Completes clinical trials required by FDA for sNDA submission planned for the first quarter of 2026NEW HAVEN, Conn., Oct. 14, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced positive results from the correlation study related to exploratory efficacy outcomes from the SERENITY At-Home trial. The results, along with the data from the SERENITY At-Home trial, wil ...

Core Insights - BioXcel Therapeutics, Inc. is celebrating a significant milestone by ringing the Nasdaq Stock Market Closing Bell, highlighting its progress in addressing unmet needs in psychiatry, particularly in treating agitation associated with bipolar disorders and schizophrenia [1][2]. Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company that leverages artificial intelligence to develop transformative medicines in neuroscience. The company also has a subsidiary, OnkosXcel Therapeutics, focused on immuno-oncology [3]. Recent Developments - The company has successfully completed its SERENITY At-Home pivotal trial, moving closer to its goal of redefining agitation management and bringing its product IGALMI® to patients in their homes [2]. - The Nasdaq Closing Bell ceremony is scheduled for October 14, 2025, and will be broadcast live [2]. Strategic Focus - BioXcel aims to transform the standard of care in neuropsychiatry by combining technology and compassion to deliver meaningful changes in patient care [2].

Core Insights - BioXcel Therapeutics, Inc. released topline exploratory efficacy data from the SERENITY At-Home Pivotal Phase 3 safety trial, showing that BXCL501 has continued effects and consistent benefits with repeat dosing [1] - The trial evaluated a 120 mcg dose of BXCL501 for agitation associated with bipolar disorders or schizophrenia, demonstrating significant efficacy compared to placebo [2] Efficacy Results - Across 2,433 treated episodes, BXCL501 showed a significant mean reduction in the modified Clinical Global Impression–Severity (mCGI-S) score from baseline compared to placebo at 2 hours [2] - Complete resolution of agitation symptoms was observed in 50% of patients in the BXCL501 arm compared to 33% in the placebo group [3] - Severe agitation episodes fully resolved in 61% of cases in the BXCL501 arm versus 18% on placebo, while moderate and mild episodes resolved in 43% and 60% of cases, respectively, compared to 34% and 40% on placebo [4] Dosage Impact - A mean reduction in mCGI-S score of 1.2 was noted after the first 12 doses, increasing to a mean reduction of 1.4 after 13 or more doses of BXCL501 [5] - Igalmi, the formulation of BXCL501, is currently FDA-approved for the acute treatment of agitation associated with bipolar I or II disorder or schizophrenia in medically supervised settings [5] Market Reaction - BioXcel Therapeutics shares increased by 3.07% to $3.69 at the time of publication, trading within a 52-week range of $1.17 to $13.36 [6]

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]

Core Insights - BioXcel Therapeutics announced positive topline exploratory efficacy data from the SERENITY At-Home Pivotal Phase 3 safety trial for BXCL501, indicating significant benefits in treating agitation associated with bipolar disorders and schizophrenia [1][3][5] Efficacy Results - BXCL501 demonstrated a significant mean reduction in the modified Clinical Global Impression–Severity (mCGI-S) score from baseline compared to placebo at 2 hours across 2,433 treated episodes (p<.05) [6] - Complete resolution of agitation symptoms was significantly higher with BXCL501 compared to placebo, with an overall resolution of 50% in the BXCL501 arm versus 33% on placebo (p <.0001) [7] - The mean reduction in mCGI-S score was maintained across repeated dosing, with a mean reduction of 1.2 after the first 12 doses and 1.4 after 13 or more doses [8] - The reduction in agitation symptoms was consistent throughout the 12-week trial duration, with a mean reduction of 1.3 in mCGI-S scores across all weeks [9] Trial Design and Patient Insights - The SERENITY At-Home trial was a double-blind, placebo-controlled study involving 246 patients, with data collected on 2,628 agitation episodes over 12 weeks [4][16] - The trial included patients with bipolar disorders (45%) and schizophrenia (55%), allowing for concomitant interventions to manage agitation episodes [4] - Patients reported an average of 11.7 agitation episodes during the trial, with 81% completing the full 12-week study [4] Market Opportunity - The total addressable market for at-home agitation treatment is estimated to be significantly higher than previously anticipated, with 57-77 million agitation episodes annually compared to an earlier estimate of 23 million [12] - The lack of FDA-approved therapies for at-home agitation treatment highlights a substantial unmet need in the market [11][12] Future Plans - Based on the positive trial results and FDA feedback, the company plans to submit a supplemental New Drug Application (sNDA) in Q1 2026 for expanded usage of IGALMI® in outpatient settings [1][2][10]

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]