BioXcel Therapeutics Progresses Phase 3 Trials of BXCL501 for Agitation in Bipolar, Schizophrenia, and Alzheimer's

BioXcel Therapeutics, Inc., a biopharmaceutical firm leveraging artificial intelligence to develop innovative treatments in neuroscience, has announced promising clinical and regulatory advancements for its pivotal Phase 3 trials of BXCL501. BXCL501 is being studied for the acute treatment of agitation associated with bipolar disorders or schizophrenia and Alzheimer’s dementia. These trials, named SERENITY At-Home and TRANQUILITY In-Care, respectively, aim to address significant unmet medical needs as there are currently no FDA-approved acute therapies for these conditions.

Dr. Vincent O’Neill, Chief of Product Development and Medical Officer of BioXcel Therapeutics, expressed satisfaction with the progress of their late-stage trials. The SERENITY At-Home trial seeks to expand the label of BXCL501, known as IGALMI™ when administered under healthcare supervision, to treat agitation in patients with bipolar disorder or schizophrenia in a home setting. The TRANQUILITY In-Care trial targets agitation associated with Alzheimer’s dementia, a condition for which there is no episodic treatment precedent in the U.S.

The SERENITY At-Home trial aims to evaluate the safety of a 120 mcg dose of BXCL501 administered at home. The first patient has been randomized for this trial, which began on September 5, 2024. The trial is designed as a double-blind, placebo-controlled, 12-week outpatient study expected to enroll around 200 patients living at home. The trial duration is anticipated to last between 9 to 12 months.

The TRANQUILITY In-Care trial is designed to assess the efficacy and safety of a 60 mcg dose of BXCL501 for Alzheimer’s dementia over a 12-week period. Feedback on the trial protocol has been received from the U.S. Food and Drug Administration. This study is also a double-blind, placebo-controlled trial and plans to enroll approximately 150 patients aged 55 years and older with varying degrees of Alzheimer’s disease severity, from mild to severe dementia. The trial will include patients residing in skilled nursing facilities, memory care units, or assisted living facilities who experience episodic agitation. Patients will self-administer 60 mcg of BXCL501 or a placebo during agitation episodes throughout the trial period. The primary endpoint is a change from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) score two hours after the first dose. This endpoint has been used in previous TRANQUILITY trials and supported the FDA approval of IGALMI™.

Continued efficacy evaluations for the TRANQUILITY In-Care trial will include additional PEC assessments and complementary efficacy measures, such as the global impression of change in agitation. The company has made a corporate presentation available on its website, which includes information on the SERENITY and TRANQUILITY trials.

IGALMI™ is a sublingual film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist, approved for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. It is administered under the supervision of a healthcare provider. Side effects of IGALMI™ can include decreased blood pressure, low blood pressure upon standing, slower heart rate, heart rhythm changes, and sleepiness, among others. The safety and effectiveness of IGALMI™ have not been studied beyond 24 hours from the first dose, and it is not known if it is safe for use in children.

BXCL501, the investigational form of IGALMI™, is being studied for its potential to treat agitation in Alzheimer’s dementia and for use in bipolar I or II disorder and schizophrenia in home settings. It has received Breakthrough Therapy designation from the FDA for treating agitation associated with dementia and Fast Track designation for agitation associated with schizophrenia, bipolar disorders, and dementia.

BioXcel Therapeutics continues its efforts to develop and potentially commercialize these transformative treatments, addressing significant gaps in current medical care.