Ankündigung • Apr 24
AtaiBeckley Inc. Announces Additional Phase 2A Results for EMP-01 Oral R-M Showing Large and Consistent Improvements in Social Anxiety Disorder AtaiBeckley Inc. announced expanded Phase 2a results for EMP-01 (oral R-MDMA) in adults with Social Anxiety Disorder (SAD) (n=70), demonstrating clinically meaningful and consistent improvements across clinician-rated symptoms, patient-reported experience, and real-world behavioral outcomes. At Day 43, EMP-01 achieved a 38% reduction vs 15% on placebo (Hedges’ g=0.84) on the patient-reported Social Phobia Inventory (SPIN), a 32% reduction vs 14% on placebo on the Subtle Avoidance Frequency Examination (SAFE), and a previously reported -11.9-point LS mean difference (LSMD) on the Liebowitz Social Anxiety Scale (LSAS) versus placebo (g=0.45), with 49% responder rates on both Clinical Global Impression-Improvement (CGI-I) (previously reported) and Patient Global Impressions of Change (PGI-C). EMP-01 was well tolerated, with no severe or serious adverse events. EMP-01 produced consistent and clinically meaningful improvements across all major symptom domains of SAD: LSAS: EMP-01 demonstrated a clinically meaningful LSMD of -11.9-points vs placebo (g=0.45) at Day 43 (previously reported), with both total and subscale improvements. Improvements were observed across both fear and avoidance subscales on the clinician-rated 24-item Liebowitz Social Anxiety Scale, indicating that patients experienced fewer social situations as distressing and were more able to engage in them. This degree of change is considered clinically meaningful and reflects broad symptom improvement across core features of social anxiety disorder including fear and anxiety of social situations. SPIN: EMP-01 produced a large, clinically meaningful patient-reported improvement of -18.3-points (38% reduction vs 15% on placebo), 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. Patients treated with 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. SAFE: 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. 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 on the Subtle Avoidance Frequency Examination, a 32-item questionnaire that measures safety behaviors, suggest that participants were more willing to participate in everyday activities such as social interactions without engaging in avoidant coping behaviors. Clinical impression: 49% CGI-I responders (NNT=2.95) at Day 43 compared to 15% on placebo (previously reported). This measure reflects clinicians’ overall judgment of meaningful improvement in a patient’s condition, considering symptom severity, functioning, and overall clinical presentation. Patient perception: 49% PGI-C responders (NNT=2.72) at Day 43 compared to 12% on placebo. This result indicates that patients themselves perceived the treatment-associated improvements as noticeable and meaningful in their daily lives, reinforcing the clinical and functional outcomes observed on other measures. Safety & Tolerability: EMP-01 was generally safe and well tolerated: No SAEs and no severe TEAEs in any participant; 97% retention, with 0% study dropouts attributed to TEAEs; TEAEs were expected and consistent with the class, transient, and predominantly mild-to-moderate. The multi-center study enrolled 71 adults with moderate-to-severe SAD across 7 clinical sites in the UK. Participants were randomized to receive two in-clinic administrations of EMP-01 (225 mg) or placebo, given 28 days apart, with no adjunctive psychotherapy. 70 participants received at least one dose of study drug, and 69 completed the Day 43 efficacy assessments, indicating high patient acceptability and retention. All clinician-rated assessments were conducted by blinded central raters. Ankündigung • Apr 23
AtaiBeckley Inc., Annual General Meeting, Jun 04, 2026 AtaiBeckley Inc., Annual General Meeting, Jun 04, 2026. Ankündigung • Apr 08
AtaiBeckley Inc. Shows Rapid And Durable Antidepressant Response With BPL-003 In Treatment-Resistant Depression Patients AtaiBeckley Inc. announced peer-reviewed Phase 2a results (NCT05660642) in CNS Drugs demonstrating that a single intranasal dose of BPL-003 (mebufotenin benzoate), which holds FDA Breakthrough Therapy Designation, achieved rapid and sustained reductions in MADRS scores from baseline in participants with treatment-resistant depression (TRD) who remained on stable SSRI therapy throughout the study (n=12). A 66.7% antidepressant response rate (=50% reduction from baseline MADRS score) was observed at Day 2 in both the 10 mg (n=6) and 12 mg (n=6) cohorts, with 83% (5/6) of participants in the 10 mg cohort and 66.7% (4/6) of participants in the 12 mg cohort maintaining a response at Week 12. BPL-003 was generally well tolerated with no serious adverse events reported, and participants achieved a mean discharge approximately 100 minutes post-dose. Phase 3 studies are on track to initiate in Second Quarter 2026 following recent