Core Insights - BioXcel Therapeutics, Inc. will host an investor call on August 27, 2025, to review topline data from the SERENITY At-Home Pivotal Phase 3 trial for BXCL501, aimed at treating agitation associated with bipolar disorders or schizophrenia in a home setting [1][2] Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company that leverages artificial intelligence to develop transformative medicines in neuroscience [11] - The company’s subsidiary, OnkosXcel Therapeutics, focuses on immuno-oncology [11] SERENITY At-Home Phase 3 Trial - The trial involved 200 patients with agitation episodes despite stable treatment for bipolar disorder or schizophrenia, requiring self-administration of 120 mcg of BXCL501 or placebo during episodes over a 12-week period [3] - Safety data was collected, and patients or caregivers completed a modified global impression of severity (mCGIs) two hours post-dosing as an exploratory endpoint [3] Product Information - BXCL501 is an investigational sublingual film formulation of dexmedetomidine, currently approved for use under medical supervision as IGALMI for acute treatment of agitation associated with schizophrenia and bipolar disorder [4][5] - BXCL501 is also being investigated for agitation associated with Alzheimer's dementia and has received Breakthrough Therapy and Fast Track designations from the FDA for various indications [4] Upcoming Events - The topline data from the SERENITY trial will be disseminated in a press release and presented during the investor call/webcast [2]

Core Insights - BioXcel Therapeutics has completed the database lock for its SERENITY At-Home pivotal Phase 3 safety trial, with topline results expected in August 2025 [2][3] - The trial aims to evaluate the safety of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in a home setting, addressing a significant unmet medical need [3][5] Company Overview - BioXcel Therapeutics is a biopharmaceutical company focused on developing transformative medicines in neuroscience using artificial intelligence [2][15] - The company has a subsidiary, OnkosXcel Therapeutics, which is dedicated to immuno-oncology [15] Trial Details - The SERENITY At-Home trial is a double-blind, placebo-controlled study involving 200 patients with a history of agitation episodes while on stable treatment for bipolar disorder or schizophrenia [6][8] - Patients self-administered a 120 mcg dose of BXCL501 or placebo during agitation episodes over a 12-week period, with safety data collected throughout [6][8] Collaboration and Oversight - BioXcel partnered with Worldwide Clinical Trials (WCT) to conduct the SERENITY At-Home trial, implementing robust oversight controls to ensure patient safety [4] - Independent industry experts were engaged for additional oversight of high enrolling sites to ensure compliance with Good Clinical Practice (GCP) [4] Product Information - BXCL501 is an investigational orally dissolving film formulation of dexmedetomidine, currently under investigation for treating agitation associated with Alzheimer's dementia and bipolar disorders in the at-home setting [7] - BXCL501 has received Fast Track Designation from the FDA for the acute treatment of agitation associated with bipolar disorders and schizophrenia [5][9] Patient Enrollment and Data Collection - The trial enrolled over 200 patients across 22 sites nationwide, with no single site contributing more than 11% of the total patient population [8] - Data from more than 2,600 agitation episodes was collected during the trial [8]

Core Insights - BioXcel Therapeutics has achieved alignment with the FDA on the supplemental New Drug Application (sNDA) package to expand the IGALMI label for at-home use, with submission expected in Q1 2026 [1][2][3] Group 1: FDA Interaction and Regulatory Progress - The company received positive feedback from the FDA during a pre-sNDA meeting, confirming that the planned regulatory package is sufficient for submission [2][4] - The objectives of the pre-sNDA meeting were accomplished, leading to the cancellation of the originally scheduled meeting [3] - The acceptance of the sNDA will depend on the FDA's review of the complete filing [3] Group 2: Clinical Trials and Product Details - The SERENITY At-Home Phase 3 trial is designed to evaluate the safety of a 120 mcg dose of BXCL501 for treating agitation associated with bipolar disorders or schizophrenia in an at-home setting [6] - The trial involves 200 patients and is a double-blind, placebo-controlled study, with topline data expected soon [4][6] - IGALMI is currently FDA-approved for acute treatment of agitation in medically supervised settings, available in 120 mcg and 180 mcg doses [4][9] Group 3: Market Potential and Designation - BXCL501 has received Fast Track Designation for the acute treatment of agitation associated with bipolar disorders and schizophrenia, with no FDA-approved therapies for at-home treatment currently available [5][8] - The company aims to address the urgent needs of patients suffering from bipolar disorder or schizophrenia, potentially transforming the treatment paradigm [4][5]

Core Insights - BioXcel Therapeutics has completed the last patient last visit (LPLV) in the pivotal Phase 3 SERENITY At-Home clinical trial, with topline data expected to be released this month to support a supplemental New Drug Application (sNDA) for IGALMI in the at-home setting [1][3] Group 1: Clinical Trial Details - The SERENITY At-Home Phase 3 trial is a double-blind, placebo-controlled study evaluating the safety of a 120 mcg dose of BXCL501 for treating agitation associated with bipolar disorders or schizophrenia in an at-home setting [2][4] - The trial enrolled over 200 patients across 22 sites nationwide, ensuring no single site enrolled more than 11% of the total patient population, with a balanced distribution between bipolar disorders and schizophrenia [2][4] Group 2: Market Opportunity - There are approximately 23 million episodes of agitation related to bipolar disorders or schizophrenia annually in the U.S. that occur at home, with no FDA-approved therapies currently available for acute treatment in this setting [3][4] - The company aims to provide a much-needed at-home treatment option for individuals experiencing such agitation, marking a significant milestone in its development efforts [3] Group 3: Product Information - BXCL501, known as IGALMI, is an investigational orally dissolving film formulation of dexmedetomidine, currently approved for use under healthcare supervision for acute treatment of agitation associated with schizophrenia and bipolar disorder [5][6] - The product has received Breakthrough Therapy designation from the FDA for treating agitation associated with dementia and Fast Track designation for agitation related to schizophrenia and bipolar disorders [5]

Core Points - BioXcel Therapeutics is focused on obtaining FDA approval for outpatient use of IGALMI® for treating agitation associated with bipolar disorders or schizophrenia [1][2] - A pre-sNDA meeting with the FDA is scheduled for August 20, 2025, to align on the submission process [2] - The SERENITY At-Home Phase 3 trial is nearing completion, with top-line results expected to be reported this quarter [3] Company Overview - BioXcel Therapeutics, Inc. utilizes artificial intelligence to develop transformative medicines in neuroscience [1][13] - The company has received Breakthrough Therapy designation for BXCL501 for treating agitation associated with dementia and Fast Track designation for schizophrenia and bipolar disorders [5] Clinical Development - The SERENITY At-Home trial is a double-blind, placebo-controlled study evaluating the safety of a 120 mcg dose of BXCL501 for acute agitation in the home setting [6] - The trial involves 200 patients with a history of agitation episodes, self-administering the treatment during episodes over a 12-week period [6] Market Opportunity - An estimated 23 million annual episodes of agitation associated with bipolar disorders or schizophrenia occur in the at-home setting in the U.S., with no FDA-approved therapies currently available for this treatment [3][17]

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]