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, Inc. announced that the independent Data Safety Monitoring Board (DSMB) recommended the continuation of the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 without modification, with topline data expected in Q3 2025 [2][9] Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company focused on utilizing artificial intelligence to develop transformative medicines in neuroscience [15] - The company is also involved in immuno-oncology through its subsidiary, OnkosXcel Therapeutics [15] 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 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting [4][7] - The trial is fully enrolled with more than 205 patients dosed, and over 150 patients have received multiple doses during the 12-week trial period [3][9] Product Information - BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, which is under investigation for the acute treatment of agitation associated with Alzheimer's dementia, bipolar disorders, and schizophrenia [6] - BXCL501 has received Breakthrough Therapy designation from the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for agitation associated with schizophrenia and bipolar disorders [6] Future Prospects - The results from the SERENITY trial are intended to support a potential supplemental New Drug Application (sNDA) submission for label expansion of IGALMI in the at-home setting, addressing a significant unmet medical need [4]

Core Viewpoint - BioXcel Therapeutics, Inc. has received approval from the Nasdaq Hearings Panel to continue its listing on The Nasdaq Stock Market, with a deadline to regain compliance by September 16, 2025 [1][2]. Company Overview - BioXcel Therapeutics is a biopharmaceutical company that leverages artificial intelligence to develop innovative medicines in the field of neuroscience [12]. - The company is also focused on immuno-oncology through its subsidiary, OnkosXcel Therapeutics [12]. Compliance and Listing - The Nasdaq Panel reviewed BioXcel's compliance plan during a hearing on May 1, 2025, and granted an extension for compliance with the MVLS Rule [2]. - The company is committed to executing its compliance plan to meet the necessary requirements within the specified timeframe [2][3]. Product Development - BioXcel is advancing multiple strategic initiatives, including the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501, aimed at supporting a supplemental New Drug Application (sNDA) to expand the IGALMI label [3]. - BXCL501 is an investigational sublingual film formulation of dexmedetomidine, currently under investigation for treating agitation associated with Alzheimer's dementia and bipolar disorders [4]. SERENITY At-Home Phase 3 Trial - The SERENITY At-Home trial is a double-blind, placebo-controlled study evaluating the safety of a 120 mcg dose of BXCL501 for treating agitation in patients with bipolar disorders or schizophrenia [5]. - The trial involves 200 patients self-administering BXCL501 or placebo during agitation episodes over a 12-week period, with safety data collected throughout [5]. IGALMI Overview - IGALMI (dexmedetomidine) is a prescription medicine used for the acute treatment of agitation associated with schizophrenia and bipolar disorder in adults [6]. - The safety and effectiveness of IGALMI have not been established beyond 24 hours from the first dose [6]. Safety Information - IGALMI can cause serious side effects, including decreased blood pressure and slower heart rate, particularly in patients with certain pre-existing conditions [7][8]. - Common side effects reported in clinical studies include sleepiness, dizziness, and dry mouth [9].

Core Insights - BioXcel Therapeutics is advancing its SERENITY At-Home pivotal Phase 3 trial, with patient enrollment exceeding 60% of the required 200 patients for evaluating BXCL501 in treating agitation associated with bipolar disorders or schizophrenia [1][2][8] - The company has strengthened its cash position to approximately $35 million following a $14 million equity financing, which will support the SERENITY program [6][15] - Topline data from the SERENITY trial is expected in the second half of 2025, which may support a supplemental New Drug Application (sNDA) for label expansion of IGALMI in the at-home setting [1][2][8] Financial Performance - Net revenue from IGALMI for Q4 2024 was $366,000, a slight decrease from $376,000 in Q4 2023, while full-year revenue increased to $2.3 million from $1.4 million in 2023 [7][9] - Cost of Goods Sold (COGS) for Q4 2024 was $832,000, up from $714,000 in Q4 2023, primarily due to higher reserves for excess or obsolete inventory [10] - Research and Development (R&D) expenses decreased to $5.9 million in Q4 2024 from $9.9 million in Q4 2023, and full-year R&D expenses were $30.4 million, down from $84.3 million in 2023 [12] - Selling, General and Administrative (SG&A) expenses for Q4 2024 were $4.1 million, compared to $9.6 million in Q4 2023, with full-year SG&A expenses at $34.5 million, down from $83.4 million in 2023 [13][14] - The company reported a net loss of $10.9 million for Q4 2024, an improvement from a net loss of $22.3 million in Q4 2023, and a full-year net loss of $59.6 million compared to $179.1 million in 2023 [15] Clinical Programs - 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 for acute treatment of agitation in patients with bipolar disorders or schizophrenia [8][18] - The TRANQUILITY In-Care Phase 3 trial is also underway, focusing on the efficacy and safety of a 60 mcg dose of BXCL501 for agitation associated with Alzheimer's dementia [19] - A Data Safety Monitoring Board (DSMB) is planned to assess safety during the SERENITY trial [8] Market Potential - The SERENITY program aims to address a significant unmet medical need, with an estimated 23 million episodes of agitation related to bipolar and schizophrenia occurring annually in the U.S. [2] - The company continues to supply IGALMI through existing distribution channels with minimal commercial support, indicating a focus on expanding market presence [5]

