BioXcel Therapeutics(BTAI) - 2023 Q4 - Annual Report

Part I Forward Looking Statements This section outlines forward-looking statements regarding sales, strategy, capital, and clinical plans, subject to inherent risks and uncertainties - The report contains forward-looking statements regarding IGALMITM sales, strategic reprioritization, going concern ability, clinical trials, and market estimates[194][24] - These statements are subject to significant risks and uncertainties, with key factors detailed in Items 1A and 7[24] Summary Risk Factors This section highlights principal risks, including going concern doubt, operating losses, product dependence, clinical trial challenges, and the unproven AI platform - The company identified conditions raising substantial doubt about its ability to continue as a going concern[6] - Near-term success depends heavily on IGALMITM commercialization and clinical development of BXCL501, BXCL502, BXCL701, and BXCL702[7] - The company incurred significant operating losses since inception and anticipates continued losses[28] - Significant indebtedness and contractual obligations could impair liquidity and restrict operations[29] - The AI-based drug discovery approach is novel and unproven, with no guarantee of commercially valuable products[10] Business BioXcel Therapeutics uses AI for drug development in neuroscience and immuno-oncology, with lead product IGALMITM and a strategic reprioritization in August 2023 - The company utilizes AI platforms to identify new therapeutic indications for existing drugs, aiming to reduce development costs and timelines[209] - IGALMITM (dexmedetomidine) sublingual film was FDA-approved on April 6, 2022, for acute agitation in schizophrenia or bipolar disorder[221] - The most advanced neuroscience candidate is BXCL501, developed for agitation in Alzheimer's disease and at-home use for bipolar/schizophrenia[180] - The most advanced immuno-oncology candidate is BXCL701, an oral innate immune activator for aggressive prostate cancer and other tumors[18] - In August 2023, the company implemented a strategic reprioritization, reducing workforce by approximately 60% and shifting IGALMITM commercial strategy to reduce cash burn[234][531] Neuroscience Programs The neuroscience division focuses on AI-enabled therapeutics for agitation, with BXCL501 (IGALMITM) as its core asset, targeting significant market opportunities and undergoing strategic reprioritization - The company aims to be the leading AI-enabled neuroscience therapeutics company, focused on rapidly identifying medications[52][41] - The primary neuroscience asset is BXCL501, an orally dissolving film of dexmedetomidine, marketed as IGALMITM for agitation in schizophrenia/bipolar disorder and developed for Alzheimer's-related agitation[180][64] IGALMITM Net Revenue | Period | Net Revenue (USD) | | :--- | :--- | | FY 2023 | $1.4 million | | FY 2022 | $375,000 | | Q4 2023 | $376,000 | | Q3 2023 | $341,000 | - The company estimates a significant market opportunity with approximately 1.9 million U.S. patients with Alzheimer's-related dementia and 1.6 million with schizophrenia or bipolar disorders experiencing frequent agitation[58] - The company holds a robust intellectual property portfolio for its neuroscience program, with eight U.S. utility patents for IGALMI™ listed in the FDA's Orange Book, providing protection until 2039-2043[95] Immuno-Oncology Programs Immuno-oncology programs, managed by OnkosXcel, focus on BXCL701 for aggressive cancers, with promising trial results, but development is paused post-August 2023 strategic reprioritization - The immuno-oncology subsidiary, OnkosXcel, develops therapeutics using proprietary AI capabilities, with strategic options including a potential IPO under evaluation[139] - The lead candidate, BXCL701, is an oral innate immune activator designed to enhance checkpoint inhibitor efficacy in "cold" tumors[140][119] - BXCL701 received FDA Fast Track designation in February 2024 for use with a CPI in metastatic small cell neuroendocrine prostate cancer (mCRPC SCNC)[140][348] BXCL701 Phase 2a mCRPC Trial Results | Patient Cohort | Composite Response Rate | | :--- | :--- | | SCNC Phenotype | 25% | | Adenocarcinoma Phenotype | 21% | - Following the August 2023 strategic reprioritization, further development on immuno-oncology programs has been paused[123][139] Our Relationship with BioXcel LLC BioXcel LLC, a 29% stockholder and former parent, provides R&D and IP services, with an option for future collaboration on product identification - BioXcel LLC holds approximately 29% ownership and provides R&D and IP management services[387][388] - The company has an option until December 31, 2024, for a new collaborative services agreement with BioXcel LLC for product identification, with a $18,000 monthly fee[388] Service Charges from BioXcel LLC | Year | Amount (USD) | | :--- | :--- | | 2023 | $1.3 million | | 2022 | $1.4 million | Government Regulation and Product Approval The company is subject to extensive U.S. and international drug development, manufacturing, and marketing regulations, including FDA approval processes, expedited programs, and healthcare reform impacts - The company's