BXCL501 shows significant agitation reduction in at-home trials with FDA feedback.

BXCL501 significantly reduced mean mCGI-S score from baseline compared to placebo at 2 hours in 2,433 treated episodes (p<.05). Complete resolution of agitation was higher with BXCL501 compared to placebo across agitation episode severity (p<.0001). The company plans to submit a sNDA in Q1 2026 for expanded usage of IGALMI in the outpatient setting.

New Haven, Conn., Sept. 10, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced positive topline exploratory efficacy data from the SERENITY At-Home Pivotal Phase 3 safety trial. The trial demonstrated that BXCL501, a proprietary sublingual film formulation of dexmedetomidine, IGALMI®, showed significant reductions in agitation symptoms compared to placebo.

The SERENITY At-Home trial, which evaluated 120 mcg dose of BXCL501 for the treatment of agitation associated with bipolar disorders or schizophrenia in an at-home setting, found that BXCL501 demonstrated a significant mean reduction in the modified Clinical Global Impression–Severity (mCGI-S) score from baseline compared to placebo at 2 hours across 2,433 treated episodes (p.05) BioXcel Therapeutics Reports Positive Topline Exploratory Efficacy Data from SERENITY At-Home Pivotal Phase 3 Safety Trial for Agitation Associated with Bipolar Disorders or Schizophrenia^[1]. Complete resolution of agitation was significantly higher with BXCL501 compared to placebo across agitation episode severity (p.0001). The drug showed a similar reduction in agitation symptoms over both the duration of the trial and the number of treated episodes, indicating continued effects and consistent benefit with repeat dosing BioXcel Therapeutics Reports Positive Topline Exploratory Efficacy Data from SERENITY At-Home Pivotal Phase 3 Safety Trial for Agitation Associated with Bipolar Disorders or Schizophrenia^[1].

The company plans to submit a sNDA in Q1 2026 for expanded usage of IGALMI® in the outpatient setting, leveraging the positive results from the SERENITY At-Home trial. The trial was not powered for efficacy assessments but provided valuable insights into the drug's potential in the outpatient setting.

Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics, commented, "The SERENITY At-Home results mark a pivotal step in advancing the potential for outpatient use of BXCL501 for the acute treatment of agitation in bipolar disorders and schizophrenia. These positive results, along with the safety and tolerability data previously announced, reinforce BXCL501’s potential in the at-home setting where there is substantial unmet need with no FDA-approved options currently available."

The company will host a virtual KOL call with Dr. Leslie Citrome on the SERENITY At-Home program today at 2 p.m. . The webcast and accompanying presentation materials are available on the company's investor section website .

BioXcel Therapeutics is committed to redefining the treatment paradigm for patients with agitation in bipolar disorders and schizophrenia by expanding the use of IGALMI® in the outpatient setting. This represents a large addressable market and a catalyst for long-term growth and value creation.

About BioXcel Therapeutics, Inc.

BioXcel Therapeutics, Inc. is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. The company is focused on developing novel, AI-driven therapeutic approaches to treat a range of neurological and psychiatric disorders.