Glomerulosclerosis, Focal Segmental

Phase 2a trial is on track to begin in the first half of 2024.Cholesterol Efflux MediatorTM VAR 200 is in development to ameliorate renal lipid accumulation that damages the kidneys’ filtration system, leading to chronic kidney disease and its progression. WESTON, Fla., March 18, 2024 (GLOBE NEWSWIRE) -- ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for the treatment of renal and inflammatory diseases with high unmet medical needs, announces Institutional Review Board (IRB) approval of the Phase 2a clinical trial protocol to evaluate the efficacy and safety of Cholesterol Efflux Mediator VAR 200 in patients with diabetic kidney disease. The clinical trial is on track to begin in the first half of 2024. Initiation of this clinical trial is a key milestone for ZyVersa. It is the first in human trial for VAR 200 intended to substantiate that the promising preclinical results demonstrated in three different animal models of kidney disease (diabetic kidney disease, focal segmental glomerulosclerosis, and Alport syndrome) translate to patients with kidney disease. The preclinical data demonstrated across all three kidney disease models that VAR 200: Reduced cholesterol and lipid levels in the kidneys’ filtration system.Protected against kidney injury and fibrosis.Significantly reduced protein in the urine (proteinuria). For more information about VAR 200, Click Here. “A large body of evidence reinforces the importance of addressing kidney accumulation of cholesterol and lipids to help attenuate progression of chronic kidney disease, which affects over 35 million adults in the United States,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO, and President. “Currently, over 130,000 patients with kidney disease progress to renal failure each year, and more than 800,000 patients are living with renal failure requiring dialysis or transplant to sustain life. We are excited about the potential of Cholesterol Efflux MediatorTM VAR 200 to protect against kidney injury and reduce kidney disease progression.” About ZyVersa Therapeutics, Inc. ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and peripheral inflammatory diseases. For more information, please visit www.zyversa.com. Cautionary Statement Regarding Forward-Looking Statements Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing. New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law. This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities. Corporate, Media, and IR Contact:Karen CashmereChief Commercial Officerkcashmere@zyversa.com786-251-9641

Conference Call and Webcast at 8:30 a.m. Eastern Time Today SAN DIEGO--(BUSINESS WIRE)-- Ligand Pharmaceuticals Inc. (Nasdaq: LGND) today reported financial results for the three and twelve months ended December 31, 2023, and provided an operating forecast and business updates. Ligand management will host a conference call and webcast today beginning at 8:30 a.m. Eastern time to discuss this announcement and answer questions. “2023 was a transformative year for Ligand, both operationally and financially. We refocused the company to be a lean-infrastructure, high-margin business,” said Todd Davis, CEO of Ligand. “We enhanced our deal making capabilities with the strengthening of our senior team and opening of a Boston office. These initiatives will help us execute on a larger scale and continue to expand our portfolio through a focus on life science royalty opportunities. We are now well positioned and resourced to close on multiple new investments. We also saw important clinical and regulatory events across our existing partnered assets and expect this momentum will continue in 2024 and beyond.” Fourth Quarter 2023 Financial Results Total revenues for the fourth quarter of 2023 were $28.1 million. Revenues for the same period in 2022 excluding sales related to COVID-19 were to $26.8 million. Total revenues for the fourth quarter of 2022 including COVID-19 related sales were $50.4 million. Royalties for the fourth quarter of 2023 were $22.5 million, compared with $22.0 million for the same period in 2022. Core Captisol® sales (excluding sales of Captisol related to COVID-19) were $3.9 million for the fourth quarter of 2023, compared with $3.3 million for the same period in 2022. There were no Captisol sales related to treatments for COVID-19 for the fourth quarter of 2023, compared with $23.5 million for the same period in 2022. Contract revenue was $1.7 million for the fourth quarter of 2023, compared with $1.5 million for the same period in 2022, with the difference due to the timing of partner milestone events. Costs of Captisol sales were $1.6 million for the fourth quarter of 2023, compared with $21.6 million for the same period in 2022, with the decrease due to lower total Captisol sales during the fourth quarter of 2023 and $9.8 million in accelerated depreciation on Captisol manufacturing equipment during the fourth quarter of 2022. Amortization of intangibles was $8.3 million for the fourth quarter of 2023, compared with $8.5 million for the same period in 2022. Research and development expenses were $5.5 million for the fourth quarter of 2023, compared with $9.2 million for the same period in 2022, with the decrease attributed to lower stock-based compensation, employee related expenses and lab supply expenses. General and administrative expenses were $16.0 million for the fourth quarter of 2023, compared with $31.1 million for the same period in 2022, with the decrease primarily attributed to lower stock-based compensation and employee related expenses for the fourth quarter of 2023 and a one-time stock compensation expense associated with the retirement of our former CEO in the fourth quarter of 2022. Net income from continuing operations for the fourth quarter of 2023 was $18.2 million, or $1.03 per diluted share, compared to a net loss of $14.5 million, or $0.86 per share, for the same period in 2022. Net income from continuing operations for the fourth quarter of 2023 included a $16.0 million gain in short term investment primarily driven by an increase in the value of our holdings in Viking Therapeutics (Nasdaq: VKTX) stock. The net loss for the fourth quarter of 2022 was impacted by the aforementioned Captisol equipment accelerated depreciation, a one-time stock compensation expense and a $24.8 million deferred tax asset valuation allowance during the fourth quarter of 2022, which was partially offset by a non-cash gain of $44.2 million from the value of Ligand's short-term investments. Adjusted net income from continuing operations for the fourth quarter of 2023 was $24.4 million, or $1.38 per diluted share, compared to $23.5 million, or $1.36 per diluted share, for the same period in 2022. Excluding the impact of gains from sales of Viking Therapeutics stock and gross profit from Captisol sales related to COVID-19, core adjusted net income for the fourth quarter of 2023 was $18.6 million, or $1.05 per diluted share, compared with $13.0 million, or $0.75 per diluted share, for the same period in 2022. The table below shows a reconciliation of net income (loss) from continuing operations to adjusted net income from continuing operations. As of December 31, 2023, Ligand had cash, cash equivalents and short-term investments of $170.3 million. Full Year 2023 Financial Results Total revenues for 2023 were $131.3 million. Revenues for 2022, excluding Captisol sales related to COVID-19, were $108.2 million. Total revenues for 2022 including COVID-19 related sales were $196.2 million. Royalties for 2023 were $83.9 million, compared with $72.5 million for 2022, with