Nitisinone

GENEVA, SWITZERLAND / ACCESSWIRE / March 22, 2024 / RELIEF THERAPEUTICS Holding SA (SIX:RLF)(OTCQB:RLFTF)(OTCQB:RLFTY ) (Relief, or the Company), a biopharmaceutical company committed to delivering innovative treatment options for select specialty, unmet and rare diseases, today announced it has granted an exclusive license to Eton Pharmaceuticals, Inc. (Nasdaq: ETON) (Eton) for the commercialization of GOLIKE ® family of products in the United States. "This transition is fully aligned with Relief's strategy of moving the GOLIKE products into partnership models in the U.S. and Europe. We are very pleased to be establishing this partnership with Eton for GOLIKE patients in the U.S. given their valuable experience in the metabolic area," said Michelle Lock, interim CEO of Relief. "We are excited to be partnering with Relief on PKU GOLIKE in the United States. After extensive discussions with metabolic geneticists, dieticians, and PKU patients, we believe PKU GOLIKE is the best product in the estimated $100 million U.S. PKU medical formula market. With our sales force and existing relationships in the metabolic community, we believe we can significantly increase the awareness, education, and adoption of this important product," said Sean Brynjelsen, CEO of Eton Pharmaceuticals. Under the terms of the agreement, Relief will receive an upfront payment of $2.2 million and is eligible to receive an additional $2.0 million in sales milestones payments as well as mid-teens royalties on net sales. In the fourth quarter of 2023, PKU GOLIKE's annualized net sales exceeded $1 million in the U.S., continuing its growth trajectory since its launch in late 2022. As part of the agreement, Eton also received U.S. rights to Relief's GOLIKE Medical Food line extensions under development for the management of other inherited rare metabolic diseases such as tyrosinemia and homocystinuria, which are both expected to launch in 2025 and 2026 under the same trademark. Relief is also in discussions with Eton related to additional development assets, including RLF-OD032. RLF‑OD032 is an innovative drug product candidate under development for the treatment of PKU expected to be filed for approval before the FDA in H2/2025 as 505(b)(2) application. Relief will continue to fully own GOLIKE rights outside the United States. About Relief Relief is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety, and convenience to benefit the lives of patients living with select specialty and rare diseases. Relief's portfolio offers a balanced mix of marketed, revenue-generating products, our proprietary, globally patented Physiomimic™ and TEHCLO™ platform technologies and a targeted clinical development pipeline consisting of risk-mitigated assets focused in three core therapeutic areas: rare metabolic disorders, rare skin diseases and rare respiratory diseases. In addition, Relief is commercializing several legacy products via licensing and distribution partners. Relief's mission is to provide therapeutic relief to those suffering from rare diseases and is being advanced by an international team of well-established, experienced biopharma industry leaders with extensive research, development and rare disease expertise. Relief is headquartered in Geneva, with additional offices in Balerna, Switzerland, Offenbach am Main, Germany and Monza, Italy. Relief is listed on the SIX Swiss Exchange under the symbol RLF and quoted in the U.S. on OTCQB under the symbols RLFTF and RLFTY. For more information, please visit our website or follow Relief on LinkedIn . About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. It currently has five commercial rare disease products: ALKINDI SPRINKLE ® , PKU GOLIKE ® , Carglumic Acid, Betaine Anhydrous, and Nitisinone. Eton has three additional product candidates in late-stage development: ET-400, ET-600, and ZENEO ® hydrocortisone autoinjector. For more information, visit . About Phenylketonuria (PKU) Phenylketonuria (PKU) is caused by a defect of the enzyme needed to break down phenylalanine (Phe), leading to a toxic buildup of Phe from the consumption of foods containing protein or aspartame. Untreated PKU can result in global developmental delay or severe irreversible intellectual disability, as well as growth failure, hypopigmentation, motor deficits, ataxia and seizures. Living with PKU requires a limited diet and very