Nitisinone

Q4 2025 product sales of $21.3 million, representing 83% growth over Q4 2024 Q4 2025 basic GAAP EPS of $0.06, fully diluted GAAP EPS of $0.05, basic non-GAAP EPS of $0.21, fully diluted non-GAAP EPS of $0.19, and Adjusted EBITDA of $6.2 million Launched DESMODA™, the first and only FDA-approved desmopressin oral solution Licensed U.S. rights to Orphan Drug HEMANGEOL ® ; expected to be accretive to 2026 earnings Company expects full year 2026 revenue to exceed $110 million with an Adjusted EBITDA margin of over 30% Management to hold conference call today at 4:30pm ET DEER PARK, Ill., March 19, 2026 (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 December 31, 2025. “It was another phenomenal quarter for Eton with meaningful contributions from key products across our portfolio including INCRELEX, ALKINDI SPRINKLE, KHINDIVI, and GALZIN. The strong fourth quarter helped us cap off a transformational 2025. During the year we launched three major products, INCRELEX, GALZIN, and KHINDIVI, and delivered $80 million in total revenue, more than doubling our 2024 revenue,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “The recent FDA approval of DESMODA and the acquisition of HEMANGEOL have further accelerated our 2026 growth trajectory. DESMODA’s approval was highly anticipated by the endocrinology community, and the product received a very strong reception in its first week of launch. Our commercial team is fired up and fully mobilized, executing on the launch plan. Our entire team is also hard at work on the integration of HEMANGEOL and eager for our scheduled May 1 st relaunch. Both of these products will be key growth contributors in 2026 and beyond. In addition, this year is poised to be our most active year yet on the clinical front, with key studies initiating or already initiated for the INCRELEX label expansion, the KHINDIVI reformulation, ET-700, and AMGLIDIA.” “We are poised for another year of record financial results in 2026. For the full year, we expect to see revenue exceed $110 million and at least a 30% Adjusted EBITDA margin,” concluded Brynjelsen. Fourth Quarter and Recent Business Highlights 83% growth in product sales year-over-year. Eton reported fourth quarter 2025 product sales of $21.3 million, compared to $11.6 million in the prior year period, driven primarily by strong growth in ALKINDI SPRINKLE and the addition of revenues from INCRELEX, GALZIN, and KHINDIVI. Commercial launch of DESMODA. Eton launched DESMODA on March 9 th , leveraging its existing team of pediatric endocrinology rare disease specialists. The product has received a strong reception from the patient and healthcare professional community, and numerous patients have already started on treatment. DESMODA is expected to significantly contribute to the Company’s long-term growth, with potential peak sales of $30-50 million annually. Acquired U.S. rights to Orphan Drug HEMANGEOL. The acquisition establishes a third call point for Eton’s sales team, pediatric dermatology. Eton anticipates integrating the product into its rare disease commercial infrastructure and extending its best-in-class Eton Cares patient support program to all HEMANGEOL patients, which includes the $0 co-pay for all commercially insured patients. Eton will begin commercializing HEMANGEOL on May 1 st . The transaction was financed with cash on hand and is expected to be accretive to 2026 earnings. Advancement of INCRELEX label harmonization study . In December, the company held a Type C meeting with the FDA to discuss the clinical pathway required to expand the U.S. FDA definition of Severe Primary IGF-1 Deficiency (SPIGFD) to match the broader E.U. definition. The Company believes the meeting was positive, and as a result, Eton submitted the proposed final study protocol to the FDA in February. The Company expects FDA feedback on the protocol by the end of March. If cleared, Eton would proceed with study initiation, with the goal of dosing the first patient in the third quarter of 2026. Continued strong GALZIN growth, now exceeding 300 active patients. GALZIN growth continued to trend ahead of forecast in the fourth quarter of 2025 and so far in the first quarter of 2026. The Company’s targeted investment in rare disease specialists and education has increased awareness, access, and adoption and, earlier this month, the Company eclipsed 300 active patients on therapy. Eton continues to see a large growth opportunity to expand the