In a significant development for mental health treatment, BioXcel Therapeutics, Inc. has received a second positive recommendation from an independent Data Safety Monitoring Board (DSMB) to continue its SERENITY At-Home pivotal Phase 3 safety trial. This trial focuses on the acute treatment of agitation associated with bipolar disorders or schizophrenia using BXCL501, a sublingual film medication. The DSMB’s recommendation follows a review of unblinded safety data from 178 patients as of May 28, 2025. The trial is fully enrolled, and data collection will continue over a 12-week period, with topline results expected in the third quarter of 2025.
Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics, expressed optimism about the DSMB’s recommendation, emphasizing the unmet medical need for at-home treatment options for agitation related to bipolar and schizophrenia disorders. “We are highly motivated to address this need for millions of patients,” Mehta stated, noting that the trial data could support a potential supplemental New Drug Application (sNDA) to expand the IGALMI® label for at-home use.
Understanding the SERENITY At-Home Trial
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. The trial involves 200 patients who self-administer the medication or a placebo during episodes of agitation over a 12-week period. The study aims to address the challenges faced by patients managing agitation at home, either alone or with caregivers.
Key exploratory endpoints include modified global impression of severity (mCGIs) and clinical global impression of change (mCGI-C) assessments conducted two hours post-dosing. These assessments help evaluate the medication’s efficacy in an outpatient setting.
BXCL501: A Promising Treatment
BXCL501 is an investigational, orally dissolving film formulation of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist. While it is already approved by the FDA as IGALMI® for the acute treatment of agitation in clinical settings, its potential for at-home use is under investigation. The FDA has granted BXCL501 Breakthrough Therapy designation for agitation associated with dementia and Fast Track designation for agitation related to schizophrenia, bipolar disorders, and dementia.
BioXcel Therapeutics’ innovative approach involves leveraging artificial intelligence to identify new therapeutic indications for existing drugs, aiming to transform treatment options in neuroscience.
Implications and Future Prospects
The continuation of the SERENITY At-Home trial represents a significant step forward in addressing the needs of patients who experience agitation due to bipolar disorders or schizophrenia. The potential expansion of IGALMI® for at-home use could provide a much-needed solution for managing these conditions outside of clinical settings.
As the trial progresses, the biopharmaceutical industry and mental health professionals will be closely monitoring the outcomes. The success of this trial could pave the way for new regulatory approvals and expanded treatment options, potentially improving the quality of life for millions of patients worldwide.
BioXcel Therapeutics continues to innovate in the field of neuroscience, with a focus on developing transformative medicines. The company’s use of artificial intelligence and big data to re-innovate existing drugs highlights its commitment to advancing medical science and addressing unmet medical needs.
As the trial moves forward, stakeholders and investors will be keenly awaiting the release of topline data in Q3 2025, which could significantly impact the company’s market position and the future of mental health treatment.