careful management. If left unmanaged, PKU can lead to devastating consequences, such as brain damage. People living with PKU do not have the ability to metabolize Phe, which is found in many foods, and they require supplementation of amino acid-based phenylalanine-free Medical Foods to prevent protein deficiency and optimize metabolic control. Currently available Medical Foods may lead to poor or suboptimal clinical outcomes and compliance because they are rapidly absorbed and are characterized by an unpleasant odor and aftertaste. Such factors contribute to barriers to social interaction for PKU patients, further limiting Medical Foods compliance and exposing patients to the risks of poor disease control. About PKU GOLIKE PKU GOLIKE ® products are Medical Foods for the dietary management of PKU in both children and adults. Developed with the Relief proprietary, patent-protected Physiomimic Technology™ drug delivery platform, PKU GOLIKE ® products are the first prolonged-release amino acid Medical Food, characterized by a special coating that ensures physiological absorption of the amino acids mirroring that of natural proteins. The special coating also masks the unpleasant taste, odor and aftertaste of the amino acids. PKU GOLIKE ® granules are flavorless and can be mixed with many foods. PKU GOLIKE ® granule products contain all 19 amino acids that people with PKU need to maintain neurological and muscular health and is fortified with vitamins and minerals, including ones normally found in protein-rich foods like iron, calcium, and vitamin B12. The PKU GOLIKE ® line of products is available in convenient packets (PKU GOLIKE Plus ® 3-16 and 16+) and medical food bars (PKU GOLIKE BAR ® ). PKU GOLIKE ® products have been commercially available in the U.S. since October 2022. For more information, visit (this site is intended for U.S. audiences only). CONTACT : RELIEF THERAPEUTICS Holding SA Avenue de Sécheron 15 1202 Geneva Switzerland contact@relieftherapeutics.com DISCLAIMER This press release contains forward-looking statements. Forward-looking statements involve known and unknown risks, uncertainties, including its ability to achieve its corporate, development and commercial goals, and other factors which could cause the actual results, financial condition, performance or achievements of Relief to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. A number of factors, including those described in Relief's filings with the SIX Swiss Exchange and the U.S. Securities and Exchange Commission (SEC), could adversely affect Relief. Copies of Relief's filings with the SEC are available on the SEC EDGAR database at . Relief does not undertake any obligation to update the information contained herein, which speaks only as of this date. SOURCE: Relief Therapeutics Holdings AG View the original press release on accesswire.com

Q4 2023 revenue of $7.3 million, representing a 109% increase from Q4 2022, marking the twelfth consecutive quarter of sequential product sales growthFull year 2023 revenues reached $31.6 million with product sales and royalty revenues of $26.1 million, a 132% increase from 2022Announced successful results from ET-400’s pivotal bioequivalence study, NDA submission planned for Q2 2024Management to hold conference call today at 4:30pm ET DEER PARK, Ill., March 14, 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 and year ended December 31, 2023. “We finished 2023 by delivering our twelfth straight quarter of sequential product sales growth and another quarter of positive operating cash flow. The growth trajectory continues for ALKINDI SPRINKLE® and Carglumic Acid, and we are excited for the Company’s prospects for 2024 and beyond,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “In addition to the strong performance of our commercial products, we are pleased to report favorable developments in our pipeline. In recent weeks, we received positive study results for both ET-400 and ET-600, which brings both products one step closer to NDA submission. We look forward to submitting our NDA for ET-400 in the second quarter of this year and ET-600 early next year,” concluded Brynjelsen. Fourth Quarter and Recent Business Highlights Twelfth straight quarter of sequential growth in product sales. Eton reported fourth quarter 2023 product sales and royalty revenue of $7.3 million, representing a 109% year-over-year