BioXcel Begins Phase 3 Safety Trial of BXCL501 for Bipolar and Schizophrenia Agitation

BioXcel Therapeutics, Inc., a biopharmaceutical company, has embarked on a new clinical trial to address a significant gap in the treatment of agitation in patients with bipolar disorders and schizophrenia. This trial, known as the SERENITY At-Home pivotal Phase 3 trial, will investigate the safety of BXCL501, a proprietary, orally dissolving film formulation of dexmedetomidine, in an at-home setting. With an estimated 23 million annual agitation episodes occurring at home, there are currently no FDA-approved therapies available for such acute treatments.

Dr. John Krystal, a professor at Yale School of Medicine, emphasized the importance of managing agitation at home, noting that early treatment can alleviate patient suffering and reduce emergency room visits. He highlighted the need for new therapeutic options that could fill this treatment void.

Vincent J. O’Neill, the Executive Vice President and Chief of Product Development at BioXcel Therapeutics, expressed optimism about initiating the SERENITY At-Home trial. He emphasized the company’s extensive prior experience with BXCL501 and favorable safety results observed in earlier programs. The primary goal of the trial is to characterize the safety profile of the 120 mcg dose, which is an approved dose of BXCL501.

The SERENITY At-Home Phase 3 trial is designed as a double-blind, placebo-controlled study. It will enroll approximately 200 patients who live at home, either alone or with caregivers. These patients will self-administer BXCL501 or a placebo when they experience agitation episodes over a 12-week period. Safety data will be collected through patient and caregiver reports, as well as telephone interviews and agitation episode diaries. Adverse events will be recorded at all in-clinic visits. In addition to safety, an exploratory endpoint will evaluate the patients’ or caregivers’ impressions of using BXCL501 at home, utilizing a modified clinical global impression of change (mCGI-C) two hours after dosing.

BXCL501 is an investigational, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. Apart from its approved indication as IGALMI™ for acute agitation treatment, BXCL501 is being explored for its potential in treating agitation associated with Alzheimer’s dementia as well as bipolar I or II disorder and schizophrenia in home settings. While safety and efficacy for these uses are yet to be established, BXCL501 has received Breakthrough Therapy designation for agitation related to dementia and Fast Track designation for agitation associated with schizophrenia, bipolar disorders, and dementia.

BioXcel Therapeutics, Inc. leverages artificial intelligence to develop transformative medicines in neuroscience. The company’s approach involves drug re-innovation, combining big data and machine learning algorithms to identify new therapeutic indications for existing approved drugs and clinically validated product candidates. Its subsidiary, OnkosXcel Therapeutics, focuses on immuno-oncology.

The initiation of the SERENITY At-Home trial marks a significant step toward addressing the unmet need for effective at-home treatments for agitation in patients with bipolar disorders and schizophrenia. By potentially reducing patient suffering and healthcare costs, this trial could pave the way for new therapeutic options that improve the quality of life for millions of individuals experiencing agitation episodes annually.