Protein Deficiency

PKU GOLIKE® is a commercial ultra-rare disease medical formula complementary to Eton's existing metabolic franchise and specialty call pointTransaction is expected to be accretive to 2024 earningsEstimated peak sales of more than $10 million annuallyU.S. phenylketonuria ("PKU") medical formula market estimated to be $100 million annually DEER PARK, Ill., March 22, 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 announced that it has acquired U.S. rights to PKU GOLIKE® from RELIEF THERAPEUTICS Holding SA (“Relief”) (SIX: RLF, OTCQB: RLFTF, RLFTY). “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. “We are very pleased to gain this partnership with Eton for PKU GOLIKE patients in the US as it is aligned with RELIEF’s strategy to enhance patient access to our innovative products,” said Michelle Lock, interim CEO of Relief. “Eton brings valuable know-how in the metabolic space as well as commercialization expertise for products in rare diseases.” PKU is a rare inherited disorder caused by a defect in the enzyme needed to break down phenylalanine, leading to a toxic buildup of phenylalanine when eating foods that contain proteins. Treatment of PKU requires patients to follow a strict diet that severely limits phenylalanine content and typically requires low protein foods supplemented by phenylalanine-free medical formulas. Excessive levels of phenylalanine in the blood accumulate in the brain and inhibit proper brain development. It is estimated that 8,000 PKU patients in the U.S. utilize medical formulas to manage their diet. PKU GOLIKE® is a next generation medical formula product engineered with the patent protected, pharmaceutical grade Physiomimic™ technology for the dietary management of PKU under medical supervision. PKU GOLIKE’s taste-masked, odor-free coating technology is designed to provide a better taste and a superior experience compared to alternative PKU medical formulas. In addition, PKU GOLIKE’s delayed amino acid release formulation is designed to keep patients full for a longer period of time. Relief launched PKU GOLIKE Granules in the United States in the fourth quarter of 2022 and the PKU GOLIKE Tropical Bar in the second quarter of 2023. Only a few months into the product’s launch, PKU GOLIKE’s fourth quarter net sales in the U.S. were at an annual run rate of more than $1 million. Relief, through its subsidiary APR, has commercialized PKU GOLIKE in Europe since 2019 and seen strong adoption. Eton plans to promote PKU GOLIKE with its existing metabolic sales force, which currently promotes Eton’s Carglumic Acid, Betaine, and Nitisinone products. PKU patients’ care is typically overseen by metabolic geneticists and their support staff of nurse practitioners and registered dieticians. Medical formulas for PKU are frequently covered by insurance and are regulated by the FDA as medical food products. PKU GOLIKE is exclusively distributed in the United States by Pentec Health. Patients and healthcare professionals seeking additional information on the product can visit www.PKUGOLIKE.com. The transaction is expected to be accretive to Eton’s 2024 earnings and the company expects peak sales of more than $10 million annually. As part of the transaction, Eton also received U.S. rights to Relief’s GOLIKE medical formulas line extensions under development for the management of the metabolic conditions tyrosinemia and homocystinuria, which are expected to launch in 2025 and 2026, respectively. Eton has also been granted a right of first negotiation for Relief’s RLF-OD032 development candidate. RLF-OD032 is an innovative drug product candidate under development for the treatment of PKU and is expected to be filed with the FDA in the second half of 2025 as a 505(b)(2) New Drug Application. Relief will continue to own PKU GOLIKE rights outside 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 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. About RELIEF THERAPEUTICS Holding SA 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, 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, visit www.relieftherapeutics.com. 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 formulas as part of an effort to prevent protein deficiency and optimize metabolic control. Medical formulas used in PKU are challenged to provide a range of amino acids slowly and without a medicinal aftertaste. About PKU GOLIKE PKU GOLIKE® products are foods for special medical purposes (FSMPs) for the dietary management of PKU in both children and adults for use under medical supervision. Developed with Relief’s proprietary, patent-protected Physiomimic Technology™ drug delivery platform, PKU GOLIKE® products are the first prolonged-release amino acid FSMPs, 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® products contain all 19 amino acids that people with PKU need to maintain neurological and muscular health and is fortified with 27 essential vitamins and minerals, including ones normally found in protein-rich foods like iron, calcium and vitamin B12. The PKU GOLIKE® line of products are available in convenient packets (PKU GOLIKE Plus® 3-16 and 16+) and medical formula bars (PKU GOLIKE BAR®). PKU GOLIKE® products have been commercially available in the U.S. since October 2022. For more information, visit www.pkugolike.com (Please note this site is intended for U.S. audiences only). 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