Core Insights - BioXcel Therapeutics, Inc. has successfully raised $14 million in equity financing, resulting in a cash position of approximately $35 million to support its clinical trials [1][2] - The company is advancing its SERENITY At-Home Phase 3 trial, which aims to evaluate the safety of BXCL501 for treating agitation associated with bipolar disorders or schizophrenia in a home setting [1][3] - Topline data from the trial is expected in the second half of 2025, which may support a supplemental New Drug Application (sNDA) for expanding the label of IGALMI for at-home use [2][3] Company Overview - BioXcel Therapeutics is a biopharmaceutical company focused on developing transformative medicines in neuroscience using artificial intelligence [1][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] - IGALMI (dexmedetomidine) is a prescription medicine used for the acute treatment of agitation in adults with schizophrenia and bipolar disorder [4][12] SERENITY At-Home Phase 3 Trial - The trial is a double-blind, placebo-controlled study enrolling 200 patients with a history of agitation episodes, who will self-administer BXCL501 or placebo during the trial [3] - Safety data will be collected throughout the 12-week trial period, with exploratory endpoints including modified global impression scales to assess treatment effectiveness [3] Financial Position - Following the recent equity financing, the company is well-positioned financially with $35 million in cash to support ongoing clinical trials and operational needs [1][2]

Core Insights - BioXcel Therapeutics is progressing with its SERENITY At-Home trial, having reached 33% enrollment of the targeted 200 patients, aimed at evaluating the safety of BXCL501 for treating agitation associated with bipolar disorders or schizophrenia in a home setting [1][2][3] - Topline data results are anticipated in the second half of 2025, which will support a potential supplemental new drug application (sNDA) for expanding the label of IGALMI [1][3] Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company focused on developing transformative medicines in neuroscience using artificial intelligence [13] - The company’s approach involves leveraging existing approved drugs and big data to identify new therapeutic indications [13] SERENITY At-Home 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 in home settings [4] - The trial will collect safety data over a 12-week period, with patients self-administering the drug during agitation episodes [4] Market Context - There are approximately 23 million annual episodes of agitation related to bipolar disorders or schizophrenia occurring in the U.S. home setting, with no FDA-approved therapies currently available for acute treatment in this context [3][4]

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]

Core Viewpoint - The FDA has concluded the inspection of a site involved in BioXcel Therapeutics' TRANQUILITY II trial, indicating that the data from this trial is reliable and supports the company's potential sNDA submission for BXCL501 [1][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] BXCL501 Overview - BXCL501 is an investigational sublingual film formulation of dexmedetomidine, currently approved as IGALMI by the FDA [3] - It is being investigated for the acute treatment of agitation associated with Alzheimer's dementia and bipolar disorders or schizophrenia in at-home settings [3] - BXCL501 has received Breakthrough Therapy designation for agitation associated with dementia and Fast Track designation for agitation related to schizophrenia and bipolar disorders [3] TRANQUILITY In-Care Phase 3 Trial - 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 with varying degrees of dementia [4] - The primary endpoint is a change in the Positive and Negative Syndrome Scale-Excitatory Component total score at two hours post-first dose [4]