Menkes Kinky Hair Syndrome

– Eton expects to significantly expand patient access to this critical treatment for Wilson disease patients – – $0 co-pay for all eligible patients and best-in-class patient support services – – Now available exclusively through Optime Care – DEER PARK, Ill., March 03, 2025 (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 launched Galzin® (zinc acetate) capsules. Galzin is FDA-approved for the maintenance treatment of patients with Wilson disease who have been initially treated with a chelating agent. It is the only FDA-approved zinc therapy for Wilson disease. “We are excited to launch Galzin with our dedicated sales force and robust Eton Cares patient support services. Due to historic availability and affordability challenges, many patients have turned to unapproved compounded products or over-the-counter supplements to manage their Wilson disease. We are dedicated to increasing both access to and awareness of this critical medication. Through Eton Cares, we intend to ensure that every patient that needs Galzin will have access to it,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. “As part of our commitment to the Wilson disease community, Eton is also actively investing in further research and development in an effort to drive meaningful advancements in treatment.” “Galzin is an important medication for Wilson disease patients, and we look forward to partnering with Eton to ensure patients have continued access to this drug,” said Rhonda Rowland, President, Wilson Disease Association. Galzin is now available exclusively through Optime Care, a specialty pharmacy dedicated to helping patients with rare diseases manage their conditions. Optime Care will administer the Eton Cares Program in partnership with Eton Pharmaceuticals. The program will provide prescription fulfillment, insurance benefits investigation, educational support, and qualified patient financial assistance, along with other services designed to help eligible patients access treatment. Eton Cares will offer $0 co-pay for all eligible patients. Clinicians seeking to prescribe Galzin can e-prescribe by selecting Optime Care as the pharmacy or fax in a patient referral form to 866-318-2990. Additional product details can be found on the product website, www.galzin.com. IMPORTANT SAFETY INFORMATION Warning and Precautions  Copper Deficiency: Several postmarketing cases reported that zinc acetate taken over extended periods of time (i.e., months to years) may result in decreased enteral copper absorption and copper deficiency. The cases reported the following complications of copper deficiency: anemia, granulocytopenia, leukopenia, neutropenia, pancytopenia, thrombocytopenia, and myeloneuropathy. If a patient develops signs and/or symptoms of copper deficiency during treatment with zinc acetate, interrupt zinc treatment and measure zinc, 24-hr urinary copper, and non-ceruloplasmin bound copper (NCC) levels. Consider restarting zinc acetate treatment based on periodic monitoring of 24-hr urinary copper and NCC levels. Gastric Ulcer: There have been postmarketing reports of gastric ulcers with long-term use of zinc acetate. The cases reported the complications of anemia and gastric ulcer perforation with peritonitis. In some cases, ulcers persisted after treatment until zinc acetate was discontinued. If a patient develops signs and/or symptoms of gastric ulcer during treatment with zinc acetate, discontinue zinc treatment. Most patients showed improvement after cessation of zinc treatment. General: Zinc acetate is not recommended for the initial therapy of symptomatic patients because of the delay required for zinc-induced increase in enterocytic metallothionein and blockade of copper uptake. Symptomatic patients should be treated initially, using chelating agents. During initial therapy, neurological deterioration may occur as stores of copper are mobilized. Once initial therapy has been completed, and the patient is clinically stable, maintenance treatment with zinc acetate can be considered, but patients may be continued on initial therapy as clinically indicated. Information for Patients: Patients should take GALZIN® on an empty stomach, at least one hour before or two to three hours after meals. Capsules should be swallowed whole, not opened or chewed. In the rare event of gastric intolerance of zinc, generally occurring with the morning dose, this dose may be taken between breakfast and lunch. Patients must be clinically monitored to determine the adequacy of zinc acetate therapy. Since strict adherence to the zinc regimen is essential for optimal control of copper distribution and metabolism, the physician must reinforce the need for compliance at each contact with the patient.  Monitoring Patients: Patients should be monitored primarily by assessment of existing signs and symptoms of Wilson’s disease and 24-hour urine copper. Neuropsychiatric evaluations including speech as well as liver function tests including bilirubin and aminotransferases, should be done as appropriate. Adverse Reactions The following adverse reactions associated with the use of zinc acetate were identified from postmarketing reports. Because these reactions were reported voluntarily from a population of uncertain size, it is not possible to reliably estimate their frequency or establish a causal relationship to drug exposure. Gastrointestinal disorders: gastric irritation Investigations: elevations of serum alkaline phosphatase, amylase, and lipase lasting from weeks to months suggesting pancreatitis; the levels usually return to high normal within the first one or two years of zinc therapy. INDICATION Zinc acetate therapy is indicated for maintenance treatment of patients with Wilson’s disease who have been initially treated with a chelating agent Please see Full Prescribing Information for more information. About Eton Pharmaceuticals Eton is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The Company currently has seven commercial rare disease products: INCRELEX® (mecasermin) injection, ALKINDI SPRINKLE® (hydrocortisone) oral granules, GALZIN® (zinc acetate) capsules, PKU GOLIKE®, Carglumic Acid tablets, Betaine Anhydrous oral solution, and Nitisinone capsules. The Company has four additional product candidates in late-stage development: ET-400, ET-600, Amglidia® (glyburide oral suspension), 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 Source: Eton Pharmaceuticals, Inc. Source: Eton Pharmaceuticals

