AtaiBeckley’s social anxiety disorder (SAD) drug has shown both safety and early signs of efficacy in a Phase IIa trial.
In the study (NCT06693609), patients treated with EMP-01 showed a 28.53-point reduction in the Liebowitz Social Anxiety Scale (LSAS), compared to a 16.67-point reduction in the placebo cohort after 43 days.
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While this secondary endpoint was not powered for statistical significance, the placebo-adjusted least squares mean reduction for EMP-01 was 11.85 points on the LSAS, which AtaiBeckley said is consistent with a clinically meaningful improvement and a moderate treatment effect size.
On the CGI-I scale, which reflects a global impression of overall patient improvement, 49% of patients receiving EMP-01 were rated as “very much improved” or “much improved” compared to 15% in the placebo group. The company said again that this difference would indicate a clinically meaningful level of global improvement in the EMP-01 group.
The primary endpoint of safety was also achieved, with EMP-01 demonstrating a favourable and manageable safety and tolerability profile. No serious adverse events (AEs) and no treatment-emergent suicidal behaviour or intent were observed. Most AEs were mild or moderate and resolved without intervention.
Professor Emeritus of Psychiatry at the University of California, San Diego, and consultant to AtaiBeckley, Dr David Feifel, said: “Several findings from this preliminary study are highly encouraging, particularly that LSAS improvements became apparent after dosing was completed and the drug was no longer present, suggesting a sustained biological therapeutic effect.
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By GlobalData“If confirmed in larger trials, this would represent a meaningful departure from current first-line pharmacotherapies like SSRIs, which require continuous daily dosing and frequently produce tolerability concerns. An intervention delivering durable symptom improvement with intermittent drug exposure would be a potentially transformative development for patients with this disorder.”
While Feifel believes the drug shows promise, investors were seemingly less satisfied with the data, with the company’s stock, listed on the Nasdaq exchange, dropping 14% after the data was announced, from a 25 February close of $4.34 to a 26 February close of $3.73.
The double-blind, placebo-controlled trial enrolled 71 adults with moderate-to-severe SAD across seven clinical sites in the UK. Participants were randomised to receive two in-clinic administrations of EMP-01 (225 mg) or placebo, given 28 days apart, with no adjunctive psychotherapy.
EMP-01 is an oral formulation of the R-enantiomer of 3,4-methylenedioxy-methamphetamine (MDMA) derivative.
This is not the only MDMA-based drug to work its way through the pipeline. In 2024, Lykos Therapeutics’ MDMA-based therapy for post-traumatic stress disorder (PTSD) was rejected for approval by the US Food and Drug Administration (FDA), with the agency requesting more trials. One of the reasons of concern was study blinding, with it being noted that patients would likely have known if they had received the study drug due to the psychoactive nature of the candidate.
This is something that companies developing psychedelic drugs have taken note of, with companies adapting their trial designs to better mitigate this.
