With our lead product candidate, GH001 an inhalation formulation of synthetic mebufotenin (5-methoxy-N,N-dimethyltryptamine, 5-MeO-DMT), we have completed two Phase 1 clinical trials in healthy volunteers (GH001-HV-101 and GH001-HV-103) and a Phase 1/2 clinical trial in patients with treatment-resistant depression (TRD) (GH001-TRD-102).
In the latter trial, remission of depressive symptoms (as defined by a Montgomery–Åsberg Depression Rating Scale (MADRS) of ≤10) at Day 8 of the trial was achieved in 25% to 50% when using a single dose of GH001 and in 87.5% of patients when using an individualized dosing regimen (IDR) of GH001, with an ultra-rapid onset of anti-depressant effects.
In the three completed phase 1 clinical trials of GH001 and one completed clinical trial of GH002, where GH001 and GH002 were administered to a total of 78 and to 64 participants respectively, as a single dose and using the IDR, both product candidates were well tolerated, and no severe or serious adverse events occurred.
For more recent clinical trial results, see our latest News Release.
With our ongoing and planned clinical trials, we are seeking to further advance our mission of improving the treatment of patients with difficult-to-treat depression.
Completed Clinical Trials
Healthy Volunteer Trials
GH001-HV-101
Aim of the Trial
The aim of this trial was to investigate the safety, tolerability, and psychoactive effects of GH001 when it was inhaled as single or multiple doses by healthy volunteers.
Trial Design
This trial was a Phase 1, open-label, non-randomized trial with 22 healthy volunteers, aged between 18 and 45 years. The trial was designed in two parts:
- A single-dose, single-arm part where trial participants inhaled one of four possible doses of GH001 (2 mg, 6, mg, 12 mg, and 18 mg).
- A multiple dose, single-arm part where trial participants were administered the IDR, involving up to three doses of GH001 of increasing strength (6 mg as first dose, 12 mg as second dose, 18 mg as third dose) by inhalation in a single day.
Key Results
- Inhaled GH001 produced a well-tolerated psychedelic experience when administered as single or multiple doses in healthy volunteers.
- There were no serious adverse events.
- The IDR was more effective in inducing an intense psychedelic experience, or “peak experience” than a single dose of GH001.
To learn more about this trial, you can read the publication here.
GH001-HV-103
Aim of the Trial
The aim of this trial was to study the pharmacokinetics (what the body does to a drug) and pharmacodynamics (what a drug does to the body) of single or multiple doses of inhaled GH001 in healthy volunteers.
Trial Design
This trial was a Phase 1 trial in 46 healthy volunteers, aged between 18 and 45 years. The trial was designed in two parts:
- A single dose part, which was a double-blind, placebo-controlled, randomized, parallel-group design where trial participants inhaled one of three possible doses of GH001 (6 mg, 12 mg, or 18mg) or placebo.
- A multiple dose part which was an open-label, single-arm design where trial participants were administered the IDR of up to three doses of GH001 of increasing strength (6 mg as first dose, 12 mg as second dose, and 18 mg as third dose) by inhalation in a single day.
Key Results
- Inhaled GH001 produced a well-tolerated psychedelic experience when administered as single or multiple doses in healthy volunteers.
- There were no serious adverse events.
- Pharmacokinetic analyses and psychoactive effect assessments support that an interval down to 1 hour between individual doses of the IDR is feasible for future clinical trials.
Further trial results can be found in our corporate presentation, linked here.
GH002-HV-105
Aim of the Trial
The aim of this trial was to determine the safety, tolerability, and pharmacokinetics of intravenous GH002 following single and multiple doses.
Trial Design
A Phase 1 trial in healthy volunteers aged between 18-45 years, designed in two parts:
- A single dose part that was a double-blind, placebo-controlled, randomized, parallel-group design with single doses of GH002 administered intravenously, or placebo.
- A multiple dose part that was an open-label, non-randomized design where the IDR was applied with up to three increasing doses of GH002 administered intravenously in a single day.
This trial included a total of 64 participants and was conducted at a single site in the Netherlands.
Key Results
- GH002 was found to be well-tolerated with no severe or serious adverse events.