the increase primarily attributable to the increase in sales of Kyprolis®, Rylaze®, Vaxneuvance™ and Pneumosil®. Core Captisol sales were $28.4 million for 2023, compared with $16.4 million for 2022. The difference in sales was due to the timing of customer orders. There were no Captisol sales related to COVID-19 in 2023, compared with $88.1 million in 2022. Contract revenue for 2023 was $19.0 million, compared to $19.2 million for 2022. Cost of Captisol sales were $10.5 million for 2023, compared to $52.8 million for 2022, with the decrease due to lower total sales of Captisol during 2023 and $9.8 million in accelerated depreciation on Captisol manufacturing equipment during 2022. Amortization of intangibles was $33.7 million for 2023, compared with $34.2 million for 2022. Research and development expenses were $24.5 million for 2023, compared with $36.1 million for 2022, with the decrease primarily attributed to lower share-based compensation and employee-related expenses, partially offset by an increase in R&D expenses due to the Novan acquisition. General and administrative expenses were $52.8 million for 2023, compared with $70.1 million for 2022, with the decrease primarily attributable to decreases in share-based compensation expenses including a one-time charge associated with the retirement of our former CEO in the fourth quarter of 2022, and employee-related expenses. In September 2023, Ligand spun out its Pelican subsidiary through a merger with Primordial Genetics, to form a privately held company, Primrose Bio, which resulted in a gain on the sale of Pelican in the amount of $2.1 million for 2023. Net income from continuing operations for 2023 was $53.8 million, or $3.03 per diluted share, compared to a net loss from continuing operations of $5.2 million, or $0.31 per share for 2022. The increase in net income from the prior year is due primarily to the aforementioned Captisol equipment accelerated depreciation in 2022, a one-time stock compensation expense and a $24.8 million deferred tax asset valuation allowance during 2022; partially offset by an increase in gain from short-term investments which increased to $46.4 million in 2023 from $28.5 million in 2022. Adjusted net income from continuing operations for 2023 was $107.4 million or $6.09 per diluted share, compared with $82.2 million, or $4.79 per diluted share for 2022. Excluding the impact of gains from sales of Viking Therapeutics stock and gross profit from Captisol sales related to COVID-19, core adjusted net income for 2023 was $71.7 million, or $4.06 per diluted share, compared with $41.9 million, or $2.44 per diluted share, for 2022. The table below shows a reconciliation of net income (loss) from continuing operations to adjusted net income from continuing operations. 2024 Financial Guidance Ligand is reaffirming 2024 financial guidance introduced at its Investor and Analyst Day held on December 12, 2023. The Company expects 2024 royalties ranging from $90 million to $95 million, sales of Captisol ranging from $25 million to $27 million and contract revenue ranging from $15 million to $20 million. These revenue components result in total revenue forecast of $130 million to $142 million. Ligand notes that with total revenue of $130 million to $142 million, adjusted earnings per diluted share are expected to range from approximately $4.25 to $4.75. Fourth Quarter 2023 and Recent Business Highlights On January 5, 2024, the U.S. Food and Drug Administration (FDA) approved ZELSUVMI™ (berdazimer topical gel, 10.3%) as a first-in-class medication for the treatment of molluscum contagiosum in adults and pediatric patients one year of age or older. Ligand acquired ZELSUVMI through its acquisition of assets from Novan, along with other assets. ZELSUVMI is the first and only topical prescription medication that can be applied by patients, parents, or caregivers at home to treat this highly contagious viral skin infection. Merck (NYSE: MRK) announced the FDA accepted for priority review a new BLA for V116, its investigational 21-valent pneumococcal conjugate vaccine specifically designed to help prevent invasive pneumococcal disease and pneumonia in adults. The FDA grants priority review to medicines and vaccines that, if approved, would provide a significant improvement in the safety or effectiveness of the treatment or prevention of a serious condition. The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 17, 2024. If approved, Ligand is entitled to a low single digit royalty on worldwide net sales. Sermonix Pharmaceuticals (private) announced it entered into a strategic collaboration and exclusive license agreement with Henlius for the rights to develop, manufacture and commercialize Sermonix's lead investigational drug, lasofoxifene, in China. Under the terms of the agreement, Henlius will receive exclusive rights and sublicenses to lasofoxifene for at least two estrogen receptor-positive (ER+)/HER2- breast cancer indications in the territory, with Sermonix retaining all other global rights. Sermonix plans to work with Henlius to accelerate the clinical development of the Phase 3 ELAINE-3 multi-regional clinical trial in China, making lasofoxifene available to Chinese patients as soon as possible. Travere Therapeutics (Nasdaq: TVTX) announced a successful pre-NDA meeting for FILSPARI® in IgA Nephropathy (IgAN). After meeting with the FDA, Travere plans to submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. Accelerated Approval of FILSPARI to full approval. Travere also completed regulatory engagement on focal segmental glomerulosclerosis (FSGS) in which the FDA communicated that the Phase 3 DUPLEX study results alone are not sufficient to support an sNDA submission for an FSGS indication for FILSPARI. As a result, Travere plans to conduct additional analyses of FSGS data with plans to re-engage the FDA in 2024 and is implementing a strategic reorganization to focus near-term resources on the ongoing FILSPARI launch in IgAN. Travere also announced fourth quarter 2023 results reporting that it received 459 new patient start forms for FILSPARI in the fourth quarter of 2023 and net product sales of $14.9 million for the fourth quarter. Eisai Co., Ltd. has obtained marketing authorization approval from the Japanese Ministry of Health, Labour and Welfare for the Captisol-enabled™ injection formulation of its antiepileptic drug (AED) Fycompa® (perampanel) in Japan as an alternative therapy when oral administration is temporarily not possible. Fycompa is a first-in-class AED discovered and developed by Eisai. Aldeyra Therapeutics (Nasdaq: ALDX) announced receipt of a Complete Response Letter (CRL) from the FDA for the NDA of reproxalap, an investigational drug candidate, for the treatment of dry eye disease. The potential NDA resubmission is anticipated in the first half of 2024, pending FDA special protocol assessment (SPA) feedback and positive results from the proposed trial. Aldeyra intends to include in the potential NDA resubmission a draft label describing chronic and acute symptomatic benefit, in addition to acute reduction in ocular redness of reproxalap. The review period for the potential NDA resubmission is expected to be six months. Palvella Therapeutics (private) announced that the FDA has granted Breakthrough Therapy Designation to QTORIN rapamycin™ for the treatment of microcystic lymphatic malformations (Microcystic LMs). Microcystic LMs is a chronically debilitating and lifelong genetic disease affecting an estimated more than 30,000 patients in the U.S. There are currently no FDA-approved treatments for Microcystic LMs. In