AtaiBeckley announces peer-reviewed Phase 2a results of BPL-003 study

AtaiBeckley (ATAI) announced peer-reviewed Phase 2a results in CNS Drugs demonstrating that a single intranasal dose of BPL-003, or mebufotenin benzoate, which holds FDA Breakthrough Therapy Designation, achieved rapid and sustained reductions in MADRS scores from baseline in participants with treatment-resistant depression, or TRD, who remained on stable SSRI therapy throughout the study. A 66.7% antidepressant response rate was observed at Day 2 in both the 10 mg and 12 mg cohorts, with 83% of participants in the 10 mg cohort and 66.7% of participants in the 12 mg cohort maintaining a response at Week 12. BPL-003 was generally well tolerated with no serious adverse events reported, and participants achieved a mean discharge approximately 100 minutes post-dose. Phase 3 studies are on track to initiate in Q2 2026 following recent FDA End-of-Phase 2, or EOP2, alignment. The 12-week, open-label, single-center, ascending-dose trial enrolled 12 adults aged 18 to 75 years with moderate-to-severe major depressive disorder and TRD. All participants had failed at least two prior antidepressants and remained on a stable dose of one of four SSRIs – citalopram, escitalopram, sertraline or fluoxetine – throughout the study. Six participants received a single intranasal 10 mg dose of BPL-003 and six received a single intranasal 12 mg dose of BPL-003, with psychological support before, during and after dosing. This publication represents Part 2 of the four-part Phase 2a open-label study. BPL-003 is a patent-protected, proprietary intranasal formulation of mebufotenin benzoate (5-MeO-DMT), administered via a nasal spray device used in a previously approved drug product. BPL-003 is designed to deliver rapid and durable effects from a single dose, with a short psychedelic duration, and is being investigated as a potential therapy for treatment-resistant depression and alcohol use disorder (AUD). BPL-003 has been granted Breakthrough Therapy designation by the FDA. Treatment-resistant depression affects an estimated 30% of the nearly 300 million people living with depression around the globe, representing one of the largest areas of unmet need in psychiatry.