Atai Beckley announces data from Phase 2b OLE study of BPL-003

Atai Beckley (ATAI) announced topline results from the open-label extension study of its Phase 2b clinical trial of BPL-003 in patients with treatment-resistant depression. Findings show that a 12 mg dose of BPL-003 administered eight weeks after a 0.3 mg, 8 mg or 12 mg dose of BPL-003 was generally well-tolerated and provided additional rapid, clinically meaningful antidepressant effects, which were sustained for up to eight weeks. The Phase 2b clinical trial of BPL-003 was conducted in two parts: an eight-week, quadruple-masked, dose-finding core study designed to evaluate the efficacy and safety of a single 0.3 mg, 8 mg, or 12 mg dose of BPL-003 in patients with TRD, followed by an eight-week OLE study to assess the safety and efficacy of a second 12 mg dose, given eight weeks after the initial dose, regardless of the patient’s Montgomery-Asberg Depression Rating Scale score. Of the 126 patients who completed the blinded core study and were eligible to enroll, 107 continued into the extension study. Patients who initially received a 0.3 mg dose of BPL-003 in the core study of the Phase 2b trial: Mean reduction in MADRS score of 14.0 points at Day 57 in the OLE compared to their baseline at the start of the core study, in line with the antidepressant effects seen in patients who received a single active dose in the core study. Patients who initially received an 8 mg dose of BPL-003 in the core study of the Phase 2b trial: Mean reduction in MADRS score of 22.3 points at Day 57 in the OLE compared to their baseline at the start of the core study. Responder rate in the OLE was 81% at Day 57 and remission rate was 67% at the same timepoint. Pooled population of patients who received an active dose of BPL-003 in the core study of the Phase 2b trial: Mean reduction in MADRS score of 19.0 points at Day 57 in the OLE compared to their baseline at the start of the core study. Responder rate in the OLE was 63% at Day 57 and remission rate was 48% at the same timepoint. Topline safety findings: Safety and tolerability profile was largely consistent with prior studies of BPL-003 and is in line with other previously reported studies of the psychedelic class, showing BPL-003 to be generally well-tolerated. The majority of adverse events occurred on the day of dosing and were classified as mild or moderate in severity and transient in nature. Most commonly reported side effects included nausea, headache, administration site pain, administration site discomfort, blood pressure increases and anxiety. One serious drug-related adverse event was reported 8 days following administration of the second dose and was resolved with additional in-patient monitoring and support. No other drug-related serious adverse events were reported in the study. Average time to meet readiness-for-discharge criteria was within 2 hours of dosing, supporting the potential of BPL-003 to fit within the existing interventional psychiatry treatment paradigm. The topline results from the OLE study are in line with the previously announced topline results from the eight-week, blinded core study, which demonstrated that both 8 mg and 12 mg single doses of BPL-003 showed statistically significant and clinically meaningful reductions in depressive symptoms when compared to a 0.3 mg low-dose active control for up to eight weeks. Safety and efficacy data from the core and OLE studies of the Phase 2b clinical trial support the selection of the 8 mg dose to advance into Phase 3 clinical development and also support the potential for continued and increased antidepressant effects with repeat dosing. AtaiBeckley is scheduled to meet with the FDA at an End-of-Phase 2 meeting to align on clinical trial designs and other aspects of the Phase 3 development program. The Company anticipates providing guidance on the Phase 3 clinical program in the first quarter of 2026 with Phase 3 clinical trial initiation in the second quarter of 2026, pending the outcome of the FDA meeting.