BioXcel reports results from SERENITY At-Home Pivotal Phase 3 safety trial

BioXcel Therapeutics (BTAI) announced positive topline exploratory efficacy data from the SERENITY At-Home Pivotal Phase 3 safety trial, which demonstrated BXCL501 had continued effects and consistent benefit with repeat dosing. The trial in the home setting evaluated 120 mcg dose of BXCL501, the Company’s proprietary, sublingual film formulation of dexmedetomidine, IGALMI, for treatment of agitation associated with bipolar disorders or schizophrenia. BioXcel previously reported positive topline safety results, the primary objective of the trial, on August 27 and is reporting additional findings today. While the trial was not powered for efficacy assessments, continued effects and consistent benefits with repeat dosing seen in the trial further support the potential of BXCL501 for use in the outpatient setting. The data from this successful trial will form the basis of the sNDA submission for label expansion of IGALMI in the at-home setting planned for the Q1 of 2026. The SERENITY At-Home trial is a Pivotal Phase 3, double-blind, placebo-controlled, 12-week clinical trial designed to evaluate the safety of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in an at-home setting. The trial also included an exploratory objective of assessing the continued efficacy of BXCL501 in the treatment of episodes of agitation. Data collected on 2,628 agitation episodes in 215 patients over a 12-week period; Treated 2,437 episodes in 208 patients 168 patients completed the full 12-week trial; Average of 11.7 agitation episodes recorded per treated patient; Reported agitation episodes were classified as mild, moderate or severe; All patients were able to successfully self-administer the film. The efficacy of IGALMI has already been established in the institutional setting in the SERENITY I and II trials that led to the approval by FDA in April 2022. In addition to the primary objective of evaluating the safety of BXCL501, the SERENITY At-Home trial also had an exploratory objective of assessing continued efficacy of BXCL501 with repeat dosing. The trial was not powered for these assessments. The positive results demonstrated continued effects and consistent benefits with repeat dosing of BXCL501, reinforcing the potential of BXCL501 in the outpatient setting. Across 2,433 treated episodes in the trial, BXCL501 demonstrated a significant mean reduction in the modified Clinical Global Impression-Severity score from baseline compared to placebo at 2 hours. Patients experienced a complete resolution of agitation symptoms measured by mCGI-S at significantly higher rates with BXCL501 compared to placebo across severity of agitation episodes, with an overall resolution of 50% in the BXCL501 arm, compared to 33% on placebo. Severe agitation episodes fully resolved in 61% of episodes in the BXCL501 arm, compared to 18% on placebo.