- GH002 exhibited potent pharmacodynamic effects, as assessed by psychoactive effect intensity, with an ultra-rapid onset and a short duration of the psychoactive experience.
- The pharmacokinetic profile of GH002 correlated with the ultra-rapid profile of the psychoactive effects.
Further trial results can be found in our corporate presentation, linked here.
Trials in Patients with Treatment-Resistant Depression
GH001-TRD-102
Aim of the Trial
The aim of this trial was to investigate the safety and potential antidepressant effects of GH001 in patients with TRD.
Trial Design
This trial was a Phase 1/2 trial of GH001 in 16 patients with TRD, aged between 18-64 years, designed in two parts:
- A single dose, Phase 1 part that was an open-label, single-arm trial evaluating two doses (12mg, 18mg) of GH001 administered by inhalation.
- A multiple dose, Phase 2 part that was an open-label, single-arm trial where trial participants were administered the IDR of up to three increasing doses of GH001 (6 mg as first dose, 12 mg as second dose, 18 mg as third dose) by inhalation in a single day.
Key Results
- Inhaled GH001 was well-tolerated in patients with TRD when administered as single or multiple doses.
- There were no serious adverse events.
- In the Phase 2 part of the trial, 25% to 50% of patients in the single dose group and 87.5% of patients in the IDR group experienced a remission in their depressive symptoms by Day 7.
- In the IDR group, remission was achieved by Day 1 in all patients.
To learn more about this trial, you can read the publication here
Trials in Patients with Bipolar II Disorder
GH001-BD-202
Aim of the Trial
The aim of this trial is to determine the effect of a single day IDR of GH001 on depressive symptoms in patients with bipolar II disorder (BDII) and a current major episode of depression.
Trial Design
A Phase 2, open-label, single-arm trial of GH001 in patients with BDII and a current major depressive episode. This trial enrolled 6 patients aged between 18-64 years.
Key Results
- The primary endpoint was met with a significant reduction from baseline of -16.8 points (51.9%) in MADRS total score on Day 8 after administration of GH001 (p<0.0099).
- On Day 8, 33.3% of patients were in remission (MADRS ≤ 10).
- GH001 led to an ultra-rapid antidepressant effect with a significant reduction in MADRS score at 2 hours after administration of -16.3 points (p=0.0006) and on Day 2 of -13.3 points (p=0.0299).
- GH001 was well tolerated and no treatment-related serious adverse events were reported. The majority of treatment-emergent adverse events (TEAEs) were mild or moderate and there were no reported TEAEs of hypomania or mania.
For more information on this trial please click here.
Trials in Patients with Postpartum Depression
GH001-PPD-203
Aim of the Trial
The aim of this trial is to determine the effect of a single day IDR of GH001 on depressive symptoms in patients with postpartum depression (PPD).
Trial Design
A Phase 2, open-label, single-arm trial of GH001 in female patients with PPD. This trial enrolled 10 patients aged between 18-45 years.
Key Results
- The primary endpoint was met with a significant reduction from baseline of -35.4 points (96.3%) in MADRS total score on Day 8 after administration of GH001 (p<0.0001).
- On Day 8, 100% of patients were in remission (MADRS ≤ 10).
- GH001 led to an ultra-rapid antidepressant effect with a significant reduction in MADRS score at 2 hours after administration of -31.4 points (p<0.0001) and on Day 2 of -36.0 points (p<0.0001).
- GH001 was well tolerated and no treatment-related serious adverse events were reported. All treatment-emergent adverse events (TEAEs) were mild or moderate.
For more information on this trial please click here.
Ongoing Clinical Trials
Trials in Patients with TRD
GH001-TRD-201
Aim of the Trial
The aim of this trial is to determine the effect of a single-day IDR with GH001 compared to placebo in improving depressive symptoms in patients with TRD.
Trial Design
A Phase 2b, randomized, double-blind, placebo-controlled trial of GH001 in patients with TRD. This trial includes patients aged between 18-64 years and is being conducted at multiple centers across the EU and the United Kingdom.
For results from the double-blind part of this trial, see our latest News Release
For more information on this trial click here.