United Therapeutics(UTHR) - 2023 Q4 - Annual Report

PART I Business Overview United Therapeutics is a public benefit corporation developing therapies for PAH/PH-ILD and advancing organ manufacturing, marketing products like Tyvaso DPI and Remodulin Overview United Therapeutics, a public benefit corporation, develops pharmaceutical therapies for PAH/PH-ILD and manufactured organs, marketing several commercial products - United Therapeutics operates as the first publicly-traded biotech or pharmaceutical public benefit corporation (PBC), with a dual purpose: developing novel pharmaceutical therapies and expanding the availability of transplantable organs[9] - The company's commercial portfolio includes Tyvaso DPI, nebulized Tyvaso, Remodulin, Orenitram, and Adcirca for PAH/PH-ILD, and Unituxin for high-risk neuroblastoma[10] - Key R&D areas include new indications/delivery devices for existing products, new PAH products, and manufactured organ products (xenotransplantation, regenerative medicine, bio-artificial organs, 3D organ bioprinting, ex vivo lung perfusion)[10] Our Commercial Products The company's commercial portfolio features treprostinil-based therapies for PAH/PH-ILD, with Tyvaso DPI driving growth amid generic competition for older products Products to Treat Pulmonary Hypertension The PH product line, including Tyvaso DPI and nebulized Tyvaso (only FDA-approved inhaled PH-ILD therapies), shows Tyvaso growth, but Remodulin and Adcirca face generic competition - Tyvaso DPI and nebulized Tyvaso are the only FDA-approved therapies for PH-ILD, a rare condition impacting at least 30,000 patients in the United States[19] Net Product Sales for PH Products (2021-2023) | Product | 2023 Sales ($M) | 2022 Sales ($M) | 2021 Sales ($M) | 2023 % of Total Revenue | 2023 YoY Change | | :------ | :-------------- | :-------------- | :-------------- | :---------------------- | :-------------- | | Tyvaso (DPI & Nebulized) | 1,233.7 | 873.0 | 607.5 | 53% | +41% | | Remodulin | 494.8 | 500.2 | 513.7 | 21% | -1% | | Orenitram | 359.4 | 325.1 | 306.1 | 15% | +11% | | Adcirca | 28.9 | 41.3 | 55.9 | 1% | -30% | - Generic versions of Adcirca launched in August 2018 and February 2019, resulting in a material adverse impact on Adcirca net product sales[41] Product to Treat Cancer — Unituxin Unituxin, an oncology product for high-risk neuroblastoma, generated $198.9 million in 2023 sales (9% of total revenue) but has severe side effects - Unituxin is approved for the treatment of high-risk neuroblastoma in the U.S., Canada, and Japan, and is a chimeric monoclonal antibody that induces antibody-dependent cell-mediated cytotoxicity[43] Unituxin Net Product Sales (2021-2023) | Year | Net Product Sales ($M) | % of Total Revenues | | :--- | :--------------------- | :------------------ | | 2023 | 198.9 | 9% | | 2022 | 182.9 | 9% | | 2021 | 202.3 | 12% | - Unituxin therapy is associated with severe side effects, including infections, infusion reactions, hypokalemia, hypotension, pain, fever, and capillary leak syndrome[43] Research and Development R&D focuses on new indications for existing products (e.g., nebulized Tyvaso for IPF/PPF), new therapies like ralinepag, and organ manufacturing via xenotransplantation and bioprinting - The company is conducting Phase 3 TETON studies for nebulized Tyvaso to treat Idiopathic Pulmonary Fibrosis (IPF) and Progressive Pulmonary Fibrosis (PPF), targeting enrollment of 576 and 698 patients, respectively[47][50] - In organ manufacturing, key accomplishments include successful xenotransplants of UHearts into two living human patients and a 61-day study of a UThymoKidney in a brain-dead organ donor, marking the longest documented case of a xenotransplanted organ functioning in a human body[63][64][66] - United Therapeutics acquired Miromatrix Medical Inc. (Dec 2023) and IVIVA Medical, Inc. (Oct 2023) to advance manufactured kidney and liver products, with Miromatrix's miroliverELAP® planning a Phase 1 human clinical trial in 2024[66][72] Sales and Marketing The marketing strategy for PAH and PH-ILD products aims to increase disease awareness, emphasize PAH progression, highlight PH-ILD risks, and promote commercial products - The marketing strategy aims to increase awareness of PAH and PH-ILD, communicate the progressive nature of PAH and the importance of early treatment, and highlight the increased risks for ILD patients who develop PH-ILD[75] Distribution of Commercial Products U.S. distribution of treprostinil-based products is via Accredo and CVS Specialty, Unituxin via ASD, and Adcirca via Lilly, with international sales through exclusive distributors - U.S. distribution of treprostinil-based products is handled by Accredo Health Group, Inc. and Caremark, L.L.C. (CVS Specialty), while Unituxin is distributed by ASD Specialty Healthcare, Inc., and Adcirca by Eli Lilly and Company's wholesaler network[76][78][80] - Internationally, Remodulin is sold through distributors like Grupo Ferrer Internacional, S.A., and nebulized Tyvaso through distributors in Argentina, Israel, and Mochida Pharmaceutical Co., Ltd. in Japan[82] Patents and Other Proprietary Rights, Strategic Licenses, and Market Exclusivity The company protects products with patents, licenses, and regulatory exclusivities, but faces ongoing generic competition and patent litigation, impacting future revenues - The company holds numerous patents for its treprostinil-based products (Tyvaso DPI, nebulized Tyvaso, Remodulin, Orenitram) with expiration dates ranging from 2024 to 2042, and a large portfolio for its organ manufacturing programs[86][87][90][93][95] - Tyvaso has three-year clinical trial exclusivity for PH-ILD (expires March 2024) and orphan drug designation for IPF, potentially granting seven-year exclusivity upon approval[94] - Ongoing patent litigation with Liquidia concerning Yutrepia (a dry powder treprostinil formulation) has resulted in a bar on FDA approval until May 2027 for the PAH indication, with further