Nitisinone

Product sales and royalty revenue of $9.1 million, representing 40% growth over Q2 2023 and the 14th straight quarter of sequential product sales growthNew drug application (NDA) for ET-400 accepted by the U.S. Food and Drug Administration (FDA) and assigned a February 28, 2025 PDUFA dateQ2 2024 Cash Flow from Operations of $1.3 million, including the payment of one-time $2.0 million NDA filing fee for ET-400Management to hold conference call today at 4:30pm ET DEER PARK, Ill., Aug. 08, 2024 (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 June 30, 2024. “We are pleased to report another quarter of record product sales as Carglumic Acid and ALKINDI SPRINKLE® continue to deliver impressive growth. During the quarter we also launched PKU GOLIKE under our commercial infrastructure and received a favorable reception from the PKU community. Our five existing commercial products have the potential to deliver strong organic growth for many years to come, and we plan to accelerate that growth with our internal pipeline and business development activities,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “We have made tremendous recent progress advancing our pipeline, which we believe will set us up to deliver even greater growth in 2025 and beyond. Our NDA for ET-400 was recently accepted for review and assigned a target action date in early 2025. Pre-launch commercialization activities for the product are underway and we anticipate launching it shortly after the expected approval. In addition, our ET-600 product candidate is now ready to start its pivotal study in Q3, after passing a pilot study earlier this year. This should allow for an early 2025 NDA submission, which would further boost the Company’s long-term growth prospects” concluded Brynjelsen. Second quarter and Recent Business Highlights 14th straight quarter of sequential growth in product sales and royalty revenue. Eton reported second quarter 2024 product sales of $9.1 million, representing 40% growth over the prior year period, and 14% over the first quarter of 2024, driven primarily by the ongoing growth of ALKINDI SPRINKLE and Carglumic Acid. The Company expects to continue to see sequential quarter-over-quarter growth of product sales through the remainder of 2024 and beyond. NDA for ET-400 submitted and accepted by the FDA. During the second quarter of 2024, Eton submitted an NDA for ET-400, its proprietary patented formulation of hydrocortisone oral solution. The FDA accepted the NDA and assigned it a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2025. Eton anticipates initiating production of launch quantities in the fourth quarter of 2024, to allow for a commercial launch promptly after the expected approval. Eton believes the introduction of ET-400 will allow the Company to capture a greater percentage of the oral hydrocortisone market and, together with ALKINDI SPRINKLE, can achieve combined peak sales of more than $50 million annually. Acceleration of growth for ALKINDI SPRINKLE. ALKINDI SPRINKLE reported 63% year over year revenue growth in Q2 2024. Product sales are benefiting from the Company’s internal sales force, which has now been in place for more than a year and has seen increased production, as well as recent initiatives including the launch of the sampling program and the Company’s strong presence at Endocrinology medical conferences and patient advocacy events in the first half of 2024. Continued Carglumic Acid outperformance. Sales of Carglumic Acid continue to exceed the Company’s expectations. The product reported strong growth in the second quarter and has already added additional patients in the third quarter of 2024. The recent launches of Betaine Anhydrous, Nitisinone, and now PKU GOLIKE have also helped increase the frequency of the Company’s interactions with potential Carglumic Acid prescribers. Launched PKU GOLIKE, a medical formula for patients with phenylketonuria (PKU). After acquiring PKU GOLIKE in the first quarter, the Company launched the product under Eton’s commercial infrastructure in April at the Genetic Metabolic Dieticians International Conference. The product has been well received and the Company has already seen an increased number of patient referrals since the initiation of its promotional activities. Anticipated initiation