BioXcel completes last patient visit in pivotal Phase 3 SERENITY At-Home trial.

BioXcel Therapeutics announced completion of the Phase 3 SERENITY At-Home clinical trial, with top-line data expected this month. The trial evaluated the safety of BXCL501 for treating agitation in bipolar disorders or schizophrenia in an at-home setting. Data from over 2,200 agitation episodes was collected. The results will support a planned supplemental New Drug Application to expand IGALMI's label.

New Haven, Conn. — BioXcel Therapeutics, Inc. (Nasdaq: BTAI) has completed the last patient last visit (LPLV) in its pivotal Phase 3 SERENITY At-Home clinical trial. The trial, 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, has now reached its endpoint [1].

The SERENITY At-Home Phase 3 trial enrolled more than 200 patients across 22 sites nationwide, with no single site enrolling more than 11% of the total patient population. The trial collected data from over 2,200 agitation episodes, with topline data expected to be released this month. The results are anticipated to support a planned supplemental New Drug Application (sNDA) to potentially expand the label of IGALMI® (dexmedetomidine) for use in the at-home (outpatient) setting [1].

Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics, stated, “This marks a major milestone in our efforts to bring a much-needed at-home (outpatient) treatment option to the millions of individuals who experience agitation related to bipolar disorders or schizophrenia. We’re grateful to the patients, caregivers, investigators, CRO, and our internal teams who made this possible in a very timely manner. With this final visit complete, we look forward to sharing topline results soon. This is another critical step toward advancing BXCL501 (IGALMI®) as the first FDA-approved therapy for this indication in the home setting” [1].

The SERENITY At-Home Phase 3 trial is 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 enrolled patients with a history of agitation episodes residing at home either alone or with caregivers/informants. Patients self-administered 120 mcg of BXCL501 or placebo when agitation episodes occurred over the 12-week trial period, with safety data (adverse events) collected during the trial. In addition, patients or caregivers/informants completed a modified global impression of severity (mCGIs) and a clinical global impression of change (mCGI-C) two hours after dosing as an exploratory endpoint to evaluate use in the outpatient setting [1].

Outside of its approved indication by the U.S. Food and Drug Administration as IGALMI® (dexmedetomidine) sublingual film, BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. BXCL501 is under investigation by BioXcel Therapeutics for the acute treatment of agitation associated with Alzheimer’s dementia and for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in the at-home setting. The safety and efficacy of BXCL501 for these investigational uses have not been established. BXCL501 has been granted Breakthrough Therapy designation by the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for the acute treatment of agitation associated with schizophrenia, bipolar disorders, and dementia [1].

About BioXcel Therapeutics, Inc.
BioXcel Therapeutics, Inc. (Nasdaq: BTAI) is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience. Its wholly owned subsidiary, OnkosXcel Therapeutics, is focused on the development of medicines in immuno-oncology. The Company’s drug re-innovation approach leverages existing approved drugs and/or clinically validated product candidates together with big data and proprietary machine learning algorithms to identify new therapeutic indications. For more information, please visit bioxceltherapeutics.com [1].

References:
[1] https://www.globenewswire.com/news-release/2025/08/01/3125705/0/en/BioXcel-Therapeutics-Announces-Last-Patient-Last-Visit-in-SERENITY-At-Home-Pivotal-Phase-3-Safety-Trial-for-Acute-Treatment-of-Agitation-Associated-with-Bipolar-Disorders-or-Schizo.html