litigation regarding a PH-ILD indication[102][106] - Generic competition for Remodulin has led to decreased international revenues, and generic versions of Adcirca launched in 2018-2019 have materially impacted its sales[97][98][99] - Generic versions of nebulized Tyvaso and Orenitram are permitted to launch in 2026 and 2027, respectively, under settlement agreements[110] Manufacturing and Supply The company manufactures key drug products internally and maintains inventory, but relies heavily on third-party contract manufacturers for specialized products, posing supply chain risks - The company synthesizes treprostinil and dinutuximab, and manufactures drug products for nebulized Tyvaso, Remodulin, Unituxin, and Orenitram at its Silver Spring, Maryland, and Research Triangle Park, North Carolina facilities[125] - A minimum two-year inventory of nebulized Tyvaso, Remodulin, and Orenitram is maintained, supplemented by third-party contract manufacturers like Simtra BioPharma Solutions and Woodstock Sterile Solutions[126][127] - The company relies entirely on MannKind for Tyvaso DPI finished drug product and inhalers, Lilly for Adcirca, and DEKA for the Remunity Pump, with plans to build its own Tyvaso DPI manufacturing facility[128][129][130] Competition The company faces intense competition in PAH, PH-ILD, IPF, PPF, and neuroblastoma from numerous approved and investigational therapies, including generic and novel drugs - The company faces competition in PAH from over fifteen branded and generic drugs, including ERAs, prostacyclin-class therapies, PDE-5 inhibitors, and sGC stimulators, marketed by large pharmaceutical companies like Johnson & Johnson, Gilead Sciences, Inc., and Bayer Schering Pharma AG[132] - Investigational PAH therapies in late-stage development include Liquidia's Yutrepia (dry powder treprostinil) and Merck's sotatercept (activin signaling inhibitor), both of which could significantly impact sales of existing treprostinil-based products[133][135] - For PH-ILD, Tyvaso DPI and nebulized Tyvaso are currently the only approved therapies, but several PAH drug candidates are also being developed for this indication[139] - For IPF/PPF, the company's investigational Tyvaso therapies will compete with existing drugs like Ofev and Esbriet, and numerous other therapies in advanced clinical development[140] Governmental Regulation Operations are extensively regulated from R&D to marketing, including pharmaceutical, biologic, and medical device approvals, with unique challenges for organ manufacturing and compliance with reimbursement and privacy laws - The pharmaceutical product approval process involves preclinical testing, IND submission, multi-phase clinical studies, and FDA approval of an NDA or BLA, with special expedited pathways available for certain drugs[144][151] - Organ manufacturing programs face unique regulatory challenges, with xenotransplantation products regulated as biologics and genetic modifications as new animal drugs, requiring DPF facilities and cGMP compliance[191] - Government reimbursement programs like Medicaid and Medicare, along with the Inflation Reduction Act (IRA), significantly impact product pricing and reimbursement rates, potentially leading to price negotiations, inflation rebates, and increased Part D discounting obligations[195][199][208] - The company must comply with federal and state anti-kickback, false claims, and privacy laws (HIPAA, CCPA, GDPR), with violations potentially leading to significant penalties, fines, and exclusion from federal healthcare programs[213][214][223][226] Environmental Matters The company materially complies with environmental regulations, with no expected material impact on capital expenditures, cash flows, earnings, or competitive position - The company's operations comply in all material respects with federal, state, and local environmental laws and regulations, with no expected material effect on capital expenditures, cash flows, earnings, or competitive position[232] Human Capital United Therapeutics employs 1,168 'Unitherians' globally, maintaining low voluntary turnover (5.0% in 2023) through a diverse, inclusive culture and competitive rewards - As of December 31, 2023, United Therapeutics had approximately 1,168 employees ('Unitherians') worldwide, with 82 joining from recent acquisitions[233] - The company maintains a low voluntary turnover rate of 5.0% in 2023, significantly below the industry average of 13.5%, attributed to its focus on challenging work, career advancement, autonomy, inspiring environment, and competitive pay/benefits[235][237] - Diversity, Equity, and Inclusion (DEI) efforts are strong, with women representing 52% of Unitherians and racial/ethnic minorities 35% of the workforce, supported by resource groups and training initiatives[238] Corporate Website The company's website, unither.com, provides free access to SEC filings (Form 10-K, 10-Q, 8-K) promptly after filing - SEC filings, including Form 10-K, 10-Q, 8-K, and amendments, are available free of charge on the company's website (http://www.unither.com) and the SEC's website (http://www.sec.gov/edgar/searchedgar/companysearch.html)[242](index=242&type=chunk) INFORMATION ABOUT OUR EXECUTIVE OFFICERS Key executive officers as of February 21, 2024, include the Chairperson and CEO, President and COO, CFO and Treasurer, and EVP, General Counsel, and Corporate Secretary Executive Officers (as of Feb 21, 2024) | Name | Age | Position | | :-------------------------------- | :-- | :------------------------------------------ | | Martine Rothblatt, Ph.D., J.D., M.B.A. | 69 | Chairperson and Chief Executive Officer | | Michael Benkowitz | 52 | President and Chief Operating Officer | | James C. Edgemond | 56 | Chief Financial Officer and Treasurer | | Paul A. Mahon, J.D. | 60 | Executive Vice President, General Counsel, and Corporate Secretary | Risk Factors The company faces risks from reliance on treprostinil therapies, clinical trial failures, intense