In a Phase IIb trial, GH001 elicited significant drops in treatment-resistant depression. The news comes less than two weeks after J&J secured FDA monotherapy approval for its esketamine nasal spray Spravato in the same indication.
GH Research’s lead molecule, an inhaled 5-MeO-DMT psychedelic named GH001, met its primary endpoint in a mid-stage clinical trial for treatment-resistant depression (TRD), the Dublin-based biotech announced Monday.
In a Phase IIb trial of 81 participants, 40 patients in the treatment arm saw a -15.2 drop on the Montgomery-Åsberg Depression Rating Scale (MADRS), compared to a +0.3 gain for the 40 patients in the placebo group after eight days.
While the primary endpoint was measured response by day eight, the company saw what it called “ultra-rapid responses.” At two hours, GH001achieved a -17.8 reduction in MADRS, and a -18.6 reduction after two days.
In the open-label extension portion of the trial, GH001 led to a 77.8% remission rate at six months in patients who completed this section.
The company reported no serious adverse events during the trial. Side effects included nausea, salivary hypersecretion, paresthesia, headache and distortions in sense of taste. There were no treatment-related events of suicidal ideation or reports of flashbacks. Most patients were ready to be discharged one hour after treatment.
“Overall, the data look remarkably consistent and are essentially a best-case scenario for [GH Research], offering another dataset that supports the promise of psychedelic drugs in treating depression,” Stifel analysts wrote in an investor note Tuesday morning.
Michael Thase, a professor of psychiatry at the University of Pennsylvania and scientific advisor to the company, said in a statement that GH001 “has the potential to be a practice-changing treatment,” citing a “large and rapid effect, particularly one that may require only infrequent, short 1-3 hours clinic visits.”
On a conference call Monday, GH CEO Velichka Valcheva compared the results to those generated by Johnson & Johnson’s Spravato, an inhaled esketamine treatment for TRD that recently won FDA approval as a monotherapy. When given in combination with antidepressants, Spravato elicited a drop of about -4 points in MADRS, Valcheva noted, additionally pointing to a superior remission rate for GH001.
GH’s results are just the latest to be released in a psychedelics space that has been active so far this year. Last week, atai Life Sciences reported positive mid-stage data for its molecule BPL-003, a DMT-based psychedelic, in alcohol-use disorders. Atai is also testing BPL-003 in patients with TRD in a Phase IIb trial. In a Tuesday investor note, Jefferies analysts said GH’s data could derisk atai’s eventual data readout for that indication, expected mid-2025.
