Core Insights - BioXcel Therapeutics has completed the last patient last visit (LPLV) in the pivotal Phase 3 SERENITY At-Home clinical trial, with topline data expected to be released this month to support a supplemental New Drug Application (sNDA) for IGALMI in the at-home setting [1][3] Group 1: Clinical Trial Details - 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 associated with bipolar disorders or schizophrenia in an at-home setting [2][4] - The trial enrolled over 200 patients across 22 sites nationwide, ensuring no single site enrolled more than 11% of the total patient population, with a balanced distribution between bipolar disorders and schizophrenia [2][4] Group 2: Market Opportunity - There are approximately 23 million episodes of agitation related to bipolar disorders or schizophrenia annually in the U.S. that occur at home, with no FDA-approved therapies currently available for acute treatment in this setting [3][4] - The company aims to provide a much-needed at-home treatment option for individuals experiencing such agitation, marking a significant milestone in its development efforts [3] Group 3: Product Information - BXCL501, known as IGALMI, is an investigational orally dissolving film formulation of dexmedetomidine, currently approved for use under healthcare supervision for acute treatment of agitation associated with schizophrenia and bipolar disorder [5][6] - The product has received Breakthrough Therapy designation from the FDA for treating agitation associated with dementia and Fast Track designation for agitation related to schizophrenia and bipolar disorders [5]

Core Points - BioXcel Therapeutics is focused on obtaining FDA approval for outpatient use of IGALMI® for treating agitation associated with bipolar disorders or schizophrenia [1][2] - A pre-sNDA meeting with the FDA is scheduled for August 20, 2025, to align on the submission process [2] - The SERENITY At-Home Phase 3 trial is nearing completion, with top-line results expected to be reported this quarter [3] Company Overview - BioXcel Therapeutics, Inc. utilizes artificial intelligence to develop transformative medicines in neuroscience [1][13] - The company has received Breakthrough Therapy designation for BXCL501 for treating agitation associated with dementia and Fast Track designation for schizophrenia and bipolar disorders [5] Clinical Development - The SERENITY At-Home trial is a double-blind, placebo-controlled study evaluating the safety of a 120 mcg dose of BXCL501 for acute agitation in the home setting [6] - The trial involves 200 patients with a history of agitation episodes, self-administering the treatment during episodes over a 12-week period [6] Market Opportunity - An estimated 23 million annual episodes of agitation associated with bipolar disorders or schizophrenia occur in the at-home setting in the U.S., with no FDA-approved therapies currently available for this treatment [3][17]

Core Insights - BioXcel Therapeutics, Inc. received a second positive recommendation from an independent Data Safety Monitoring Board (DSMB) to continue the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia [1][2] - The trial is fully enrolled with 178 patients dosed as of the May 28, 2025 cutoff date, and topline data is expected in Q3 2025 [2][7] - BXCL501 is an investigational sublingual film formulation of dexmedetomidine, currently under investigation for treating agitation in various conditions, including Alzheimer's dementia and bipolar disorders [5][6] Company Overview - BioXcel Therapeutics utilizes artificial intelligence to develop transformative medicines in neuroscience and has a subsidiary focused on immuno-oncology [13] - The company aims to address a significant unmet medical need for patients experiencing agitation related to bipolar disorders and schizophrenia, with no FDA-approved therapies currently available for at-home treatment [3][5] Trial Details - The SERENITY At-Home Phase 3 trial is a double-blind, placebo-controlled study designed to evaluate the safety of a 120 mcg dose of BXCL501 in 200 patients [6] - Patients are self-administering the drug during agitation episodes over a 12-week period, with safety data collected throughout the trial [6][3] - The trial's exploratory endpoints include assessments of severity and change in agitation two hours post-dosing [6] Regulatory and Market Potential - The trial data is intended to support a potential supplemental New Drug Application (sNDA) submission to expand the IGALMI® label for at-home use [3][16] - BXCL501 has received Breakthrough Therapy designation for treating agitation associated with dementia and Fast Track designation for agitation related to schizophrenia and bipolar disorders [5]

