BioXcel Therapeutics' BXCL501 Meets Primary Endpoint in SERENITY At-Home Phase 3 Trial

BioXcel Therapeutics' sublingual dexmedetomidine film, BXCL501, has met its primary endpoint in the SERENITY At-Home Phase 3 trial for acute agitation in bipolar disorder or schizophrenia. The results show consistent symptom reduction across 2,400+ episodes and will support an FDA supplemental filing to expand Igalmi's label for at-home use in Q1 2026. The trial found that a higher percentage of patients receiving BXCL501 achieved full resolution of mild-to-severe agitation compared to placebo, with consistent benefits observed on repeat dosing. The 120 mcg dose was well-tolerated, met the primary endpoint, and caused no discontinuations. Adverse events aligned with the approved Igalmi label and prior institutional trials.

BioXcel Therapeutics, Inc. (Nasdaq: BTAI) has announced that its sublingual dexmedetomidine film, BXCL501, has successfully met its primary endpoint in the SERENITY At-Home Phase 3 trial. The trial, designed to evaluate the safety of a 120 mcg dose of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting, demonstrated consistent symptom reduction across 2,400+ episodes.

The primary endpoint of the trial was met, with the 120 mcg dose of BXCL501 being well-tolerated and causing no discontinuations due to tolerability. This outcome was observed across repeat dosing and throughout the duration of the 12-week trial. The adverse event profile was consistent with the approved Igalmi label and prior institutional trials, with no drug-related serious adverse events, syncopes, or falls reported.

The trial also provided preliminary exploratory data that showed a greater percentage of patients experiencing mild, moderate, or severe agitation had full resolution of symptoms in the BXCL501 arm compared to placebo. This indicates continued effects and consistent benefit with repeat dosing across the course of the trial.

Based on these results, BioXcel plans to submit a supplemental New Drug Application (sNDA) in Q1 2026 to expand Igalmi's label for at-home use. This would position BXCL501 to potentially become the first at-home treatment for this critical unmet need, affecting patients and their families.

The SERENITY At-Home Pivotal Phase 3 trial was a double-blind, placebo-controlled 12-week study designed to evaluate the safety of a 120 mcg dose of BXCL501. The trial included 246 patients randomized, with data collected from 2,628 agitation episodes in 215 patients. Of these, 2,437 episodes were treated in 208 patients, with 168 patients (81%) completing the full 12-week trial. The average number of agitation episodes recorded per treated patient was 11.7.

The trial's primary endpoint data showed that the 120 mcg dose of BXCL501 was well-tolerated in patients with episodes of agitation in the outpatient setting. No discontinuations due to tolerability were reported in the BXCL501 arm, and the adverse event profile was consistent with the approved Igalmi label and multiple clinical trials in the institutional setting.

The total addressable market for at-home agitation treatment is significantly larger than previously reported. Market research and published survey data indicate that episodes may occur 3-4 times a month on average, with the majority being moderate or severe. Data from the SERENITY At-Home Pivotal Phase 3 trial align with these higher frequency estimates, suggesting that patients experience an estimated 57 million to 77 million agitation episodes annually.

References:
[1] https://ir.bioxceltherapeutics.com/news-releases/news-release-details/bioxcel-therapeutics-announces-serenity-home-pivotal-phase-3