increase and 4% growth over the third quarter of 2023, driven primarily by the ongoing momentum of ALKINDI SPRINKLE and Carglumic Acid. The Company anticipates continued growth of product sales in 2024. Recorded positive cash flow from operations. Eton generated $0.4 million of operating cash during the fourth quarter of 2023, with total cash and cash equivalents of $21.4 million as of December 31, 2023. Passed pivotal bioequivalence study for ET-400. In February, Eton received preliminary data showing that ET-400, its proprietary formulation of oral hydrocortisone, passed its pivotal bioequivalence study. All other major filing pre-requisites have been completed, and the Company expects to submit the NDA to the FDA for ET-400 when the final clinical study report is available in the second quarter of 2024. This would allow for a potential FDA approval as early as the first quarter of 2025. Awarded U.S. patent for ET-400. The patent, which covers the Company’s proprietary formulation, expires in 2043 and is expected to be listed in the FDA’s Orange Book upon the product’s approval. The Company has additional patent applications related to the product under review with the United States Patent and Trademark Office. Passed pilot bioequivalence study for ET-600. The Company plans to run the pivotal study in the second half of 2024, which would allow for a potential NDA submission in early 2025. Initiated ALKINDI SPRINKLE sampling program. The Company’s sampling program went live in February 2024, allowing pediatric endocrinology offices to stock ALKINDI SPRINKLE samples so that newly diagnosed or converting patients can immediately start therapy on ALKINDI SPRINKLE in the presence of their physician. Eton has already seen strong demand for samples from physicians and expects the sampling program to have a positive impact on adoption and 2024 revenue growth. Launched ultra-rare disease product Nitisinone capsules. The product was launched in February 2024 with full patient and provider support services. Eton believes its commercial advantages, existing relationships with prescribers, and experienced sales force will allow the Company to capture a meaningful percentage of the $50 million market. Fourth Quarter Financial Results Net Revenue: Net product sales and royalty revenues for the fourth quarter of 2023 increased by 109% to $7.3 million compared to $3.5 million in the prior year period, driven primarily by growth in ALKINDI SPRINKLE and Carglumic Acid. Total net revenues were $7.3 million for the fourth quarter of 2023, compared to $8.5 million for the fourth quarter of 2022. The prior year period included $5.0 million in licensing revenue resulting from a milestone payment received from Azurity Pharmaceuticals. Gross Profit: Gross profit for the fourth quarter of 2023 was $3.6 million. Fourth quarter 2023 gross profit was negatively impacted by a $1.0 million one-time payment to Diurnal PLC as a result of ALKINDI SPRINKLE net sales triggering a commercial success-based milestone under terms of the product’s licensing agreement. Gross profit for the fourth quarter of 2022 was $6.4 million and benefited from a $5.0 million milestone payment received from Azurity Pharmaceuticals. Research and Development (R&D) Expenses: R&D expenses for the fourth quarter of 2023 were $1.0 million compared to $0.9 million in the prior year period. General and Administrative (G&A) Expenses: G&A expenses for the fourth quarter of 2023 were $4.6 million compared to $4.4 million in the prior year period. The slight increase in G&A expenses was primarily due to personnel additions. Net Loss: Net loss for the fourth quarter of 2023 was $2.3 million or $0.09 per basic and diluted share compared to net income of $0.9 million or $0.04 per diluted share in the prior year period. Cash Position: As of December 31, 2023, the Company had cash and cash equivalents of $21.4 million. Conference Call and Webcast Information As previously announced, Eton Pharmaceuticals will host its fourth quarter 2023 conference call as follows: Date March 14, 2024 Time 4:30 p.m. ET (3:30 p.m. CT) Register* (Audio Only) 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 four FDA-approved rare disease products: ALKINDI SPRINKLE®, 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: 212-452-2793 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 (Unaudited) December 