Hyperhomocysteinemia

Posters to be presented highlighting research investigating pegtibatinase as the first potential disease-modifying treatment for classical homocystinuriaSAN DIEGO, April 04, 2024 (GLOBE NEWSWIRE) -- Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced that the Company will present eight posters in classical homocystinuria (HCU) at Society for Inherited Metabolic Disorders (SIMD) in Charlotte, North Carolina from April 14-17, 2024, and Genetic Metabolic Dieticians International (GMDI) in Charlotte, North Carolina from April 17-20, 2024. At SIMD and GMDI, the Company will present the trial design of the pivotal Phase 3 HARMONY Study of pegtibatinase, a novel investigational enzyme replacement therapy for the treatment of classical HCU. Additionally, the Company will share insights on the development of an innovative tool used for dietary management and monitoring in the Phase 3 HARMONY Study and open-label extension ENSEMBLE Study, and the positive results from cohort 6 in the placebo-controlled Phase 1/2 COMPOSE Study of pegtibatinase in classical HCU. “Classical HCU is an isolating and devastating rare disorder with very limited treatment options, including adherence to highly restrictive diets, leaving patients and their caregivers with immense challenges,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. “We look forward to participating at both of these prestigious metabolic medical meetings, and we are excited about our work pioneering the first potential disease-modifying treatment for the thousands of people affected by classical HCU.” SIMD Poster Presentations Latest Results From the COMPOSE Phase 1/2 Trial For the Treatment of Classical Homocystinuria (HCU) Using Pegtibatinase, a Novel Investigational Enzyme Replacement TherapyPoster: 139Abstract Category: Innovative TherapiesSymphony Ballrooms 4-7; April 15, 2024, 8-9 p.m. ET Pegtibatinase, an Investigational Enzyme Replacement Therapy, For The Treatment of Classical Homocystinuria (HCU): Design of the HARMONY Phase 3 Study Poster: 147Abstract Category: Innovative Therapies Symphony Ballrooms 4-7; April 15, 2024, 8-9 p.m. ET Economic Burden of Classical Homocystinuria in the United StatesPoster: 48Abstract Category: Clinical Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET Clinical Burden of Classical Homocystinuria in the United States: A Retrospective Claims Analysis Poster: 64Abstract Category: Clinical Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET Association Between Homocysteine and Clinical Outcomes in Patients with Classical Homocystinuria: A Systematic Literature ReviewPoster: 68Abstract Category: Clinical Care/ResearchSymphony Ballrooms 4-7; April 15, 2024, 7-8 p.m. ET GMDI Poster Presentations Latest Results from the COMPOSE Phase 1/2 Trial of Pegtibatinase, a Novel Investigational Enzyme Replacement Therapy for Classical Homocystinuria (HCU) Poster: 10April 18, 2024, 4:30-5:15 p.m. ET Novel Tool for Dietary Management and Monitoring in Clinical Trials of Pegtibatinase, an Investigational Enzyme Replacement Therapy for Classical Homocystinuria Poster: 16April 18, 2024, 4:30-5:15 p.m. ET Novel Dietary Management Strategies for Classical Homocystinuria (HCU) in HARMONY/ENSEMBLE Phase 3 Studies of Pegtibatinase, an Investigational Enzyme Replacement Therapy Poster: 33April 18, 2024, 5:15-6 p.m. ET About Classical Homocystinuria Classical homocystinuria (HCU) is a rare genetic metabolic disorder caused by a deficiency in the enzyme cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is essential for the management of methionine and cysteine in the body. Classical HCU leads to toxic levels of homocysteine that can result in life-threatening thrombotic events such as stroke, pulmonary embolism and deep vein thrombosis, ophthalmologic and skeletal complications, as well as developmental delay. Current treatment options are limited to protein-restricted diet and use of vitamin B6 and betaine. About Pegtibatinase Pegtibatinase is an investigational PEGylated, recombinant enzyme replacement therapy designed to address the underlying cause of classical homocystinuria (HCU). In preclinical studies, pegtibatinase has demonstrated an ability to reduce total homocysteine levels and improve clinical parameters. Pegtibatinase is currently advancing in the ongoing Phase 1/2 COMPOSE Study to assess its safety, tolerability, pharmacokinetics, pharmacodynamics and clinical effects in patients with classical HCU. In May 2023, the Company announced that data from four patients treated with the highest dose of pegtibatinase showed a clinically meaningful 67.1% mean relative reduction in total homocysteine from baseline and was generally well-tolerated after 12 weeks of treatment. To date, the pegtibatinase program has been granted Breakthrough Therapy designation, Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designation in the U.S. and Europe. About Travere Therapeutics At Travere Therapeutics, we