Core Insights - BioXcel Therapeutics, Inc. announced that the independent Data Safety Monitoring Board (DSMB) recommended the continuation of the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501 without modification, with topline data expected in Q3 2025 [2][9] Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company focused on utilizing artificial intelligence to develop transformative medicines in neuroscience [15] - The company is also involved in immuno-oncology through its subsidiary, OnkosXcel Therapeutics [15] Trial Details - The SERENITY At-Home Phase 3 trial is a double-blind, placebo-controlled study designed to evaluate the safety of a 120 mcg dose of BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia in the at-home setting [4][7] - The trial is fully enrolled with more than 205 patients dosed, and over 150 patients have received multiple doses during the 12-week trial period [3][9] Product Information - BXCL501 is an investigational proprietary, orally dissolving film formulation of dexmedetomidine, which is under investigation for the acute treatment of agitation associated with Alzheimer's dementia, bipolar disorders, and schizophrenia [6] - BXCL501 has received Breakthrough Therapy designation from the FDA for the acute treatment of agitation associated with dementia and Fast Track designation for agitation associated with schizophrenia and bipolar disorders [6] Future Prospects - The results from the SERENITY trial are intended to support a potential supplemental New Drug Application (sNDA) submission for label expansion of IGALMI in the at-home setting, addressing a significant unmet medical need [4]

Core Viewpoint - BioXcel Therapeutics, Inc. has received approval from the Nasdaq Hearings Panel to continue its listing on The Nasdaq Stock Market, with a deadline to regain compliance by September 16, 2025 [1][2]. Company Overview - BioXcel Therapeutics is a biopharmaceutical company that leverages artificial intelligence to develop innovative medicines in the field of neuroscience [12]. - The company is also focused on immuno-oncology through its subsidiary, OnkosXcel Therapeutics [12]. Compliance and Listing - The Nasdaq Panel reviewed BioXcel's compliance plan during a hearing on May 1, 2025, and granted an extension for compliance with the MVLS Rule [2]. - The company is committed to executing its compliance plan to meet the necessary requirements within the specified timeframe [2][3]. Product Development - BioXcel is advancing multiple strategic initiatives, including the SERENITY At-Home pivotal Phase 3 safety trial of BXCL501, aimed at supporting a supplemental New Drug Application (sNDA) to expand the IGALMI label [3]. - BXCL501 is an investigational sublingual film formulation of dexmedetomidine, currently under investigation for treating agitation associated with Alzheimer's dementia and bipolar disorders [4]. SERENITY At-Home Phase 3 Trial - The SERENITY At-Home trial is a double-blind, placebo-controlled study evaluating the safety of a 120 mcg dose of BXCL501 for treating agitation in patients with bipolar disorders or schizophrenia [5]. - The trial involves 200 patients self-administering BXCL501 or placebo during agitation episodes over a 12-week period, with safety data collected throughout [5]. IGALMI Overview - IGALMI (dexmedetomidine) is a prescription medicine used for the acute treatment of agitation associated with schizophrenia and bipolar disorder in adults [6]. - The safety and effectiveness of IGALMI have not been established beyond 24 hours from the first dose [6]. Safety Information - IGALMI can cause serious side effects, including decreased blood pressure and slower heart rate, particularly in patients with certain pre-existing conditions [7][8]. - Common side effects reported in clinical studies include sleepiness, dizziness, and dry mouth [9].