competition, pricing pressures, manufacturing complexities, IP challenges, cybersecurity threats, and PBC obligations - The company's revenues are heavily reliant on sales of treprostinil-based therapies (Tyvaso DPI, nebulized Tyvaso, Remodulin, Orenitram), making it vulnerable to sales declines from competitive challenges, regulatory changes, or pricing pressures[249] - Significant risks include potential failures or delays in clinical trials, intense competition from numerous approved and investigational drugs (e.g., Yutrepia, sotatercept), and the successful commercialization of products being dependent on adequate coverage and reimbursement from third-party payers, which are subject to increasing pricing pressures and healthcare reforms like the IRA[250][253][256][257] - Manufacturing strategy exposes the company to risks related to capacity expansion, cGMP compliance, supply chain disruptions, and the unprecedented complexities of organ manufacturing processes[259][260][261] - Dependence on third-party manufacturers and distributors is also a critical risk[265] - Intellectual property rights are continuously challenged by generic competitors and through patent litigation, which can be costly and lead to loss of market exclusivity[309][311] - Cybersecurity incidents also pose a material adverse effect on business operations and data integrity[316] - As a Delaware Public Benefit Corporation (PBC), directors must balance shareholder financial interests with public benefit purpose and stakeholder interests, which may lead to decisions not maximizing short-term shareholder value and could increase litigation risk[328][330] Unresolved Staff Comments There are no unresolved staff comments from the SEC - No unresolved staff comments[331] Cybersecurity The company's NIST-aligned cybersecurity program, overseen by the Audit Committee, includes an IMT for incident response, with no material incidents to date - The Audit Committee oversees cybersecurity risks, receiving regular reports from the Security, Risk and Compliance Director (SRC Director) and Chief Information Officer (CIO)[333] - The cybersecurity program follows the National Institute of Standards and Technology (NIST) framework, encompassing policies, platforms, procedures, and processes for assessing, identifying, and managing risks, including third-party vendor risks[336] - The incident response process involves five phases: Identify, Protect, Detect, Respond, and Recover, with a Testing, Training, & Exercise (TT&E) program for all IRT members[338] - To date, no cybersecurity incidents have had a material impact on the company's operations or financial condition, but future cyberattacks are recognized as a potential risk[341] Properties The company owns and leases facilities in multiple states for co-headquarters, manufacturing, R&D (organ regeneration, bioprinting, xenotransplantation), and ex vivo lung perfusion - The Silver Spring, Maryland campus serves as co-headquarters, manufactures active ingredients (treprostinil, dinutuximab) and drug products (nebulized Tyvaso, Remodulin, Unituxin), and houses an ex vivo lung perfusion center, with plans for manufactured lung production[342] - The Research Triangle Park, North Carolina facility is a co-headquarters, manufactures Orenitram, and handles packaging, warehousing, and distribution for several products[343] - A new Tyvaso DPI manufacturing facility and a warehouse/logistics hub are under construction nearby[344] - A 65,000 square foot designated pathogen-free (DPF) facility in Virginia was recently completed to produce porcine hearts and kidneys for xenotransplantation clinical trials[345] - Other key locations include Minnesota (Miromatrix subsidiary for manufactured kidney/liver R&D) and New Hampshire (3D organ bioprinting R&D)[346] Legal Proceedings The company is involved in various legal proceedings, not expected to materially harm financial position, but adverse rulings could have a material adverse effect - The company is subject to claims in legal proceedings arising in the normal course of business, with the ultimate outcome not presently believed to materially harm financial position, cash flows, or results of operations[348] - Legal proceedings are inherently uncertain, and unfavorable rulings could, individually or in aggregate, have a material adverse effect on the business, financial condition, or operating results[348] Mine Safety Disclosures This item is not applicable to United Therapeutics Corporation - Not applicable[349] PART II Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities UTHR common stock trades on Nasdaq; the company has 31 record holders, does not pay dividends, and made no equity repurchases in 2023 - The company's common stock trades on the Nasdaq Global Select Market under the symbol "UTHR"[350] - As of February 14, 2024, there were 31 holders of record of the company's common stock[351] - The company has never paid and does not intend to pay cash dividends, planning to retain any earnings for use in business operations[352] - No outstanding equity securities were repurchased during the year ended December 31, 2023[353] Item 6. [Reserved] This item is reserved and contains no information - This item is reserved[355] Management's Discussion and Analysis of Financial Condition and Results of Operations This section reviews financial performance, liquidity, and capital resources, noting 2023 revenue growth driven by Tyvaso DPI, increased R&D, and strong liquidity Overview of Marketed Products The company markets Tyvaso DPI, nebulized Tyvaso, Remodulin, Orenitram, Unituxin, and Adcirca for PAH, PH-ILD, and high-risk neuroblastoma - Tyvaso DPI and nebulized Tyvaso are approved for PAH and PH-ILD, with Tyvaso DPI commercially launched in June 2022[363] - Remodulin is a continuously-infused treprostinil for PAH, with the Remunity Pump launched in February 2021 for subcutaneous delivery[363] - Orenitram is an oral extended-release treprostinil for PAH, indicated