November, Viking Therapeutics, Inc. (Nasdaq: VKTX) announced the presentation of new results from the ongoing Phase 2b clinical trial of VK2809, a novel liver-selective thyroid hormone receptor beta agonist, in patients with biopsy-confirmed non-alcoholic steatohepatitis (NASH). The latest findings from the VOYAGE study were featured in a late breaking poster presentation at the Liver Meeting® 2023, the annual meeting of the American Association for the Study of Liver Diseases (AASLD). The newly reported findings demonstrated robust and comparable liver fat reductions among patients with or without Type 2 diabetes, as well as patients with either F2 or F3 fibrosis. Verona Pharma (Nasdaq: VRNA) announced they have entered into a debt financing facility providing the company with access to up to $400 million from funds managed by Oxford Finance LLC and Hercules Capital, Inc. The debt facility provides non-dilutive capital and further financial flexibility to support Verona Pharma’s continued growth, including the planned commercial launch of ensifentrine. The debt facility replaces the existing facility of up to $150 million with an affiliate of Oxford. Adjusted Financial Measures Ligand reports adjusted net income and adjusted net income per diluted share in addition to, and not as a substitute for, or superior to, financial measures calculated in accordance with GAAP. The Company’s financial measures under GAAP include share-based compensation expense, amortization of debt-related costs, amortization related to acquisitions and intangible assets, changes in contingent liabilities, mark-to-market adjustments for amounts relating to its equity investments in public companies, excess tax benefit from share-based compensation, transaction costs, income tax affect of adjusted reconciling items and others that are listed in the itemized reconciliations between GAAP and adjusted financial measures included at the end of this press release. However, the Company does not provide reconciliations of such forward-looking adjusted measures to GAAP due to the inherent difficulty in forecasting and quantifying certain amounts that are necessary for such reconciliation, including adjustments that could be made for changes in contingent liabilities, changes in the market value of its investments in public companies, share-based compensation expense and the effects of any discrete income tax items. Management has excluded the effects of these items in its adjusted measures to assist investors in analyzing and assessing the Company’s past and future core operating performance. Additionally, adjusted earnings per diluted share is a key component of the financial metrics utilized by the Company’s board of directors to measure, in part, management’s performance and determine significant elements of management’s compensation. Conference Call Ligand management will host a conference call and webcast today beginning at 8:30 a.m. Eastern time (5:30 a.m. Pacific time) to discuss this announcement and answer questions. To participate via telephone, please dial (800) 715-9871 (North America toll-free number) using the conference ID 875533. International participants outside of Canada may use the toll number (647) 932-3411 and use the same conference ID. To participate via live or replay webcast, a link is available at . About Ligand Pharmaceuticals Ligand is a biopharmaceutical company enabling scientific advancement through supporting the clinical development of high-value medicines. Ligand does this by providing financing, licensing our technologies or both. Our business model seeks to generate value for stockholders by creating a diversified portfolio of biotech and pharmaceutical product revenue streams that are supported by an efficient and low corporate cost structure. Our goal is to offer investors an opportunity to participate in the promise of the biotech industry in a profitable and diversified manner. Our business model is based on funding programs in mid- to late-stage drug development in return for economic rights and licensing our technology to help partners discover and develop medicines. We partner with other pharmaceutical companies to attempt to leverage what they do best (late-stage development, regulatory management and commercialization) in order to generate our revenue. Our Captisol® platform technology is a chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. We have established multiple alliances, licenses and other business relationships with the world’s leading pharmaceutical companies including Amgen, Merck, Pfizer, Jazz, Takeda, Gilead Sciences and Baxter International. For more information, please visit . Follow Ligand on X (f/k/a Twitter) @Ligand_LGND. We use our investor relations website and X as a means of disclosing material non-public information and for complying with our disclosure obligations under Regulation FD. Investors should monitor our website and our X account, in addition to following our press releases, SEC filings, public conference calls and webcasts. Forward-Looking Statements This news release contains forward-looking statements by Ligand that involve risks and uncertainties and reflect Ligand's judgment as of the date of this release. Words such as “plans,” “believes,” “expects,” “anticipates,” and “will,” and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding: Ligand’s ability to expand its portfolio with life sciences royalty opportunities; the timing of clinical and regulatory events of Ligand’s partners; the timing of the initiation or completion of preclinical studies and clinical trials by Ligand and its partners; the timing of product launches by Ligand or its partners; and guidance regarding the full-year 2024 financial results. Actual events or results may differ from Ligand's expectations due to risks and uncertainties inherent in Ligand’s business, including, without limitation: Ligand relies on collaborative partners for milestone payments, royalties, materials revenue, contract payments and other revenue projections and may not receive expected revenue; Ligand may not receive expected revenue from Captisol material sales; Ligand and its partners may not be able to timely or successfully advance any product(s) in its internal or partnered pipeline or receive regulatory approval and there may not be a market for the product(s) even if successfully developed and approved; Ligand may not achieve its guidance for 2024; Ligand faces competition in acquiring royalties and locating suitable royalties to acquire; Ligand may not be able to create future revenues and cash flows through the acquisition of royalties or by developing innovative therapeutics; products under development by Ligand or its partners may not receive regulatory approval; the total addressable market for our partners’ products may be smaller than estimated; Ligand faces competition with respect to its technology platforms which may demonstrate greater market acceptance or superiority; Ligand is currently dependent on a single source sole supplier for Captisol and failures by such supplier may result in delays or inability to meet the Captisol demands of its partners; Ligand’s partners may change their development focus and may not execute on their sales and marketing plans for marketed products for which Ligand has an economic interest; Ligand’s and its partners’ products may not be proved to be safe and efficacious and may not perform as expected and uncertainty regarding the commercial performance of such products; Ligand or its partners may not be able to protect their intellectual property and patents covering certain