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GH001

Phase 2

Treatment-resistant Depression | Small molecule | Psychiatry |GH Research PLC|Last Updated: May 31, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment81
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05800860A Trial of GH001 in Patients With Treatment-resistant DepressionPHASE2 COMPLETED 81May 24, 2023Mar 11, 2025May 31, 202511 Czechia, Germany +4
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Study Endpoints
Primary Endpoints
Mean change in MADRS from Baseline to Day 7
Baseline to Day 7

The assessment is done with the MADRS, a diagnostic questionnaire with ten items for measuring the severity of depressive episodes in patients with mood disorders. A higher MADRS score indicates more severe depression, and each item is scored from 0 to 6. The overall score ranges from 0 to 60.

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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
GH001 - Part 1EXPERIMENTALGH001 is administered via inhalation, as an IDR consisting of up to 3 increasing doses of GH001 (6 mg, 12 mg, and 18 mg), on a single day. The second and third doses are only administered if the patient did not achieve intense psychoactive effects (a peak experience \[PE\]) at the previously administered dose.
Placebo - Part 1PLACEBO_COMPARATORPlacebo is administered via inhalation, as an IDR consisting of up to 3 doses of Placebo, on a single day. The second and third doses are only administered if the patient did not achieve intense psychoactive effects (a PE) at the previously administered dose.
Open-Label Extension (OLE) - Part 2OTHERPatients can receive up to five GH001 IDRs as needed during the OLE based on the patient's clinical response.
Interventions
NameTypeDescription
GH001DRUGGH001 administered via inhalation
PlaceboDRUGPlacebo administered via inhalation
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Eligibility Criteria
Age Range18 Years — 64 Years
SexALL
Healthy VolunteersNo
Study Sites11

Main Inclusion Criteria: 1. Is in the age range between 18 and 64 years (inclusive) at the time of informed consent; 2. Meets the trial criteria for TRD as assessed by a study psychiatrist: 1. Meets the Diagnostic and Statistical Manual of Mental Disorders 5 (DSM-5) criteria for single-episode ...

Countries:CzechiaGermanyIrelandNetherlandsPolandSpain
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