SERENITY At-Home Trial Overview - The SERENITY At-Home trial investigates IGALMI® for agitation associated with bipolar disorders or schizophrenia in the at-home setting[2] - The trial aims for sNDA submission for IGALMI® label expansion, as there are currently no FDA-approved therapies for this indication in the at-home setting[12] - The study design is a double-blind, placebo-controlled trial evaluating the safety of a 120 mcg dose of BXCL501 in the home setting[28] Safety and Tolerability Results - The trial collected data from 2,628 agitation episodes[22] - 2,437 episodes were treated in 208 patients, who self-administered the film[23] - No drug-related serious adverse events, syncopes, or falls were reported in the BXCL501 arm[24,38] - The adverse event profile was consistent with the approved IGALMI® label and previous clinical trials[24,38] - The most common treatment-emergent adverse event (TEAE) for the first dose of BXCL501 120 mcg was somnolence, occurring in 23 out of 102 patients (22.5%)[39] Efficacy and Future Steps - Preliminary results suggest continued treatment effect with repeat BXCL501 dosing across the trial[24,42] - A greater percentage of patients with mild, moderate, or severe agitation experienced full resolution of symptoms in the BXCL501 arm compared to placebo[42] - The company plans to submit an sNDA in Q1 2026[13,24]

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]

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]

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].

Core Insights - BioXcel Therapeutics, Inc. reported a quarterly loss of $2.45 per share, which was worse than the Zacks Consensus Estimate of a loss of $2.3, but an improvement from a loss of $3.36 per share a year ago, indicating a year-over-year improvement in losses [1] - The company posted revenues of $0.12 million for the quarter, missing the Zacks Consensus Estimate by 52% and down from $1.1 million in the same quarter last year [2] - The stock has underperformed the market with a gain of about 7% since the beginning of the year, compared to the S&P 500's gain of 8.4% [3] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$2.57 on revenues of $0.3 million, and for the current fiscal year, it is -$8.60 on revenues of $1.07 million [7] - The estimate revisions trend for BioXcel Therapeutics was favorable ahead of the earnings release, resulting in a Zacks Rank 2 (Buy) for the stock, suggesting it is expected to outperform the market in the near future [6] Industry Context - The Medical - Biomedical and Genetics industry, to which BioXcel Therapeutics belongs, is currently ranked in the bottom 43% of over 250 Zacks industries, indicating potential challenges for stocks in this sector [8]

Summary of BioXcel Therapeutics Conference Call Company Overview - **Company**: BioXcel Therapeutics - **Industry**: Biotechnology - **Key Product**: EGALMI, approved for agitation in schizophrenia and bipolar disorder Key Points and Arguments Upcoming Clinical Trials and Data - BioXcel is preparing for pivotal Phase III trial data related to schizophrenia and bipolar agitation, expected in August [3][4] - The company has already proven safety and efficacy of EGALMI in care settings and is now testing it in home settings [4][6] - The primary endpoint of the home setting trial is safety, with exploratory endpoints focusing on repeat dosing efficacy [10][18] Market Opportunity - Initial estimates indicated 23 million episodes of agitation, but further research suggests a larger market opportunity [5][6] - The company has collected data from 2,200 episodes over a 12-week period, indicating a representative sample of real-world experiences [13][14] - Pricing strategy suggests a potential price of up to $1,400 per prescription, with packaging considerations for home use [26][27] FDA Interactions and Approval Process - Upcoming FDA meeting on August 20 will focus on the format and content of the supplemental new drug application (sNDA) [17][18] - The primary concern for FDA approval is demonstrating safety comparable to previous clinical settings [18][23] - The company has a robust intellectual property portfolio with 13 patents, ensuring market exclusivity until 2043 [35][36] Commercialization Strategy - BioXcel is evaluating options for commercialization, including potential partnerships to expand market reach [32][34] - The company acknowledges the need for education and promotion in the neuropsychiatric market, similar to migraine treatments [32] Alzheimer's Program - BioXcel is also developing a product for acute agitation in Alzheimer's, with plans for a second confirmatory Phase III trial [7][39] - The protocol for the Alzheimer's trial has been agreed upon with the FDA, and the company is in the process of selecting a contract research organization (CRO) [40][41] Financial Position - As of the latest update, BioXcel has $18 million in cash, with additional proceeds expected from recent financing activities [42][44] Additional Important Insights - The trial's real-world setting allows patients to self-administer the treatment, potentially leading to more accurate data on compliance and effectiveness [14][16] - The company is aware of the placebo effect in psychiatric trials but believes prior efficacy data will mitigate its impact [12][18] - The potential for prodromal use of the product is acknowledged, but it will not be part of the official label due to lack of specific study [28][29] This summary encapsulates the critical aspects of BioXcel Therapeutics' conference call, highlighting the company's strategic focus, market potential, and regulatory interactions.