AtaiBeckley (ATAI) Study Result summary

Study design and population

• Phase II-B/2b trial of BPL-003 in treatment-resistant depression (TRD) was conducted globally across six countries, with 193 participants randomized to 0.3 mg, 8 mg, or 12 mg arms and a core 8-week randomized, quadruple-masked study.

• Eligible patients had moderate to severe TRD, HAM-D score ≥19, and were washed out of prior medications before receiving monotherapy.

• After the double-blind period, 107 participants entered an open-label extension (OLE) for a second 12 mg dose and eight-week follow-up.

Efficacy results

• Both 8 mg and 12 mg doses produced rapid, statistically significant, and durable reductions in MADRS scores compared to 0.3 mg, sustained through 8 weeks.

• In the OLE, a second 12 mg dose led to further improvement, with responder rates up to 81% and remission rates up to 67% for the 8 mg group at Day 57.

• Mean MADRS reduction at Day 57 in the OLE: 14.0 points (0.3 mg), 22.3 points (8 mg), and 19.0 points (pooled active dose group).

• The antidepressant effect was durable, with benefits observed for at least four months after two doses.

• Progressive improvement in remission was noted post-redose, possibly due to increased social connectedness and virtuous cycles in patient well-being.

Safety and tolerability

• BPL-003 was generally well tolerated; over 99% of adverse events were mild or moderate and transient, mostly occurring on the day of dosing.

• The most common side effects were nasal discomfort, headache, nausea, administration site pain/discomfort, blood pressure increases, and anxiety.

• No drug-related serious adverse events occurred in the core study; one serious event in the extension involved a patient with pre-existing suicidal ideation, which resolved with monitoring.

• The 8 mg dose showed a more favorable safety profile than 12 mg, with fewer patients requiring extended observation post-dose.

• Most patients were ready for discharge within 2 hours post-dose.