BioXcel Therapeutics, Inc. (Nasdaq: BTAI) has announced progress in its pivotal Phase 3 clinical trials for BXCL501, targeting acute agitation associated with bipolar disorders, schizophrenia, and Alzheimer’s dementia (AAD). The SERENITY At-Home trial has randomized its first patient, while the company has received FDA feedback on the TRANQUILITY In-Care trial protocol. These trials aim to address the estimated 140 million annual acute agitation episodes associated with these conditions, for which there are currently no FDA-approved acute therapies.
SERENITY At-Home Trial
The SERENITY At-Home trial is a double-blind, placebo-controlled, 12-week outpatient study designed to evaluate the safety of a 120 mcg dose of BXCL501 in treating agitation associated with bipolar disorders or schizophrenia in the at-home setting. Approximately 200 patients will be enrolled. BioXcel Therapeutics initiated the trial on September 5, 2024, with an expected duration of 9 to 12 months. The trial seeks to expand the label for BXCL501, which is currently approved as IGALMI™ for use under healthcare provider supervision.
TRANQUILITY In-Care Trial
The TRANQUILITY In-Care trial is designed to assess the efficacy and safety of a 60 mcg dose of BXCL501 for AAD over a 12-week period. The double-blind, placebo-controlled study aims to enroll approximately 150 patients aged 55 years and older with mild, moderate, and severe Alzheimer’s disease (MMSE scores of 0 to 25) residing in skilled nursing facilities, memory care units, or assisted living facilities. Patients will self-administer 60 mcg of BXCL501 or placebo during agitation episodes.
The primary endpoint is the change from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at two hours post-first dose, consistent with previous TRANQUILITY trials and the FDA approval studies for IGALMI™ (dexmedetomidine) sublingual film. Continued efficacy evaluations will include additional PEC scores and complementary measures, such as the global impression of change in agitation.
About IGALMI™ (dexmedetomidine) Sublingual Film
IGALMI™ is a prescription medicine used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults, administered under the supervision of a healthcare provider. Its safety and effectiveness have not been studied beyond 24 hours from the first dose, and it is not known if IGALMI is safe and effective in children.
About BXCL501
BXCL501 is an investigational orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. It is being studied for the acute treatment of agitation associated with Alzheimer’s dementia and for at-home use in patients with bipolar I or II disorder or schizophrenia. The FDA has granted Breakthrough Therapy designation for BXCL501 in treating agitation associated with dementia and Fast Track designation for agitation associated with schizophrenia, bipolar disorders, and dementia.