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

– Helps to detect circulating tumor DNA (ctDNA) as a biomarker in clinical research and clinical trials for solid tumor malignancies – – May provide real-time data on therapy response, support patient prognostic stratification and enable early detection of residual disease – SAN FRANCISCO, Nov. 6, 2023 /PRNewswire/ -- Invitae (NYSE: NVTA), a leading medical genetics company, today announced enhanced chemistry of its Invitae Personalized Cancer Monitoring™ platform that helps detect circulating tumor DNA (ctDNA) as a biomarker for solid tumor malignancies. ctDNA can provide real-time insights on patient response or disease progression, support patient prognostic stratification and enable early detection of residual disease. The enhanced assay, which received Clinical Laboratory Improvement Amendments (CLIA) approval, features a flexible patient-specific panel design of between 18 and 50 variants, demonstrating a sensitivity of >99% for detecting ctDNA at variant allele frequencies as low as 0.005%. In addition, the company has also received CLIA approval on the whole exome sequencing component of PCM as a stand-alone comprehensive genomic profiling (CGP) assay to permit reporting of tumor profiles. In our commitment to continual innovation, the enhanced assay achieves a number of benefits. Compared to our prior version, the enhanced assay permits a new lower mass input limit that results in the same sensitivity as the prior version with less cell-free DNA (cfDNA), enabling Invitae to test samples that might have been previously rejected. The enhanced assay also involves fewer steps that will increase capacity and reduce the burden of materials and labor costs. Lastly, the enhanced chemistry is automation friendly enabling scale and supporting future growth. "This focused launch and enhanced chemistry will enable biopharma partners to detect recurring disease early," said Bob Daber, Ph.D., chief scientific officer of Invitae. "This will potentially help set MRD as a standard practice in clinical studies and increase the velocity of drug development. Our vision is that insights gained from Invitae Personalized Cancer Monitoring will lead to research breakthroughs that positively influence patient journeys." "With Invitae Personalized Cancer Monitoring, biopharma researchers will have information they need to inform clinical trial design and study patient outcomes, ultimately to help bring new cancer therapies that benefit patients to market," added Dr. Daber. To learn about Invitae Personalized Cancer Monitoring, please contact [email protected]. About Invitae Personalized Cancer Monitoring™ Invitae Personalized Cancer Monitoring is a multi-cancer, tumor-informed, long-term monitoring assay that incorporates next-generation sequencing libraries featuring both unique molecular identifiers (UMIs) and updated anchored multiplex PCR (AMP) chemistry. Invitae Personalized Cancer Monitoring provides personalized ctDNA detection and quantitation from the peripheral blood in patients with solid tumors. Biopharma researchers can utilize the validated Invitae Personalized Cancer Monitoring platform to detect residual cancer at the molecular level. By assessing the presence and levels of ctDNA over time, Invitae Personalized Cancer Monitoring provides a highly sensitive approach to detect residual cancer in patients who have undergone surgery and other definitive therapies, thus informing further diagnostic and therapeutic decisions, leading to potentially improved outcomes. The assay can also provide molecular information on the extent of response to therapeutic interventions, in various clinical settings, which may assist in optimizing clinical studies and accelerating drug development. Currently established clinical practice involves utilizing imaging as a means to detect residual disease and tumor changes, which might result in delayed detection of disease recurrence and inaccurate assessment of therapeutic response. Biopharma researchers need a faster and more reliable way to understand therapeutic responses to novel drug regimens. The Invitae Personalized Cancer Monitoring assay may provide such information. For example, in a study of non-small cell lung cancer, a ctDNA assay based on the Invitae Personalized Cancer Monitoring platform detected relapse prior to standard clinical diagnosis via imaging by almost four months. About Invitae Invitae (NYSE: NVTA) is a leading medical genetics company trusted by millions of patients and their providers to deliver timely genetic information using digital technology. We aim to provide accurate and actionable answers to strengthen medical decision-making for individuals and their families. Invitae's genetics experts apply a rigorous approach to data and research, serving as the foundation of their mission to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. To learn more, visit invitae.com and follow for updates on Twitter, Instagram, Facebook and LinkedIn @Invitae. Safe Harbor Statement This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to the potential benefits of the enhanced chemistry of the Invitae Personalized Cancer MonitoringTM test; that the company's focused launch and enhanced chemistry will enable biopharma partners to detect recurring disease early, potentially help set MRD as a standard practice in clinical studies and increase the velocity of drug development; and the company's vision that insights gained from its test will lead to research breakthroughs that positively influence patient journeys. Forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially, and reported results should not be considered as an indication of future performance. These risks and uncertainties include, but are not limited to: the company's ability to grow its business in a cost-efficient manner; the company's history of losses; the company's ability to maintain important customer relationships; the company's ability to compete; the company's need to scale its infrastructure in advance of demand for its tests and to increase demand for its tests; the risk that the company may not obtain or maintain sufficient levels of reimbursement for its tests; the applicability of clinical results to actual outcomes; risks associated with litigation; the company's ability to use rapidly changing genetic data to interpret test results accurately and consistently; security breaches, loss of data and other disruptions; laws and regulations applicable to the company's business; and the other risks set forth in the company's filings with the Securities and Exchange Commission, including the risks set forth in the company's Quarterly Report on Form 10-Q for the quarter ended June 30, 2023. These forward-looking statements speak only as of the date hereof, and Invitae Corporation disclaims any obligation to update these forward-looking statements. Invitae PR contact: Renee Kelley [email protected] SOURCE Invitae Corporation