treated population through conversion of patients currently taking over-the-counter products that are not FDA approved for Wilson disease. Planned ET-700 pilot study. ET-700, the Company’s extended-release formulation of zinc acetate, will be tested in a proof-of-concept positron emission tomography (PET) study that is expected to initiate in April. The study will compare ET-700 to GALZIN and a placebo. If successful, the study would support the initiation of a dose ranging and pivotal study in early 2027. Initiation of bioequivalence study to support KHINDIVI label expansion. The Company has initiated the bioequivalence study required to support submission of Eton’s revised KHINDIVI formulation designed to expand the product’s approved indication beyond its current restriction of children ages five and older. The study has been initiated, with the first patient dosed last week. The Company expects to receive preliminary top-line results late in the second quarter and receive the final study report needed for submission of the supplement in the third quarter of 2026. The Company anticipates a 10-month review for the submission, leading to a potential mid-2027 approval. 2026 Financial Guidance The Company expects 2026 revenues to exceed $110 million with an Adjusted EBITDA margin of at least 30%. Fourth Quarter Financial Results Net Revenue: Net revenues for the fourth quarter of 2025 were $21.3 million compared to $11.6 million in the prior year period, an increase of 83%. The growth was driven primarily by increased sales of ALKINDI SPRINKLE and the addition of sales from INCRELEX, GALZIN, and KHINDIVI. Gross Profit : Gross profit for the fourth quarter of 2025 was $13.1 million compared to $6.5 million in the prior year period, an increase of 102%, driven by the increase in revenues. Adjusted gross profit, which adjusts for the impact of acquired inventory step-up adjustments and intangible amortization , was $15.5 million in the fourth quarter of 2025, representing an adjusted gross margin of 73% compared to adjusted gross profit of $6.8 million and adjusted gross margin of 59% in the prior year period. Research and Development (R&D) Expenses : R&D expenses for the fourth quarter of 2025 were $1.8 million compared to $(0.9) million in the prior year period, due primarily to increased expenses associated with the Company’s pipeline development activities. In addition, during the fourth quarter of 2024, Eton’s ET-400 product was granted Orphan Drug Designation by the FDA, which resulted in Eton receiving a refund of the NDA filing fee that was paid and expensed in a prior quarter. General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2025 were $8.9 million compared to $6.7 million in the prior year period, due primarily to an increase in product advertising and launch-year promotional expenses, higher stock-based compensation expense, and an increase in compensation and benefit expenses due to an increase in general and administrative headcount. Adjusted G&A expense, which removes share-based compensation, transaction-related costs, and other one-time expenses, was $7.8 million in the quarter, compared to $5.8 million the prior year period. The fourth quarter of 2025 included $0.9 million of annual FDA Program Fees related to the company’s approved New Drug Applications. Historically, Eton qualified for a waiver of annual fees for its Orphan Designated products, however, as of October 1, 2025, the Company’s revenue level was above the waiver threshold, and it began paying the fees. Adjusted Earnings Before Interest, Taxes, Depreciation and Amortization (Adjusted EBITDA): Adjusted EBITDA for the fourth quarter of 2025 was $6.2 million compared to $2.1 million in the prior year period, an increase of 195%. Net Income/Loss: Net income for the fourth quarter of 2025 was $1.5 million or $0.05 per diluted share compared to a net loss of $5.4 million or $0.02 per basic and diluted share in the prior year period. On a non-GAAP basis, the Company reported net income of $5.4 million or $0.19 per diluted share, for the fourth quarter of 2025 compared to net income of $0.7 million, or $0.02 per diluted share in the prior year period. For a reconciliation 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 December 31, 2025, the Company had cash and cash equivalents of $25.9 million. Conference Call and Webcast Information As previously announced, Eton Pharmaceuticals will host its fourth quarter 2025 conference call as follows: Date: March 19, 2026 Time: 4:30 p.m. ET (3:30 p.m. CT) Participant Webcast Link: Click Here Participant Call Link: 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 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 ten commercial rare disease products: KHINDIVI M , INCRELEX ® , ALKINDI SPRINKLE ® , DESMODA™, GALZIN ® , HEMANGEOL ® , PKU GOLIKE ® , Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has four additional product candidates in late-stage development: Amglidia ® , ET-700, ET-800 and ZENEO ® hydrocortisone autoinjector. For more information, please visit our website at . 