Company Performance - BioXcel Therapeutics reported a quarterly loss of $1.50 per share, significantly better than the Zacks Consensus Estimate of a loss of $2.72, and an improvement from a loss of $13.92 per share a year ago, resulting in an earnings surprise of 44.85% [1] - The company posted revenues of $0.17 million for the quarter ended March 2025, missing the Zacks Consensus Estimate by 44%, and down from $0.58 million in the same quarter last year [2] - Over the last four quarters, BioXcel has surpassed consensus EPS estimates four times and topped consensus revenue estimates two times [2] Stock Performance - BioXcel Therapeutics shares have declined approximately 74.4% since the beginning of the year, contrasting with the S&P 500's decline of 3.8% [3] - The current consensus EPS estimate for the upcoming quarter is -$2.24 on revenues of $0.4 million, and for the current fiscal year, it is -$16.44 on revenues of $3.51 million [7] Industry Outlook - The Medical - Biomedical and Genetics industry, to which BioXcel belongs, is currently ranked in the top 34% of over 250 Zacks industries, indicating a favorable outlook compared to the bottom 50% [8] - Empirical research suggests a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can impact stock performance [5]

PART I - FINANCIAL INFORMATION [Forward-Looking Statements](index=3&type=section&id=Forward%20Looking%20Statements) The report contains forward-looking statements about the company's strategy, financial needs, and clinical programs, which are subject to significant risks and uncertainties - The report includes forward-looking statements concerning various aspects of the business, including sales strategy for IGALMI®, cost savings from its strategic reprioritization, ability to raise capital and continue as a going concern, clinical trial plans, and intellectual property strategy[11](index=11&type=chunk)[14](index=14&type=chunk) - These statements are based on current management expectations and are subject to known and unknown risks, directing readers to detailed discussions in risk factor sections[12](index=12&type=chunk) [Summary Risk Factors](index=5&type=section&id=Summary%20Risk%20Factors) This section summarizes principal risks, including significant operating losses, potential Nasdaq delisting, critical funding needs, and substantial doubt about going concern - The Company has a history of significant operating losses and anticipates they will continue, raising substantial doubt about its ability to continue as a going concern[17](index=17&type=chunk) - There is a risk of being delisted from The Nasdaq Capital Market, which would harm stock liquidity and the ability to raise capital[17](index=17&type=chunk) - The business is dependent on the success of its sole approved product, IGALMI®, and its key product candidates, with failure to develop or commercialize them substantially harming the business[17](index=17&type=chunk) - The Company has significant indebtedness under its Credit Agreement, where failure to raise additional capital could trigger an event of default and force program delays or eliminations[17](index=17&type=chunk) - The company is subject to legal proceedings and investigations which could be costly and result in unfavorable outcomes[22](index=22&type=chunk) [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents the unaudited condensed consolidated financial statements for Q1 2025 and 2024, including balance sheets, statements of operations, and cash flows, with accompanying notes on key financial aspects [Condensed Consolidated Balance Sheets](index=10&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) As of March 31, 2025, cash and cash equivalents slightly increased to **$31.0 million**, total assets remained stable, and total liabilities decreased to **$128.7 million**, improving stockholders' deficit Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | March 31, 2025 (unaudited) | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $31,013 | $29,854 | | Total current assets | $37,777 | $37,394 | | Total assets | $38,566 | $38,338 | | **Liabilities & Equity** | | | | Total current liabilities | $25,546 | $22,233 | | Long-term debt | $99,918 | $102,508 | | Total liabilities | $128,738 | $131,439 | | Total stockholders' (deficit) equity | $(90,172) | $(93,101) | [Condensed Consolidated Statements of Operations](index=12&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Net loss significantly reduced to **$7.3 million** in Q1 2025 from **$26.8 million** in Q1 2024, driven by substantial decreases