PresentationGreetings, and welcome to the BioXcel Therapeutics. [Operator Instructions] Please note that this call is being recorded. I will now turn the conference over to our host, Vimal Mehta. Thank you. You may begin.Vimal MehtaFounder, CEO, President, & Director Thank you, operator. Good afternoon, everyone, and thank you for joining us today. I'd like to start by welcoming our distinguished participants. First, Dr. Leslie Citrome, Clinical Professor of Psychiatry and Behavioral Sciences at New York Me ...

BioXcel Therapeutics, Inc. BTAI on Wednesday released topline exploratory efficacy data from the SERENITY At-Home Pivotal Phase 3 safety trial, which demonstrated that BXCL501 had continued effects and consistent benefit with repeat dosing.The trial in the home setting evaluated a 120 mcg dose of BXCL501, the company’s proprietary, sublingual film formulation of dexmedetomidine, Igalmi, for agitation associated with bipolar disorders or schizophrenia.Recently, BioXcel reported topline safety results and is ...

Summary of BioXcel Therapeutics Conference Call Company Overview - **Company**: BioXcel Therapeutics (NasdaqCM:BTAI) - **Product**: IGALMI (dexmedetomidine) sublingual film - **Focus**: Treatment of acute agitation associated with schizophrenia and bipolar disorder Key Industry Insights - **Market Need**: There is a significant burden of agitation in patients with bipolar disorder and schizophrenia, with an estimated 60 to 80 million episodes occurring among 23 million patients in home settings [10] - **Current Treatment Gap**: Prior to IGALMI, there were no FDA-approved therapies for at-home treatment of agitation associated with these disorders [11] Core Findings from SERENITY At-Home Trial - **Trial Design**: The SERENITY At-Home trial was a double-blind, placebo-controlled study evaluating the safety and efficacy of a 120 microgram dose of IGALMI in an at-home setting [21] - **Patient Demographics**: Included 200 patients with a history of agitation, primarily diagnosed with schizophrenia or bipolar disorder, aged 18 to 75 [23][25] - **Efficacy Results**: - The trial demonstrated a significant reduction in agitation symptoms, with a responder rate of 40% for placebo and double that for IGALMI [14][15] - The number needed to treat (NNT) was 3, indicating that for every three patients treated, one additional patient experienced a significant reduction in agitation [17] - Complete resolution of agitation was significantly higher with IGALMI compared to placebo [28] Safety and Tolerability - **Adverse Events**: The safety profile was consistent with previous studies, showing no serious adverse events related to IGALMI. Somnolence rates were 22% for IGALMI and 6% for placebo in single doses [27] - **Repeated Dosing**: The drug was well tolerated with repeated dosing, maintaining efficacy without increased adverse events over time [30] Future Outlook - **Regulatory Submission**: A supplemental NDA for at-home use is planned for submission in Q1 2026 [12] - **Market Potential**: If approved, IGALMI could provide a new treatment option for managing acute agitation at home, potentially reducing hospital admissions and associated costs [70] Additional Considerations - **Patient Compliance**: The likelihood of patient compliance with at-home use is high if the drug is effective in managing agitation [72] - **Cost Sensitivity**: While cost may be a concern for inpatient providers, outpatient providers are generally less sensitive to price as long as the drug is accessible [70] Conclusion - BioXcel Therapeutics is positioned to address a significant unmet need in the management of acute agitation with IGALMI, supported by promising trial results and a clear regulatory pathway ahead. The focus on at-home treatment could transform patient care and reduce the burden on healthcare systems.

