BXCL501 achieved SERENITY At-Home’s primary endpoint of being well tolerated in the at-home treatment of agitation episodes in patients with bipolar disorders or schizophrenia No discontinuations for tolerability in the BXCL501 arm While not the primary objective of the SERENITY At-Home trial, the preliminary results demonstrate continued effects and consistent benefit with repeat dosing across the course of the trial Following previously disclosed positive FDA feedback, BioXcel plans to submit a sNDA in Q1 ...

Company to host conference call at 8 a.m. ET, August 27, 2025NEW HAVEN, Conn., Aug. 26, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that it will host an investor call and live webcast at 8 a.m. ET, August 27, 2025 to review topline data from the SERENITY At-Home Pivotal Phase 3 trial evaluating the safety of BXCL501, the Company’s proprietary, sublingual ...

Data from more than 2,600 agitation episodes collected Topline data readout is on track for August BXCL501 was granted Fast Track Designation for the acute treatment of agitation associated with bipolar disorders or schizophrenia. There are no FDA-approved therapies for the acute treatment of agitation in the at- home setting. About the SERENITY At-Home Phase 3 Trial The trial was designed to study 200 patients with a history of agitation episodes despite being on stable treatment for their underlying bipol ...

The primary purpose of the planned meeting was to gain alignment with the FDA regarding the content and format of the Company's planned supplemental New Drug Application (sNDA) submission for the at-home (outpatient) use of BXCL501, including the clinical, nonclinical, and chemistry and manufacturing and controls (CMC) requirements. The Company concludes that the objectives of the pre-sNDA meeting have been accomplished based on the FDA's written responses and has determined that the meeting, originally sch ...

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]

NEW HAVEN, Conn., Aug. 13, 2025 (GLOBE NEWSWIRE) -- BioXcel Therapeutics, Inc. (Nasdaq: BTAI), a biopharmaceutical company utilizing artificial intelligence to develop transformative medicines in neuroscience, today announced that Vimal Mehta, Ph.D., CEO of BioXcel Therapeutics, will participate in a virtual fireside chat on Thursday, August 14, 2025 at 1:00 p.m. ET as part of the H.C. Wainwright & Co. "HCW@Home" series. Date: Tuesday, August 14, 2025 Time: 1 p.m. Eastern Time Webcast Link: Register here Ab ...

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)