in operating expenses, despite lower net product revenue Statement of Operations Summary (in thousands, except per share data) | Metric | Three months ended March 31, 2025 | Three months ended March 31, 2024 | | :--- | :--- | :--- | | Product revenue, net | $168 | $582 | | Research and development | $4,554 | $11,401 | | Selling, general and administrative | $5,699 | $13,264 | | Loss from operations | $(10,099) | $(24,163) | | Net loss | $(7,254) | $(26,791) | | Basic and diluted net loss per share | $(1.50) | $(13.89) | [Condensed Consolidated Statements of Cash Flows](index=14&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities improved to **$12.0 million** in Q1 2025, with **$13.2 million** provided by financing activities, resulting in a **$1.2 million** net cash increase and **$31.0 million** cash balance Cash Flow Summary (in thousands) | Activity | Three months ended March 31, 2025 | Three months ended March 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(12,043) | $(17,706) | | Net cash from investing activities | $0 | $0 | | Net cash provided by financing activities | $13,202 | $26,626 | | **Net increase in cash** | **$1,159** | **$8,920** | | **Cash and cash equivalents, end of period** | **$31,013** | **$74,141** | [Notes to Condensed Consolidated Financial Statements](index=15&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) These notes detail the company's AI-driven drug development, critical **going concern** doubt due to losses and financing dependency, restructuring, complex debt terms, equity transactions, and ongoing legal proceedings including an SEC investigation - The company's management has concluded there is substantial doubt about its ability to continue as a going concern for at least 12 months from the financial statement issuance date, citing a history of significant losses, negative cash flows, and dependence on obtaining additional financing[44](index=44&type=chunk) - The company has undergone multiple restructuring actions, including workforce reductions in May and September 2024, to preserve cash and prioritize core clinical programs[57](index=57&type=chunk)[58](index=58&type=chunk) - The company has a complex Credit Agreement with Oaktree, amended six times, with the Fifth Amendment waiving the minimum revenue covenant, fixing the interest rate at **13%**, and establishing new capital raising requirements, all in compliance as of March 31, 2025[84](index=84&type=chunk)[85](index=85&type=chunk)[91](index=91&type=chunk)[96](index=96&type=chunk) - In March 2025, the company raised approximately **$13.0 million** in net proceeds through a registered direct offering of common stock, pre-funded warrants, and accompanying warrants[125](index=125&type=chunk) - The company is involved in several legal proceedings, including a securities class action lawsuit and stockholder derivative complaints, with the class action parties scheduled for private mediation[183](index=183&type=chunk)[184](index=184&type=chunk)[185](index=185&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=64&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section provides management's analysis of financial condition and operations, detailing the AI-driven drug development, commercial status of IGALMI®, clinical program progress, reduced operating expenses, and critical liquidity challenges including **going concern** status [Overview and Business Update](index=64&type=section&id=Overview%20and%20Business%20Update) The company, an AI-driven biopharmaceutical firm, focuses on neuroscience with lead product IGALMI® and key programs like BXCL501, while facing a Nasdaq delisting notice for market value non-compliance - The company received a Nasdaq delisting notice for failing to meet the **$35 million** minimum market value of listed securities requirement, with an appeal decision pending[200](index=200&type=chunk)[201](index=201&type=chunk) - The SERENITY At-Home Phase 3 trial for BXCL501 is fully enrolled, with topline data expected in the second half of 2025, intended to support an sNDA for IGALMI's at-home label expansion[205](index=205&type=chunk)[206](index=206&type=chunk) - The company has undergone significant restructuring, including workforce reductions in May and September 2024, to preserve cash and prioritize core clinical programs[210](index=210&type=chunk)[211](index=211&type=chunk) [Clinical Programs and Intellectual Property](index=67&type=section&id=Clinical%20Programs%20and%20Intellectual%20Property) The company focuses on neuroscience candidate BXCL501, with the