SERENITY At-Home Trial Overview - The SERENITY At-Home trial evaluated the safety and tolerability of BXCL501 (120 mcg) for agitation associated with bipolar disorder or schizophrenia in the at-home setting[2, 27, 28] - The trial design was a double-blind, placebo-controlled study with a 1:1 randomization to BXCL501 (N=100) or placebo (N=100) over a 12-week period[27] - The study included patients aged 18-75 years with bipolar I or II disorder, schizophrenia, schizoaffective, or schizophreniform disorder on a stable psychotropic regimen[30] Key Results - The trial collected data from 2,628 agitation episodes in 215 patients, with 2,437 episodes treated in 208 patients[21, 22, 36] - 168 (81%) of treated patients completed the full 12-week study, with an average of 117 agitation episodes recorded per treated patient[36] - No drug-related serious adverse events (SAEs), falls, or syncopes were reported in the BXCL501 arm[23, 37] - The adverse event profile was consistent with the approved IGALMI® label and other clinical trials in institutional settings[23, 37] - Somnolence was the most common treatment-emergent adverse event (TEAE) in the BXCL501 group, occurring in 23 (225%) of patients after the first dose and 161 (139%) of all doses[38] Clinical Benefit - BXCL501 demonstrated a significant mean reduction in mCGI-S score from baseline compared to placebo at 2 hours across 2,433 treated episodes (P<005)[41] - Complete resolution of agitation was significantly higher with BXCL501 compared to placebo across agitation episode severity (P<0001)[41] - For fully resolved episodes by baseline severity, BXCL501 showed higher percentages than placebo across mild, moderate, and severe agitation episodes[43] Next Steps - BioXcel Therapeutics plans to prepare and submit an sNDA (supplemental New Drug Application) in Q1 2026 for label expansion of IGALMI®[12, 23]

BXCL501 demonstrated a significant mean reduction in mCGI-S score from baseline compared to placebo at 2 hours across 2,433 treated episodes (p<.05) Complete resolution of agitation was significantly higher with BXCL501 compared to placebo across agitation episode severity (p<.0001) BXCL501 showed a similar reduction in agitation symptoms over both the duration of the trial and number of treated episodes, demonstrating continued effects and consistent benefit with repeat dosing of BXCL501 Based on the large ...

Summary of BioXcel Therapeutics FY Conference Call Company Overview - **Company**: BioXcel Therapeutics (NasdaqCM:BTAI) - **Founded**: Became a public company in March 2018 - **Focus**: Development of transformative medicines in neuroscience utilizing artificial intelligence platforms [2][3] Key Product Information - **Product**: IGALMI - **Indication**: Acute treatment of agitation related to schizophrenia and bipolar disorder - **Current Approval**: Approved under medical supervision, not available in retail settings [3][4] - **Market Potential**: Initially estimated at 23 million episodes, revised to 57 to 77 million episodes in home settings [5][9] Recent Developments - **Phase 3 Trial**: Announced pivotal Phase 3 data for IGALMI, with plans to file a supplemental New Drug Application (sNDA) [2][8] - **Alzheimer's Agitation Program**: Positive Phase 3 trial completed; FDA has requested another confirmatory Phase 3 trial [5][20] Market Expansion Strategy - **Home Setting**: Focus on expanding IGALMI's use into home settings, targeting a larger patient population [5][9] - **Commercial Strategy**: Developing a strategy to bring IGALMI to patients, with potential approval in 2026 [9][19] Clinical Trial Insights - **SERENITY At-Home Trial**: Completed safety trial using the lowest approved dose of IGALMI; demonstrated safety and tolerability in home settings [8][16] - **Patient Data**: 2,437 agitation episodes treated in 208 patients; average of 11.7 episodes over 12 weeks [15][16] - **Safety Profile**: No treatment-emergent adverse events leading to discontinuation; consistent with approved IGALMI label [16][18] Future Plans - **sNDA Submission**: Expected in Q1 of 2026; non-clinical sections completed, clinical and CMC sections in progress [9][18] - **Alzheimer's Trial**: Plans to conduct a second confirmatory Phase 3 trial for Alzheimer's agitation in in-care settings [20] Market Opportunity - **Agitation as a Medical Need**: Significant unmet medical need for agitation treatment, especially in home settings; no FDA-approved therapies currently available for home use [10][17] - **Patient Distribution**: Approximately 50% of agitation episodes occur in in-care settings, highlighting the need for effective home treatment options [19][20] Conclusion - **Overall Outlook**: Positive momentum with ongoing trials and market expansion strategies; significant potential for IGALMI in both schizophrenia and Alzheimer's agitation [20][21]

NEW HAVEN, Conn., Sept. 04, 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 present a company overview at the H.C. Wainwright 27th Annual Global Investment Conference to be held September 8 - 10, 2025. Date: Monday, September 8, 2025Time: 4:30 p.m. Eastern TimeWebcast Link: HERE The live and archived ...

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 ...