to delay disease progression and improve exercise capacity[363] - Unituxin is a monoclonal antibody for high-risk neuroblastoma, and Adcirca is an oral PDE-5 inhibitor for PAH[363] Research and Development R&D focuses on new indications for existing products (e.g., nebulized Tyvaso for IPF/PPF), new PAH products like ralinepag, and organ transplantation technologies - R&D efforts include new indications and delivery devices for existing products, such as the Remunity Pump for Remodulin and TETON studies for nebulized Tyvaso in IPF and PPF[360] - The company is developing ralinepag, a new product for PAH, and is heavily engaged in organ transplantation-related technologies like xenotransplantation, regenerative medicine, 3D organ bioprinting, and ex vivo lung perfusion[361] Revenues Revenues derive from commercial product sales and devices, distributed via specialty channels, subject to distributor order fluctuations and gross-to-net deductions - Total revenues consist primarily of sales of commercial products and associated administration devices, distributed through contracted specialty pharmaceutical distributors in the U.S. (Accredo, CVS Specialty, ASD) and internationally[362] - Sales of treprostinil-based therapies can vary based on the timing and magnitude of distributor orders and do not always precisely reflect changes in patient demand[363] Operating Expenses Operating expenses comprise cost of sales, R&D, SG&A, and share-based compensation, with STAP awards introducing volatility due to re-measurement - Cost of sales includes manufacturing costs, royalties, distribution costs, and inventory reserves[365] - Research and development expenses cover internal and external R&D, preclinical and clinical studies, clinical manufacturing, regulatory costs, and upfront fees/milestone payments for development-stage therapies[366] - Share-based compensation, including cash-settled STAP awards, is re-measured at each reporting period, leading to potential volatility in operating expenses based on stock price fluctuations[369] Future Prospects Near-term growth is expected from Tyvaso DPI and PH-ILD expansion, with long-term growth from pipeline products and significant capital expenditures for new manufacturing facilities - Near-term revenue growth is anticipated from Tyvaso DPI sales, nebulized Tyvaso (PH-ILD label expansion), Orenitram, and modest price increases[370] - The company has budgeted approximately $500 million for capital expenditures during 2024-2026, primarily for constructing a new Tyvaso DPI manufacturing facility in Research Triangle Park, North Carolina[372] - The market is highly competitive, with potential erosion of market share from investigational products like Liquidia's Yutrepia and Merck's sotatercept, especially if Yutrepia is approved for PH-ILD[373] Results of Operations In 2023, total revenues grew 20% to $2,327.5 million, driven by Tyvaso DPI, while operating expenses increased due to R&D and manufacturing investments Revenues Total revenues increased 20% to $2,327.5 million in 2023, primarily from 41% growth in Tyvaso sales, driven by Tyvaso DPI launch Total Revenues and Net Product Sales (2021-2023) | Product | 2023 ($M) | 2022 ($M) | 2021 ($M) | 2023 vs 2022 Change ($M) | 2023 vs 2022 Change (%) | | :---------------- | :-------- | :-------- | :-------- | :----------------------- | :---------------------- | | Tyvaso DPI | 731.1 | 158.3 | — | 572.8 | 362% | | Nebulized Tyvaso | 502.6 | 714.7 | 607.5 | (212.1) | (30)% | | Total Tyvaso | 1,233.7 | 873.0 | 607.5 | 360.7 | 41% | | Remodulin | 494.8 | 500.2 | 513.7 | (5.4) | (1)% | | Orenitram | 359.4 | 325.1 | 306.1 | 34.3 | 11% | | Unituxin | 198.9 | 182.9 | 202.3 | 16.0 | 9% | | Adcirca | 28.9 | 41.3 | 55.9 | (12.4) | (30)% | | Other | 11.8 | 13.8 | — | (2.0) | (14)% | | Total Revenues | 2,327.5 | 1,936.3 | 1,685.5 | 391.2 | 20% | - Total Tyvaso net product sales grew 41% in 2023, primarily due to the commercial launch of Tyvaso DPI in June 2022 and continued growth in utilization by PH-ILD patients[377] - Nebulized Tyvaso net product sales decreased in the U.S. in 2023 following the Tyvaso DPI launch, but international sales increased due to the commercial launch in Japan[377] Gross-to-Net Deductions Gross-to-net deductions, including rebates and chargebacks, increased to $108.4 million in 2023, based on estimates and contractual requirements Rebates & Chargebacks Liability (2021-2023) | Year Ended December 31, | Balance, January 1 ($M) | Provisions Current Period ($M) | Provisions Prior Periods ($M) | Payments Current Period ($M) | Payments Prior Periods ($M) | Balance, December 31 ($M) | | :---------------------- | :---------------------- | :----------------------------- | :---------------------------- | :--------------------------- | :-------------------------- | :-------------------------- | | 2023 | 81.3 | 278.0 | (2.5) | (169.8) | (78.6) | 108.4 | | 2022 | 67.8 | 202.8 | (4.3) | (121.1) | (63.9) | 81.3 | | 2021 | 65.3 | 217.0 | 1.6 | (151.8) | (64.3) | 67.8 | - Gross-to-net deductions are primarily based on estimates reflecting historical experiences and contractual/statutory requirements, with adjustments for prior periods historically being less than one percent of total revenues[381][411] Cost of Sales Total cost of sales increased 70% to $257.5 million in 2023, mainly due to higher Tyvaso DPI royalty and product costs, and increased Remunity sales Cost of Sales (2021-2023) | Category | 2023 ($M) | 2022 ($M) | 2021 ($M) | 2023 vs 2022 Change ($M) | 2023 vs 2022 Change (%) | | :--------------------- | :-------- | :-------- | :-------- | :----------------------- | :---------------------- | | Cost of sales | 255.1 | 146.7 | 116.7 | 108.4 | 74% | | Share-based compensation | 2.4 | 4.9 | 5.8 | (2.5) | (51)% | | Total cost of sales | 257.5 | 151.6 | 122.5 | 105.9 | 70% | - The increase in cost of sales in 2023 was primarily due to higher Tyvaso DPI royalty expense and product costs following its commercial launch, and