SERENITY At-Home trial fully enrolled and TRANQUILITY In-Care trial pending funding, while immuno-oncology programs are largely paused, supported by a robust IP portfolio - The TRANQUILITY program for Alzheimer's agitation requires additional Phase 3 efficacy and safety data, with the TRANQUILITY In-Care trial planned upon securing funding[222](index=222&type=chunk) - Investigator-sponsored trials are evaluating BXCL501 for Opioid Use Disorder (OUD), Alcohol Use Disorder (AUD) with PTSD, and Acute Stress Disorder (ASD), funded by NIDA and the Department of Defense[229](index=229&type=chunk)[230](index=230&type=chunk)[231](index=231&type=chunk)[234](index=234&type=chunk) - Immuno-oncology programs, including BXCL701, have been paused to conserve resources, despite FDA Fast Track designation for BXCL701 in a specific prostate cancer indication[247](index=247&type=chunk)[248](index=248&type=chunk) - The neuroscience patent portfolio for BXCL501 includes **13 U.S. patents** listed in the FDA's Orange Book for IGALMI®, with expiration dates between **2037** and **2043**[240](index=240&type=chunk) [Results of Operations](index=90&type=section&id=Results%20of%20Operations) Q1 2025 net product revenue for IGALMI® decreased, while R&D and SG&A expenses significantly fell by **60%** and **57%** respectively due to restructuring, narrowing the net loss to **$7.3 million** Comparison of Operating Results (in thousands) | Line Item | Q1 2025 | Q1 2024 | Change | % Change | | :--- | :--- | :--- | :--- | :--- | | Product Revenue, Net | $168 | $582 | $(414) | (71)% | | R&D Expense | $4,554 | $11,401 | $(6,847) | (60)% | | SG&A Expense | $5,699 | $13,264 | $(7,565) | (57)% | - The decrease in R&D expense was mainly due to reduced costs for the TRANQUILITY II and III studies and lower personnel costs from the Clinical Prioritization[297](index=297&type=chunk) - The decrease in SG&A expense was primarily driven by reduced personnel costs, lower legal fees, and decreased commercial and marketing expenses as a result of restructuring[300](index=300&type=chunk)[302](index=302&type=chunk) [Liquidity and Capital Resources](index=94&type=section&id=Liquidity%20and%20Capital%20Resources) With **$31.0 million** cash as of March 31, 2025, the company faces substantial **going concern** doubt, with liquidity dependent on raising additional capital and complying with stringent debt covenants, including a **$29.0 million** net proceeds requirement - The company has substantial doubt about its ability to continue as a going concern, with **$31.0 million** in cash and cash equivalents as of March 31, 2025, projected to fund operations only into the third quarter of 2025[306](index=306&type=chunk)[393](index=393&type=chunk) - The Fifth Amendment to the Credit Agreement requires the company to raise at least **$29.0 million** in net cash proceeds by mid-2025, of which **$19.1 million** has been met as of the filing[393](index=393&type=chunk) - The minimum liquidity covenant under the Credit Agreement increased to **$10.0 million** on March 31, 2025, and will further increase to **$15.0 million** on September 30, 2025[394](index=394&type=chunk) - On February 10, 2025, the company effected a **1-for-16 reverse stock split**, with all share and per-share amounts in the report retrospectively adjusted[305](index=305&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=109&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) This section is not applicable for the reporting period - This section is not applicable for the reporting period[365](index=365&type=chunk) [Item 4. Controls and Procedures](index=109&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded the company's disclosure controls and procedures were effective as of March 31, 2025, with no material changes in internal control over financial reporting during the quarter - The CEO and CFO concluded that as of March 31, 2025, the company's disclosure controls and procedures were effective at the reasonable assurance level[368](index=368&type=chunk) - No changes in internal control over financial reporting occurred during the quarter ended March 31, 2025, that have materially affected, or are reasonably likely to materially affect, internal controls[369](index=369&type=chunk) PART II - OTHER INFORMATION [Item 1. Legal Proceedings](index=112&type=section&id=Item%201.