Core Insights - BioXcel Therapeutics, Inc. is leveraging artificial intelligence to develop transformative medicines in neuroscience and has a subsidiary focused on immuno-oncology [2] Group 1: Company Overview - BioXcel Therapeutics will present a company overview at the H.C. Wainwright 27th Annual Global Investment Conference on September 8, 2025, at 4:30 p.m. Eastern Time [1] - The company utilizes a drug re-innovation approach, combining existing approved drugs with big data and proprietary machine learning algorithms to identify new therapeutic indications [2] Group 2: Upcoming Events - The presentation at the investment conference will be available via a live and archived webcast on the company's website [1]

Summary of BioXcel Therapeutics (BTAI) Conference Call - August 27, 2025 Company Overview - **Company**: BioXcel Therapeutics - **Focus**: Development of BXCL501 for the treatment of agitation associated with bipolar disorders and schizophrenia in home settings Key Points Industry Context - **Agitation in Bipolar Disorders and Schizophrenia**: - A significant unmet medical need exists as there are currently no FDA-approved therapies for treating agitation in home settings [5][8] - Most agitation episodes occur at home, leading to emergency room visits and hospitalizations [5] Clinical Trial Results - **SERENITY at Home Phase III Trial**: - The trial evaluated the 120 microgram dose of BXCL501, which was well tolerated and met its primary objective [4][6] - An estimated 57 million to 77 million agitation episodes occur annually in the U.S. [6] - The trial involved 2,628 agitation events with over 2,400 treated episodes, with patients self-administering the drug successfully [10][19] - No drug-related serious adverse events were reported, and the adverse event profile was consistent with the approved label [14][19] Efficacy and Safety - **Efficacy**: - Patients experienced consistent benefits with repeat dosing throughout the study [19] - The incidence of somnolence, the most common adverse event, was 22% and did not increase with repeated dosing [15][16] - The study showed no evidence of tachyphylaxis, indicating sustained efficacy over time [70] Regulatory and Commercial Strategy - **sNDA Submission**: - The company plans to submit a supplemental New Drug Application (sNDA) in Q1 2026 for label expansion to include home use [7][20] - The FDA has provided alignment on the protocol for the sNDA submission [36][68] Market Opportunity - **Commercialization Strategy**: - The company is developing a multi-pronged commercialization strategy, potentially including partnerships and self-commercialization [39][54] - Initial estimates suggest a need for 50 to 70 sales representatives to effectively launch the drug in the U.S. market [81] Future Development - **Potential for Other Indications**: - There is a significant opportunity to explore BXCL501 for treating agitation in Alzheimer's patients, with an estimated 100 million episodes annually [43][46] - A second confirmatory Phase III trial for Alzheimer's agitation is planned, expanding the patient population to include various care settings [44][47] Financial Considerations - **Funding and Financial Strategy**: - The positive trial data opens multiple financial options for the company, including strategic partnerships and potential royalty deals [53][54] - The company is evaluating its cash position and operational expenditures to ensure sufficient runway for upcoming initiatives [52] Additional Insights - **Patient Self-Management**: - The drug allows patients to self-manage their agitation episodes, which is crucial for maintaining home stability and caregiver relationships [64][66] - **Real-World Application**: - The drug's ease of use and tolerability may encourage its adoption in real-world settings, addressing the needs of patients experiencing agitation [80] Conclusion - The results from the SERENITY at Home trial represent a significant milestone for BioXcel Therapeutics, paving the way for potential FDA approval and commercialization of BXCL501 for home use in managing agitation associated with bipolar disorders and schizophrenia. The company is well-positioned to address a large unmet need in the market while exploring future indications for the drug.

® BioXcel Therapeutics | 555 Long Wharf Drive, 12th Floor | New Haven, CT 06511 | www.bioxceltherapeutics.com Agitation Associated with Bipolar Disorders or Schizophrenia in the At-Home Setting SERENITY At-Home Pivotal Phase 3 Safety Trial Topline Results August 27, 2025 Nasdaq: BTAI Forward-Looking Statements This presentation includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe ...