an increase in Remunity product sales[384] Research and Development Total R&D expense increased 26% to $408.0 million in 2023, driven by the IVIVA acquisition, TETON studies, and organ manufacturing projects Research and Development Expense (2021-2023) | Category | 2023 ($M) | 2022 ($M) | 2021 ($M) | 2023 vs 2022 Change ($M) | 2023 vs 2022 Change (%) | | :----------------------------- | :-------- | :-------- | :-------- | :----------------------- | :---------------------- | | External R&D | 192.0 | 168.8 | 156.7 | 23.2 | 14% | | Internal R&D | 146.6 | 131.4 | 117.2 | 15.2 | 12% | | Share-based compensation | 15.6 | 23.8 | 24.4 | (8.2) | (34)% | | Impairments | — | — | 130.0 | — | —% | | Other | 53.8 | (1.1) | 111.8 | 54.9 | NM | | Total R&D expense | 408.0 | 322.9 | 540.1 | 85.1 | 26% | - The increase in R&D expense in 2023 was driven by increased IPR&D expense from the IVIVA acquisition, higher expenditures for the TETON 1 and TETON 2 clinical studies, and increased spending on organ manufacturing projects[387] Selling, General, and Administrative Total SG&A expense slightly decreased by 1% to $477.1 million in 2023, with increases in G&A and sales/marketing offset by other factors Selling, General, and Administrative Expense (2021-2023) | Category | 2023 ($M) | 2022 ($M) | 2021 ($M) | 2023 vs 2022 Change ($M) | 2023 vs 2022 Change (%) | | :----------------------------- | :-------- | :-------- | :-------- | :----------------------- | :---------------------- | | General and administrative | 374.2 | 333.2 | 294.3 | 41.0 | 12% | | Sales and marketing | 81.8 | 70.8 | 64.4 | 11.0 | 16% | | Share-based compensation | 21.1 | 78.1 | 108.3 | (57.0) | (73)% | | Total SG&A expense | 477.1 | 482.1 | 467.0 | (5.0) | (1)% | - General and administrative expense increased primarily due to increases in office expenses, personnel expense from headcount growth, and sponsorships and grants[389] - Sales and marketing expense increased primarily due to increases in personnel expense from headcount growth and consulting expenses[390] Share-Based Compensation Total share-based compensation decreased 63% to $39.1 million in 2023, primarily due to a 21% stock price decrease impacting STAP awards Share-Based Compensation Expense by Category (2021-2023) | Category | 2023 ($M) | 2022 ($M) | 2021 ($M) | 2023 vs 2022 Change ($M) | 2023 vs 2022 Change (%) | | :---------------------- | :-------- | :-------- | :-------- | :----------------------- | :---------------------- | | Stock options | 15.4 | 22.6 | 25.4 | (7.2) | (32)% | | Restricted stock units | 52.4 | 35.7 | 24.7 | 16.7 | 47% | | STAP awards | (30.7) | 46.7 | 86.6 | (77.4) | (166)% | | Employee stock purchase plan | 2.0 | 1.8 | 1.8 | 0.2 | 11% | | Total | 39.1 | 106.8 | 138.5 | (67.7) | (63)% | - The decrease in share-based compensation expense in 2023 was primarily due to an increase in STAP benefit, driven by a 21% decrease in the company's stock price during 2023 (compared to a 29% increase in 2022), and fewer outstanding stock options[391] Other (Expense) Income, Net The change in other (expense) income, net in 2023 was primarily due to net unrealized and realized gains/losses on equity securities - The change in other (expense) income, net for 2023 was primarily due to net unrealized and realized gains and losses on equity securities[392] Income Tax Expense Income tax expense increased to $289.5 million in 2023 from $223.3 million in 2022, with an effective tax rate of approximately 23% Income Tax Expense (2021-2023) | Year | Income Tax Expense ($M) | Effective Income Tax Rate | | :--- | :---------------------- | :------------------------ | | 2023 | 289.5 | 23% | | 2022 | 223.3 | 23% | | 2021 | 118.1 | 20% | Financial Condition, Liquidity, and Capital Resources The company maintains strong liquidity with $4,903.9 million in cash and investments, increased operating cash flow, and manages a $2.0 billion credit facility Cash and Cash Equivalents and Marketable Investments Total cash, cash equivalents, and marketable investments increased 18% to $4,903.9 million in 2023, driven by rises in cash and non-current investments Cash and Marketable Investments (2022-2023) | Category | 2023 ($M) | 2022 ($M) | Dollar Change ($M) | Percentage Change (%) | | :------------------------------------------ | :-------- | :-------- | :----------------- | :-------------------- | | Cash and cash equivalents | 1,207.7 | 961.2 | 246.5 | 26% | | Marketable investments—current | 1,786.4 | 1,877.5 | (91.1) | (5)% | | Marketable investments—non-current | 1,909.8 | 1,316.2 | 593.6 | 45% | | Total | 4,903.9 | 4,154.9 | 749.0 | 18% | Cash Flows Net cash from operations increased 22% to $978.0 million, investing cash use decreased 11% to $719.6 million, and financing cash use was $11.9 million in 2023 Cash Flows Summary (2021-2023) | Activity | 2023 ($M) | 2022 ($M) | 2021 ($M) | 2023 vs 2022 Change ($M) | 2023 vs 2022 Change (%) | | :----------------------------------- | :-------- | :-------- | :-------- | :----------------------- | :---------------------- | | Net cash provided by operating activities | 978.0 | 802.5 | 598.2 | 175.5 | 22% | | Net cash used in investing activities | (719.6) | (811.5) | (486.9) | 91.9 | 11% | | Net cash (used in) provided by financing activities | (11.9) | 75.4 | 44.8 | (87.3) | (116)% | - The increase in net cash from operating activities was primarily due to a $53.5 million decrease in cash paid to settle STAP awards and changes in other assets and liabilities[399] - The decrease in net cash used in investing activities was primarily due to a $306.3 million decrease in cash used for marketable investments, partially offset by increased capital expenditures ($91.6 million), acquisitions ($89.2 million), and deposits ($23.0 million)[400] Unsecured Revolving Credit Facilities The company has a $2.0 billion unsecured revolving credit facility, with $700.0 million outstanding as of December 31, 2023, and $400.0 million reclassified as current - The company has a $2.0 