%20Legal%20Proceedings) The company is subject to various legal proceedings, including class action and derivative lawsuits, and a formal SEC investigation initiated in February 2024 regarding public disclosures and securities trading - In February 2024, the company became aware of a formal SEC investigation involving the company and certain directors and officers[373](index=373&type=chunk) - The SEC investigation relates to public disclosures about product sales, a Form 483 at a clinical trial site for the TRANQUILITY II study, and trading in the company's securities, with the company cooperating[373](index=373&type=chunk) [Item 1A. Risk Factors](index=112&type=section&id=Item%201A.%20Risk%20Factors) This section extensively discusses risks, including precarious financial position, drug development uncertainties, commercialization challenges, reliance on third parties, intellectual property protection, and risks associated with common stock ownership - Financial risks are paramount, including a history of significant losses, substantial doubt about its ability to continue as a going concern, and the need for additional funding to avoid defaulting on its Credit Agreement[377](index=377&type=chunk)[389](index=389&type=chunk)[403](index=403&type=chunk) - Development of BXCL501 for Alzheimer's agitation is at risk due to principal investigator misconduct in the TRANQUILITY II trial and FDA requirements for additional efficacy and safety data, requiring significant time and funding[412](index=412&type=chunk)[418](index=418&type=chunk)[420](index=420&type=chunk) - The company faces a risk of being delisted from The Nasdaq Capital Market for not meeting the minimum market value requirement, which would harm liquidity and the ability to raise capital[382](index=382&type=chunk)[385](index=385&type=chunk)[386](index=386&type=chunk) - The company is substantially dependent on third parties for manufacturing and conducting clinical trials, where any failure could stop or delay development and commercialization[576](index=576&type=chunk)[588](index=588&type=chunk) - The company is subject to ongoing legal proceedings, including a securities class action, derivative lawsuits, and a formal SEC investigation, which could result in substantial costs and divert management's attention[703](index=703&type=chunk)[709](index=709&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=239&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) There were no unregistered sales of equity securities or use of proceeds during the reporting period - None[725](index=725&type=chunk) [Item 3. Defaults Upon Senior Securities](index=239&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) There were no defaults upon senior securities during the reporting period - None[726](index=726&type=chunk) [Item 5. Other Information](index=239&type=section&id=Item%205.%20Other%20Information) The company reports no other material information, confirming no directors or officers adopted or terminated Rule 10b5-1 trading arrangements during the quarter - During the first quarter of 2025, no directors or officers as defined by Rule 16a-1(f) adopted or terminated a Rule 10b5-1 trading arrangement[729](index=729&type=chunk)

[Business Update and Clinical Program Highlights](index=1&type=section&id=Business%20Update%20and%20Clinical%20Program%20Highlights) BioXcel completed SERENITY At-Home Phase 3 enrollment for BXCL501, expecting H2 2025 data to support at-home use - **Enrollment is complete** in the SERENITY At-Home pivotal Phase 3 safety trial for BXCL501 for the acute treatment of agitation associated with bipolar disorders or schizophrenia[1](index=1&type=chunk) - Topline data from the SERENITY At-Home trial is expected in the **second half of 2025**, which is intended to support a potential supplemental New Drug Application (sNDA) for at-home use[1](index=1&type=chunk)[6](index=6&type=chunk) - The company is continuing to supply its commercial product, IGALMI, and build brand awareness using **minimal commercial resources**[5](index=5&type=chunk) [SERENITY Program (BXCL501 for Bipolar/Schizophrenia Agitation)](index=1&type=section&id=SERENITY%20Program%20%28BXCL501%20for%20Bipolar%2FSchizophrenia%20Agitation%29) SERENITY At-Home Phase 3 safety trial for BXCL501 completed enrollment, evaluating a 120 mcg dose for at-home agitation - The trial is a double-blind, placebo-controlled study to evaluate the safety of a **120 mcg dose** of BXCL501 in **200 patients** in an at-home setting[6](index=6&type=chunk)[14](index=14&type=chunk) - Trial **enrollment is complete**, with over **165 patients dosed** and more than **115 patients** having received multiple doses over the 12-week trial period[6](index=6&type=chunk) [TRANQUILITY Program (BXCL501 for Alzheimer's