Hydrocortisone

« Back to news DEER PARK, Ill., Dec. 17, 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 announced the full readout and compelling results from the clinical trial evaluating PKU GOLIKE as a protein substitute for the treatment of phenylketonuria (PKU) in patients during prolonged fasting periods. The study demonstrated that PKU GOLIKE, administered as the last daily dose and compared to standard amino acid protein substitutes, improved metabolic control by reducing harmful phenylalanine (Phe) levels and increasing beneficial tyrosine (Tyr) levels, both essential for brain function and metabolic health. PKU patients often experience significant fluctuations in blood Phe levels during prolonged fasting periods, particularly at night, when protein breakdown causes Phe concentrations to peak in the early morning. These fluctuations are associated with cognitive difficulties and overall health impacts, making nighttime metabolic control an important focus in PKU management. The study was sponsored by Relief Therapeutics Holding SA, and was a randomized, crossover, controlled clinical study conducted by the Inherited Metabolic Disorders Unit at Birmingham Children’s Hospital, UK, on pediatric patients with classical PKU, the condition’s most severe form. The trial compared PKU GOLIKE to standard amino acid protein substitutes in managing metabolic parameters during overnight fasting, the longest fasting period within 24 hours. At the end of the one-week treatment period, patients receiving PKU GOLIKE as the last daily protein substitute dose showed a statistically significant reduction in blood Phe levels compared to those receiving standard amino acid substitutes (P=0.0002) and a statistically significant increase in blood Tyr levels (P=0.0113). Compared to baseline levels measured prior to the start of treatment, the PKU GOLIKE group achieved an average 17.8% reduction in blood Phe levels (P=0.0484) and an average 33.8% increase in blood Tyr levels (P=0.0008) upon awakening after the overnight fasting period. In comparison, when treated with standard amino acid protein substitutes, the same patients experienced an average 27.6% increase in blood Phe levels (P=0.0063) and no significant improvement in blood Tyr levels. Blood sample analysis at three early morning time points across the two groups revealed no significant differences in peak Phe levels upon reawakening in either group. Highlighting the clinical significance of the findings, Prof. Anita MacDonald, principal investigator and leading dietitian in inherited metabolic disorders at Birmingham Children’s Hospital, stated: “Giving one dose of PKU GOLIKE as the final daily dose of protein substitute resulted in consistently better metabolic control in our cohort of patients with PKU. They all had classical PKU and were a particularly challenging group to control.” These results confirm that PKU GOLIKE’s prolonged-release profile provides clinically and statistically significant improvements in metabolic control during extended fasting periods compared to standard amino acid protein substitutes. Eton expects these findings to support the adoption of PKU GOLIKE among healthcare providers and within the PKU community. The study findings will be presented in a poster titled A Prolonged-Release Formula Has a Positive Impact on Morning Phenylalanine and Tyrosine Fluctuations in Patients with Classical Phenylketonuria at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting, March 18-22, 2025, in Los Angeles, California. Eton promotes PKU GOLIKE with its existing metabolic sales force, which also 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. Patients and healthcare professionals seeking additional information or requesting a product sample can visit pkugolike.com. For more information on this study (NCT05487378), please visit clinicaltrials.gov. 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 etonpharma.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 PLUS® 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 PKU GOLIKE PLUS® granules are 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 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: 212-452-2793 E: lwilson@insitecony.com Source: Eton Pharmaceuticals, Inc. Source: Eton Pharmaceuticals

