BioXcel Therapeutics' BXCL501 Meets Primary Goal in SERENITY Phase 3 Study

BioXcel Therapeutics announced that its SERENITY At-Home Phase 3 study of BXCL501 met its primary goal, demonstrating the sublingual film formulation's efficacy in treating agitation associated with bipolar disorders or schizophrenia in an at-home setting. The study showed BXCL501 was well-tolerated with no discontinuations for tolerability. The company plans to submit a supplemental new drug application to expand the IGALMI label for at-home use in Q1 2026.

BioXcel Therapeutics, Inc. (Nasdaq: BTAI) has announced that its SERENITY At-Home Phase 3 study of BXCL501 has met its primary goal, demonstrating the sublingual film formulation's efficacy in treating agitation associated with bipolar disorders or schizophrenia in an at-home setting. The study, which involved over 2,600 episodes of agitation, showed that BXCL501 was well-tolerated with no discontinuations for tolerability. The company plans to submit a supplemental new drug application (sNDA) to expand the IGALMI label for at-home use in Q1 2026.

The SERENITY At-Home trial was a double-blind, placebo-controlled study 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. The trial involved 246 patients, with 2628 agitation episodes recorded. The study showed that the 120 mcg dose of BXCL501 was well-tolerated, with no discontinuations due to tolerability in the BXCL501 arm. The adverse event profile was consistent with the approved IGALMI label and multiple clinical trials in the institutional setting.

The successful outcome of the SERENITY At-Home trial underscores the potential to address a significant unmet medical need, as there are currently no FDA-approved therapies for at-home agitation treatment. The trial's success also suggests a significantly larger market opportunity for BXCL501 than previously estimated, with over 2400 episodes of agitation treated.

BioXcel plans to submit the sNDA in Q1 2026 for expanded outpatient use of BXCL501, which could provide broader patient access. The company is committed to changing the treatment paradigm and providing patients with access to IGALMI in the home setting.

The SERENITY At-Home trial's success comes after previously disclosed positive FDA feedback. The company believes the total addressable market is significantly larger than previously reported.

References:
[1] https://www.quiverquant.com/news/BioXcel+Therapeutics+Reports+Successful+Results+from+SERENITY+At-Home+Trial+for+BXCL501+as+At-Home+Treatment+for+Agitation+in+Bipolar+Disorders+and+Schizophrenia