products undergo a rigorous and lengthy approval process by the FDA and foreign authorities, involving preclinical studies and three phases of clinical trials[390][405][431] - The company utilized expedited development programs, receiving Fast Track designation for BXCL501 and BXCL701, and Breakthrough Therapy designation for BXCL501 in dementia agitation[440][420] - Post-approval, products like IGALMITM are subject to ongoing FDA regulation, including cGMP, adverse event reporting, and off-label promotion restrictions[464][467] - The company is subject to healthcare reform measures like the ACA and IRA, impacting drug pricing, reimbursement, and marketability[520][522] - Compliance with fraud and abuse laws, including the Anti-Kickback Statute and False Claims Act, is required, with significant penalties for violations[526][545][527] Risk Factors This section details substantial risks, including going concern doubt, operational challenges from reprioritization, product dependency, clinical trial uncertainties, debt, and an unproven AI platform - The company has a limited operating history, significant operating losses of $179.1 million in 2023, and expects continued losses, raising substantial doubt about its going concern ability[575][578][851] - The August 2023 strategic reprioritization and 60% workforce reduction may not achieve cost savings and could harm institutional knowledge, morale, and reputation[599][600] - Near-term success relies heavily on IGALMITM commercialization and successful clinical development and approval of key candidates, especially BXCL501 for Alzheimer's-related agitation[663] - Clinical trials are expensive and uncertain; the TRANQUILITY II trial was impacted by investigator misconduct, potentially jeopardizing data use and requiring new trials[676][639][617] - The company has significant indebtedness of $102.7 million as of December 31, 2023, with restrictive covenants including minimum cash and capital raising requirements, which could trigger default[609][592][588] - The proprietary AI platform drug discovery approach is novel and unproven, with no guarantee of commercially viable products beyond IGALMITM[685][686] Unresolved Staff Comments The company reports no unresolved staff comments from the Securities and Exchange Commission - There are no unresolved staff comments[983] Cybersecurity The company implements a cybersecurity risk management program based on the NIST Framework, overseen by the Audit Committee, with no material incidents identified to date - The company's cybersecurity risk management program is based on the NIST Cybersecurity Framework[913][1022] - Oversight is provided by the Audit Committee of the Board of Directors, receiving periodic management reports[1009][1010] - A Security Incident Management Team, including senior executives, manages material cybersecurity risks[965] - The company has not identified any cybersecurity threats materially affecting its operations, business strategy, or financial condition[1008] Properties The company leases 18,285 square feet for its corporate headquarters in New Haven, Connecticut, with the current lease expiring in February 2026 - The company leases 18,285 square feet for its corporate headquarters in New Haven, Connecticut[1016] - The lease expires in February 2026, with a five-year renewal option[1016] Legal Proceedings The company faces consolidated securities class action and stockholder derivative lawsuits related to the TRANQUILITY II trial, and is cooperating with an SEC investigation - The company is a defendant in a consolidated securities class action lawsuit alleging false or misleading statements related to the TRANQUILITY II trial[960] - Multiple stockholder derivative complaints with similar claims have been filed in federal courts in Connecticut and Delaware[942][943] - The company is cooperating with an SEC investigation and seeking document production[1017] Mine Safety Disclosures This item is not applicable to the company - Not applicable[1024] Part II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities The company's common stock trades on Nasdaq under BTAI, with 12 stockholders of record, and no cash dividends have been paid or are anticipated - The company's common stock trades on The Nasdaq Capital Market under the ticker symbol BTAI[1019] - The company has never declared or paid cash dividends and does not plan to do so in the foreseeable future[1020] Reserved This item is reserved Management's Discussion and Analysis of Financial Condition and Results of Operations This section discusses financial condition and operations, highlighting a $179.1 million net loss in 2023, increased operating expenses, and substantial doubt about going concern due to liquidity issues - Management concluded substantial doubt exists about the company's ability to continue as a going concern for at least 12 months due to recurring losses, negative cash flows, and limited liquidity[1096][1106] Comparison of Operations (Years Ended Dec 31) | Metric | 2023 (in thousands) | 2022 (in thousands) | | :--- | :--- | :--- | | Product Revenue, Net | $1,380 | $375 | | Research & Development | $84,326 | $91,239 | | Selling, General & Admin | $83,413 | $68,761 | | Restructuring Costs | $4,163 | $0 | | Net Loss | ($179,053) | ($165,757) | | Net Loss Per Share | ($6.15) | ($5.92) | - In August 2023, the company initiated a strategic reprioritization, reducing its workforce by ~60% and incurring $4.2 million in restructuring costs to reduce annualized operating expenses by approximately $80 million[1045][1052][1077] - As of December 31, 2023, the company had $65.2 million in cash and cash equivalents and $102.7 million in aggregate principal indebtedness under its Credit Agreement[1096][592] Quantitative and Qualitative Disclosures About Market Risk The company's primary market risk is interest rate fluctuations from variable-rate debt tied to SOFR, partially mitigated by a contractual floor and ceiling - The company has material interest rate risk exposure from its variable-rate debt under the Credit Agreement, tied to SOFR[1153] - This interest rate risk is partially hedged by a SOFR floor of 2.5% and a ceiling of 5.5% within the credit agreement terms[1153] - The company does not participate in foreign currency hedging and has limited exposure to other derivative financial instruments[1001] Financial Statements and Supplementary Data This section incorporates consolidated financial statements and the auditor's report from Ernst & Young LLP, which includes a going concern explanatory paragraph - The financial statements and the report of the independent auditor, Ernst & Young LLP, are included by reference starting on page F-1[1154] - The auditor's report contains an explanatory paragraph regarding the company's ability to continue as a going concern[1335] Changes in and Disagreements with Accountants on Accounting and Financial Disclosure The company reports no changes in or disagreements with its accountants on accounting and financial disclosure - None reported[1155] Controls and Procedures As of December 31, 2023, the company's disclosure controls and internal control over financial reporting were deemed effective, with no material changes reported - Management concluded that as of December 31, 2023, the company's disclosure controls and procedures were effective[1156] - Management concluded that as of December 31, 2023, the company's internal control over financial reporting was effective[1175] - There were no material changes in internal control over financial reporting during the quarter ended December 31, 2023[1003] Other Information This section discloses a March 20, 2024, Credit Agreement amendment waiving a going concern default and establishing new capital raising covenants, alongside executive Rule 10b5-1 trading plans - On March 20, 2024, the company amended its Credit Agreement to waive a default triggered by the "going concern" qualification in the 2023 audit report[1157] - The amendment introduced new covenants requiring the company to raise specified capital amounts by certain dates in 2024 to avoid default[1197] - Several executive officers, including the CEO, adopted Rule 10b5-1 trading plans in December 2023 to sell shares for tax purposes related to vested RSUs[1004][1184][1013] Disclosure Regarding Foreign Jurisdictions that Prevent Inspections This item is not applicable to the company - Not applicable[1185] Part III Directors, Executive Officers and Corporate Governance Information on directors, executive officers, and corporate governance is incorporated by reference from the forthcoming 2024 proxy statement - Information is incorporated by reference from the forthcoming 2024 proxy statement[989][990] Executive Compensation Information detailing executive compensation is incorporated by reference from the forthcoming 2024 proxy statement - Information is incorporated by reference from the forthcoming 2024 proxy statement[1014] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters Information on security ownership by beneficial owners and management, and equity compensation plans, is incorporated by reference from the forthcoming 2024 proxy statement - Information is incorporated by reference from the forthcoming 2024 proxy statement[1188] Certain Relationships and Related Transactions, and Director Independence Information on related-party transactions and director independence is incorporated by reference from the forthcoming 2024 proxy statement - Information is incorporated by reference from the forthcoming 2024 proxy statement[1188] Principal Accounting Fees and Services Information detailing principal accounting fees and services is incorporated by reference from the forthcoming 2024 proxy statement - Information is incorporated by reference from the forthcoming 2024 proxy statement[1189] Part IV Exhibits and Financial Statement Schedules This section lists documents filed with the Form 10-K, including consolidated financial statements, auditor's report, and an index of all exhibits - This section contains the list of financial statements and exhibits filed with the Form 10-K[1190] - Financial statement schedules have been omitted as not applicable or information is provided elsewhere[1233] Form 10-K Summary This section indicates that a summary of the Form 10-K is not applicable or has not been provided - Not applicable[1332]