products and technologies may be challenged or invalidated; Ligand's partners may terminate any of its agreements or development or commercialization of any of its products; Ligand and its partners may experience delays in the commencement, enrollment, completion or analysis of clinical testing for its product candidates, or significant issues regarding the adequacy of its clinical trial designs or the execution of its clinical trials, challenges, costs and charges associated with integrating acquisitions with Ligand’s existing businesses; Ligand may not be able to successfully commercialize Novan's berdazimer program and may not be able to outlicense or sell Novan's programs or assets; Ligand may not be able to successfully implement its strategic growth plan and continue the development of its proprietary programs; restrictions under Ligand's credit agreement may limit its flexibility in operating its business and a default under the agreement could result in a foreclosure of the collateral securing such obligations; changes in general economic conditions, including as a result of war, conflict or epidemic diseases and ongoing or future litigation could expose Ligand to significant liabilities and have a material adverse effect on the company. The failure to meet expectations with respect to any of the foregoing matters may reduce Ligand's stock price. Additional information concerning these and other risk factors affecting Ligand can be found in prior press releases available at as well as in Ligand's public periodic filings with the Securities and Exchange Commission available at . Ligand disclaims any intent or obligation to update these forward-looking statements beyond the date of this release, including the possibility of additional license fees and milestone revenues we may receive. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Other Disclaimers and Trademarks The information in this press release regarding certain third-party products and programs, including V116, a Merck product candidate, FILSPARI, a Travere Therapeutics product, Fycompa, an Eisai Co. Ltd., product, reproxalap, an Aldeyra Therapeutics product candidate, and QTORIN, a Palvella Therapeutics product candidate, comes from information publicly released by the owners of such products and programs. Ligand is not responsible for, and has no role in, the development of such products or programs. Ligand owns or has rights to trademarks and copyrights that it uses in connection with the operation of its business including its corporate name, logos and websites. Other trademarks and copyrights appearing in this press release are the property of their respective owners. The trademarks Ligand owns include Ligand®, Captisol® , and ZELSUVMI, a Novan product. Solely for convenience, some of the trademarks and copyrights referred to in this press release are listed without the ®, © and ™ symbols, but Ligand will assert, to the fullest extent under applicable law, its rights to its trademarks and copyrights. LIGAND PHARMACEUTICALS INCORPORATED CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited, in thousands, except per share amounts) Three Months Ended December 31, Year Ended December 31, 2023 2022 2023 2022 Revenues: Royalties $ 22,463 $ 22,019 $ 83,910 $ 72,527 Captisol - Core 3,922 3,347 28,372 16,429 Captisol - COVID — 23,533 — 88,066 Contract revenue 1,716 1,483 19,032 19,223 Total revenues 28,101 50,382 131,314 196,245 Operating costs and expenses: Cost of Captisol 1,641 21,614 10,512 52,827 Amortization of intangibles 8,338 8,539 33,654 34,237 Research and development 5,488 9,197 24,537 36,082 General and administrative 15,992 31,131 52,790 70,062 Total operating costs and expenses 31,459 70,481 121,493 193,208 Gain on sale of Pelican — — (2,121 ) — (Loss) income from operations (3,358 ) (20,099 ) 11,942 3,037 Gain from short-term investments 16,025 44,248 46,365 28,540 Interest income, net 1,562 783 7,055 247 Gain on derivative instruments 250 — 250 — Other income (expense), net 2,618 (792 ) (1,952 ) 4,187 Total other income, net 20,455 44,239 51,718 32,974 Income before income taxes 17,097 24,140 63,660 36,011 Income tax benefit (expense) 1,091 (38,674 ) (9,841 ) (41,230 ) Net income (loss) from continuing operations 18,188 (14,534 ) 53,819 (5,219 ) Net loss from discontinued operations — (2,951 ) (1,665 ) (28,142 ) Net income (loss): $ 18,188 $ (17,485 ) $ 52,154 $ (33,361 ) Basic net income (loss) from continuing operations per share $ 1.04 $ (0.86 ) $ 3.11 $ (0.31 ) Basic net loss from discontinued operations per share $ — $ (0.17 ) $ (0.10 ) $ (1.67 ) Basic net income (loss) per share $ 1.04 $ (1.04 ) $ 3.02 $ (1.98 ) Shares used in basic per share calculation 17,466 16,890 17,298 16,868 Diluted net income (loss) from continuing operations per share $ 1.03 $ (0.86 ) $ 3.03 $ (0.31 ) Diluted net loss from discontinued operations per share $ — $ (0.17 ) $ (0.09 ) $ (1.67 ) Diluted net income (loss) per share $ 1.03 $ (1.04 ) $ 2.94 $ (1.98 ) Shares used in diluted per share calculation 17,676 16,890 17,757 16,868 LIGAND PHARMACEUTICALS INCORPORATED CONDENSED CONSOLIDATED BALANCE SHEETS (unaudited, in thousands) December 31, 2023 December 31, 2022 Assets Current assets: Cash and cash equivalents $ 22,954 $ 45,006 Short-term investments 147,355 166,864 Accounts receivable, net 32,917 30,424 Inventory 23,969 13,294 Income tax receivable 6,395 4,614 Prepaid expenses 1,182 1,132 Other current assets 2,657 2,267 Total current assets 237,429 263,601 Deferred income taxes, net 46,062 8,530 Goodwill and other identifiable intangible assets, net 402,976 448,128 Commercial license and other economic rights, net 67,291 10,182 Operating lease right-of-use assets 6,062 10,914 Finance lease 3,393 4,095 Equity method investment in Primrose Bio 12,595 — Other investments 35,726 3,000 Other assets 21,530 14,218 Total assets $ 833,064 $ 762,668 Liabilities and Stockholders' Equity Current liabilities: Accounts payable and accrued liabilities $ 14,894 $ 20,988 Current contingent liabilities 256 57 Current operating lease liabilities 403 670 Current finance lease liabilities 7 45 Deferred revenue 1,222 355 2023 convertible senior notes, net — 76,695 Total current liabilities 16,782 98,810 Long-term contingent liabilities 2,942 3,456 Long-term operating lease liabilities 5,755 10,336 Long-term deferred revenue 1,444 44 Deferred income taxes, net 77,470 30,615 Other long-term liabilities 27,758 21,922 Total liabilities 132,151 165,183 Total stockholders' equity 700,913 597,485 Total liabilities and stockholders' equity $ 833,064 $ 762,668 LIGAND PHARMACEUTICALS INCORPORATED ADJUSTED FINANCIAL MEASURES (Unaudited, in thousands, except per share amounts) Three months ended December 31, Year ended December 31, 2023 2022 2023 2022 Net income (loss) from continuing operations $ 18,188 $ (14,534 ) $ 53,819 $ (5,219 ) Adjustments: Share-based compensation expense 5,721 27,664 25,743 50,881 Finance lease impairment charge and other — 10,821 — 10,821 Non-cash interest expense (1) 81 95 240 734 Amortization related to acquisitions and intangible assets 8,338 8,539 33,654 34,237 Amortization of commercial license and other economic rights (2) (23 ) (32 ) (1,049 ) (355 ) Change in contingent liabilities (3) (397 ) 698 (265 ) (144 ) Novan operating loss 5,183 — 5,520 — Transaction costs (210 ) — 3,078 — Gain from short-term investments (16,025 ) (44,248 ) (46,365 ) (28,540 ) Realized gain (loss) from short-term investments 7,180 — 44,377 (288 ) Gain on sale of Pelican — — (2,121 ) — Credit losses and impairment charges for commercial license rights 405 — 4,519 — Loss from equity