AtaiBeckley Inc. (NASDAQ:ATAI, XETRA:9VC) announced that patients in a mid-stage trial of its experimental depression treatment BPL-003 showed improvement within two days of a single dose, with effects lasting up to 12 weeks.
The Phase 2a study found that 66.7% of participants achieved a clinically significant reduction in symptoms by Day 2.
Responses were sustained through Day 85 in 83% of patients in the 10 mg group and 66.7% in the 12 mg group.
The trial included 12 adults with treatment-resistant depression who continued taking stable doses of selective serotonin reuptake inhibitors. Participants received a single intranasal dose of either 10 mg or 12 mg of BPL-003.
Reductions in depressive symptoms were measured using the Montgomery-Åsberg Depression Rating Scale, with average core symptom scores declining to levels consistent with remission in the 10 mg group and near-remission in the 12 mg group.
No serious adverse events were reported. Most side effects were transient and resolved on the same day, and participants were discharged on average about 100 minutes after dosing.
“This study provides the first Phase 2a evidence that BPL‑003 can be administered alongside SSRIs without compromising efficacy or safety, a meaningful advance given that many TRD patients remain on chronic SSRI therapy,” AtaiBeckley’s chief medical officer Dr. Kevin Craig said in a statement.
“The rapid resolution of acute effects and ~100‑minute discharge readiness further support the feasibility of integrating BPL‑003 into existing interventional psychiatry settings.”
The results were published in CNS Drugs and are part of a four-part Phase 2a program. Additional study data have been previously released, and another cohort remains ongoing, with initial results expected in the fourth quarter of 2026.
BPL-003 has received Breakthrough Therapy Designation from the US Food and Drug Administration. The company said it plans to begin Phase 3 trials in the second quarter of 2026.
"A 66.7% Day‑2 response rate with a single intranasal dose of BPL‑003, maintained through Week 12 in the 10 mg cohort, represents a compelling clinical signal in patients who remained on their baseline SSRI therapy,” AtaiBeckley CEO Srinivas Rao said.
“Combined with our statistically significant double-blind, randomized Phase 2b results in 193 participants and our recent FDA End‑of‑Phase 2 alignment, these Phase 2a data reinforce the potential of BPL‑003 to transform the treatment paradigm for TRD as we prepare to initiate Phase 3 in Q2 2026.”