billion unsecured revolving credit facility (2022 Credit Agreement) maturing in March 2028[402] - As of December 31, 2023, $700.0 million was outstanding under the credit agreement, with $400.0 million reclassified as a current liability due to the intent to repay within one year[402][403][524] Contractual Obligations Total contractual obligations were $1,719.6 million as of December 31, 2023, including debt, leases, STAP, SERP, and purchase obligations, plus royalties Contractual Obligations as of December 31, 2023 ($M) | Obligation Type | Total | Less than 1 year | 2-3 Years | 4-5 Years | More than 5 Years | | :---------------------- | :---- | :--------------- | :-------- | :-------- | :---------------- | | Operating lease obligations | 32.8 | 4.7 | 8.5 | 8.1 | 11.5 | | Long-term debt obligations | 822.3 | 441.5 | 49.7 | 331.1 | — | | Obligations under the STAP | 31.3 | 31.3 | — | — | — | | Obligations under the SERP | 61.0 | 30.5 | — | 11.2 | 19.3 | | Purchase obligations | 772.2 | 586.6 | 150.8 | 26.1 | 8.7 | | Total | 1,719.6 | 1,094.6 | 209.0 | 376.5 | 39.5 | - The company has ongoing royalty obligations for products like Tyvaso DPI (10% to MannKind), ralinepag (low double-digit tiered royalty to Arena), Adcirca (10% to Lilly), Orenitram (single-digit to Supernus), and Unituxin (1% to Scripps), plus potential milestone payments[405][577] Off-Balance Sheet Arrangements The company holds an interest in an unconsolidated VIE for Remunity Pump manufacturing, with a maximum loss exposure of $7.1 million as of December 31, 2023 - The company has an interest in an unconsolidated VIE, an affiliate of DEKA, which manufactures the Remunity Pump[406] - The company is not the primary beneficiary but has a maximum exposure to loss of $7.1 million as of December 31, 2023[510] Summary of Critical Accounting Policies and Estimates Critical accounting policies involve significant estimates for revenue recognition (gross-to-net deductions) and share-based compensation (STAP awards), impacting recognized expense - Revenue recognition involves significant estimates for gross-to-net deductions, especially rebates and chargebacks, due to time lags between accrual and invoicing, which can extend up to several years for Medicaid programs[408][410][411] - Share-based compensation for STAP awards (cash-settled liabilities) is re-measured at each reporting date, and its fair value estimation using the Black-Scholes-Merton model relies on subjective assumptions like expected volatility and term, which can materially impact recognized expense[413] Recently Issued Accounting Standards FASB issued ASU 2023-07 (Segment Reporting) and ASU 2023-09 (Income Tax Disclosures), effective after December 15, 2023, and 2024, respectively, with impact under assessment - ASU 2023-07 (Segment Reporting) requires enhanced disclosures about significant segment expenses and interim period disclosures, effective for fiscal years beginning after December 15, 2023[493] - ASU 2023-09 (Income Tax Disclosures) enhances required disclosures for income tax rate reconciliation and disaggregated income taxes paid, effective for annual periods beginning after December 15, 2024[494] Quantitative and Qualitative Disclosures About Market Risk The company faces investment risk from its $3.8 billion debt portfolio and interest rate risk on $700.0 million variable-rate debt, mitigated by short-maturity, highly-rated securities - The company has $3.8 billion invested in corporate debt securities and U.S. government and agency securities, which are subject to market and interest rate risk[415] - Investment risk is mitigated by investing in unstructured, highly-rated securities with relatively short maturities (no longer than three years), typically held to maturity[415] - The company has $700.0 million outstanding under its 2022 Credit Agreement, which bears variable interest[416] - A 100-basis point increase in the variable interest rate would increase annual interest expense by approximately $7.0 million (12%) for 2023[416] Financial Statements and Supplementary Data This section presents audited consolidated financial statements, including balance sheets, statements of operations, comprehensive income, stockholders' equity, cash flows, and detailed notes - The section includes the Report of Independent Registered Public Accounting Firm, expressing an unqualified opinion on the consolidated financial statements[419] - It also contains the Report of Independent Registered Public Accounting Firm on Internal Control Over Financial Reporting, expressing an unqualified opinion on the effectiveness of internal control as of December 31, 2023[420][427] - Key financial statements presented are the Consolidated Balance Sheets, Statements of Operations, Comprehensive Income, Stockholders' Equity, and Cash Flows for the years ended December 31, 2023, 2022, and 2021[418] - Detailed notes to the consolidated financial statements provide information on accounting policies, investments, debt, share-based compensation, income taxes, employee benefit plans, commitments and contingencies, segment information, litigation, and acquisitions[418] Report of Independent Registered Public Accounting Firm Ernst & Young LLP issued an unqualified opinion on the consolidated financial statements and internal control over financial reporting, noting rebate accounting as a critical audit matter - Ernst & Young LLP issued an unqualified opinion on the consolidated financial statements for the three years ended December 31, 2023, stating they are presented fairly in all material respects in conformity with U.S. GAAP[419] - The firm also expressed an unqualified opinion on the effectiveness of the company's internal control over financial reporting as of December 31, 2023[420] - A critical audit matter identified was the accounting for rebates, due to the judgmental nature of assumptions, time lags in third-party reporting, and complexities in government pricing calculations, requiring specialist