EMP-01 for Social Anxiety Disorder Demonstrates Meaningful Improvements in Phase 2a Study

AtaiBeckley today announced expanded phase 2a results for oral R-MDMA (EMP-01) in adults with social anxiety disorder (SAD), which show that EMP-01 demonstrated clinically meaningful and consistent improvements across clinician-rated symptoms, patient-reported experience, and real-world behavioral outcomes.¹

“[SAD] is a chronic and highly impairing condition for which many patients do not achieve adequate benefit from currently available treatments. The consistent improvements observed across clinician-rated symptoms, patient-reported experience, and avoidance behavior—alongside a favorable tolerability profile—provide encouraging evidence that EMP-01 may address multiple important dimensions of this disorder,” said Kevin Craig, MD, the chief medical officer at AtaiBeckley.

The multi-center study enrolled 71 adult participants with moderate-to-severe SAD across 7 clinical sites in the UK. Participants were randomly assigned to receive 2 in-clinic administrations of EMP-01 (225 mg) or placebo, given 28 days apart, with no adjunctive psychotherapy; 70 participants received at least 1 dose of study drug and 69 completed the day 43 efficacy assessments. All clinician-rated assessments were conducted by blinded central raters. Topline results from this study were previously reported in February 2026.²

EMP‑01 produced consistent and clinically meaningful improvements across all major symptom domains of SAD. Notably, at day 43, EMP-01 demonstrated a clinically meaningful LS mean difference (LSMD) of −11.9‑points on the clinician-rated 24-item Liebowitz Social Anxiety Scale (LSAS) compared with placebo (g=0.45), with both total and subscale improvements. Investigators noted improvements on both fear and avoidance subscales on LSAS, indicating that patients experienced fewer social situations as distressing and were more able to engage in them. Additionally,

EMP-01 demonstrated a large, clinically meaningful patient-reported improvement of -18.3-points (38% reduction vs 15% on placebo) on the Social Phobia Inventory (SPIN), which corresponded to a large between-group standardized effect size (g=0.84), in self-reported SAD symptoms from baseline to day 43. Additional model-based analyses further supported treatment benefits at day 43, showing statistically significant improvements with a placebo-adjusted LSMD of -11.5 points (95% CI: -18.5, -4.6; P=0.002) at day 43. Participants receiving EMP-01 moved from severe baseline symptom severity to substantially lower symptom burden by day 43. These results on the 17-item SPIN are equivalent to being able to initiate conversations, attend social events, and perform at work with substantially less fear and avoidance.

EMP-01 demonstrated a large, clinically meaningful improvement of −25.9‑points (32% reduction vs 14% on placebo) in real-world behavioral avoidance at day 43 on the Subtle Avoidance Frequency Examination (SAFE). Additional model-based analyses further supported treatment benefits at day 43, showing statistically significant improvements with a placebo-adjusted LSMD of -15.6 points at day 43 (95% CI: -26.0, -5.2; p=0.004). These results suggest that participants were more willing to participate in everyday activities such as social interactions without engaging in avoidant coping behaviors.

As to clinical impression and patient perception, there were 49% responder rates on both Clinical Global Impression-Improvement (CGI-I) and Patient Global Impressions of Change (PGI-C) at day 43 compared with 15% and 12% respectively on placebo. These measures show that overall, both clinicians and participants saw a meaningful improvement in a the participants’ daily lives, symptom severity, functioning, and overall clinical presentation.

“I was particularly struck by the consistency of the findings across clinician-rated and patient-reported outcomes. The improvement on the SAFE is especially interesting because reductions in subtle avoidance behaviors are seen as an important goal of cognitive behavioral therapies and yet are seldom measured in medication trials for [SAD]. Taken together, these findings provide strong support for continued development of EMP-01,” said Murray Stein, MD, MPH, FRCPC, Distinguished Professor of Psychiatry and Public Health at the University of California San Diego (UCSD) and consultant to AtaiBeckley.

As to safety profile, EMP‑01 was generally safe and well tolerated. There were no SAEs and no severe TEAEs in any participant. The study had 97% retention, with 0% study dropouts attributed to TEAEs. TEAEs were expected and consistent with the class, transient, and predominantly mild‑to‑moderate.

“In this phase 2a trial of 70 patients, EMP-01 delivered a 49% clinician-rated and patient-reported responder rate alongside significant improvements across LSAS, SPIN, and real-world avoidance behavior. Unlike current standards of care, which require daily, chronic dosing, the durability observed in this study following just 2 administrations suggests that EMP-01 could be a differentiated treatment option that could meaningfully improve outcomes for people living with [SAD],” said Srinivas Rao, MD, PhD, the cofounder and chief executive officer at AtaiBeckley.

References

1. AtaiBeckley announces additional phase 2a results for EMP-01 (oral R-MDMA) showing large and consistent improvements in social anxiety disorder. News release. April 22, 2026. Accessed April 22, 2026. https://ir.ataibeckley.com/news-releases/news-release-details/ataibeckley-announces-additional-phase-2a-results-emp-01-oral-r

2. AtaiBeckley announces positive topline results from an exploratory phase 2a trial of EMP-01 (oral R-MDMA) in social anxiety disorder. News release. February 26, 2026. Accessed April 22, 2026. https://ir.ataibeckley.com/news-releases/news-release-details/ataibeckley-announces-positive-topline-results-exploratory-phase