CUTX-101 is intended to treat Menkes disease. Credit: luchschenF / Shutterstock. The US Food and Drug Administration (FDA) has accepted for filing and priority review of the new drug application (NDA) for Sentynl Therapeutics’ CUTX-101 (copper histidinate) for the treatment of Menkes disease. Sentynl announced the acceptance with Fortress Biotech, with the former taking over complete responsibility for the therapy’s development and commercialisation from Fortress Biotech subsidiary Cyprium Therapeutics as of December 2023. The therapy may be eligible for a rare pediatric disease priority review voucher (PRV) if approved. Cyprium would retain ownership of this voucher, which could be used for a subsequent marketing application or sold or transferred to a third party. Additionally, Cyprium is positioned to obtain royalties and aggregate development and sales milestones of up to $129m from Sentynl. Fortress Biotech chairman, president and CEO and Cyprium Therapeutics chairman Lindsay Rosenwald said: “We congratulate Sentynl on the NDA acceptance for filing and review of CUTX-101, a programme that Cyprium advanced and proudly supports. “The drug has a demonstrated safety and efficacy profile for the treatment of Menkes disease and, if approved, CUTX-101 will fill a significant unmet need for children suffering from this rare, fatal paediatric disease.” The NDA submission for the therapy is backed by positive topline clinical efficacy outcomes, which have shown a statistically ‘significant improvement’ in overall survival (OS) among patients when treated early with the therapy. According to the data, a nearly 80% decrease is observed in the mortality risk when compared to an untreated historical control group. CUTX-101 has received the FDA fast track, rare pediatric disease, orphan drug, and breakthrough designations. The European Medicines Agency (EMA) has also granted it orphan designation. The FDA, under the Prescription Drug User Fee Act (PDUFA), has set a six-month review period with a target action date of 30 June this year. A rare genetic disorder affecting copper levels in the body, Menkes disease is caused by mutations in the ATPase Copper Transporting Alpha (ATP7A) gene.

January 6, 2025 Six-month priority review granted for CUTX-101 copper histidinate with PDUFA target action date set for June 30, 2025 CUTX-101 has potential to be the first FDA-approved treatment for Menkes disease, a rare and fatal pediatric disease Solana Beach, CA, Ahmedabad, India, and Miami, FL – January 6, 2025 — Sentynl Therapeutics, Inc. (“Sentynl”), a U.S.-based biopharmaceutical company wholly-owned by Zydus Lifesciences, Ltd. (“Zydus Group”), and Fortress Biotech, Inc. (Nasdaq: FBIO) (“Fortress”) announced that the U.S. Food and Drug Administration (FDA) has accepted for filing and Priority review Sentynl’s New Drug Application (NDA) for CUTX-101, the product candidate for the treatment of Menkes disease. Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. Recent estimates suggest a prevalence of 1 in 34,810 to as high as 1 in 8,664 live male births. Sentynl’s NDA is supported by positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment with CUTX-101, with a nearly 80% reduction in the risk of death compared to an untreated historical control cohort. Median overall survival was 177.1 months for CUTX-101 early treatment cohort compared to 16.1 months for the untreated historical control cohort. “The NDA acceptance of CUTX-101 marks an important step towards our vision to transform lives and meaningfully impact patients, caregivers, and the rare disease community at large,” said Punit Patel, President and CEO, Zydus Americas. “We remain committed to providing access to path-breaking discoveries that can address unmet needs globally, as evident in the potential impact of CUTX-101 on Menkes disease patients and their families.” “Menkes disease presents a difficult journey for patients and their caregivers, as ATP7A mutations impact the transport of copper to a range of organs and systems, such as the lungs, brain and heart. With no known cure or current FDA-approved treatments, death typically occurs between 2 to 3 years of age,” said Matt Heck, President & Chief Executive Officer of Sentynl. “We are eager for the FDA to review our application for CUTX-101, which has the potential to be the first FDA-approved therapy for this devastating condition.” CUTX-101 was granted Breakthrough Therapy, Fast Track, Rare Pediatric Disease and Orphan Drug Designations by the FDA and Orphan Designation by the European Medicines Agency (EMA). Under the Prescription Drug User Fee Act (PDUFA), the FDA set a six-month period with a target action date of June 30, 2025. In December 2023, Sentynl assumed full responsibility for the development and commercialization of CUTX-101 from Cyprium Therapeutics (“Cyprium”), a Fortress subsidiary company focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. “We congratulate Sentynl on the NDA acceptance for filing and review of CUTX-101, a