FDA End-of-Phase 2 (EOP2) alignment. BPL-003 (mebufotenin benzoate intranasal spray) is a 5-HT1A and 5-HT2A agonist associated with rapid onset and treatment experience of approximately 2 hours. The indication is treatment-resistant depression (TRD); failure to respond to =2 prior antidepressants at adequate dose and duration. U.S. FDA Breakthrough Therapy Designation was granted October 2025. NCT05660642 is a four-part Phase 2a open-label study. Part 2 (SSRI-concomitant, n=12) reported here; Part 1 (monotherapy, single dose, n=12) published in Journal of Psychopharmacology in March 2026. Part 3 (8 mg + 12 mg, monotherapy, n = 12) topline announced September 2025. Part 4 (8 mg + 8 mg, adjunctive) ongoing; initial data expected Fourth Quarter 2026. Endpoint is safety and tolerability; exploratory Montgomery-Asberg Depression Rating Scale (MADRS) total score change from baseline. 66.7% of participants achieved =50% MADRS reduction from a single dose at Day 2. 83% responders (10 mg cohort); 66.7% (12 mg cohort) at Day 85. MADRS-6 Core Symptoms: 19.2 ? 6.2 (10 mg); 21.0 ? 9.3 (12 mg); =10 = remission. No serious adverse events; majority of drug-related AEs transient and resolved on same day. Mean discharge readiness approximately 100 minutes post-dose. End-of-Phase 2 meeting with U.S. FDA completed; Phase 3 initiation on track for Second Quarter 2026. The 12-week, open-label, single-center, ascending-dose trial enrolled 12 adults aged 18 to 75 years with moderate-to-severe major depressive disorder (baseline MADRS =24) and TRD. All participants had failed at least two prior antidepressants and remained on a stable dose of one of four SSRIs - citalopram, escitalopram, sertraline or fluoxetine - throughout the study. Six participants received a single intranasal 10 mg dose of BPL-003 and six received a single intranasal 12 mg dose of BPL-003, with psychological support before, during and after dosing. Participants were followed for 12 weeks. This publication represents Part 2 of the four-part Phase 2a open-label study. Data from Part 1 (BPL-003 monotherapy, 10 mg single dose, n=12) was published in the Journal of Psychopharmacology in March 2026, reporting a mean 12.6-point MADRS reduction by Day 2, sustained through 12 weeks. Topline data from Part 3 (8 mg + 12 mg, BPL-003 monotherapy, n=12) were announced in September 2025, showing that a second dose of BPL-003 at Week 2 has the potential to induce additional reductions in MADRS scores without impact on the safety and tolerability profile of the treatment. Part 4 of the program - evaluating a two-dose induction regimen of BPL-003 (8 mg + 8 mg) in participants with TRD who are also receiving defined antidepressants – is ongoing. Initial data from that cohort is expected in Fourth Quarter 2026. Ankündigung • Mar 17
AtaiBeckley Inc Demonstrates Rapid And Durable Antidepressant Effects In Treatment-Resistant Depression AtaiBeckley Inc. announced the peer-reviewed publication of results from its ongoing four-part Phase 2a study evaluating BPL-003 (mebufotenin benzoate nasal spray) in patients with treatment-resistant depression (TRD). Newly reported data come from Cohort 1 – a 12 week, open-label trial of a single 10 mg intranasal dose of BPL-003 in 12 patients with moderate-to-severe TRD who were not taking concomitant antidepressants. BPL-003 produced a mean Montgomery–Åsberg Depression Rating Scale (MADRS) total score reduction of 12.6 points by Day 2 (from a baseline mean of 27.5 to 14.8), which was sustained over 12 weeks to a mean MADRS score of 14.5 at Day 85. A response rate (=50% MADRS reduction) of 54.5% was observed from the day after dosing through Day 85, and 63.6% of patients achieved remission (MADRS =10) at one or more timepoints. Mean Snaith-Hamilton Pleasure Scale (SHAPS) scores also improved from 8.4 at baseline to 1.5 at Day 85, indicating an absence of anhedonia. BPL-003 was well tolerated with no serious adverse events and no treatment withdrawals due to adverse events. Most adverse events were transient and mild-to-moderate in severity. Following a successful End-of-Phase 2 meeting with the U.S. Food and Drug Administration announced in March 2026, the Phase 3 program for BPL-003 in TRD remains on track for initiation in Second Quarter 2026. The Phase 2a trial comprises four cohorts. Results from Parts 1, 2 and 3 have been previously announced. The first patient has been dosed in the Part 4 cohort, evaluating a two-dose induction regimen (8 mg + 8 mg) of BPL-003 in TRD patients receiving defined antidepressants, with initial data expected in Fourth Quarter 2026. BPL-003 is a patent-protected, proprietary intranasal formulation of mebufotenin benzoate, administered via a nasal spray device used in a previously approved drug product. BPL-003 is designed to deliver rapid and durable effects from a single dose, with a short psychedelic duration, and is being investigated as a potential therapy for treatment-resistant depression (TRD) and alcohol use disorder (AUD). BPL-003 has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration and is covered by granted US, UK and European composition-of-matter patents, with multiple further claims pending in various jurisdictions. Ankündigung • Mar 03
