AtaiBeckley (ATAI) Study result summary

Study background and objectives

• EMP-01, an oral R-MDMA candidate, was evaluated in its first controlled clinical trial for social anxiety disorder (SAD), a prevalent and chronic psychiatric condition with significant unmet treatment needs, affecting approximately 32 million adults in the US.

• The study aimed to assess safety, tolerability, feasibility, and initial efficacy of two in-clinic doses in a real-world SAD population, without psychotherapy.

• 71 participants were randomized, 70 dosed, and 69 completed the primary endpoint at day 43, with high retention and adherence across seven UK sites.

• The trial enrolled a highly severe, treatment-resistant population, with a mean baseline LSAS score of 108, higher than historical SAD trials.

• Central raters were used for all clinical measures to ensure rigor and consistency.

Study design and patient characteristics

• Exploratory, double-blind, randomized, placebo-controlled Phase 2a study with two doses (225 mg EMP-01 or placebo) given on day 1 and day 29, with no adjunctive psychotherapy.

• Primary focus was on safety and feasibility; efficacy endpoints included LSAS and CGI-I scores.

• Demographics and baseline characteristics were well balanced between groups, including age, sex, and prior medication use.

• The patient group was characterized by severe SAD and high disease burden.

Safety and tolerability

• EMP-01 demonstrated a favorable and manageable safety profile, with only mild or moderate adverse events and no severe or serious adverse events or treatment-emergent suicidal behavior or intent.

• Three participants discontinued due to mild/moderate adverse events, mainly related to predefined vital sign criteria or pre-existing suicidal ideation.

• No suicidal intent or behavior was reported during the study; challenging experiences were minimal.

• Common adverse events included nausea, headache, fatigue, dizziness, decreased appetite, and typical psychedelic effects such as sensory disturbance and euphoria.

• Subjective effects were robustly psychedelic but resolved within six hours on average.