atai Life Sciences and Beckley Psytech have secured a significant regulatory milestone with the FDA's Breakthrough Therapy designation for BPL-003 (mebufotenin benzoate) nasal spray in adult patients with treatment-resistant depression (TRD). This designation is reserved for therapies targeting serious or life-threatening conditions where preliminary clinical evidence suggests substantial improvement over existing treatments.
Clinical Trial Results Drive FDA Recognition
The breakthrough designation follows positive topline results from Beckley Psytech's Phase 2b core, blinded study in patients with TRD. In the trial, single administrations of 8 mg or 12 mg of BPL-003 produced clinically meaningful and statistically significant reductions in depressive symptoms within 24 hours, with effects maintained throughout the eight-week study period.
The study also demonstrated practical advantages for clinical implementation, with most patients considered ready for discharge 90 minutes after dosing. This finding highlights BPL-003's potential to integrate into a two-hour, in-clinic interventional psychiatry model, according to atai.
Accelerated Development Pathway
The FDA's Breakthrough Therapy designation enables enhanced collaboration with the agency and may help accelerate development timelines and regulatory review processes. BPL-003 is designed to provide rapid and durable antidepressant effects following a single dose, with a short in-clinic therapeutic experience.
"We are now among a select group of mental health companies with clinical programs that have been recognized by the FDA with Breakthrough Therapy designations," said atai CEO Dr. Srinivas Rao. "BPL-003 is well-positioned for Phase 3 trials which are expected to initiate in the second quarter of 2026, subject to alignment with the FDA. This is great news for patients."
Strategic Partnership and Market Position
Cosmo Feilding Mellen, Beckley CEO, emphasized the designation's significance for addressing unmet medical needs. "We believe the Breakthrough Therapy designation supports the strength of our clinical data and, importantly, it will help to ensure the pivotal Phase 3 clinical program will be as expedited and efficient as possible, guided by the FDA," Mellen said.
The two companies have previously announced a planned strategic combination aimed at creating a global leader in transformative mental health therapies. This partnership positions the combined entity to advance BPL-003 through late-stage development with enhanced regulatory support.
Funding Development
Separately, atai announced the commencement of a proposed registered underwritten offering of its common shares. The company intends to use the net proceeds, together with existing cash and short-term investments, to advance clinical development of its product candidates, as well as for working capital and general corporate purposes. Jefferies is acting as lead bookrunner, with Berenberg Capital Markets as passive bookrunner, and Canaccord Genuity and Oppenheimer as co-managers for the offering.