Agitation)](index=1&type=section&id=TRANQUILITY%20Program%20%28BXCL501%20for%20Alzheimer%27s%20Agitation%29) TRANQUILITY In-Care Phase 3 trial for BXCL501, assessing a 60 mcg dose for Alzheimer's agitation, is advancing - The TRANQUILITY program is designed to evaluate the efficacy and safety of a **60 mcg dose** of BXCL501 for acute treatment of agitation associated with Alzheimer's dementia in a care setting[6](index=6&type=chunk)[15](index=15&type=chunk) - The program remains part of the company's broader development strategy, and **plans for trial initiation are being advanced**[6](index=6&type=chunk) [First Quarter 2025 Financial Results](index=2&type=section&id=First%20Quarter%202025%20Financial%20Results) BioXcel's Q1 2025 net loss reduced to **$7.3 million** from **$26.8 million** in Q1 2024 Q1 2025 vs Q1 2024 Financial Performance (in thousands) | Metric | Q1 2025 | Q1 2024 | | :--- | :--- | :--- | | IGALMI Net Revenue | $168 | $582 | | R&D Expenses | $4,554 | $11,401 | | SG&A Expenses | $5,699 | $13,264 | | Net Loss | $(7,254) | $(26,791) | | Net Loss Per Share | $(1.50) | $(13.89) | Key Balance Sheet and Cash Flow Items | Metric | As of Mar 31, 2025 | | :--- | :--- | | Cash and cash equivalents | $31.0 million | | Operating cash used in Q1 2025 | $12.0 million | - The decrease in operating expenses was primarily attributable to **reduced clinical trial activity**, **lower personnel costs**, and **reduced commercial and marketing expenses** resulting from reprioritization actions taken in 2024[9](index=9&type=chunk)[10](index=10&type=chunk) [Product and Pipeline Overview](index=2&type=section&id=Product%20and%20Pipeline%20Overview) This section outlines BioXcel's FDA-approved IGALMI and investigational BXCL501 for agitation treatment [About BXCL501](index=2&type=section&id=About%20BXCL501) BXCL501 is an investigational orally dissolving film for agitation, with FDA Breakthrough and Fast Track designations - BXCL501 is an **investigational, orally dissolving film formulation** of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist[13](index=13&type=chunk) - It has received **FDA Breakthrough Therapy designation** for the acute treatment of agitation associated with dementia and **Fast Track designation** for agitation associated with schizophrenia, bipolar disorders, and dementia[13](index=13&type=chunk) [About IGALMI (Approved Indication)](index=3&type=section&id=About%20IGALMI%20%28Approved%20Indication%29) IGALMI is an FDA-approved sublingual film for acute agitation in schizophrenia and bipolar disorder - IGALMI is **FDA-approved** for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults, administered under medical supervision[16](index=16&type=chunk) - Serious side effects include **decreased blood pressure**, low blood pressure upon standing, **slower heart rate**, and heart rhythm changes (**QT interval prolongation**)[17](index=17&type=chunk) - Patients should **not drive or operate hazardous machinery for at least 8 hours** after taking IGALMI due to potential sleepiness/drowsiness[17](index=17&type=chunk) [Forward-Looking Statements](index=5&type=section&id=Forward-Looking%20Statements) This section outlines **forward-looking statements**, subject to **significant risks** including **limited operating history** - The press release includes **forward-looking statements** regarding the advancement of TRANQUILITY and SERENITY trials, potential market opportunity for BXCL501, and timelines for data release and regulatory submissions[23](index=23&type=chunk) - The company highlights **significant risks** that could cause actual results to differ, including its **limited operating history**, **significant losses**, **need for additional funding**, **significant indebtedness**, and its **ability to continue as a going concern**[23](index=23&type=chunk)

Core Insights - BioXcel Therapeutics has completed enrollment in its SERENITY At-Home pivotal Phase 3 safety trial for BXCL501, aimed at treating agitation associated with bipolar disorders or schizophrenia, with topline data expected in the second half of 2025 to support a potential sNDA submission for label expansion of IGALMI in the at-home setting [1][2] Company Updates - The SERENITY trial is designed as a double-blind, placebo-controlled study involving 200 patients, with over 165 patients already dosed and more than 115 receiving multiple doses during the 12-week trial period [7] - The company continues to supply IGALMI to patients and providers while building brand awareness with minimal commercial resources [4] Financial