involvement[423][426] Report of Independent Registered Public Accounting Firm on Internal Control Over Financial Reporting Ernst & Young LLP issued an unqualified opinion on the effectiveness of internal control over financial reporting as of December 31, 2023, based on COSO criteria - Ernst & Young LLP issued an unqualified opinion on United Therapeutics Corporation's internal control over financial reporting as of December 31, 2023, based on criteria established in the COSO framework[427] - Management is responsible for maintaining effective internal control over financial reporting, and the audit provides reasonable assurance that effective internal control was maintained in all material respects[429][430] Consolidated Balance Sheets As of December 31, 2023, total assets increased to $7,167.0 million, liabilities decreased to $1,182.2 million, and stockholders' equity rose to $5,984.8 million Consolidated Balance Sheet Summary (2022-2023) | Category | Dec 31, 2023 ($M) | Dec 31, 2022 ($M) | | :-------------------------------- | :---------------- | :---------------- | | Total current assets | 3,551.0 | 3,380.3 | | Total assets | 7,167.0 | 6,044.5 | | Total current liabilities | 804.4 | 343.2 | | Total liabilities | 1,182.2 | 1,247.8 | | Total stockholders' equity | 5,984.8 | 4,796.7 | - Cash and cash equivalents increased from $961.2 million in 2022 to $1,207.7 million in 2023[434] - A $400.0 million portion of the line of credit was reclassified as a current liability in 2023, reflecting the intent to repay within one year[434] Consolidated Statements of Operations In 2023, total revenues reached $2,327.5 million, operating income $1,184.9 million, and net income $984.8 million ($19.81 diluted EPS) Consolidated Statements of Operations Summary (2021-2023) | Metric | 2023 ($M) | 2022 ($M) | 2021 ($M) | | :-------------------------- | :-------- | :-------- | :-------- | | Total revenues | 2,327.5 | 1,936.3 | 1,685.5 | | Total operating expenses | 1,142.6 | 956.6 | 1,129.6 | | Operating income | 1,184.9 | 979.7 | 555.9 | | Income before income taxes | 1,274.3 | 950.6 | 593.9 | | Income tax expense | (289.5) | (223.3) | (118.1) | | Net income | 984.8 | 727.3 | 475.8 | | Diluted EPS | 19.81 | 15.00 | 10.06 | - Net income increased by $257.5 million from 2022 to 2023, reflecting a 20% increase in total revenues and changes in operating expenses[435] Consolidated Statements of Comprehensive Income Comprehensive income for 2023 was $1,027.5 million, including $984.8 million net income and $42.7 million other comprehensive income from unrealized gains Consolidated Statements of Comprehensive Income (2021-2023) | Metric | 2023 ($M) | 2022 ($M) | 2021 ($M) | | :------------------------------------ | :-------- | :-------- | :-------- | | Net income | 984.8 | 727.3 | 475.8 | | Total defined benefit pension plan, net of tax | (2.9) | 19.3 | 6.2 | | Unrealized gain (loss) on available-for-sale securities, net of tax | 45.6 | (51.8) | (15.0) | | Other comprehensive income (loss), net of tax | 42.7 | (32.5) | (8.8) | | Comprehensive income | 1,027.5 | 694.8 | 467.0 | - Other comprehensive income (loss) in 2023 was positively impacted by $45.6 million in unrealized gains on available-for-sale securities, a reversal from unrealized losses in prior years[437] Consolidated Statements of Stockholders' Equity Total stockholders' equity increased to $5,984.8 million as of December 31, 2023, driven by net income and unrealized gains on securities Consolidated Stockholders' Equity Summary (2021-2023) | Category | Dec 31, 2023 ($M) | Dec 31, 2022 ($M) | Dec 31, 2021 ($M) | | :-------------------------- | :---------------- | :---------------- | :---------------- | | Common Stock | 0.7 | 0.7 | 0.7 | | Additional paid-in capital | 2,549.0 | 2,388.4 | 2,245.4 | | Accumulated other comprehensive loss | (12.8) | (55.5) | (23.0) | | Treasury stock | (2,579.2) | (2,579.2) | (2,579.2) | | Retained earnings | 6,027.1 | 5,042.3 | 4,315.0 | | Total Stockholders' Equity | 5,984.8 | 4,796.7 | 3,958.9 | - The increase in stockholders' equity was primarily due to net income of $984.8 million and $45.6 million in unrealized gains on available-for-sale securities in 2023[440] Consolidated Statements of Cash Flows Net cash from operations increased to $978.0 million, investing cash use decreased to $719.6 million, and financing cash use was $11.9 million in 2023 Consolidated Statements of Cash Flows Summary (2021-2023) | Cash Flow Activity | 2023 ($M) | 2022 ($M) | 2021 ($M) | | :----------------------------------- | :-------- | :-------- | :-------- | | Net cash provided by operating activities | 978.0 | 802.5 | 598.2 | | Net cash used in investing activities | (719.6) | (811.5) | (486.9) | | Net cash (used in) provided by financing activities | (11.9) | 75.4 | 44.8 | | Net increase in cash and cash equivalents | 246.5 | 66.4 | 156.1 | | Cash and cash equivalents, end of year | 1,207.7 | 961.2 | 894.8 | - Net cash provided by operating activities increased by $175.5 million in 2023, primarily due to a $53.5 million decrease in cash paid to settle STAP awards[399][442] - Net cash used in investing activities decreased by $91.9 million, driven by a $306.3 million decrease in cash used for marketable investments, partially offset by increased purchases of property, plant, and equipment and acquisitions[400][442] Notes to Consolidated Financial Statements The notes detail accounting policies, investments, debt, share-based compensation, income taxes, employee benefits, commitments, segment information, litigation, and acquisitions - The notes detail the company's accounting policies, including revenue recognition, share-based compensation, and fair value measurements, emphasizing the use of estimates and assumptions[447][469] - Information on investments includes available-for-sale debt securities and equity investments in privately-held companies, with details on unrealized losses and impairment reviews[495][503] - Significant litigation