of ET-600 pivotal study. Eton expects to initiate a pivotal bioequivalence study for ET-600, the Company’s product candidate for diabetes insipidus, in the third quarter. Earlier this year, the product passed its pilot bioequivalence study, and the company anticipates submitting an NDA for the product in early 2025 Second Quarter Financial Results Net Revenue: Net sales for the second quarter of 2024 were $9.1 million compared to $12.0 million in the prior year period. Net sales in the prior year period included $5.5 million of licensing payments related to the divestment of Eton’s neurology product royalties. Product sales and royalty revenue were $9.1 million for the second quarter of 2024, an increase of 40% compared to $6.5 million in the prior year period, and an increase of 14% over the first quarter of 2024. The year-over-year increase in product sales and royalty revenue was primarily driven by growth in ALKINDI SPRINKLE and Carglumic Acid. Gross Profit: Gross profit for the second quarter of 2024 was $5.6 million compared to $9.7 million in the prior year period. Research and Development (R&D) Expenses: R&D expenses for the second quarter of 2024 were $3.0 million compared to $1.1 million in the prior year period. The increase was primarily due to the payment of a $2.0 million NDA filing fee for ET-400. General and Administrative (G&A) Expenses: G&A expenses for the second quarter of 2024 were $5.6 million compared to $4.7 million in the prior year period, mainly due to increased sales and marketing expenses, employee-related expenses, and one-time legal fees related to business development activities. Net Income: Net loss for the second quarter of 2024 was $2.9 million or $0.11 per basic and diluted share, including the $2.0 million ET-400 filing fee. In the prior year period, net income was $4.6 million, or $0.18 per basic and diluted share, and included $5.5 million from the sale of Eton’s royalty interests. Cash Position: As of June 30, 2024, Eton had cash and cash equivalents of $17.7 million. The Company generated $1.3 million of operating cash flow during the quarter, including the $2.0 million ET-400 filing fee. The company expects to continue to generate positive operating cash flow for the remainder of 2024. Conference Call and Webcast InformationAs previously announced, Eton Pharmaceuticals will host a its second quarter 2024 conference call as follows:DateAugust 8, 2024Time4:30 p.m. ET (3:30 p.m. CT)Dial in* (Audio Only)Click HereWebcast: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 also 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 five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, 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 Eton Pharmaceuticals, Inc.Condensed Statements of Operations(In thousands, except per share amounts)(Unaudited) For the three months ended For the six months ended June 30, June 30, June 30, June 30, 2024 2023 2024 2023 Revenues: Licensing revenue$— $5,500 $— $5,500 Product sales and royalties 9,074 6,497 17,040 11,801 Total net revenues 9,074 11,997 17,040 17,301 Cost of sales: Licensing revenue — — $— — Product sales and royalties 3,448 2,315 6,407 4,273 Total cost of sales 3,448 2,315 6,407 4,273 Gross profit 5,626 9,682 10,633 13,028 Operating expenses: Research and development 2,970 1,125 3,621 1,660 General and administrative 5,591 4,674 10,747 10,019 Total operating expenses 8,561 5,799 14,368 11,679 Income (loss) from operations (2,935) 3,883 (3,735) 1,349 Other income (expense): Other income — 800 — 800 Interest expense, net (52) (124) (63) (250)Total other income (expense) (52) 676 (63) 550 Income (loss) before income tax expense (2,987) 4,559 (3,798) 1,899 Income tax expense 54 — 54 — Net income (loss)$(3,041) $4,559 $(3,852) $1,899 Net income (loss) per share, basic$(0.12) $0.18 $(0.15) $0.07 Weighted average number of common shares outstanding, basic 25,778 25,593 25,771 25,560 Net income (loss) per share, diluted$(0.12) $0.18 $(0.15) $0.07 Weighted average number of common shares outstanding, diluted 25,778 25,983 25,771 25,949 Eton Pharmaceuticals, Inc.Condensed Balance Sheets(in thousands, except share and per share amounts) June 30, 2024 December 31, 2023 (Unaudited) Assets Current assets: Cash and cash equivalents$17,694 $21,388 Accounts receivable, net 4,873 3,411 