method investment in Primrose Bio 1,761 1,829 Other (4) (156 ) 1,904 530 (34 ) Income tax effect of adjusted reconciling items above 816 8,093 (9,144 ) (4,561 ) Tax benefit related to decrease in unrecognized tax benefits (5) (7,206 ) — (7,206 ) — Tax expense related to increase in valuation allowance (6) — 24,799 — 24,799 Excess tax benefit (shortfall) from share-based compensation (7) 757 (267 ) 228 (138 ) Adjusted net income from continuing operations $ 24,413 $ 23,532 $ 107,387 $ 82,193 Realized gains from sales of VKTX stock, net of tax (5,780 ) — (35,720 ) — Captisol - COVID gross profit, net of tax (8) — (10,514 ) — (40,268 ) Core adjusted net income from continuing operations $ 18,633 $ 13,018 $ 71,667 $ 41,925 Diluted per-share amounts attributable to common shareholders: Diluted net income (loss) per share from continuing operations $ 1.03 $ (0.86 ) $ 3.03 $ (0.31 ) Adjustments: Share-based compensation expense 0.32 1.60 1.46 2.96 Finance lease impairment charge and other — 0.63 — 0.63 Non-cash interest expense (1) — 0.01 0.01 0.04 Amortization related to acquisitions and intangible assets 0.47 0.49 1.91 1.99 Amortization of commercial license and other economic rights (2) — — (0.06 ) (0.02 ) Change in contingent liabilities (3) (0.02 ) 0.04 (0.02 ) (0.01 ) Novan operating loss 0.29 0.31 Transaction costs (0.01 ) — 0.17 — Loss (gain) from short-term investments (0.91 ) (2.56 ) (2.63 ) (1.66 ) Realized gain from short-term investments 0.41 — 2.52 (0.02 ) Gain on sale of Pelican — — (0.12 ) — Credit losses and impairment charges for commercial license rights 0.02 — 0.26 — Loss from equity method investment in Primrose Bio 0.10 — 0.10 — Other (4) — 0.10 0.04 0.01 Income tax effect of adjusted reconciling items above 0.05 0.48 (0.49 ) (0.27 ) Tax benefit related to decrease in unrecognized tax benefits (5) (0.41 ) — (0.41 ) — Tax expense related to increase in valuation allowance (6) — 1.44 — 1.44 Excess tax benefit (shortfall) from share-based compensation (7) 0.04 (0.02 ) 0.01 (0.01 ) Adjustment for shares excluded due to anti-dilution effect on GAAP net loss — 0.01 — 0.02 Adjusted diluted net income per share from continuing operations $ 1.38 $ 1.36 $ 6.09 $ 4.79 Realized gains from sales of VKTX stock, net of tax (0.33 ) — (2.03 ) — Captisol - COVID gross profit, net of tax (8) — (0.61 ) — (2.35 ) Core adjusted diluted net income per share from continuing operations $ 1.05 $ 0.75 $ 4.06 $ 2.44 GAAP - weighted average number of common shares - diluted 17,676 16,890 17,757 16,868 Shares excluded due to anti-dilutive effect on GAAP net loss — 390 — 298 Diluted effect of the 2023 Notes (9) — — (119 ) — Adjusted weighted average number of common shares - diluted 17,676 17,280 17,638 17,166 (1) Amounts represent non-cash debt related costs that are calculated in accordance with the authoritative accounting guidance for convertible debt instruments that may be settled in cash and for revolving credit facility. (2) Amounts represent the amortization of commercial license and other economic rights to revenue. (3) Amounts represent changes in fair value of contingent consideration related to CyDex and Metabasis transactions. (4) Amounts primarily relate to restructuring costs, losses associated with our equity investment in Nucorion, gain or loss from change in fair value or derivative assets as well as gain on debt extinguishment in the prior year period. (5) Amounts represent discrete tax benefit related to the release of FIN48 reserves associated with certain R&D tax credits during the fourth quarter of 2023 due to the lapse of applicable statute of limitation. (6) Amounts represent discrete tax expense related to the valuation allowance established during the fourth quarter of 2022 against deferred tax asset for California research and development credits and net operating losses. (7) Excess tax benefits from share-based compensation are recorded as a discrete item within the provision for income taxes on the consolidated statements of operations as a result of the adoption of an accounting pronouncement (ASU 2016-09) on January 1, 2017. Prior to the adoption, the amount was recognized in additional paid-in capital on the consolidated statement of stockholders' equity. (8) Captisol - COVID gross profit, net of tax, represents gross profit, net of tax, for Captisol supplied for use in formulation with remdesivir, an antiviral treatment for COVID-19. (9) Excluding the impact from the adoption of accounting pronouncement (ASU 2020-06) on January 1, 2022, as the Company has intended to settle the principal balance in cash. Under the standard, the Company is required to reflect the dilutive effect of the 2023 Notes by application of the if-converted method, The 2023 Notes were fully paid off on May 15, 2023, the debt maturity date.

Received 459 new patient start forms (PSFs) for FILSPARI® (sparsentan) in Q4 2023; Total of 1,452 PSFs received in 2023 Company nearing submission of sNDA to convert U.S. accelerated approval of FILSPARI in IgAN to full approval CHMP opinion on potential approval in Europe for sparsentan in IgAN anticipated in Q1 2024 Pivotal Phase 3 HARMONY Study of pegtibatinase in classical homocystinuria (HCU) enrolling; topline data anticipated in 2026 Net product sales of $39.9 million for Q4 2023; $127.5 million for FY 2023 Cash, cash equivalents, and marketable securities as of December 31, 2023, totaled $566.9 million SAN DIEGO, Feb. 15, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc. (NASDAQ: TVTX) today reported its fourth quarter and full year 2023 financial results and provided a corporate update. “With ongoing growth in demand for FILSPARI in IgAN and the initiation of the pivotal Phase 3 program of pegtibatinase in HCU, we have made strong progress towards our goal of breaking down barriers in rare kidney and metabolic diseases where there has historically been little innovation,” said Eric Dube, Ph.D., president and chief executive officer of Travere Therapeutics. "As we enter 2024, our top priority remains furthering the strength of the U.S. commercial launch of FILSPARI in IgAN. With the potential full approval of FILSPARI, anticipated inclusion into the global treatment guidelines for IgAN as well as additional data planned in 2024, we are well-positioned to build upon our current momentum. Additionally, with a potential conditional marketing approval of FILSPARI for IgAN in the EU and development progress in Japan, we look to make sound progress with our foreign partners towards the shared goal of reaching patients outside of the U.S. Beyond FILSPARI, we look forward to advancing the innovative Phase 3 HARMONY Study of pegtibatinase, which is designed to ultimately deliver the first disease modifying therapy for people living with HCU.” Financial Results for Continuing Operations for the Quarter Ended December 31, 2023 The following financial results discussion compares Travere’s continuing operations. All periods unless otherwise specified have been adjusted to exclude discontinued operations related to the divestiture of the bile acid product portfolio completed on August 31, 2023. Net product sales for the fourth quarter of 2023 were $39.9 million, compared to $25.8 million for the same period in 2022. For the full year 2023, net product sales were $127.5 million, compared to $98.0 million for the same period in 2022. The increase is primarily attributable to sales from the ongoing commercial launch of FILSPARI. Research and development (R&D) expenses for the fourth quarter of 2023 were $59.7 million, compared to $58.1 million for the same period in 2022. For the full year 2023, R&D