BioXcel Therapeutics, Inc. (BTAI) came out with a quarterly loss of $2.45 per share versus the Zacks Consensus Estimate of a loss of $2.3. This compares to a loss of $3.36 per share a year ago. These figures are adjusted for non-recurring items. This quarterly report represents an earnings surprise of -6.52%. A quarter ago, it was expected that this company would post a loss of $2.72 per share when it actually produced a loss of $1.5, delivering a surprise of +44.85%. Over the last four quarters, the compan ...

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.

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38410 BioXcel Therapeutics, Inc. (Exact name of registrant as specified in its charter) Delaware 82-1386754 (S ...

[Overview and Business Updates](index=1&type=section&id=Overview%20and%20Business%20Updates) [Q2 2025 Highlights](index=1&type=section&id=Q2%202025%20Highlights) BioXcel Therapeutics completed its SERENITY At-Home Phase 3 trial, with topline data expected in August 2025, and is preparing for an FDA meeting to discuss a potential sNDA for IGALMI's at-home use, suggesting a larger addressable market - Topline data from the SERENITY At-Home Phase 3 trial for treating agitation in bipolar disorders or schizophrenia is expected in August 2025[1](index=1&type=chunk)[3](index=3&type=chunk) - An FDA meeting is scheduled for August 2025 to support a potential sNDA submission for IGALMI's label expansion into the at-home setting[1](index=1&type=chunk) - The company's research suggests the target addressable market for at-home agitation is **meaningfully larger** than the original estimate of **23 million annual episodes**[4](index=4&type=chunk) - The company has strengthened its cash position through at-the-market sales and warrant exercises subsequent to the quarter's end[2](index=2&type=chunk)[14](index=14&type=chunk) [Clinical Program Updates](index=1&type=section&id=Clinical%20Program%20Updates) The company provided updates on its late-stage clinical programs for BXCL501, including the completed SERENITY At-Home Phase 3 trial, the advancing TRANQUILITY program, and new non-clinical data supporting BXCL501's potential in broader chronic stress-related disorders [SERENITY Program (BXCL501 for Bipolar/Schizophrenia)](index=1&type=section&id=SERENITY%20Program%20(BXCL501%20for%20Bipolar%2FSchizophrenia)) The SERENITY At-Home Phase 3 trial, evaluating a 120 mcg dose of BXCL501 for agitation in bipolar disorders or schizophrenia, has completed enrollment and dosing, collecting data from over 2,200 agitation episodes across more than 200 patients with two favorable DSMB recommendations SERENITY At-Home Phase 3 Trial Statistics | Metric | Value | | :--- | :--- | | Patients Enrolled | >200 | | Study Sites | 22 | | Agitation Episodes Data | >2,200 | | Patient Populations | Balanced (Bipolar & Schizophrenia) | | DSMB Recommendations | 2 (Favorable) | - The vast majority of patients who were dosed completed the full **12-week** study[5](index=5&type=chunk) [TRANQUILITY Program (BXCL501 for Alzheimer's Dementia)](index=2&type=section&id=TRANQUILITY%20Program%20(BXCL501%20for%20Alzheimer's%20Dementia)) The TRANQUILITY In-Care Phase 3 trial, assessing a 60 mcg dose of BXCL501 for acute agitation in Alzheimer's dementia, remains a key strategy, with BioXcel currently evaluating Contract Research Organizations to initiate the trial - The TRANQUILITY program is designed to evaluate a **60 mcg dose** of BXCL501 for agitation in Alzheimer's dementia within care settings[13](index=13&type=chunk) - The company is currently evaluating CROs for trial initiation, indicating the program is moving forward[13](index=13&type=chunk) [BXCL501 in Chronic Conditions](index=2&type=section&id=BXCL501%20in%20Chronic%20Conditions) Recently published non-clinical research in *Frontiers in Pharmacology* demonstrated that BXCL501 significantly reduced stress-induced behaviors in animal models, supporting its mechanism of action and potential utility in broader stress-related psychiatric disorders - Research published in *Frontiers in Pharmacology* showed BXCL501 reduced stress-induced