are in rare for life. We are a biopharmaceutical company that comes together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. On this path, we know the need for treatment options is urgent – that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. In pursuit of this mission, we continuously seek to understand the diverse perspectives of rare patients and to courageously forge new paths to make a difference in their lives and provide hope – today and tomorrow. For more information, visit travere.com Forward Looking Statements This press release contains “forward-looking statements” as that term is defined in the Private Securities Litigation Reform Act of 1995. Without limiting the foregoing, these statements are often identified by the words “anticipate,” “believe,” “expect,” “intend,” “may,” “might,” “objective,” “plan,” “will” or similar expressions. In addition, expressions of our strategies, intentions or plans are also forward-looking statements. Such forward-looking statements include, but are not limited to, statements regarding pegtibatinase as the first potential disease-modifying treatment for classical homocystinuria, references to the HARMONY study and the other studies described herein, including statements regarding expectations related thereto, and statements regarding the development of an innovative tool used for dietary management and monitoring. Such forward-looking statements are based on current expectations and involve inherent risks and uncertainties, including factors that could delay, divert or change any of them, and could cause actual outcomes and results to differ materially from current expectations. No forward-looking statement can be guaranteed. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are risks related to the timing and outcome of the HARMONY study and the other studies described herein; risks and uncertainties associated with the regulatory review and approval process, risks associated with enrollment of clinical trials for rare diseases, and risks that ongoing or planned clinical trials may not succeed or may be delayed for safety, regulatory or other reasons. The Company faces risks related to its business and finances in general; risks related to the commercial launch of a new product; risks associated with market acceptance of FILSPARI and other current and future products, including efficacy, safety, price, reimbursement and benefit over competing therapies; and the risk that the Company’s clinical candidates will not be found to be safe or effective and that current or anticipated future clinical trials will not proceed as planned. Specifically, the Company faces risks related to the timing and potential outcome of the FDA’s potential acceptance for filing and review of the sNDA submission for full approval of FILSPARI in IgAN, and the timing and potential outcome of the European Commission’s decision regarding conditional marketing authorization of sparsentan for IgAN. There is no guarantee that the FDA will accept the sNDA submission for filing, that the European Commission will grant conditional marketing authorization of sparsentan for IgAN, or that regulators will grant full approval of sparsentan for IgAN or FSGS. The Company also faces the risk that it will be unable to raise additional funding that may be required to complete development of any or all of its product candidates, including as a result of macroeconomic conditions; risks relating to the Company's dependence on contractors for clinical drug supply and commercial manufacturing; uncertainties relating to patent protection and exclusivity periods and intellectual property rights of third parties; risks associated with regulatory interactions; and risks and uncertainties relating to competitive products, including current and potential future generic competition with certain of the Company's products, and technological changes that may limit demand for the Company's products. The Company also faces additional risks associated with global and macroeconomic conditions, including health epidemics and pandemics, including risks related to potential disruptions to clinical trials, commercialization activity, supply chain, and manufacturing operations. You are cautioned not to place undue reliance on these forward-looking statements as there are important factors that could cause actual results to differ materially from those in forward-looking statements, many of which are beyond our control. The Company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events, or otherwise. Investors are referred to the full discussion of risks and uncertainties, including under the heading “Risk Factors”, as included in the Company’s most recent Form 10-K, Form 10-Q and other filings with the Securities and Exchange Commission. Contact Info Media:888-969-7879 mediarelations@travere.comInvestors:888-969-7879 IR@travere.com

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