Atai Life Sciences (NASDAQ: ATAI) and Beckley Psytech Limited have announced that the U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation (BTD) to BPL-003 (mebufotenin benzoate) nasal spray for adult patients with treatment-resistant depression (TRD). This designation recognizes the potential of BPL-003 to deliver substantial improvement over existing therapies for TRD, and it provides intensive FDA guidance to support the advancement of BPL-003.
The breakthrough therapy designation follows positive topline results from Beckley Psytech’s phase 2b core, blinded study of BPL-003 in TRD. The study showed that a single administration of 8 mg or 12 mg of BPL-003 led to clinically meaningful and statistically significant reductions in depressive symptoms within 24 hours, with effects sustained through the eight-week trial period. Notably, the majority of patients were deemed ready for discharge at the 90-minute post-dose assessment, underscoring the potential scalability of the BPL-003 treatment model and its potential to fit into the established two-hour, in-clinic interventional psychiatry treatment paradigm.
The CEO of Beckley Psytech, Cosmo Feilding Mellen, stated that the breakthrough therapy designation supports the strength of their clinical data and will help ensure the pivotal phase 3 clinical program will be as expedited and efficient as possible, guided by the FDA.
Srinivas Rao, M.D., Ph.D., CEO and co-founder of Atai Life Sciences, expressed that 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.
BPL-003 is Beckley Psytech’s novel, patent-protected, proprietary intranasal formulation of mebufotenin (5-meo-dmt) benzoate, administered via a nasal spray device used in a previously approved drug product. It is designed to deliver rapid and durable effects from a single dose, with a short treatment window, and is being investigated as a potential therapy for TRD and for alcohol use disorder (AUD).
Depression is a debilitating and life-changing condition affecting nearly 300 million people across the globe. Treatment-resistant depression occurs when an individual does not respond to two or more courses of antidepressants and may affect up to 50% of those living with depression. It is associated with higher rates of comorbid anxiety, sexual dysfunction, cognitive impairment, and reduced quality of life compared to non-resistant forms of depression.
Atai Life Sciences is a clinical-stage biopharmaceutical company with a pipeline of psychedelic-based therapies, including BPL-003 for TRD, VLS-01 for TRD, and EMP-01 for social anxiety disorder. All three programs are in phase 2 clinical development. Atai is also advancing a drug discovery program to identify novel, non-hallucinogenic 5-ht2ar agonists for TRD and opioid use disorder. The market has reacted to these announcements by moving the company's shares -8.05% to a price of $5.48. If you want to know more, read the company's complete 8-K report here.