program that Cyprium advanced and proudly supports,” said Lindsay A. Rosenwald, M.D., Chairman, President and Chief Executive Officer of Fortress and Chairman of Cyprium. “The drug has a demonstrated safety and efficacy profile for the treatment of Menkes disease and, if approved, CUTX-101 will fill a significant unmet need for children suffering from this rare, fatal pediatric disease.” ### About CUTX-101 (Copper Histidinate) CUTX-101 is an investigational drug currently under NDA review with the U.S. FDA to treat patients with Menkes disease. CUTX-101 is a subcutaneous injectable formulation of copper histidinate manufactured under current good manufacturing practice (“cGMP”) that is intended to improve tolerability due to its physiological pH. In a Phase 1/2 clinical trial conducted by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health (“NIH”), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. Cyprium previously reported positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (ET) with CUTX-101, compared to an untreated historical control cohort, with a nearly 80% reduction in the risk of death. Median overall survival (OS) was 177.1 months for CUTX-101 ET cohort compared to 16.1 months for the untreated historical control cohort. CUTX-101 has been granted FDA Breakthrough Therapy, Fast Track, Rare Pediatric Disease and FDA Orphan Drug Designations. Additionally, the European Medicines Agency granted Orphan Designation for CUTX-101. An expanded access protocol for patients with Menkes disease is ongoing at multiple U.S. medical centers. About Menkes Disease Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of the copper transporter ATP7A. The minimum birth prevalence for Menkes disease is believed to be 1 in 34,810 live male births, and potentially as high as 1 in 8,664 live male births, based on recent genome-based ascertainment (Kaler SG, Ferreira CR, Yam LS. Estimated birth prevalence of Menkes disease and ATP7A-related disorders based on the Genome Aggregation Database (gnomAD). Molecular Genetics and Metabolism Reports 2020 June 5;24:100602). The condition is characterized by distinctive clinical features, including sparse and depigmented hair (“kinky hair”), connective tissue problems, and severe neurological symptoms such as seizures, hypotonia, failure to thrive, and neurodevelopmental delays. Mortality is high in untreated Menkes disease, with many patients dying between 2-3 years of age. Milder versions of ATP7A mutations are associated with other conditions, including Occipital Horn Syndrome and ATP7A-related Distal Motor Neuropathy. Currently, there is no FDA-approved treatment for Menkes disease and its variants. About Sentynl Therapeutics Sentynl Therapeutics, Inc. (“Sentynl”) is a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients suffering from rare diseases. The company was acquired by the Zydus Group in 2017. Sentynl’s experienced management team has previously built multiple successful pharmaceutical companies. With a focus on commercialization, Sentynl looks to source effective and well-differentiated products across a broad spectrum of therapeutic areas to address unmet needs. Sentynl is committed to the highest ethical standards and compliance with all applicable laws, regulations and industry guidelines. For more information, visit https://sentynl.com. About Zydus Group Zydus Lifesciences Ltd. with an overarching purpose of empowering people with freedom to live healthier and more fulfilled lives, is an innovative, global lifesciences company that discovers, develops, manufactures, and markets a broad range of healthcare therapies. The group employs over 27,000 people worldwide, including 1,400 scientists engaged in R & D, and is driven by its mission to unlock new possibilities in lifesciences through quality healthcare solutions that impact lives. The group aspires to transform lives through path-breaking discoveries. For more information, visit https://www.zyduslife.com/zyduslife. About Fortress Biotech Fortress Biotech, Inc. (“Fortress”) is an innovative biopharmaceutical company focused on acquiring and advancing assets to enhance long-term value for shareholders through product revenue, equity holdings and dividend and royalty revenue. The company has seven marketed prescription pharmaceutical products and over 20 programs in development at Fortress, at its majority-owned and majority-controlled partners and subsidiaries and at partners and subsidiaries it founded and in which it holds significant minority ownership positions. Such product candidates span six large-market areas, including oncology, rare diseases and gene therapy, which allow it to create value for shareholders. Fortress advances its diversified pipeline through a streamlined operating structure that fosters efficient drug development. The Fortress model is focused on leveraging its significant biopharmaceutical industry expertise and network to further expand the company’s portfolio of product opportunities. Fortress has established partnerships with some of the world’s leading academic research institutions and biopharmaceutical companies to maximize each opportunity to its full potential, including AstraZeneca, City of Hope, Fred Hutchinson Cancer Center, Nationwide Children’s Hospital and Sentynl. For more information, visit www.fortressbiotech.com. Contacts: Media: Andrea Moody, andrea.moody@fleishman.com Sentynl: Michael Hercz, ir@sentynl.com < Back This website uses cookies for data analytics. This website may also use cookies to enhance your experience with the website. We do not use cookies to sell or share your personal information. By selecting “Accept all,” you agree to our use of cookies. 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