Inventories 2,068 911 Prepaid expenses and other current assets 799 1,129 Total current assets 25,434 26,839 Property and equipment, net 45 58 Intangible assets, net 6,122 4,739 Operating lease right-of-use assets, net 210 92 Other long-term assets, net 12 12 Total assets$31,823 $31,740 Liabilities and stockholders’equity Current liabilities: Accounts payable$2,133 $1,848 Debt, net of unamortized discount 4,658 5,380 Accrued liabilities 11,425 9,013 Total current liabilities 18,216 16,241 Operating lease liabilities, net of current portion 145 22 Total liabilities 18,361 16,263 Commitments and contingencies (Note 11) Stockholders’equity Common stock, $0.001 par value; 50,000,000 shares authorized; 25,745,802 and 25,688,062 shares issued and outstanding at June 30, 2024 and December 31, 2023, respectively 26 26 Additional paid-in capital 121,358 119,521 Accumulated deficit (107,922) (104,070)Total stockholders’equity 13,462 15,477 Total liabilities and stockholders’equity$31,823 $31,740 Eton Pharmaceuticals, Inc.Condensed Statements of Cash Flows(In thousands)(Unaudited) Six months ended Six months ended June 30, 2024 June 30, 2023 Cash flows from operating activities Net loss$(3,852) $1,899 Adjustments to reconcile net loss to net cash (used in) provided by operating activities: Stock-based compensation 1,661 1,657 Depreciation and amortization 513 385 Non-cash lease expense 36 39 Debt discount amortization 48 61 Changes in operating assets and liabilities: Accounts receivable (1,462) (1,232)Inventories (1,157) (259)Prepaid expenses and other assets 330 423 Accounts payable 284 537 Accrued liabilities 2,381 2,045 Net cash (used in) provided by operating activities (1,218) 5,555 Cash flows from investing activities Purchases of product license rights (1,868) — Purchases of property and equipment (14) — Net cash used in investing activities (1,882) — Cash flows from financing activities Repayment of long-term debt (770) (385)Proceeds from employee stock purchase plan and stock option exercises 176 272 Payment of tax withholding related to net share settlement of stock option exercises — (181)Net cash used in financing activities (594) (294) Change in cash and cash equivalents (3,694) 5,261 Cash and cash equivalents at beginning of period 21,388 16,305 Cash and cash equivalents at end of period$17,694 $21,566 Supplemental disclosures of cash flow information Cash paid for interest$362 $426 Cash paid for income taxes$— $— Supplemental disclosures of non-cash transactions in investing and financing activities Right-of-use assets and liabilities obtained due to lease renewal$153 $—

- Prescription Drug User Fee Act (PDUFA) target action date is February 28, 2025 - - Product has patent protection through 2043 - July 15, 2024 -- 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 Company’s New Drug Application (NDA) for ET-400, a proprietary patented formulation of hydrocortisone oral solution, has been accepted by the U.S. Food and Drug Administration (FDA) and has been assigned a Prescription Drug User Fee Act (PDUFA) target action date of February 28, 2025. “The acceptance of our NDA is a significant milestone toward our efforts of bringing this important and highly in-demand liquid formulation to children. Once approved, we believe ET-400 will allow us to capture a greater percentage of the oral hydrocortisone market and, together with ALKINDI SPRINKLE®, achieve combined peak sales of more than $50 million annually. We look forward to working with the FDA and preparing for a potential launch in early 2025,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. ET-400 is a proprietary room temperature stable formulation of oral hydrocortisone solution. The Company has been issued a patent related to the product by the U.S. Patent and Trademark Office that expires in 2043 and has additional patent applications currently under review. Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has five commercial rare disease products: ALKINDI SPRINKLE®, PKU GOLIKE®, Carglumic Acid, Betaine Anhydrous, and Nitisinone. The Company has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO® hydrocortisone autoinjector. For more information, please visit our website at www.etonpharma.com. The content above comes from the network. if any infringement, please contact us to modify.