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: 212-452-2793 E: lwilson@insitecony.com Eton Pharmaceuticals, Inc. Statements of Operations (In thousands, except per share amounts) For the three months ended For the years ended December 31, December 31, December 31, December 31, 2025 2024 2025 2024 Revenues: (Unaudited) (Audited) Licensing revenue $ — $ — $ 3,286 $ 500 Product sales and royalties, net 21,281 11,647 76,664 38,511 Total net revenues 21,281 11,647 79,950 39,011 Cost of sales: Licensing revenue — — 825 270 Product sales and royalties 8,181 5,171 36,385 15,330 Total cost of sales 8,181 5,171 37,210 15,600 Gross profit 13,100 6,476 42,740 23,411 Operating expenses: Research and development (1) 1,780 (871 ) 7,765 3,255 General and administrative 8,856 6,718 35,819 22,753 Total operating expenses 10,636 5,847 43,584 26,008 Income (loss) from operations 2,464 629 (844 ) (2,597 ) Other expense: Interest and other expense, net (1,050 ) (1,140 ) (3,714 ) (1,211 ) Income (loss) before income tax expense 1,414 (511 ) (4,558 ) (3,808 ) Income tax (benefit) expense (69 ) 87 43 15 Net (loss) income $ 1,483 $ (598 ) $ (4,601 ) $ (3,823 ) Net income (loss) per share, basic and diluted $ 0.06 $ (0.02 ) $ (0.17 ) $ (0.15 ) Weighted average number of common shares outstanding, basic and diluted 26,937 26,135 26,908 25,895 Net income (loss) per share, diluted $ 0.05 $ (0.02 ) $ (0.17 ) $ (0.15 ) Weighted average number of common shares outstanding, diluted 31,243 26,135 26,908 25,895 (1) During the three months ended December 31, 2024, we received $2,024 from the FDA associated with an approved PDUFA exemption and corresponding refund for a previously paid NDA filing fee. Eton Pharmaceuticals, Inc. Balance Sheets (In thousands, except share and per share amounts) (Audited) December 31, 2025 December 31, 2024 Assets Current assets: Cash and cash equivalents $ 25,942 $ 14,936 Accounts receivable, net 11,757 5,361 Inventories, net 15,419 15,232 Prepaid expenses and other current assets 7,463 5,492 Total current assets 60,581 41,021 Property and equipment, net 326 34 Intangible assets, net 30,878 34,881 Operating lease right-of-use assets, net 310 175 Other long-term assets, net 19 12 Total assets $ 92,114 $ 76,123 Liabilities and stockholders ’ equity Current liabilities: Accounts payable $ 10,976 $ 4,167 Short-term debt, net of discount 8,789 — Accrued Medicaid rebates 9,317 6,866 Accrued liabilities 9,408 8,914 Total current liabilities 38,490 19,947 Long-term debt, net of discount and including accrued fees 21,769 29,811 Operating lease liabilities, net of current portion 460 107 Other long-term liabilities 5,241 1,830 Total liabilities 65,960 51,695 Commitments and contingencies Stockholders ’ equity Common stock, $0.001 par value; 50,000,000 shares authorized; 27,047,061 and 26,709,084 shares issued and outstanding at December 31, 2025 and December 31, 2024, respectively 27 27 Additional paid-in capital 138,621 132,294 Accumulated deficit (112,494 ) (107,893 ) Total stockholders ’ equity 26,154 24,428 Total liabilities and stockholders ’ equity $ 92,114 $ 76,123 Eton Pharmaceuticals, Inc. Statements of Cash Flows (In thousands) (Audited) For the years ended December 31, 2025 December 31, 2024 Cash flows from (used in) operating activities Net loss $ (4,601 ) $ (3,823 ) Adjustments to reconcile net loss to net cash from operating activities: Stock-based compensation 5,512 3,165 Depreciation and amortization 4,044 1,146 Inventory step-up 5,094 — Excess and obsolete inventory reserve 594 529 Debt discount amortization 696 1,109 Non-cash lease expense 44 70 Changes in operating assets and liabilities, net of impact of business acquisition: Accounts receivable (6,396 ) (3,118 ) Inventories (5,876 ) (1,839 ) Prepaid expenses and other assets (1,971 ) (3,349 ) Accounts payable 6,808 2,318 Accrued Medicaid