The FDA has supported the Phase 3 design for BPL-003, an intranasal psychedelic-derived therapy for treatment-resistant depression
AtaiBeckley NV (NASDAQ:ATAI, XETRA:9VC), a clinical-stage biotechnology company focused on mental health treatments, has received backing from US regulators to advance its BPL-003 drug candidate into Phase 3 clinical trials for treatment-resistant depression, a condition affecting millions of patients for whom standard antidepressants have failed.
The company said a formal end-of-phase 2 meeting with the Food and Drug Administration (FDA) concluded successfully, with the regulator indicating support for its proposed pivotal trial programme and providing constructive feedback on study design, safety database requirements and other key components.
BPL-003 is an intranasal formulation of mebufotenin benzoate, a compound derived from psychedelic research, administered via a nasal spray and designed to deliver rapid and durable antidepressant effects from a single dose with a short psychedelic duration of around two hours in a clinical setting.
The drug was granted Breakthrough Therapy designation by the FDA in October 2025, a status awarded to treatments that show early evidence of substantial improvement over existing therapies for serious conditions.
The Phase 3 programme, which AtaiBeckley said remains on track to begin in the second quarter of 2026, will comprise two parallel pivotal studies named ReConnection 1 and ReConnection 2.
ReConnection 1 will enrol approximately 350 participants and test single doses of BPL-003 at 8mg and 4mg against placebo, aiming to replicate Phase 2b results and further characterise the dose-response relationship.
ReConnection 2 will enrol around 300 participants and investigate a two-dose induction model, with doses administered on day one and day 15, to assess whether repeat dosing increases the magnitude and durability of the antidepressant effect.
Both studies will be followed by a 52-week open-label extension phase in which patients may receive repeat treatments at eight or twelve-week intervals.
Srinivas Rao, co-founder and chief executive, said the FDA's guidance firmly positioned the company to advance a programme designed to maximise the probability of both regulatory approval and commercial success.
AtaiBeckley will host a virtual investor day on 6 March to provide further detail on the Phase 3 strategy.