expenses were $245.0 million, compared to $227.3 million for the same period in 2022. The difference is largely attributable to the continued advancement of the Company’s pegtibatinase clinical program, including clinical trial expenses and manufacturing, as well as increased headcount. On a non-GAAP adjusted basis, R&D expenses were $55.3 million for the fourth quarter of 2023, compared to $52.0 million for the same period in 2022. Selling, general, and administrative (SG&A) expenses for the fourth quarter of 2023 were $63.6 million, compared to $57.1 million for the same period in 2022. For the full year 2023, SG&A expenses were $265.5 million, compared to $197.5 million for the same period in 2022. The difference is largely attributable to commercial launch related activities following the accelerated approval of FILSPARI in February 2023, as well as legal fees. On a non-GAAP adjusted basis, SG&A expenses were $49.7 million for the fourth quarter of 2023, compared to $44.3 million for the same period in 2022. Total other income, net, for the fourth quarter of 2023 was $5.7 million, compared to $1.1 million for the same period in 2022. The difference is largely attributable to an increase in interest income during the period. Net loss including discontinued operations for the fourth quarter of 2023 was $90.2 million, or $1.18 per basic share, compared to a net loss of $65.8 million, or $1.03 per basic share for the same period in 2022. For the full year 2023, net loss including discontinued operations was $111.4 million, compared to $278.5 million for the same period in 2022. On a non-GAAP adjusted basis, net income including discontinued operations for the fourth quarter of 2023 was $71.8 million, or $0.94 per basic share, compared to a net loss of $46.9 million, or $0.73 per basic share for the same period in 2022. As of December 31, 2023, the Company had cash, cash equivalents, and marketable securities of $566.9 million. Program Updates and Anticipated Milestones FILSPARI® (sparsentan) – IgAN On February 17, 2023, the U.S. Food and Drug Administration (FDA) granted accelerated approval to FILSPARI to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a urine protein-to-creatinine ratio (UPCR) ≥1.5 g/g. FILSPARI became commercially available the week of February 27, 2023. Commercial progress in the ongoing launch has resulted in: 459 new patient start forms (PSFs) received in the fourth quarter of 2023; as of December 31, 2023 a total of 1,452 PSFs had been received since approval.Net product sales of $14.7 million during the fourth quarter, bringing the total to $29.2 million in net product sales since the beginning of the launch. The Company expects to submit a supplemental New Drug Application (sNDA) in the first quarter of 2024 for conversion of the existing U.S. accelerated approval of FILSPARI to full approval.The Company and its collaborator CSL Vifor anticipate a review opinion by the Committee for Medicinal Products for Human Use (CHMP) on the potential approval of the Conditional Marketing Authorization (CMA) application for sparsentan for the treatment of IgAN in Europe in the first quarter of 2024. If approved, sparsentan would receive CMA in all member states of the European Union, as well as in Iceland, Liechtenstein, and Norway.In January 2024, the Company announced that it had entered into an exclusive licensing agreement with Renalys Pharma, Inc., to bring sparsentan to patients in Japan and other countries in Asia. Following successful meetings with the Pharmaceuticals and Medical Devices Agency (PMDA) in 2023, Renalys plans to initiate an open label registrational study of sparsentan in Japan in the second quarter of 2024 to support potential approval of sparsentan in Japan. Results from the urine protein/creatinine ratio (UP/C) endpoint in the study are expected in the second half of 2025. In 2024, the Company expects additional data from its ongoing open-label studies evaluating the safety and efficacy of sparsentan in combination with sodium glucose cotransporter-2 inhibitors (SGLT2i) as well as from the ongoing SPARTAN Study evaluating the potential effect of FILSPARI as a first-line therapy in patients with newly diagnosed IgAN.In 2024, the Company anticipates inclusion of FILSPARI into the Kidney Disease Improving Global Outcomes (KDIGO) Clinical Practice Guideline for the Management of Glomerular Diseases. Sparsentan – Focal Segmental Glomerulosclerosis (FSGS) In 2024, the Company is conducting additional analyses of FSGS data and will engage with regulators to evaluate potential regulatory pathways for a sparsentan FSGS indication. Pegtibatinase – Classical HCU In December 2023, the Company initiated the pivotal Phase 3 HARMONY Study to support the potential approval of pegtibatinase for the treatment of HCU. The HARMONY Study is a global, randomized, multi-center, double-blind, placebo-controlled Phase 3 clinical trial designed to evaluate the efficacy and safety of pegtibatinase as a novel treatment to reduce total homocysteine (tHcy) levels. The trial is expected to enroll approximately 70 patients with a diagnosis of classical HCU and tHcy levels ≥50 μM while maintaining their standard-of-care treatment. The primary endpoint is relative geometric mean change in plasma tHcy levels from baseline compared to weeks 6 through 12. Durability of treatment response through 24 weeks of treatment will also be measured as a secondary endpoint. Topline results from the HARMONY Study are expected in 2026.The Company will also be initiating the ENSEMBLE Study, a Phase 3b, open-label, long-term extension, that will evaluate the ongoing efficacy and long-term safety of pegtibatinase in participants with HCU following their completion of the Phase 1/2 COMPOSE Study or the HARMONY Study. ENSEMBLE will include an optional protein tolerance modification sub-study that will evaluate if patients can increase their natural dietary protein intake and maintain an acceptable level of metabolic control while receiving pegtibatinase. Conference Call Information Travere Therapeutics will host a conference call and webcast today, Thursday, February 15, 2024, at 4:30 p.m. ET to discuss company updates as well as fourth quarter and full year 2023 financial results. To participate in the conference call, dial +1 (888) 256-1007 (U.S.) or +1 (323) 701-0225 (International), confirmation code 7983862 shortly before 4:30 p.m. ET. The webcast can be accessed on the Investor page of Travere’s website at ir.travere.com/events-presentations. Following the live webcast, an archived version of the call will be available for 30 days on the Company’s website. Use of Non-GAAP Financial Measures To supplement Travere’s financial results and guidance presented in accordance with U.S. generally accepted accounting principles (GAAP), the Company uses certain non-GAAP adjusted financial measures in this press release and the accompanying tables. The Company believes that these non-GAAP financial measures are helpful in understanding its past financial performance and potential future results. They are not meant to be considered in isolation or as a substitute for comparable GAAP measures and should be read in conjunction with the consolidated financial statements prepared in accordance with GAAP. Travere’s management regularly uses these supplemental non-GAAP financial measures internally to understand, manage and evaluate its business and make operating decisions. In addition, Travere believes that the use of these non-GAAP measures enhances the ability of investors to compare its results from period to period and allows for greater