Core Insights - BioXcel Therapeutics, Inc. received a second positive recommendation from an independent Data Safety Monitoring Board (DSMB) to continue the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia [1][2] - The trial is fully enrolled with 178 patients dosed as of the May 28, 2025 cutoff date, and topline data is expected in Q3 2025 [2][7] - BXCL501 is an investigational sublingual film formulation of dexmedetomidine, currently under investigation for treating agitation in various conditions, including Alzheimer's dementia and bipolar disorders [5][6] Company Overview - BioXcel Therapeutics utilizes artificial intelligence to develop transformative medicines in neuroscience and has a subsidiary focused on immuno-oncology [13] - The company aims to address a significant unmet medical need for patients experiencing agitation related to bipolar disorders and schizophrenia, with no FDA-approved therapies currently available for at-home treatment [3][5] Trial Details - 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 in 200 patients [6] - Patients are self-administering the drug during agitation episodes over a 12-week period, with safety data collected throughout the trial [6][3] - The trial's exploratory endpoints include assessments of severity and change in agitation two hours post-dosing [6] Regulatory and Market Potential - The trial data is intended to support a potential supplemental New Drug Application (sNDA) submission to expand the IGALMI® label for at-home use [3][16] - BXCL501 has received Breakthrough Therapy designation for treating agitation associated with dementia and Fast Track designation for agitation related to schizophrenia and bipolar disorders [5]

Core Insights - BioXcel Therapeutics is progressing with its SERENITY At-Home trial, which is pivotal for evaluating the safety of BXCL501 in treating agitation associated with bipolar disorders or schizophrenia in a home setting [1][2][4] - The trial aims to enroll 200 patients and has reached 33% enrollment, with topline data expected in the second half of 2025 to support a potential supplemental new drug application (sNDA) for IGALMI® [1][3][4] Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company focused on developing transformative medicines in neuroscience using artificial intelligence [13] - The company’s investigational product, BXCL501, is an orally dissolving film formulation of dexmedetomidine, currently under investigation for various indications including agitation associated with Alzheimer's dementia and bipolar disorders [12][13] Trial Details - The SERENITY At-Home trial is a double-blind, placebo-controlled study assessing the safety of a 120 mcg dose of BXCL501 for acute treatment of agitation [4] - The trial is designed for patients with a history of agitation episodes, allowing them to self-administer the treatment during episodes over a 12-week period [4] Market Context - There are an estimated 23 million annual episodes of agitation associated with bipolar disorders or schizophrenia occurring in the home setting in the U.S., with no FDA-approved therapies currently available for acute treatment in this context [3][4] - The SERENITY At-Home trial represents a significant opportunity for BioXcel to address an unmet medical need in the treatment of agitation [3]

Core Insights - The FDA has concluded the inspection of a site involved in the TRANQUILITY II Phase 3 trial of BXCL501, indicating "Voluntary Action Indicated," which supports the reliability of the trial data [1][2] - BioXcel Therapeutics is advancing its TRANQUILITY In-Care Phase 3 trial for BXCL501, having received FDA feedback on the trial protocol [2] Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company focused on developing transformative medicines in neuroscience using artificial intelligence [5] - The company has a subsidiary, OnkosXcel Therapeutics, which is dedicated to immuno-oncology [5] Product Information - BXCL501 is an investigational orally dissolving film formulation of dexmedetomidine, currently approved as IGALMI® for specific indications [3] - BXCL501 is being investigated for the acute treatment of agitation associated with Alzheimer's dementia and bipolar disorders, with Breakthrough Therapy and Fast Track designations from the FDA [3] Trial Details - The TRANQUILITY In-Care trial is a double-blind, placebo-controlled study evaluating the efficacy and safety of a 60 mcg dose of BXCL501 over 12 weeks for agitation in Alzheimer's dementia patients [4] - The trial aims to enroll approximately 150 patients aged 55 and older residing in care facilities, with the primary endpoint being a change in the Positive and Negative Syndrome Scale-Excitatory Component total score [4]