Nitisinone

Q1 2025 revenue of $17.3 million , with product sales of $14.0 million , representing 76% growth over Q1 2024 and the 17th straight quarter of sequential product sales growth Q1 2025 basic and fully diluted GAAP EPS of $(0.06) , Non-GAAP fully diluted EPS of $0.07 , and Adjusted EBITDA of $3.7 million Relaunched pediatric endocrinology biologic INCRELEX® in the U.S. and out-licensed international rights Established Wilson disease franchise with the acquisition and relaunch of GALZIN® (zinc acetate) capsules and the introduction of product candidate ET-700 Announced positive pivotal clinical study results for ET-600 and submitted its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) Management to hold conference call today at 4:30pm ET DEER PARK, Ill. , May 13, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or “the Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today reported financial results for the quarter ended March 31, 2025 . “The first quarter was another exceptional quarter for Eton. We reported record financial results, launched two recently acquired commercial products, and achieved critical milestones with the advancement of our development pipeline. In its first quarter of relaunch, INCRELEX is already tracking ahead of our expectations, with a significant number of new patients added to therapy in the first quarter, and we are confident that it has a long runway for growth in the years ahead. In March, we relaunched GALZIN with our full Eton Cares patient support program to a strong reception in the Wilson disease community. As part of our commitment to investing in Wilson disease, we also announced our ET-700 development program, which we believe has the potential to drastically improve the lives of Wilson disease patients.” said Sean Brynjelsen , CEO of Eton Pharmaceuticals . “We are now less than one month away from a potential launch of ET-400. We have been actively engaged with the FDA as part of the ongoing review, and we remain prepared to launch quickly upon potential approval on its Prescription Drug User Fee Act (PDUFA) date of May 28 th. With three expected product launches this year and several late-stage candidates progressing in development, 2025 is shaping up to be another monumental year for Eton as we further solidify our place as a leading ultra-rare disease company,” concluded Brynjelsen. First Quarter and Recent Business Highlights 17th straight quarter of sequential growth in product sales. Eton reported first quarter 2025 product sales of $14.0 million , representing 76% growth over the prior year period, driven primarily by the ongoing momentum of ALKINDI SPRINKLE® and the addition of INCRELEX, which was acquired in late December. The Company expects sequential quarter-over-quarter growth of product sales to continue throughout 2025 and beyond. Relaunched INCRELEX and increased number of active patients. INCRELEX is a biologic product used for the treatment of an ultra-rare pediatric endocrinology condition called Severe Primary IGF-1 Deficiency (SPIGFD) that is estimated to impact approximately 200 children in the United States . Eton closed the acquisition in late December and relaunched the product in the U.S. in January. Eton is making significant investments to raise awareness, increase education, and provide patient support for the product, and has already seen a significant number of new patients initiate treatment in 2025. Preparing for the anticipated second quarter launch of ET-400. With product inventory on hand, and a specialty sales force and promotional campaigns ready to go live, the Company is prepared to launch ET-400 shortly after its scheduled May 28th PDUFA goal date. If approved, Eton believes ET-400 will allow the Company to capture a greater percentage of the estimated $200 million oral hydrocortisone market opportunity. Relaunched GALZIN and announced internal development program ET-700 to further invest in advancing treatment options for Wilson disease. Eton added GALZIN to its metabolic portfolio in January and relaunched the product in March with its newly deployed metabolic sales force and its Eton Cares patient support program. Eton has increased access with Eton Cares , which provides $0 co-pays and free drug to patients not covered by insurance. The Company started the transition of GALZIN to Eton’s commercial distribution in March and expects GALZIN to start contributing material revenue during the third quarter of 2025. In addition, Eton announced a new internal development product candidate, ET- 700. ET -700 is an extended-release form of zinc acetate developed to improve adherence and tolerability. Eton expects to manufacture registration batches of ET-700 later this year and initiate a clinical study by early 2026. Out-licensed ex-US rights to INCRELEX. During the first quarter, Eton out-licensed INCRELEX commercial rights in territories outside of the U.S. to Esteve Pharmaceuticals, S.A. (“Esteve”). Esteve will license the ex- U.S. rights for up to ten years and Eton will supply product at a fixed transfer price. Under the terms of the transaction, Eton will receive a $4.3 million upfront licensing fee. $1.8 million of the upfront licensing fee was recognized as licensing revenue during the first quarter and the remainder will be recognized over the life of the license. Submitted NDA for ET-600. During the quarter, ET-600 passed its pivotal bioequivalence study, and the Company submitted an NDA for the product candidate in late April. ET-600 is a proprietary, patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. The