behaviors, supporting its potential for use in chronic stress-related disorders[6](index=6&type=chunk) [Corporate Updates](index=2&type=section&id=Corporate%20Updates) BioXcel is maintaining the market presence of its approved drug, IGALMI, for institutional use, continuing to supply the product and build brand awareness with minimal commercial resources - The company continues to supply IGALMI in the institutional setting and build brand awareness with minimal commercial resources[7](index=7&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) [Financial Performance Summary](index=2&type=section&id=Financial%20Performance%20Summary) In Q2 2025, BioXcel reported a significant decrease in IGALMI net revenue to $120 thousand from $1.1 million in Q2 2024, with net loss increasing to $19.2 million, while R&D expenses rose due to clinical trial activity and SG&A expenses fell from cost-saving measures Q2 2025 vs. Q2 2024 Financial Performance (in millions) | Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Net Revenue | $0.12 | $1.1 | -89% | | R&D Expenses | $10.3 | $8.0 | +28.8% | | SG&A Expenses | $5.6 | $9.5 | -41.1% | | Operating Loss | $15.9 | $17.3 | -8.1% | | Net Loss | $19.2 | $8.3 | +131.3% | - The increase in R&D expenses was primarily driven by clinical trial activity for the SERENITY at-home Phase 3 studies[10](index=10&type=chunk) - The decrease in SG&A expenses was attributed to lower personnel costs, professional fees, and marketing costs following reprioritization actions in **2024**[11](index=11&type=chunk) - Cost of Goods Sold in Q2 2025 included a **$95 thousand** charge for reserves for excess or obsolete inventory, which was not present in the same period of **2024**[9](index=9&type=chunk) [Financial Position](index=2&type=section&id=Financial%20Position) The company ended Q2 2025 with $18.6 million in cash, cash equivalents, and restricted cash, subsequently raising an additional $11.5 million from at-the-market sales and $3.6 million from warrant exercises, with an operating cash burn of $12.6 million for the quarter Cash Position as of June 30, 2025 | Item | Amount (in millions) | | :--- | :--- | | Cash and cash equivalents (June 30, 2025) | $18.6 | | Gross proceeds from ATM (post-Q2) | $11.5 | | Proceeds from warrant exercises (post-Q2) | $3.6 | | **Pro-forma Cash** | **$33.7** | - The company used **$12.6 million** in operating cash during the second quarter of **2025**[12](index=12&type=chunk) [Detailed Financial Statements](index=7&type=section&id=Detailed%20Financial%20Statements) This section provides the unaudited condensed statements of operations for the three and six months ended June 30, 2025 and 2024, and the condensed balance sheets as of June 30, 2025, and December 31, 2024 Statements of Operations (Unaudited, in thousands) | | Three months ended June 30, | Six months ended June 30, | | :--- | :--- | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | Product revenues | $120 | $1,104 | $288 | $1,686 | | Total operating expenses | $15,972 | $18,400 | $26,239 | $43,145 | | Loss from operations | $(15,852) | $(17,296) | $(25,951) | $(41,459) | | Net loss | $(19,187) | $(8,299) | $(26,441) | $(35,090) | | Net loss per share | $(2.45) | $(3.30) | $(4.17) | $(15.78) | Condensed Balance Sheets (Unaudited, in thousands) | | **June 30, 2025** | **December 31, 2024** | | :--- | :--- | :--- | | Cash and cash equivalents and restricted cash | $18,575 | $29,854 | | Total assets | $25,789 | $38,338 | | Total liabilities | $133,456 | $131,439 | | Total stockholders' equity (deficit) | $(107,667) | $(93,101) | [Product and Trial Information](index=3&type=section&id=Product%20and%20Trial%20Information) [About BXCL501](index=3&type=section&id=About%20BXCL501) BXCL501 is an investigational, proprietary, orally dissolving film of dexmedetomidine, currently under investigation for acute agitation in Alzheimer's dementia and at-home use in bipolar disorder or schizophrenia, having received FDA Breakthrough Therapy and Fast Track designations - BXCL501 is an investigational, orally dissolving film of dexmedetomidine, a selective alpha-2 adrenergic