Core Insights - BioXcel Therapeutics announced that BXCL501 achieved the primary endpoint of being well tolerated in the SERENITY At-Home trial for treating agitation episodes in patients with bipolar disorders or schizophrenia [1][2][3] - The company plans to submit a supplemental New Drug Application (sNDA) in Q1 2026 for expanded usage of BXCL501 in outpatient settings without healthcare provider supervision [1][2][3] - The SERENITY At-Home trial treated over 2400 episodes of agitation, indicating a larger potential market opportunity for BXCL501 [1][2][3] Company Overview - BioXcel Therapeutics is a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience [2][30] - BXCL501 is a proprietary sublingual film formulation of dexmedetomidine, currently FDA-approved for use in medically supervised settings [2][23][24] - The SERENITY At-Home trial is a pivotal Phase 3 study designed to evaluate the safety of BXCL501 for acute treatment of agitation in the home setting [2][22] Trial Results - The SERENITY At-Home trial was a double-blind, placebo-controlled study involving 246 patients, with 168 patients (81%) completing the full 12-week trial [6][22] - The 120 mcg dose of BXCL501 was well-tolerated, with no discontinuations due to tolerability issues [5][13] - Adverse events were consistent with the approved IGALMI label, with no new or unexpected treatment-emergent adverse events reported [13][19] Market Insights - Previous estimates indicated 23 million annual episodes of agitation, but new data suggests the total addressable market could be significantly higher, with estimates ranging from 57 million to 77 million episodes annually in the U.S. [19] - The majority of agitation episodes are believed to be moderate or severe, highlighting a significant unmet medical need for effective at-home treatments [19][26] - Physicians report underdiagnosis and undertreatment of agitation episodes in community settings, with only a third of patients receiving prescription drugs [19]

Core Insights - BioXcel Therapeutics announced that BXCL501 achieved its primary endpoint in the SERENITY At-Home trial, demonstrating good tolerability for treating agitation in patients with bipolar disorders or schizophrenia at home [1][2][3] - The company plans to submit a supplemental New Drug Application (sNDA) in Q1 2026 for expanded use of BXCL501 in outpatient settings without healthcare provider supervision [1][2][3] - The SERENITY At-Home trial treated over 2400 episodes of agitation, indicating a significant market opportunity for BXCL501 [1][2][3] Company Overview - BioXcel Therapeutics is a biopharmaceutical company focused on developing transformative medicines in neuroscience using artificial intelligence [2][26] - BXCL501 is a proprietary sublingual film formulation of dexmedetomidine, currently FDA-approved for use in medically supervised settings [19][20] - The company aims to change the treatment paradigm for agitation associated with bipolar disorders and schizophrenia, addressing a significant unmet medical need in the at-home setting [3][14] Trial Details - The SERENITY At-Home trial was a double-blind, placebo-controlled study involving 246 patients over 12 weeks, evaluating the safety of a 120 mcg dose of BXCL501 [3][18] - The trial reported that 168 patients (81%) completed the full 12-week duration, with an average of 11.7 agitation episodes recorded per treated patient [6][9] - No discontinuations due to tolerability were reported in the BXCL501 arm, and the adverse event profile was consistent with the approved IGALMI® label [9][10] Market Insights - Previous estimates suggested 23 million annual episodes of agitation, but new data indicates the total addressable market could be significantly higher, with estimates of 57 million to 77 million episodes annually in the at-home setting [15][22] - Market research suggests that episodes may occur 3-4 times a month on average, with most being moderate or severe [15][22] - Physicians believe there is a significant unmet need for effective and fast-acting treatments for agitation in the community setting [22] Future Plans - BioXcel plans to leverage the positive results from the SERENITY At-Home trial to support its regulatory submission for BXCL501, aiming to provide patients with access to treatment in the home setting [3][13] - The company will continue to analyze the full dataset from the trial and present additional results at upcoming medical meetings and conferences [16][12]

Company to host conference call at 8 a.m. ET, August 27, 2025NEW HAVEN, Conn., Aug. 26, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that it will host an investor call and live webcast at 8 a.m. ET, August 27, 2025 to review topline data from the SERENITY At-Home Pivotal Phase 3 trial evaluating the safety of BXCL501, the Company’s proprietary, sublingual ...

