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

BioXcel Therapeutics announced that its SERENITY Phase 3 trial met its primary endpoint, demonstrating the safety of BXCL501 for treating agitation associated with bipolar disorders or schizophrenia in the at-home setting. The trial showed that the 120 mcg dose of BXCL501 was well-tolerated and met the primary objective. The company plans to submit a supplemental new drug application (sNDA) for label expansion of IGALMI in the at-home setting in Q1 2026.

BioXcel Therapeutics, Inc. (Nasdaq: BTAI) has announced that its SERENITY At-Home Pivotal Phase 3 trial has met its primary endpoint, demonstrating the safety of BXCL501 for treating agitation associated with bipolar disorders or schizophrenia in the at-home setting. The trial, which involved over 2,600 episodes of agitation, showed that the 120 mcg dose of BXCL501 was well-tolerated and met the primary objective.

The trial, conducted by BioXcel Therapeutics, focused on the safety of BXCL501 for bipolar and schizophrenia patients. It 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 over 12 weeks. The trial involved 246 patients who recorded 2,628 episodes of agitation, with 2,437 episodes treated in 208 patients [1].

The primary endpoint was met with no discontinuations due to tolerability in the BXCL501 arm, indicating a favorable safety profile. The adverse event profile was consistent with the approved IGALMI ® label and multiple clinical trials in the institutional setting. No drug-related serious adverse events (SAEs), syncopes, or falls were reported. The trial also showed that repeated dosing continued to provide symptomatic relief [1].

Following positive feedback from the FDA, BioXcel plans to submit a supplemental New Drug Application (sNDA) in Q1 2026 to expand BXCL501's use to outpatient settings. The success 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 [1].

The trial's success suggests a significantly larger market opportunity for BXCL501 than previously estimated, with over 2,400 episodes of agitation treated. However, the reliance on preliminary results and ongoing analyses may raise uncertainty about the robustness of the findings. The company's plan to submit a sNDA in Q1 2026 indicates a delay in securing approval for at-home usage, which may hinder potential market entry and profitability in the interim [1].

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