transparency with respect to key financial metrics the Company uses in making operating decisions. Investors should note that these non-GAAP financial measures are not prepared under any comprehensive set of accounting rules or principles and do not reflect all of the amounts associated with the Company’s results of operations as determined in accordance with GAAP. Investors should also note that these non-GAAP financial measures have no standardized meaning prescribed by GAAP and, therefore, have limits in their usefulness to investors. In addition, from time to time in the future the Company may exclude other items, or cease to exclude items that it has historically excluded, for purposes of its non-GAAP financial measures; because of the non-standardized definitions, the non-GAAP financial measures as used by the Company in this press release and the accompanying tables may be calculated differently from, and therefore may not be directly comparable to, similarly titled measures used by the Company’s competitors and other companies. As used in this press release, (i) the historical non-GAAP net loss measures exclude from GAAP net loss, as applicable, stock-based compensation expense, amortization and depreciation expense, and income tax; (ii) the historical non-GAAP SG&A expense measures exclude from GAAP SG&A expenses, as applicable, stock-based compensation expense, and amortization and depreciation expense; (iii) the historical non-GAAP R&D expense measures exclude from GAAP R&D expenses, as applicable, stock-based compensation expense, and amortization and depreciation expense. About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families, and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop, and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com About FILSPARI (sparsentan) FILSPARI (sparsentan) is a once-daily, oral medication designed to selectively target two critical pathways in the disease progression of IgAN (endothelin-1 and angiotensin II) and is the first and only non-immunosuppressive therapy approved for the treatment of this condition. FILSPARI is a prescription medicine indicated to reduce proteinuria in adults with primary IgAN at risk of rapid disease progression, generally a UPCR ≥1.5 g/g. FILSPARI (sparsentan) U.S. Indication FILSPARI is an endothelin and angiotensin II receptor antagonist indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid disease progression, generally a UPCR ≥1.5 g/g. This indication is granted under accelerated approval based on reduction in proteinuria. It has not been established whether FILSPARI slows kidney function decline in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial. FILSPARI (sparsentan) Important Safety Information BOXED WARNING: HEPATOTOXICITY AND EMBRYO-FETAL TOXICITYBecause of the risks of hepatotoxicity and birth defects, FILSPARI is available only through a restricted program called the FILSPARI REMS. Under the FILSPARI REMS, prescribers, patients and pharmacies must enroll in the program. HepatotoxicitySome Endothelin Receptor Antagonists (ERAs) have caused elevations of aminotransferases, hepatotoxicity, and liver failure. In clinical studies, elevations in aminotransferases (ALT or AST) of at least 3-times the Upper Limit of Normal (ULN) have been observed in up to 2.5% of FILSPARI-treated patients, including cases confirmed with rechallenge. Measure transaminases and bilirubin before initiating treatment and monthly for the first 12 months, and then every 3 months during treatment. Interrupt treatment and closely monitor patients who develop aminotransferase elevations more than 3x Upper Limit of Normal (ULN). FILSPARI should generally be avoided in patients with elevated aminotransferases (>3x ULN) at baseline because monitoring for hepatotoxicity may be more difficult and these patients may be at increased risk for serious hepatotoxicity. Embryo-Fetal Toxicity FILSPARI can cause major birth defects if used by pregnant patients based on animal data. Therefore, pregnancy testing is required before the initiation of treatment, during treatment and one month after discontinuation of treatment with FILSPARI. Patients who can become pregnant must use effective contraception before the initiation of treatment, during treatment, and for one month after discontinuation of treatment with FILSPARI. Contraindications: FILSPARI is contraindicated in patients who are pregnant. Do not coadminister FILSPARI with angiotensin receptor blockers (ARBs), endothelin receptor antagonists (ERAs), or aliskiren. Warnings and Precautions Hepatotoxicity:Hepatotoxicity: Elevations in ALT or AST of at least 3-fold ULN have been observed. To reduce the risk of potential serious hepatotoxicity, measure serum aminotransferase levels and total bilirubin prior to initiation of treatment, monthly for the first 12 months, then every 3 months during treatment.Advise patients with symptoms suggesting hepatotoxicity (nausea, vomiting, right upper quadrant pain, fatigue, anorexia, jaundice, dark urine, fever, or itching) to immediately stop treatment with FILSPARI and seek medical attention. If aminotransferase levels are abnormal at any time during treatment, interrupt FILSPARI and monitor as recommended.Consider re-initiation of FILSPARI only when hepatic enzyme levels and bilirubin return to pretreatment values and only in patients who have not experienced clinical symptoms of hepatotoxicity.Avoid initiation of FILSPARI in patients with elevated aminotransferases (>3x ULN) prior to drug initiation. Embryo-Fetal Toxicity: FILSPARI can cause fetal harm. Advise patients who can become pregnant of the potential risk to a fetus. Obtain a pregnancy test and advise patients who can become pregnant to use effective contraception prior to, during, and one month after discontinuation of FILSPARI treatment.FILSPARI REMS: FILSPARI is available only through a restricted program under a REMS called the FILSPARI REMS.Important requirements include: Prescribers must be certified with the FILSPARI REMS by enrolling and completing training.All patients must enroll in the FILSPARI REMS prior to initiating treatment and comply with monitoring requirements.Pharmacies that dispense FILSPARI must be certified with the FILSPARI REMS and must dispense only to patients who are authorized to receive FILSPARI. Further information is available at www.filsparirems.com or 1-833-513-1325. Please see Full Prescribing Information for FILSPARI here Forward-Looking Statements This press release contains "forward-looking statements" as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words “on-track”, “positioned”, “look forward to”, “will,” “would,” "may", "might", "believes", "anticipates", "plans", "expects", "intends," “potential” or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, references to: continued progress with the FILSPARI launch; the planned submission of an sNDA for full approval of FILSPARI and the anticipated timing and outcome thereof; statements regarding the potential approval of sparsentan for the treatment of IgAN in the EU and the anticipated timing thereof; the potential for pegtibatinase to become the first disease modifying therapy for people living with HCU; the timing and achievement of additional development and regulatory milestones, including expected data from additional studies; planned additional analyses of FSGS data and plans and timing for re-engaging with regulators; the advancement of the Company’s pipeline throughout the year; expectations regarding the Phase 3 HARMONY Study and the other studies described herein; the potential inclusion of FILSPARI in the KDIGO Clinical Practice Guideline for the Management of Glomerular Diseases; and statements regarding financial metrics and expectations related thereto. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks and uncertainties associated with the regulatory review and approval process, as well as risks and uncertainties associated with the Company’s business and finances in general and its recently announced strategic reorganization, the success of its commercial products and risks and uncertainties associated with the Company's preclinical and clinical stage pipeline. Specifically, the Company faces risks associated with market acceptance of its commercial products including efficacy, safety, price, reimbursement, and benefit over competing therapies, as well as risks associated with the successful development and execution of commercial strategies for such products, including FILSPARI. The risks and uncertainties the Company faces with respect to its preclinical and clinical stage pipeline include risk that the Company's clinical candidates will not be found to be safe or effective and that current or anticipated future clinical trials will not proceed as planned. Specifically, the Company faces risks related to the timing and potential outcome of its Phase 3 HARMONY Study and the other studies described herein, the timing and potential outcome of its planned sNDA submission for full approval of sparsentan in IgAN, and the risk that the results from the Phase 3 DUPLEX Study of sparsentan in FSGS will not serve as a basis for a regulatory submission for approval of sparsentan for FSGS. There is no guarantee that regulators will grant full approval of sparsentan for IgAN or FSGS. The Company also faces the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company’s products, and technological changes that may limit demand for the Company's products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading “Risk Factors”, as included in the Company's most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission. TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIESCONSOLIDATED BALANCE SHEETS(in thousands, except share amounts) December 31, 2023 December 31, 2022Assets Current assets: Cash and cash equivalents$ 58,176 $ 61,688 Marketable debt securities, at fair value 508,675 388,557 Accounts receivable, net 21,179 16,646 Inventory, net 9,410 4,523 Prepaid expenses and other current assets 19,335 12,033 Current assets of discontinued operations — 2,990 Total current assets 616,775 486,437 Long-term inventory, net 31,494 — Property and equipment, net 7,479 9,049 Operating lease right of use assets 18,061 21,000 Intangible assets, net 104,443 97,073 Other assets 10,661 10,684 Non-current assets of discontinued operations — 48,342 Total assets$ 788,913 $ 672,585 Liabilities and Stockholders' Equity Current liabilities: Accounts payable$ 41,675 $ 17,290 Accrued expenses 118,991 95,742 Deferred revenue, current portion 7,096 11,976 Operating lease liabilities, current portion 4,909 4,433 Other current liabilities 5,237 5,722 Current liabilities of discontinued operations — 7,000 Total current liabilities 177,908 142,163 Convertible debt 377,263 375,545 Deferred revenue, less current portion 1,835 10,931 Operating lease liabilities, less current portion 22,612 27,510 Other non-current liabilities 8,485 9,385 Non-current liabilities of discontinued operations — 64,200 Total liabilities 588,103 629,734 Stockholders' Equity: Preferred stock $0.0001 par value; 20,000,000 shares authorized; no shares issued and outstanding as of December 31, 2023 and 2022 — — Common stock $0.0001 par value; 200,000,000 and 200,000,000 shares authorized; 75,367,117 and 64,290,570 issued and outstanding as of December 31, 2023 and 2022, respectively 7 6 Additional paid-in capital 1,327,881 1,059,975 Accumulated deficit (1,125,622) (1,014,223)Accumulated other comprehensive loss (1,456) (2,907)Total stockholders' equity 200,810 42,851 Total liabilities and stockholders' equity$ 788,913 $ 672,585 Note: Certain adjustments / reclassifications have been made to prior periods to conform to current year presentation. TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIESCONSOLIDATED STATEMENT OF OPERATIONS(in thousands, except share and per share data) Three Months Ended December 31, Twelve Months Ended December 31, 2023 2022 2023 2022 (unaudited) Net product sales: Tiopronin products$ 25,217 $ 25,816 $ 98,329 $ 97,970 FILSPARI 14,699 — 29,208 — Total net product sales 39,916 25,816 127,537 97,970 License and collaboration revenue 5,143 3,523 17,701 11,490 Total revenue 45,059 29,339 145,238 109,460 Operating expenses: Cost of goods sold 4,564 868 11,450 4,420 Research and development 59,746 58,087 244,990 227,333 Selling, general and administrative 63,588 57,086 265,542 197,520 Restructuring 11,394 — 11,394 — Total operating expenses 139,292 116,041 533,376 429,273 Operating loss (94,233) (86,702) (388,138) (319,813) Other income (expenses), net: Interest income 7,152 3,115 21,768 6,276 Interest expense (2,821) (2,858) (11,334) (11,014)Other income, net 1,374 872 1,594 974 Loss on extinguishment of debt — — — (7,578)Total other income (expense), net 5,705 1,129 12,028 (11,342) Loss from continuing operations before income tax provision (88,528) (85,573) (376,110) (331,155)Income tax provision on continuing operations (68) (63) (223) (313) Loss from continuing operations, net of tax (88,596) (85,636) (376,333) (331,468)(Loss) income from discontinued operations, net of tax (1,577) 19,813 264,934 52,986 Net loss$ (90,173) $ (65,823) $ (111,399) $ (278,482) Per share data Basic and diluted: Net loss per common share$ (1.18) $ (1.03) $ (1.50) $ (4.37)Weighted average common shares outstanding 76,474,560 64,214,167 74,267,418 63,758,515 Note: Certain adjustments / reclassifications have been made to prior periods to conform to current year presentation. TRAVERE THERAPEUTICS, INC. AND SUBSIDIARIESRECONCILIATION OF GAAP REPORTED TO NON-GAAP ADJUSTED INFORMATION(in thousands, except share and per share data)(unaudited) Three Months Ended December 31, Twelve Months Ended December 31, 2023 2022 2023 2022 GAAP operating loss$ (94,233) $ (86,702) $ (388,138) $ (319,813) R&D operating expense (59,746) (58,087) (244,990) (227,333) Stock compensation 3,426 3,613 17,284 13,858 Amortization & depreciation 997 2,447 7,261 6,264 Subtotal non-GAAP items 4,423 6,060 24,545 20,122 Non-GAAP R&D expense (55,323) (52,027) (220,445) (207,211) SG&A operating expense (63,588) (57,086) (265,542) (197,520) Stock compensation 3,070 5,915 28,389 25,319 Amortization & depreciation 10,855 6,855 37,671 26,816 Subtotal non-GAAP items 13,925 12,770 66,060 52,135 Non-GAAP SG&A expense (49,663) (44,316) (199,482) (145,385) Subtotal non-GAAP items 18,348 18,830 90,605 72,257 Non-GAAP operating loss$ (75,885) $ (67,872) $ (297,533) $ (247,556) GAAP net loss$ (90,173) $ (65,823) $ (111,399) $ (278,482)Non-GAAP operating loss adjustments 18,348 18,830 90,605 72,257 Income tax provision 68 63 223 313 Non-GAAP net loss$ (71,757) $ (46,930) $ (20,571) $ (205,912) Per share data Basic and diluted: Net loss per common share$ (0.94) $ (0.73) $ (0.28) $ (3.23)Weighted average common shares outstanding 76,474,560 64,214,167 74,267,418 63,758,515 (1) Non-GAAP net loss includes income from discontinued operations but excludes non-GAAP adjustments for the effect of discontinued operations. Note: Certain adjustments / reclassifications have been made to prior periods to conform to current year presentation. Contact: Investors:888-969-7879 ir@travere.comMedia:888-969-7879 mediarelations@travere.com