31, December 31, December 31, December 31, 2023 2022 2023 2022 Revenues: Licensing revenue $— $5,000 $5,500 $10,000 Product sales and royalties, net 7,313 3,498 26,142 11,251 Total net revenues 7,313 8,498 31,642 21,251 Cost of sales: Licensing revenue 1,000 650 1,000 1,640 Product sales and royalties 2,683 1,488 9,581 5,293 Total cost of sales 3,683 2,138 10,581 6,933 Gross profit (loss) 3,630 6,360 21,061 14,318 Operating expenses: Research and development 1,047 944 3,322 3,996 General and administrative 4,575 4,354 18,931 18,582 Total operating expenses 5,622 5,298 22,253 22,578 Income (loss) from operations (1,992) 1,062 (1,192) (8,260) Other (expense) income: Interest and other (expense) income, net (17) (150) 503 (761)Gain on PPP loan forgiveness — — — — Gain on equipment sale — — — — Income (loss) before income tax expense (2,008) 912 (689) (9,021) Income tax expense 247 — 247 — Net income (loss) $(2,256) $912 $(936) $(9,021)Net loss per share, basic $(0.09) $0.04 $(0.04) $(0.36)Net loss per share, diluted $(0.09) $0.04 $(0.04) $(0.36) Weighted average number of common shares outstanding, basic 25,741 25,381 25,645 25,146 Weighted average number of common shares outstanding, diluted 25,741 25,691 25,645 25,146 Eton Pharmaceuticals, Inc.BALANCE SHEETS(in thousands, except share and per share amounts) December 31, December 31, 2023 2022 Assets Current assets: Cash and cash equivalents $21,388 $16,305 Accounts receivable, net 3,411 1,852 Inventories 911 557 Prepaid expenses and other current assets 1,129 1,290 Total current assets 26,839 20,004 Property and equipment, net 58 72 Intangible assets, net 4,739 4,754 Operating lease right-of-use assets, net 92 188 Other long-term assets, net 12 12 Total assets $31,740 $25,030 Liabilities and stockholders’ equity Current liabilities: Accounts payable $1,848 $1,766 Current portion of long-term debt 5,380 1,033 Accrued liabilities 9,013 3,662 Total current liabilities 16,241 6,461 Long-term debt, net of discount and including accrued fees — 5,384 Operating lease liabilities, net of current portion 22 107 Total liabilities 16,263 11,952 Commitments and contingencies (Note 14) Stockholders’ equity Common stock, $0.001 par value; 50,000,000 shares authorized; 25,688,062 and 25,353,119 shares issued and outstanding at December 31, 2023 and 2022, respectively 26 25 Additional paid-in capital 119,521 116,187 Accumulated deficit (104,070) (103,134)Total stockholders’ equity 15,477 13,078 Total liabilities and stockholders’ equity $31,740 $25,030 Eton Pharmaceuticals, Inc.STATEMENTS OF CASH FLOWS(In thousands) December 31, December 31, 2023 2022 Cash flows from operating activities Net loss $(936) $(9,021) Adjustments to reconcile net loss to net cash provided by (used in) operating activities: Stock-based compensation 3,137 4,218 Common stock issued for product candidate licensing rights — — Depreciation and amortization 901 1,774 Debt discount amortization 117 127 Gain on forgiveness of PPP loan — — Gain on sale of equipment — — Changes in operating assets and liabilities: Accounts receivable (1,559) 3,619 Inventories (354) (7)Prepaid expenses and other assets 161 1,902 Accounts payable 53 (8)Accrued liabilities 5,295 2,217 Net cash provided by (used in) operating activities 6,815 4,821 Cash used in investing activities Proceeds from sale of equipment — — Purchases of property and equipment — (38)Purchase of product licensing rights (775) (2,750)Net cash used in investing activities (775) (2,788) Cash flows from financing activities Proceeds from issuance of long-term debt, net of issuance costs — — Proceeds from sales of common stock, net of offering costs — — Proceeds from PPP and EIDL loans — — Debt paydown (1,155) (385)Proceeds from employee stock purchase plan and stock option and stock warrant exercises 198 251 Net cash (used in) provided by financing activities (957) (134) Change in cash and cash equivalents 5,083 1,899 Cash and cash equivalents at beginning of year 16,305 14,406 Cash and cash equivalents at end of year $21,388 $16,305 Supplemental disclosures of cash flow information Cash paid for interest $842 $730 Cash paid for income taxes $247 $— Supplemental disclosures of non-cash investing and financing activities: Adjustment of operating lease right-of-use assets and liabilities due to tenant allowance $29 $— Right-of-use assets obtained in exchange for lease liabilities $— $188