AtaiBeckley Inc Announces Successful End-Of-Phase 2 Meeting For BPL-003 In Treatment-Resistant Depression AtaiBeckley Inc. had a successful End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) regarding the development of BPL-003, the Company’s proprietary intranasal formulation of mebufotenin benzoate, for treatment-resistant depression (TRD). Minutes from the meeting show that the FDA indicated support for AtaiBeckley’s proposed BPL-003 pivotal program and the compound’s advancement into Phase 3 studies in adults with TRD. The FDA also provided constructive feedback on the overall design and key components of the proposed program, including the overall size of the safety database for this chronic indication. BPL-003 was previously granted Breakthrough Therapy designation by the FDA for treatment-resistant depression. The Phase 3 program is designed to include two pivotal studies, ReConnection 1 and ReConnection 2, conducted in parallel. Both will include a 12-week, randomized, double-blind, placebo-controlled core study followed by a 52-week open-label extension (OLE) in which participants may receive individualized retreatment with BPL-003, subject to pre-specified eligibility criteria. ReConnection 1: The core study will enroll approximately 350 participants and will evaluate a single-dose of BPL-003 across three treatment arms - 8 mg, 4 mg, and placebo (randomized 2:1:2). This trial is designed to replicate and extend the treatment response observed in the Phase 2b study and to further characterize the dose–response relationship for BPL-003. ReConnection 2: The core study will enroll approximately 300 participants and will evaluate BPL-003 administered on Day 1 and Day 15 across two arms - 8 mg BPL-003 and placebo (randomized 1:1). This trial is designed to investigate a two-dose induction model of BPL-003 as a potential treatment option to increase magnitude and durability of initial response. The primary endpoint in both pivotal trials will be the change from baseline in the MADRS (Montgomery-Åsberg Depression Rating Scale) total score at Week 4 (Day 29) of the response in the 8 mg BPL-003 treatment arm compared to placebo. In the OLE, participants may receive 8 mg BPL-003 at 8- or 12-week intervals, subject to certain conditions for re-treatment eligibility, with the goal of maintaining remission and assessing long-term safety, durability of effects, and treatment patterns. BPL-003 is a patent-protected, proprietary intranasal formulation of mebufotenin benzoate, administered via a nasal spray device used in a previously approved drug product. BPL-003 is designed to deliver rapid and durable antidepressant effects from a single dose with a short psychedelic duration and is being investigated as a potential therapy for treatment-resistant depression (TRD). BPL-003 has been granted Breakthrough Therapy designation by the U.S. Food and Drug Administration and is covered by granted US, UK and European composition-of-matter patents, with multiple further claims pending in various jurisdictions. BPL-003 is an investigational product and has not been approved by the FDA. Ankündigung • Feb 26
AtaiBeckley Inc. Announces Positive Topline Results from an Exploratory Phase 2a Trial of EMP-01 (oral R-MDMA) in Social Anxiety Disorder AtaiBeckley Inc. announced topline results from its exploratory, double-blind, placebo-controlled, first-in-patient Phase 2a study (NCT06693609) evaluating EMP-01 (oral R-MDMA) in adults with Social Anxiety Disorder (SAD). The study met its primary safety objective and generated encouraging secondary and exploratory efficacy signals in a highly severe, difficult-to-treat population. Participants were randomized to receive two in-clinic administrations of EMP-01 (225 mg) or placebo, given 28 days apart, with no adjunctive psychotherapy. 70 participants received at least one dose of study drug, and 69 completed the Day 43 efficacy assessments, indicating high patient acceptability and retention. All clinician-rated assessments were conducted by blinded central raters. Because avoidance behaviors typically change gradually and often require prolonged real-world exposure, the early, parallel improvements in both Fear and Avoidance - after two dosing sessions and without psychotherapy - suggest that EMP-01 could influence both the emotional and behavioral dimensions of social anxiety disorder. Dr. David Feifel, Professor Emeritus of Psychiatry at the University of California, San Diego, Founder and President of the Kadima Neuropsychiatry Institute, and consultant to AtaiBeckley, 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. 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". 
