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 ...

BXCL501 achieved SERENITY At-Home’s primary endpoint of being well tolerated in the at-home treatment of agitation episodes in patients with bipolar disorders or schizophrenia No discontinuations for tolerability in the BXCL501 arm While not the primary objective of the SERENITY At-Home trial, the preliminary results demonstrate continued effects and consistent benefit with repeat dosing across the course of the trial Following previously disclosed positive FDA feedback, BioXcel plans to submit a sNDA in Q1 ...

BXCL501 achieved SERENITY At-Home’s primary endpoint of being well tolerated in the at-home treatment of agitation episodes in patients with bipolar disorders or schizophrenia No discontinuations for tolerability in the BXCL501 arm While not the primary objective of the SERENITY At-Home trial, the preliminary results demonstrate continued effects and consistent benefit with repeat dosing across the course of the trial Following previously disclosed positive FDA feedback, BioXcel plans to submit a sNDA in Q1 ...

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 ...

The primary purpose of the planned meeting was to gain alignment with the FDA regarding the content and format of the Company's planned supplemental New Drug Application (sNDA) submission for the at-home (outpatient) use of BXCL501, including the clinical, nonclinical, and chemistry and manufacturing and controls (CMC) requirements. The Company concludes that the objectives of the pre-sNDA meeting have been accomplished based on the FDA's written responses and has determined that the meeting, originally sch ...

NEW HAVEN, Conn., Aug. 13, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics, will participate in a virtual fireside chat on Thursday, August 14, 2025 at 1:00 p.m. ET as part of the H.C. Wainwright & Co. "HCW@Home" series. Date: Tuesday, August 14, 2025 Time: 1 p.m. Eastern Time Webcast Link: Register here Ab ...

Core Insights - BioXcel Therapeutics is set to release topline data from its SERENITY At-Home Phase 3 trial for the acute treatment of agitation associated with bipolar disorders or schizophrenia in August [1][2] - The company is preparing for a meeting with the FDA to support a potential supplemental New Drug Application (sNDA) for label expansion of IGALMI in the at-home setting [1][2] - Recent research indicates potential applications of BXCL501 in chronic conditions, enhancing its market prospects [1][4] Clinical Trials - 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 in bipolar disorders or schizophrenia at home [4][15] - The trial enrolled over 200 patients across 22 sites, with a balanced distribution between bipolar disorder and schizophrenia populations [6] - The TRANQUILITY In-Care Phase 3 trial is also underway, focusing on agitation associated with Alzheimer's dementia [6][16] Financial Performance - For Q2 2025, net revenue from IGALMI was $120 thousand, a significant decrease from $1.1 million in Q2 2024 [8] - Cost of Goods Sold for Q2 2025 was $107 thousand, up from $62 thousand in the same period last year [9] - Research and Development (R&D) expenses rose to $10.3 million in Q2 2025, compared to $8.0 million in Q2 2024, primarily due to increased clinical trial activities [10] Losses and Cash Position - The company reported an operating loss of $15.9 million and a net loss of $19.2 million for Q2 2025, compared to an operating loss of $17.3 million and a net loss of $8.3 million in Q2 2024 [12] - As of June 30, 2025, cash and cash equivalents totaled $18.6 million, not including an additional $11.5 million from sales and $3.6 million from warrant exercises [13][30]

NEW HAVEN, Conn., Aug. 11, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics, will participate in a fireside chat at the Canaccord Genuity 45th Annual Growth Conference in Boston on Tuesday, Aug. 12, 2025, at 12 p.m. ET. To access a webcast of the fireside chat, please visit theEvents & Presentations page und ...