receptor agonist[15](index=15&type=chunk) - It is being investigated for acute treatment of agitation in Alzheimer's dementia and at-home use for bipolar disorder or schizophrenia[15](index=15&type=chunk) - BXCL501 has received FDA Breakthrough Therapy designation for agitation in dementia and Fast Track designation for agitation in schizophrenia, bipolar disorders, and dementia[15](index=15&type=chunk) [About IGALMI (Approved Drug)](index=4&type=section&id=About%20IGALMI%20(Approved%20Drug)) IGALMI, the FDA-approved dexmedetomidine sublingual film, is indicated for acute agitation in adults with schizophrenia and bipolar I or II disorder, requiring healthcare provider supervision due to potential risks like low blood pressure and significant drowsiness - IGALMI is a prescription medicine for the acute treatment of agitation associated with schizophrenia and bipolar disorder I or II in adults[18](index=18&type=chunk) - It must be administered under the supervision of a healthcare provider, and its safety has not been studied beyond **24 hours** from the first dose[18](index=18&type=chunk) - Important safety risks include decreased blood pressure, slower heart rate, QT interval prolongation, and sleepiness/drowsiness; patients should not drive or operate machinery for at least **8 hours** after taking it[24](index=24&type=chunk) [Clinical Trial Designs](index=3&type=section&id=Clinical%20Trial%20Designs) This section outlines the design of two pivotal Phase 3 trials for BXCL501: the SERENITY At-Home trial evaluating the safety of a 120 mcg dose, and the TRANQUILITY In-Care trial assessing the efficacy of a 60 mcg dose for Alzheimer's-related agitation [SERENITY At-Home Phase 3 Trial Design](index=3&type=section&id=SERENITY%20At-Home%20Phase%203%20Trial%20Design) The SERENITY At-Home trial is a double-blind, placebo-controlled study focused on the safety of a 120 mcg dose of BXCL501, enrolling 200 patients with bipolar disorders or schizophrenia to self-administer the drug at home over a 12-week period, with efficacy as an exploratory endpoint - The trial is a double-blind, placebo-controlled study evaluating the safety of a **120 mcg dose** of BXCL501 for at-home use[16](index=16&type=chunk) - It involves **200 patients** self-administering the drug or placebo during agitation episodes over a **12-week period**, with safety data as the primary collection point[16](index=16&type=chunk) [TRANQUILITY In-Care Phase 3 Trial Design](index=3&type=section&id=TRANQUILITY%20In-Care%20Phase%203%20Trial%20Design) The TRANQUILITY In-Care trial is a double-blind, placebo-controlled study designed to evaluate the efficacy and safety of a 60 mcg dose of BXCL501, aiming to enroll approximately 150 patients aged 55 and older with Alzheimer's dementia in care facilities, with the primary endpoint being the change in PEC score two hours after the first dose - The trial is designed to evaluate the efficacy and safety of a **60 mcg dose** of BXCL501 for agitation in Alzheimer's dementia in a care setting[17](index=17&type=chunk) - The primary endpoint is the change from baseline in the Positive and Negative Syndrome Scale-Excitatory Component (PEC) total score at **two hours** post-dose[17](index=17&type=chunk)

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]

Core Insights - BioXcel Therapeutics, Inc. is participating in a fireside chat at the Canaccord Genuity 45th Annual Growth Conference on August 12, 2025 [1] - The company utilizes artificial intelligence to develop transformative medicines in neuroscience and has a subsidiary focused on immuno-oncology [3] Company Overview - BioXcel Therapeutics, Inc. is a biopharmaceutical company listed on Nasdaq under the ticker BTAI [3] - The company employs a drug re-innovation approach, leveraging existing approved drugs and big data to identify new therapeutic indications [3] Event Details - The fireside chat will take place at 12 p.m. ET and will be accessible via a webcast on the company's website [1][2] - A replay of the webcast will be available for 90 days following the event [2]

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]