AtaiBeckley NV (NASDAQ:ATAI, ETR:9VC) has unveiled positive topline results from the open-label extension (OLE) study of its Phase 2b clinical trial evaluating BPL-003 in patients with treatment-resistant depression (TRD).
The company said that a 12 mg dose of BPL-003 (mebufotenin benzoate nasal spray) administered eight weeks after an initial 0.3 mg, 8 mg, or 12 mg dose produced additional rapid and clinically meaningful antidepressant effects that were sustained for up to eight weeks.
The Phase 2b trial was conducted in two parts: an eight-week, quadruple-masked, dose-finding core study, followed by an eight-week open-label extension in which a 12 mg dose was administered regardless of a patient’s Montgomery-Asberg Depression Rating Scale (MADRS) score.
Of the 126 patients who completed the core study, 107 continued into the OLE study.
Patients who initially received 0.3 mg in the core study recorded a mean reduction in MADRS score of 14.0 points at Day 57 in the OLE, compared to baseline.
Those who received 8 mg in the core study showed a mean reduction of 22.3 points, with an 81% responder rate, defined as a 50% or greater improvement in MADRS score, and a 67% remission rate (MADRS ≤10) at Day 57.
In a pooled population of patients who received either 8 mg or 12 mg in the core study, the mean reduction in MADRS score was 19 points, with a 63% responder rate and a 48% remission rate at Day 57.
The safety and tolerability profile of BPL-003 in the OLE was consistent with previous studies and aligned with findings from other compounds in the psychedelic class, the company noted.
“These new data provide compelling support that redosing with BPL-003 may deliver additional and durable antidepressant effects in patients with treatment-resistant depression while maintaining a favourable safety and tolerability profile,” AtaiBeckley CEO Dr Srinivas Rao said in a statement.
“Importantly, two doses, given eight weeks apart, provided antidepressant effects lasting up to four months, further supporting the viability of an intermittent-dose treatment paradigm with a short psychedelic duration, which has the potential to fit within the existing healthcare infrastructure and could minimize the burden on patients and providers.”
AtaiBeckley said that the Phase 2b core and OLE data support advancing the 8 mg dose of BPL-003 into Phase 3 development and suggest the potential for continued and increased antidepressant effects with repeat dosing.
The company has scheduled an End-of-Phase 2 meeting with the US Food and Drug Administration (FDA) to discuss clinical trial design and other aspects of the Phase 3 development program.
Guidance on the Phase 3 program is expected in the first quarter of 2026, with trial initiation planned for the second quarter, pending FDA discussions.
“With a significant need for more effective therapies, our priority, following End-of-Phase 2 discussions with the FDA, is to advance BPL-003 into Phase 3 clinical trials with the hope of bringing this promising treatment option to patients as swiftly as possible,” Dr Rao said.
The announcement follows the FDA’s recent granting of Breakthrough Therapy designation to BPL-003 for TRD.
Shares of AtaiBeckley moved higher on the update, adding almost 8% before Monday’s opening bell in New York.