Data from more than 2,600 agitation episodes collected Topline data readout is on track for August BXCL501 was granted Fast Track Designation for the acute treatment of agitation associated with bipolar disorders or schizophrenia. There are no FDA-approved therapies for the acute treatment of agitation in the at- home setting. About the SERENITY At-Home Phase 3 Trial The trial was designed to study 200 patients with a history of agitation episodes despite being on stable treatment for their underlying bipol ...

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]

Summary of BioXcel Therapeutics (BTAI) Conference Call Company Overview - BioXcel Therapeutics is a publicly traded company listed on NASDAQ under the ticker symbol BTAI, focusing on AI-driven drug re-innovation [1][2] - The company has successfully brought to market a drug named EGALMI, a sublingual formulation of dexmedetomidine for treating acute agitation in neuropsychiatric contexts [4] Clinical Trials and Drug Development - The ongoing pivotal Phase III trial, SERENITY AT HOME, is designed to evaluate the safety and efficacy of BXCL501 in a home setting for patients with schizophrenia and bipolar disorder [6][8] - The trial involves 200 patients, with safety as the primary endpoint and exploratory efficacy endpoints [6][7] - The trial is on track to announce top-line data shortly, having passed two data safety monitoring board reviews [9][10] - The expected safety profile is comparable to that observed in institutional settings, where the drug has shown a favorable safety and tolerability profile [12][14] Market Opportunity - The outpatient market for acute agitation treatment is significantly larger than the institutional market, with estimates suggesting up to 140-150 million episodes of agitation annually when including both neuropsychiatric and dementia-related contexts [36][69] - The inpatient market is estimated at around 60 million episodes per year, while the outpatient market is estimated at 23 million episodes, which may be conservative [36][37][69] - The company anticipates that the at-home setting market could be three to four times larger than the institutional market [39] Regulatory Process - BioXcel is preparing for a meeting with the FDA to discuss the sNDA application, focusing on operational aspects and confirming the sufficiency of the clinical program for filing [30][31] - The timeline for preparing the sNDA is estimated to take about four to six months, with the company already working on nonclinical sections [32][33] Competitive Landscape - BXCL501 is positioned to treat breakthrough agitation episodes, differentiating itself from other treatments like Rexulti, which is approved for chronic agitation but not for acute episodes [45][46][49] - The company believes it will occupy a distinct niche in the market, particularly for treating acute agitation in patients with schizophrenia and bipolar disorder [49] Future Directions - BioXcel is exploring additional indications for BXCL501, including agitation in Alzheimer's patients, with a focus on conducting further studies in both in-care and at-home settings [50][52] - The company is leveraging its AI-driven platform to identify and develop new drug candidates, aiming to expand its pipeline beyond BXCL501 [88][90] Financial Position - As of the latest reports, BioXcel has a strong balance sheet with sufficient capital to support the completion of the SERENITY AT HOME study and the preparation of the sNDA [78][80] - The company is focused on maintaining capital efficiency while exploring strategic partnerships to enhance market access and commercialization efforts [72][73] Conclusion - BioXcel Therapeutics is at a pivotal moment with the upcoming data readout from the SERENITY AT HOME trial and its interaction with the FDA, which could significantly impact its market position and shareholder value [96][95]

Core Viewpoint - BioXcel Therapeutics, Inc. is leveraging artificial intelligence to develop innovative medicines in the field of neuroscience and has announced a virtual fireside chat featuring its CEO, Vimal Mehta, on August 14, 2025 [1][2]. Company Overview - BioXcel Therapeutics, Inc. (Nasdaq: BTAI) focuses on utilizing artificial intelligence for the development of transformative medicines in neuroscience [2]. - The company has a subsidiary, OnkosXcel Therapeutics, which is dedicated to immuno-oncology drug development [2]. - BioXcel employs a drug re-innovation strategy that combines existing approved drugs and clinically validated candidates with big data and proprietary machine learning algorithms to discover new therapeutic uses [2].