A rare, inherited endocrine disorder whose standard treatments are steroids that introduce a wide range of side effects now has a new FDA-approved therapy, a non-steroidal Neurocrine Biosciences drug that could give the company another blockbuster seller. The FDA late Friday handed out two approvals for the Neurocrine drug crinecerfont as a treatment for classic congenital adrenal hyperplasia (CAH), a disease that leads to a hormone imbalance that can become fatal. A pill formulation of the San Diego-based biotech’s drug was approved for adults. An oral solution was approved for children age 4 and older. Both formulations will be marketed under the brand name Crenessity. The company claims it’s the first new CAH treatment in 70 years. CAH is an inherited disorder that affects the ability of adrenal glands to produce cortisol, a glucocorticoid hormone that plays a role in tissues and organs throughout the body. Low cortisol levels lead to increased secretion of two other hormones, adrenocorticotropic hormone (ACTH) and androgens. Without treatment, CAH leads to salt wasting (low levels of sodium that affect the brain and kidneys) and dehydration. Extremely low blood levels of cortisol can lead to adrenal crisis, a life-threatening complication. presented by Market Trends to Watch for Health Systems and Their Specialty Pharmacies The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients. By Jake Gaffey, MBA, Chief Strategy Officer at Shields Health Solutions Standard CAH treatment includes synthetic versions of glucocorticoids intended to make up for deficient cortisol. But patients need higher doses than what is normally produced by the body to lower levels of ACTH and adrenal androgens. These supraphysiologic doses bring a wide range of complications associated with steroids, including weight gain, diabetes, cardiovascular disease, and osteoporosis. High GC doses can also have psychological and cognitive impacts. Crenessity is an oral small molecule designed to block the corticotropin-releasing factor type 1 receptor. Doing so inhibits secretion of ACTH from the pituitary gland, which in turn reduces production of adrenal androgens. FDA approval of Crenessity is based on results of a global Phase 3 study in adults and children that showed the drug led to significant glucocorticoid reductions compared to placebo after 24 weeks of treatment. The most common adverse reactions reported in the study included fatigue, headache, dizziness, and muscle and joint pain. Neurocrine said these side effects were temporary and mild to moderate in severity. The FDA approval covers the use of Crenessity as an adjunct to glucocorticoid replacement. For adults, the drug is twice daily pill. For children age 4 and older, dosing of the oral solution is according to patient weight. In a note sent to investors Monday, William Blair analyst Myles Minter said the drug’s label is nonrestrictive, permitting treatment of a broad range of CAH patients whether or not clinicians adjust dosing of glucocorticosteroids in patients receiving the two therapies together. Neurocrine said it would disclose Crenessity’s price when the product becomes available later this week. William Blair projects Crenessity will carry a $1,116 per dose price, which works out to about $264,784 per year —falling within the price range of orphan disease drugs. The new Neurocrine drug could achieve about $150 million in 2025 revenue, the firm projects. Peak global sales could reach $1.47 billion depending on regulatory approvals in other markets. Consumer / Employer Health Executives on Digital Transformation in Healthcare Hear executives from Quantum Health, Surescripts, EY, Clinical Architecture and Personify Health share their views on digital transformation in healthcare. By MedCity News “We see significant potential in the CAH market but acknowledge that for this to be a blockbuster $1 billion-plus commercial opportunity, there will need to be both patient and clinician education that supraphysiological glucocorticoid dosing would no longer be the only way to control androgen levels and prevent adrenal crises,” Minter wrote. “With this in mind, we view education activities and partnering with patient advocacy groups like the CARES Foundation as prudent steps ahead of the launch.” Neurocine expanded in endocrine disorders with the 2022 acquisition of Diurnal Group, a Wales-based company whose commercialized products include CAH drug Efmody, a hydrocortisone drug approved for treating CAH in adults and adolescents. At the time of the deal, Crenessity had reached Phase 3 testing. Neurocrine described the Diurnal acquisition as a way to add clinical development and commercial infrastructure in the U.K. The main revenue driver for Neurocrine is Ingrezza, which in 2017 became the first FDA-approved drug for the chronic movement disorder tardive dyskinesia. In the first nine months of 2024, the company reported $1.69 billion in Ingrezza sales, a 27% increase compared to the same period in the prior year. That drug alone accounts for 99% of Neurocrine product revenue, according to the company’s financial reports. FDA approval of Crenessity comes with a priority review voucher that Neurocrine may apply toward speedier regulatory review of a future rare disease drug. But most companies awarded these vouchers sell them to big pharmaceutical companies. Acadia Pharmaceuticals and PTC Therapeutics both received vouchers along with the recent FDA approvals of their respective rare disease drugs. Each voucher sold for $150 million. Illustration: QAI Publishing/Universal Images Group, via Getty Images