AtaiBeckley (ATAI) investor relations material

Strategic vision and clinical pipeline

• Focus on rapid-acting, durable, and convenient mental health treatments for high unmet needs such as treatment-resistant depression (TRD) and social anxiety disorder (SAD), using next-generation psychedelic-based neuroplastogens.

• Lead asset BPL-003, an intranasal dry powder formulation of mebufotenin benzoate, is advancing into phase III pivotal studies after positive phase IIb results and FDA breakthrough therapy designation.

• Additional assets include VLS-01 (buccal DMT for TRD) and EMP-01 (MDMA derivative for SAD), both showing promising early clinical data.

• Emphasis on commercial scalability, practical dosage forms, and comprehensive intellectual property protection, with U.S. patents extending exclusivity potentially into the mid-2040s.

• Positioned in the growing US depression and anxiety market, projected at a 5.02% CAGR from 2025 to 2033.

BPL-003 clinical development and results

• Phase IIb trial showed BPL-003 is fast-acting, durable, highly effective, and well-tolerated, with significant MADRS score reductions and high response/remission rates after single and repeat dosing.

• 81% response and 67% remission rates at Week 16 after two doses; 90% completed the core study, and 85% of eligible participants received a second dose in the open-label extension.

• The 8 mg dose was selected for phase III due to comparable efficacy and a better safety profile versus 12 mg.

• Efficacy will be assessed by independent, blinded raters, and no psychotherapy is required during the study, isolating pharmacological effects.

• Top-line readouts from both phase III trials are expected in early 2029, with interim phase IIa data anticipated this year and cash runway through planned readouts.

Phase III pivotal program and regulatory alignment

• Two global, randomized, double-blind, placebo-controlled phase III studies (ReConnection-1 and 2) with long-term open-label extensions will evaluate both single and two-dose induction regimens.

• Phase III designed to maximize clinical, regulatory, and commercial success, with individualized retreatment every 8–12 weeks and primary endpoint as change from baseline in MADRS total score at Week 4 for 8 mg vs. placebo.

• FDA feedback incorporated; program aims for a broad label and operational efficiency.

• Efficacy will be assessed by independent, blinded raters, and no psychotherapy is required during the study.

• The FDA and DEA process for down-scheduling is expected to follow approval, with a 90-day statutory window for DEA action.