includes the Sandoz litigation regarding generic Remodulin, ongoing patent and trade secret litigation with Liquidia concerning Yutrepia, and lawsuits against the FDA and HHS regarding Yutrepia's PH-ILD indication and 340B program policies[584][589][598][601][609] - The company completed the acquisitions of IVIVA Medical, Inc. (asset acquisition) and Miromatrix Medical Inc. (business combination) in Q4 2023, with details on purchase price allocation and contingent consideration[616][617] Changes in and Disagreements with Accountants on Accounting and Financial Disclosure There have been no changes in or disagreements with the company's accountants on accounting and financial disclosure - None[629] Controls and Procedures Management assessed disclosure controls and internal control over financial reporting as effective as of December 31, 2023, with no material changes in Q4 2023 - Disclosure controls and procedures were evaluated and deemed effective as of December 31, 2023[630] - Management concluded that internal control over financial reporting was effective as of December 31, 2023, with an unqualified attestation report from Ernst & Young LLP[632][633] - No material changes in internal control over financial reporting occurred during the quarter ended December 31, 2023[635] Other Information Director Raymond Dwek adopted a Rule 10b5-1(c) trading plan on November 27, 2023, to exercise up to 10,000 STAP awards before June 26, 2024 - Raymond Dwek, a director, adopted a Rule 10b5-1(c) trading plan on November 27, 2023, to exercise up to 10,000 STAP awards between April 8, 2024, and April 26, 2024, which are set to expire on June 26, 2024[636] Disclosure Regarding Foreign Jurisdictions that Prevent Inspections This item is not applicable to United Therapeutics Corporation - Not applicable[638] PART III Directors, Executive Officers, and Corporate Governance Information on directors, executive officers, and corporate governance is incorporated from the 2024 Proxy Statement, including the Code of Conduct and diverse Board - Information on directors, executive officers, and corporate governance is incorporated by reference from the company's definitive proxy statement for the 2024 annual meeting of shareholders[639] - The company has a written Code of Conduct and Business Ethics that applies to all directors, officers, and employees, available on its investor relations website[641] - The Board of Directors includes individuals such as Martine Rothblatt (Chairperson and CEO), Raymond Dwek (Oxford Glycobiology Institute), Ray Kurzweil (Google Inc.), and Louis Sullivan (Former Secretary, U.S. Department of Health and Human Services)[642][643][644][645] Executive Compensation Executive compensation information, including director compensation and analysis, is incorporated by reference from the 2024 Proxy Statement - Information concerning executive compensation is incorporated by reference from the 2024 Proxy Statement under headings such as Director Compensation, Compensation Discussion and Analysis, and Executive Compensation Data[646] Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters Beneficial ownership information is from the 2024 Proxy Statement; 7,178,612 securities were authorized under equity plans as of December 31, 2023 - Information regarding beneficial ownership of common stock is incorporated by reference from the 2024 Proxy Statement[647] Securities Authorized for Issuance Under Equity Compensation Plans (as of Dec 31, 2023) | Plan Category | Number of securities to be issued upon exercise of outstanding options and RSUs (a) | Weighted average exercise price of outstanding options ($) (b) | Number of securities remaining available for future issuance (c) | | :------------------------------------------ | :---------------------------------------------------------------- | :----------------------------------------- | :--------------------------------------------------------------- | | Equity compensation plan approved by security holders | 7,153,777 | 136.60 | 5,273,018 | | Equity compensation plan not approved by security holders | 24,835 | — | 24,586 | | Total | 7,178,612 | 136.60 | 5,297,604 | Certain Relationships and Related Transactions, and Director Independence Information on certain relationships, related party transactions, and director independence is incorporated by reference from the 2024 Proxy Statement - Information concerning related party transactions and director independence is incorporated by reference from the 2024 Proxy Statement[651] Principal Accountant Fees and Services Information on principal accountant fees and Audit Committee pre-approval policies is incorporated by reference from the 2024 Proxy Statement - Information regarding principal accountant fees and Audit Committee's pre-approval policies is incorporated by reference from the 2024 Proxy Statement[652] PART IV Exhibits and Financial Statement Schedules This section lists all exhibits and financial statement schedules filed with Form 10-K, including a disclaimer on contractual representations and availability of copies - The section lists exhibits and financial statement schedules, including the company's financial statements and notes, as part of the Form 10-K[656] - A disclaimer notes that representations and warranties in agreements are for allocating risk between parties and may not reflect actual affairs or be material to investors[653][654] - Copies of individual exhibits can be furnished to shareholders upon written request and payment of a reasonable fee[655] Form 10-K Summary This item indicates that no Form 10-K Summary is provided - None[665] Signatures The report is duly signed by the Chairperson and CEO, CFO and Treasurer, and all Directors of United Therapeutics Corporation as of February 21, 2024 - The report is signed by Martine Rothblatt (Chairperson and Chief Executive Officer), James C. Edgemond (Chief Financial Officer and Treasurer), and all Directors as of February 21, 2024[666][668]