Promising Early Outcomes: Beckley Psytech's BPL-003 for Treatment-Resistant Depression

On March 27, 2024, atai Life Sciences, a clinical-stage biopharmaceutical company focusing on mental health disorders, announced promising early results from Beckley Psytech’s Phase 2a open label study of BPL-003 for patients with Treatment Resistant Depression (TRD). TRD affects nearly 100 million individuals globally.

BPL-003 is a novel, synthetically formulated benzoate salt of 5-MeO-DMT, which is administered intranasally. Initial findings revealed that a single 10mg dose was well-tolerated, displaying a rapid onset and sustained antidepressant effect in TRD patients.

The Phase 2a open-label study evaluated the safety, tolerability, and efficacy of a single 10mg dose of BPL-003 in conjunction with psychological support for patients with moderate-to-severe TRD who were not concurrently on antidepressants. Twelve subjects were enrolled, and eleven were included in the per-protocol analysis. The study followed patients for 12 weeks post-dosing, with regular assessments, utilizing the Montgomery-Åsberg Depression Rating Scale (MADRS) to measure efficacy.

The initial analysis indicated that 55% of patients experienced a rapid antidepressant response within a day of dosing. This response rate was maintained at 55% at week four and continued through week twelve. Remission rates were 55% at week four and 45% at week twelve. These results mark the longest known follow-up period in a clinical trial involving 5-MeO-DMT for depression.

BPL-003 was generally well tolerated, with most adverse events (AEs) being mild or moderate. The most common AEs (occurring in more than 10% of patients) included nasal discomfort, headaches, nausea, and vomiting, consistent with Phase 1 findings. No serious AEs were reported. The acute effects generally resolved within two hours on average, suggesting a shorter in-clinic treatment duration compared to other psychedelic treatments being developed.

Florian Brand, CEO and Co-Founder of atai Life Sciences, expressed enthusiasm about the progress of the BPL-003 program, emphasizing the positive data from the Phase 2a study. With around half of the TRD patients showing remission three months post a single dose, the study underpins the drug's potential for durable antidepressant effects. Brand highlighted that BPL-003 could provide a scalable, single-dose treatment option that fits within a two-hour clinical visit paradigm.

The study extends into a Part 2 phase, now enrolling TRD patients on stable oral antidepressants to assess the safety and efficacy of BPL-003 co-administration. Additionally, a randomized, quadruple-masked, controlled Phase 2b study of BPL-003 is underway. This study involves 225 TRD patients, comparing single doses of 12mg or 8mg against a 0.3mg sub-perceptual dose, with efficacy assessed at various time points using MADRS. The primary endpoint is at week four, with final results expected in the second half of 2024.