Performance - For Q1 2025, net revenue from IGALMI was $168,000, a decrease from $582,000 in Q1 2024 [5] - Cost of Goods Sold for Q1 2025 was $14,000, down from $80,000 in the same period last year [6] - Research and Development (R&D) expenses were $4.6 million for Q1 2025, significantly lower than $11.4 million in Q1 2024, primarily due to reduced clinical trial activity [8] - Selling, General and Administrative (SG&A) expenses decreased to $5.7 million in Q1 2025 from $13.3 million in Q1 2024, reflecting cost-cutting measures [9] - The company reported a net loss of $7.3 million for Q1 2025, compared to a net loss of $26.8 million for the same period in 2024, with cash and cash equivalents totaling $31 million as of March 31, 2025 [10] Product Development - BXCL501 is under investigation for the acute treatment of agitation associated with Alzheimer's dementia and bipolar disorders or schizophrenia in the at-home setting, with Breakthrough Therapy and Fast Track designations granted by the FDA [11] - The TRANQUILITY In-Care Phase 3 trial is also in progress, designed to evaluate the efficacy and safety of BXCL501 for agitation associated with Alzheimer's dementia [13]

Clinical Trial Progress - Patient enrollment in the SERENITY At-Home pivotal Phase 3 trial has exceeded 60%, with 127 out of 200 patients enrolled, representing 63% of the required enrollment[5] - Topline data from the SERENITY At-Home trial is expected in the second half of 2025, which may support a potential sNDA submission for label expansion of IGALMI[1][3] Financial Performance - Net revenue from IGALMI for Q4 2024 was $366,000, a slight decrease from $376,000 in Q4 2023; for the full year 2024, net revenue was $2.3 million, up from $1.4 million in 2023[8] - Product revenues for the year ended December 31, 2024, were $2,266,000, compared to $1,380,000 for the year ended December 31, 2023, representing a 64.3% increase[31] Cost and Expenses - Cost of Goods Sold (COGS) for Q4 2024 was $832,000, compared to $714,000 in Q4 2023; for the full year 2024, COGS was $2.1 million, up from $1.3 million in 2023[9][10] - Research and Development (R&D) expenses for Q4 2024 were $5.9 million, down from $9.9 million in Q4 2023; for the full year 2024, R&D expenses totaled $30.4 million, significantly lower than $84.3 million in 2023[11] - Selling, General and Administrative (SG&A) expenses for Q4 2024 were $4.1 million, down from $9.6 million in Q4 2023; for the full year 2024, SG&A expenses were $34.5 million, compared to $83.4 million in 2023[12][13] - Total operating expenses for the year ended December 31, 2024, were $69,511,000, down from $173,162,000 for the year ended December 31, 2023, indicating a 60.1% reduction[31] - Research and development expenses for the year ended December 31, 2024, were $30,435,000, down from $84,326,000 for the year ended December 31, 2023, a reduction of 64.1%[31] - Selling, general and administrative expenses decreased to $34,492,000 for the year ended December 31, 2024, from $83,413,000 for the year ended December 31, 2023, a decline of 58.6%[31] Net Loss and Financial Position - BioXcel Therapeutics reported a net loss of $10.9 million for Q4 2024, an improvement from a net loss of $22.3 million in Q4 2023; for the full year 2024, the net loss was $59.6 million, down from $179.1 million in 2023[14] - The net loss for the year ended December 31, 2024, was $59,599,000, compared to a net loss of $179,053,000 for the year ended December 31, 2023, reflecting a 66.7% improvement[31] - The net loss per share for the year ended December 31, 2024, was $23.51, compared to $98.33 for the year ended December 31, 2023, a 76.1% improvement[31] - The company reported a loss from operations of $67,245,000 for the year ended December 31, 2024, compared to a loss of $171,782,000 for the year ended December 31, 2023, a 60.8% decrease[31] Cash Position - Cash and cash equivalents totaled $29.9 million as of December 31, 2024, a decrease from $65.2 million on December 31, 2023[15] - Cash and cash equivalents decreased to $29,854,000 as of December 31, 2024, from $65,221,000 as of December 31, 2023, a decline of 54.2%[31] - The company closed a $14 million equity financing, strengthening its cash position to approximately $35 million as of March 4, 2025[7] Liabilities and Going Concern - Total liabilities increased slightly to $131,439,000 as of December 31, 2024, from $130,210,000 as of December 31, 2023[31] - The company has identified conditions that raise substantial doubt about its ability to continue as a going concern[31]