BioXcel Therapeutics' lead asset BXCL501 achieves Phase 3 trial goals, shares fluctuate in premarket trading.

BioXcel Therapeutics' lead asset BXCL501 achieved the primary endpoints in a Phase 3 trial, according to the company. Shares of the biotech initially rose and then fell in the premarket. The trial, named SERENITY, aimed to evaluate BXCL501 in patients with schizophrenia. Further details on the trial results and implications for the company's future prospects are not specified in the article.

BioXcel Therapeutics, Inc. (Nasdaq: BTAI) announced that its lead asset, BXCL501, achieved the primary endpoint in the SERENITY At-Home Phase 3 trial, which evaluated the safety of BXCL501 for the treatment of agitation associated with bipolar disorders or schizophrenia in an at-home setting [1].

The trial, which enrolled 246 patients and collected data on 2628 agitation episodes, demonstrated that the 120 mcg dose of BXCL501 was well-tolerated and met its primary objective of safety. No discontinuations due to tolerability were reported in the BXCL501 arm, indicating a favorable safety profile. Preliminary results also suggest that repeated dosing continues to provide symptomatic relief.

BioXcel plans to submit a supplemental New Drug Application (sNDA) in Q1 2026 to expand BXCL501's use to outpatient settings. The trial's success underscores the potential to address a significant unmet medical need, as there are currently no FDA-approved therapies for at-home agitation treatment.

However, the reliance on preliminary results and ongoing analyses may suggest that the final data may not confirm these positive outcomes, raising uncertainty about the robustness of the findings. The company’s plan to submit a supplemental New Drug Application (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.

The SERENITY At-Home trial treated over 2400 episodes of agitation, suggesting a significantly larger market opportunity for BXCL501 than previously estimated. Potential Positives BXCL501 met its primary endpoint as a well-tolerated at-home treatment for agitation in patients with bipolar disorders or schizophrenia, indicating a successful clinical trial outcome. The trial demonstrated no discontinuations due to tolerability in the BXCL501 arm, which enhances confidence in the medication's safety profile for outpatient use. BioXcel plans to submit a supplemental New Drug Application (sNDA) for expanded usage of BXCL501 in the outpatient setting in Q1 2026, paving the way for broader patient access. The SERENITY At-Home trial treated over 2400 episodes of agitation, suggesting a significantly larger market opportunity for BXCL501 than previously estimated.

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