Analysts consider AtaiBeckley’s MDMA candidate a “high-risk, high-reward” bet after the drug demonstrated reductions in anxiety severity in a phase 2a study.
AtaiBeckley—the new name for atai Life Sciences after its merger with Beckley Psytech last year—evaluated the asset, dubbed EMP-01, in a trial of 71 adults in the U.K. with moderate-to-severe social anxiety disorder (SAD). Patients received two in-clinic administrations of a 225 mg oral dose of the drug, or placebo, 28 days apart with no psychotherapy.
While the trial wasn’t powered for efficacy, the biotech reported a placebo-adjusted mean reduction of 11.8 points on the Liebowitz Social Anxiety Scale, which the biotech considered “clinically meaningful.”
On a clinician-rated assessment, 49% of patients receiving EMP-01 were rated as “very much improved” or “much improved” compared to 15% of the placebo cohort, according to AtaiBeckley’s Feb. 26 press release.
MDMA, commonly known as ecstasy, consists of two enantiomers—R-MDMA and S-MDMA—which have distinct pharmacological profiles. EMP-01 is R-MDMA, which means it should release less dopamine in the brain than S-MDMA and result in reduced stimulatory activity compared to S-MDMA.
The trial was the first to assess R-MDMA in patients with social anxiety disorder, AtaiBeckley CEO Srinivas Rao said in a statement.
“The consistent pattern of improvement observed across secondary and exploratory efficacy endpoints, together with a generally favorable safety and tolerability profile, provides meaningful validation of both the compound and our clinical development model as we assess the next phase of advancement,” Rao added.
In a note to clients, Berenberg analysts described EMP-01 as a “high-risk, high-reward” candidate. The analysts modeled a 5% chance of $4 billion global peak sales for the drug, noting the lack of new treatments for SAD in the last 30 years, despite the opportunity to treat around 20 million patients in the U.S. alone.
The analysts’ ambitious sales forecast could be achieved if EMP-01 reached 5% of domestic SAD patients who don’t respond to existing therapies, and could equate to $3 billion in U.S. sales, they explained in the note.
Even though the trial’s primary endpoint was safety rather than efficacy, some experts are bullish on the candidate and its ability to change the lives of millions of patients. Murray Stein, M.D., a professor of psychiatry and public health at the University of California, San Diego, and a consultant to AtaiBeckley, called the findings “remarkable” and expressed hope that future studies would be conducted to support EMP-01’s advancement.
Related
Hopes for MDMA to treat a variety of mental health conditions received a knock-back in 2024 when the FDA turned down Lykos Therapeutics’ approval application for its MDMA-assisted treatment for post-traumatic stress disorder (PTSD). Last year, Lykos agreed to run another phase 3 trial of the drug, but not before the biotech had shed 75% of its employees.
Other companies continue to explore the space. Last year, Transcend Therapeutics’ MDMA analog, which is not hallucinogenic, achieved its primary endpoint in a phase 2 trial that yielded findings competitive to Lykos’ PTSD data.
Despite the bumpy road for MDMA development in recent years, Berenberg analysts remained positive about the future of the modality and AtaiBeckley’s role in it. They expect the biotech to launch a phase 3 study of its lead program, a psychedelic called 5-MeO-DMT, in the first half of this year.
“AtaiBeckley is set to be a leader in the highly promising emerging class of psychedelics for the treatment of severe mental health conditions with high unmet need and large patient populations, in our view,” the analysts wrote in their note this morning. “Success for just one of AtaiBeckley’s short-acting psychedelic candidates should offer substantial upside to the current valuation.”
