AtaiBeckley (ATAI) TD Cowen 46th Annual Health Care Conference summary

Phase III trial design and regulatory feedback

• Two pivotal Phase III trials for BPL-003 in TRD: one with single doses (4 mg, 8 mg, placebo) and one with two 8 mg doses two weeks apart.

• Four-week primary endpoint chosen to align with Phase IIb and FDA comfort; open-label extension allows redosing as early as eight weeks if needed.

• Placebo used as control arm for both trials to ensure clean analysis and robust safety database.

• FDA feedback led to use of a perceptual, putatively sub-therapeutic dose (4 mg) as comparator, not a sub-perceptual dose.

• Safety monitoring enhanced, especially for cardiovascular effects, with discharge criteria refined for clarity.

Phase IIb data insights and implications

• Phase IIb showed an 8 mg dose had better efficacy and tolerability than 12 mg, with a 6.2-point delta at four weeks versus sub-perceptual dose.

• Anxiety-related adverse events were higher in the 12 mg group (14%) but not linked to baseline characteristics.

• One case of suicidal ideation occurred in a patient with prior history, resolving after brief hospitalization.

• 85% of subjects met readiness for discharge within two hours post-dose, supporting scalability in clinical settings.

• No major changes to monitoring protocols, but some procedural streamlining and enhanced cardiovascular monitoring implemented.

EMP-01 program in social anxiety disorder

• EMP-01 (R-MDMA) showed an 11.85-point LSAS delta at six weeks, comparable to SSRIs but with faster onset.

• Both fear and avoidance behaviors improved rapidly, resembling CBT-like effects.

• Study population had higher baseline severity (LSAS 108) than historical trials.

• About half of participants had prior SSRI/SNRI exposure but discontinued due to tolerability or lack of benefit.

• Additional analyses and next development steps expected in the coming months.