Bioxcel Therapeutics Announces That An Independent Data Safety Monitoring Board Recommended That The Serenity At-Home Pivotal Phase 3 Safety Trial Of BXCL501 For Acute Treatment of Agitation Associated With Bipolar Disorders Or Schizophrenia Continue Without Modification.

- BioXcel Therapeutics, Inc. (NASDAQ:BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that an independent Data Safety Monitoring Board (DSMB) recommended that the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for acute treatment of agitation associated with bipolar disorders or schizophrenia continue without modification.

The DSMB recommendation followed a review of unblinded safety data from the first 115 patients dosed as of the May 2, 2025 cutoff date. The trial is fully enrolled and collection of data over the 12-week period is continuing.

"We are pleased with the favorable DSMB meeting outcome and are excited about the upcoming data readout for our first at-home trial with BXCL501," said Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics. "Results are intended to help support a potential sNDA submission for label expansion of IGALMI® in the at-home setting — a sizeable unmet medical need given there are no FDA-approved therapies for bipolar or schizophrenia-related agitation in this environment."

The SERENITY At-Home Phase 3 trial is designed as a double-blind, placebo-controlled study to evaluate the safety of a 120 mcg dose of BXCL501 in 200 patients for acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting.

• Trial enrollment is complete:
  • More than 205 patients have been dosed.
  • More than 150 patients have received multiple doses for agitation over the 12-week trial period.
• Topline data expected in Q3 2025.

Additional information on the SERENITY At-Home trial is included in a corporate presentation in the Investors section of the Company's website: bioxceltherapeutics.com.

About BXCL501

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.

About the SERENITY At-Home Phase 3 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 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting. The trial is designed to evaluate 200 patients with a history of agitation episodes residing at home either alone or with caregivers/informants. Patients are self-administering 120 mcg of BXCL501 or placebo when agitation episodes occur over the 12-week trial period, with safety data (adverse events) collected during the trial. In addition, patients or caregivers/informants will complete 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.

About IGALMI® (dexmedetomidine) sublingual film  

  

INDICATION 

IGALMI® (dexmedetomidine) sublingual film is a prescription medicine, administered under the supervision of a health care provider, that is placed under the tongue or behind the lower lip and is used for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults. The safety and effectiveness of IGALMI has not been studied beyond 24 hours from the first dose. It is not known if IGALMI is safe and effective in children. 

IMPORTANT SAFETY INFORMATION 

IGALMI can cause serious side effects, including: 

• Decreased blood pressure, low blood pressure upon standing, and slower than normal heart rate, which may be more likely in patients with low blood volume, diabetes, chronic high blood pressure, and older patients. IGALMI is taken under the supervision of a healthcare provider who will monitor vital signs (like blood pressure and heart rate) and alertness after IGALMI is administered to help prevent falling or fainting. Patients should be adequately hydrated and sit or lie down after taking IGALMI and instructed to tell their healthcare provider if they feel dizzy, lightheaded, or faint. 
• Heart rhythm changes (QT interval prolongation). IGALMI should not be given to patients with an abnormal heart rhythm, a history of an irregular heartbeat, slow heart rate, low potassium, low magnesium, or taking other drugs that could affect heart rhythm. Taking IGALMI with a history of abnormal heart rhythm can increase the risk of torsades de pointes and sudden death. Patients should be instructed to tell their healthcare provider immediately if they feel faint or have heart palpitations. 
• Sleepiness/drowsiness. Patients should not perform activities requiring mental alertness, such as driving or operating hazardous machinery, for at least 8 hours after taking IGALMI. 
• Withdrawal reactions, tolerance, and decreased response/efficacy. IGALMI was not studied for longer than 24 hours after the first dose. Physical dependence, withdrawal symptoms (e.g., nausea, vomiting, agitation), and decreased response to IGALMI may occur if IGALMI is used longer than 24 hours.