Company expects to receive a 10-month FDA review, which could allow for potential approval and launch in the first quarter of 2026. First Quarter Financial Results Net Revenue: Net revenues for the first quarter of 2025 were $17.3 million compared with $8.0 million in the prior year period, an increase of 117%. Net revenues included $3.3 million of licensing revenue recognized during the quarter, including $1.8 million from the out-licensing of INCRELEX rights outside of the U.S. and $1.5 million from a regulatory milestone event associated with the Company’s previous divestiture of DS-200. There were no licensing revenues in the first quarter of 2024. Product sales and royalty revenue were $14.0 million during the first quarter of 2025 compared with $8.0 million in the prior year period, an increase of 76%. The growth was driven primarily by increased sales of ALKINDI SPRINKLE and the addition of INCRELEX, which was acquired in late December 2024 . The company continues to expect to exit 2025 at an approximately $80 million annual revenue run rate. Gross Profit: Gross profit for the first quarter of 2025 was $9.9 million compared with $5.0 million in the prior year period, an increase of 97%, primarily due to increased product sales. Adjusted gross profit, which adjusts for the impact of acquired inventory step-up adjustments and intangible amortization, was $12.0 million in the first quarter of 2025, representing an adjusted gross margin of 69.5%, compared to adjusted gross profit of $5.2 million and adjusted gross margin of 65.6% in the prior year period. The margin improvement was primarily driven by continued growth of higher margin ALKINDI SPRINKLE and the addition of higher margin licensing revenue. The Company continues to expect to report full year 2025 adjusted gross profit of approximately 70%. Research and Development (R&D) Expenses: R&D expenses for the first quarter of 2025 were $1.2 million compared to $0.7 million in the prior year period, primarily due to increased expenses associated with ET-700 and ET-800 project development activities. In April, the Company paid $2.2 million for the NDA application fee associated with ET-600, which will be recorded as an expense in the second quarter of 2025.General and Administrative (G&A) Expenses: G&A expenses for the first quarter of 2025 were $9.2 million compared to $5.2 million in the prior year period, primarily attributable to an increase in product advertising and promotional expenses, an increase in professional services and transaction-related expenses, and an increase in compensation and benefit expenses due to increased headcount. Adjusted G&A expense, which removes share-based compensation, transaction-related costs, and other one-time expenses, was $7.3 million in the quarter, compared with $4.4 million the prior year period. First quarter G&A spending was elevated due to a heavy concentration of launch and pre-launch spending activities that occurred in the first quarter. The Company expects quarterly adjusted G&A to be flat or decrease from first quarter levels for the remainder of 2025Adjusted Earnings Before Interest, Taxes, Depreciation and Amortization (Adjusted EBITDA): Adjusted EBITDA for the first quarter of 2025 was $3.7 million compared to $0.5 million in the prior year period. Net Income/Loss: Net loss for the first quarter of 2025 was $1.6 million or $0.06 per diluted share compared to a net loss of $0.8 million or $0.03 per diluted share in the prior year period. On a non-GAAP basis, the Company reported net income of $2.4 million or $0.07 per diluted share, for the first quarter of 2025 compared to $0.2 million , or $0.00 per diluted share in the prior year period. For a reconciliations of GAAP net loss to Earnings Before Interest, Taxes, Depreciation and Amortization EBITDA (“EBITDA”), Adjusted EBITDA and adjusted Non-GAAP basic and fully diluted earnings per share to the most directly comparable GAAP financial measure, please see the tables below. Cash Position: As of March 31, 2025 , the Company had cash and cash equivalents of $17.4 million and generated $2.1 million of operating cash flow during the quarter. Conference Call and Webcast Information As previously announced, Eton Pharmaceuticals will host its first quarter 2025 conference call as follows: Date: May 13, 2025 Time: 4:30 p.m. ET ( 3:30 p.m. CT ) Dial In* (Audio Only): Click Here Webcast: Click Here In addition to taking live questions from participants on the conference call, management will be answering emailed questions from investors. Investors can email questions to: investorrelations@etonpharma.com. The live webcast can be accessed on the Investors section of Eton’s website at https://ir.etonpharma.com/. An archived webcast will be available on Eton’s website approximately two hours after the completion of the event and for 30 days thereafter. * Conference call participants should register to obtain their dial-in and passcode details. Please be sure to register using a valid email address. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has six additional product candidates in late-stage development: ET- 400, ET -600, Amglidia®, ET- 700, ET -800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission . All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Non-GAAP Financial Measures In addition to the Company’s results of operations determined in accordance with U.S. generally accepted accounting principles (GAAP), which are presented and discussed above, management also utilizes Adjusted EBITDA, an unaudited financial measure that is not calculated in accordance with GAAP, to evaluate the Company’s financial results and performance and to plan and forecast future periods. Adjusted EBITDA is considered a “non-GAAP” financial measure within the meaning of Regulation G promulgated by the SEC . Management believes that this non-GAAP financial measure reflects an additional way of viewing aspects of the Company’s operations that, when viewed with GAAP results, provides a more complete understanding of the Company’s results of operations and the factors and trends affecting its business. Management believes Adjusted EBITDA provides meaningful supplemental information regarding the Company’s performance because (i) it allows for greater transparency with respect to key metrics used by management in its financial and operational decision-making; (ii) it excludes the impact of non-cash or, when specified, non-recurring items that are not directly attributable to the Company’s core operating performance and that may obscure trends in the Company’s core operating performance; and (iii) it is used by institutional investors and the analyst community to help analyze the Company’s results. However, Adjusted EBITDA and any other non-GAAP financial measures should be considered as a supplement to, and not as a substitute for, or superior to, the corresponding measures calculated in accordance with GAAP. Further, non-GAAP financial measures used by the Company and the way they are calculated may differ from the non-GAAP financial measures or the calculations of the same non-GAAP financial measures used by other companies, including the Company’s competitors. Adjusted EBITDA The Company defines Adjusted EBITDA as net loss, excluding the effects of stock-based compensation and expenses, interest, taxes, depreciation, amortization, investment loss, net, and, if any and when specified, other non-recurring income or expense items. Management believes that the most directly comparable GAAP financial measure to Adjusted EBITDA is net loss. Adjusted EBITDA has limitations and should not be considered as an alternative to gross profit or net loss as a measure of operating performance or to net cash provided by (used in) operating, investing, or financing activities as a measure of ability to meet cash needs. Investor Relations: Lisa M. Wilson , In-Site Communications, Inc. T: 212-452-2793E: lwilson@insitecony.com The following is a reconciliation of Adjusted EBITDA, a non-GAAP measure, to the most comparable GAAP measure, net loss, for the three months ended March 31, 2025 and for the same period in 2024: Source: Eton Pharmaceuticals

- NDA submitted for the treatment of central diabetes insipidus -- Expected 10-month review; commercial preparations underway for a potential Q1 2026 launch – DEER PARK, Ill., April 28, 2025 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (“Eton” or the “Company”) (Nasdaq: ETON), an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for ET-600, Eton’s proprietary, patented oral solution of desmopressin under development for the treatment of central diabetes insipidus. The Company expects the application to be assigned a 10-month FDA review, allowing for potential approval and launch in the first quarter of 2026. “The NDA submission for ET-600 is another important milestone for Eton, and for the thousands of children impacted by diabetes insipidus. If approved, ET-600 will be the only FDA-approved oral liquid formulation of desmopressin, providing the small, precise, and titratable doses required to treat pediatric patients,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “Leading pediatric endocrinologists have long expressed a need for this product, and we’re pleased to be one step closer to bringing it to patients. Pre-launch commercial activities are already underway as we plan for a potential approval in the first quarter of 2026.” Eton has been issued a patent by the U.S. Patent and Trademark Office on ET-600, which expires in 2044 and has additional patent applications under review. In a bioequivalence study conducted in 75 human subjects, ET-600 demonstrated pharmacokinetic equivalence to the FDA-approved reference product of the same active ingredient. The company estimates that central diabetes insipidus impacts approximately 3,000 pediatric patients in the United States. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX®, ALKINDI SPRINKLE®, GALZIN®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has six additional product candidates in late-stage development: ET-400, ET-600, Amglidia®, ET-700, ET-800 and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements associated with the expected ability of Eton to undertake certain activities and accomplish certain goals and objectives. These statements include but are not limited to statements regarding Eton’s business strategy, Eton’s plans to develop and commercialize its product candidates, the safety and efficacy of Eton’s product candidates, Eton’s plans and expected timing with respect to regulatory filings and approvals, and the size and growth potential of the markets for Eton’s product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Eton’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such drugs. These and other risks concerning Eton’s development programs and financial position are described in additional detail in Eton’s filings with the Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made. Eton undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made. Investor Relations:Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Source: Eton Pharmaceuticals, Inc.