BioXcel Therapeutics(BTAI) - 2025 Q2 - Quarterly Results

Overview and Business Updates Q2 2025 Highlights 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][3] - 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] - 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] - The company has strengthened its cash position through at-the-market sales and warrant exercises subsequent to the quarter's end[2][14] Clinical Program Updates 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) 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] TRANQUILITY Program (BXCL501 for Alzheimer's Dementia) 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] - The company is currently evaluating CROs for trial initiation, indicating the program is moving forward[13] BXCL501 in Chronic Conditions 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] Corporate Updates 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] Second Quarter 2025 Financial Results Financial Performance Summary 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] - The decrease in SG&A expenses was attributed to lower personnel costs, professional fees, and marketing costs following reprioritization actions in 2024[11] - 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] Financial Position 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] Detailed Financial Statements 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 About BXCL501 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] - It is being investigated for acute treatment of agitation in Alzheimer's dementia and at-home use for bipolar disorder or schizophrenia[15] - BXCL501 has received FDA Breakthrough Therapy designation for agitation in dementia and Fast Track designation for agitation in schizophrenia, bipolar disorders, and dementia[15] About IGALMI (Approved Drug) 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] - 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] - 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] Clinical Trial Designs 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 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] - 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] TRANQUILITY In-Care Phase 3 Trial Design 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] - 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]