rebates 2,451 3,239 Accrued liabilities 1,011 1,484 Other non-current assets and liabilities 3,114 38 10,524 969 Cash flows from (used in) investing activities Purchases of property and equipment (333 ) (26 ) Acquisition of business — (30,000 ) Purchase of product licensing rights — (9,988 ) Net cash from (used in) investing activities (333 ) (40,014 ) Cash flows from (used in) financing activities Net proceeds from the issuance of long-term debt — 25,309 Repayment of long-term debt — (1,155 ) Common stock issued in private placement offering — 7,000 Proceeds from stock option exercises 598 1,191 Employee stock purchase plan 217 248 Net cash from (used in) financing activities 815 32,593 Change in cash and cash equivalents 11,006 (6,452 ) Cash and cash equivalents at beginning of period 14,936 21,388 Cash and cash equivalents at end of period $ 25,942 $ 14,936 Supplemental disclosures of cash flow information Cash paid for interest $ 3,325 $ 665 Cash paid for income taxes $ 118 $ 82 Eton Pharmaceuticals, Inc. Adjusted non-GAAP EBITDA Calculation and US GAAP to Non-GAAP Reconciliation (in thousands, except per share amounts) (Unaudited) For the three months ended For the years ended December 31, December 31, December 31, December 31, 2025 2024 2025 2024 GAAP net income (loss) $ 1,483 $ (598 ) $ (4,601 ) $ (3,823 ) Depreciation 17 12 41 50 Intangible amortization expense 1,000 343 4,003 1,096 Interest expense (including debt discount amortization and non-cash interest expenses) 1,208 1,333 4,781 2,005 Income tax expense (benefit) (69 ) 87 43 15 EBITDA $ 3,639 $ 1,177 $ 4,267 $ (657 ) Other non-GAAP adjustments: Inventory step-up expense (1) 1,421 — 5,094 — Stock-based compensation (2) 1,124 782 5,512 3,165 Severance expense (3) — — 335 — Acquisition/divestiture-related costs (4) 7 140 581 415 Total of Other non-GAAP adjustments 2,552 922 11,522 3,580 Adjusted EBITDA $ 6,191 $ 2,099 $ 15,789 $ 2,923 GAAP income (loss) before income tax $ 1,414 $ (511 ) $ (4,558 ) $ (3,808 ) Non-GAAP adjustments: Depreciation (5) 17 12 41 50 Intangible amortization expense (6) 1,000 343 4,003 1,096 Inventory step-up expense (1) 1,421 - 5,094 - Share-based compensation (2) 1,124 782 5,512 3,165 Severance expense (3) — — 335 — Acquisition/divestiture-related costs (4) 7 140 581 415 Total pre-tax non-GAAP adjustments 3,569 1,277 15,566 4,726 Income tax effect of pre-tax non-GAAP adjustments (7) (372 ) 94 235 49 Total non-GAAP adjustments 3,941 1,183 15,331 4,677 Non-GAAP Net Income $ 5,355 $ 672 $ 10,773 $ 869 Weighted average number of common shares outstanding, basic 26,937 26,135 26,908 25,895 Weighted average number of common shares outstanding, diluted 31,243 29,320 31,046 27,458 GAAP income (loss) per share - Basic $ 0.06 $ (0.02 ) $ (0.17 ) $ (0.15 ) Non-GAAP adjustments 0.15 0.05 0.57 0.18 Non-GAAP earnings per share - Basic $ 0.21 $ 0.03 $ 0.40 $ 0.03 GAAP income (loss) per share - Basic $ 0.06 $ (0.02 ) $ (0.17 ) $ (0.15 ) Non-GAAP adjustments 0.13 0.04 0.49 0.17 Non-GAAP earnings per share - Diluted $ 0.19 $ 0.02 $ 0.32 $ 0.02 Eton Pharmaceuticals, Inc. Fourth Quarter 2025 GAAP to Non-GAAP Net Income (Loss) Reconciliation (in thousands) (Unaudited) Fourth Quarter 2025 GAAP Depreciation and Intangible Amortization Inventory Step-Up Expense Stock Based Compensation Severance Expense Acquisition/ Divestiture Related Costs Non-GAAP Cost of sales $ 8,181 (1,000 ) (1,421 ) - - - $ 5,760 Research and development 1,780 - - (62 ) - - 1,718 General and administrative 8,856 (17 ) - (1,062 ) - (7 ) 7,770 Interest and other expense, net (1,050 ) - - - - - (1,050 ) Fourth Quarter 2024 Cost of sales $ 5,171 (343 ) - - - - $ 4,828 Research and development (871 ) - - (31 ) - - (902 ) General and administrative 6,718 (12 ) - (751 ) - (140 ) 5,815 Interest and other expense, net (1,140 ) - - - - - (1,140 ) Eton Pharmaceuticals, Inc. Full Year 2025 GAAP to Non-GAAP Net Income (Loss) Reconciliation (in thousands) (Unaudited) Year Ended December 31, 2025 Cost of sales $ 37,210 (4,003 ) (5,094 ) - - - $ 28,113 Research and development 7,765 - - (182 ) - - 7,583 General and administrative 35,819 (41 ) - (5,330 ) (335 ) (581 ) 29,532 Interest and other expense, net (3,714 ) - - - - - (3,714 ) Year Ended December 31, 2024 Cost of sales $ 15,600 (1,096 ) - - - - $ 14,504 Research and development 3,255 - - (276 ) - - 2,979 General and administrative 22,753 (50 ) - (2,889 ) - (415 ) 19,399 Interest and other expense, net (1,211 ) - - - - - (1,211 )