptoms of depression in 7/8 patients (87.5%) with TRD GH001 was well tolerated, and no serious adverse events were reported Co