Somapacitan

June 28, 2024 (GLOBE NEWSWIRE) -- Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for approval of DCCR (diazoxide choline) extended-release tablets for the treatment of Prader-Willi syndrome (PWS) in individuals four years and older who have hyperphagia. “Submission of the DCCR NDA to the FDA marks a significant milestone not only for Soleno, but for people living with PWS,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “This regulatory submission advances us one important step closer to our goal of bringing to market a new therapeutic for individuals with PWS that addresses the life-threatening hyperphagia and other key aspects of this serious and rare condition. We look forward to working with the FDA throughout the review process. We extend sincere gratitude to the team at Soleno, investigators, study site teams, advocacy organizations and most importantly, the individuals with PWS and their families who were instrumental in completing our DCCR development program.” DCCR has Breakthrough and Fast Track Designations in the U.S., as well as Orphan Drug Designation for PWS in the U.S. and E.U. The FDA has 60 days to determine whether the NDA is accepted for review. Soleno has requested Priority Review of the NDA, which would provide a target review period of six months by the FDA after the NDA has been accepted. The Prader-Willi Syndrome Association USA estimates that PWS occurs in one in every 15,000 live births. The hallmark symptom of this disorder is hyperphagia, a chronic and life-threatening feeling of intense, persistent hunger, food pre-occupation, extreme drive to food seek and consume food that severely diminish the quality of life for individuals with PWS and their families. Additional characteristics of PWS include behavioral problems, cognitive disabilities, low muscle tone, short stature (when not treated with growth hormone), the accumulation of excess body fat, developmental delays, and incomplete sexual development. Hyperphagia can lead to significant mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior) and longer term, co-morbidities such as diabetes, obesity, and cardiovascular disease. In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parents and caregivers) rated hyperphagia and 92.9% rated body composition as either the most important or a very important symptom to be relieved by a new medicine. There are currently no approved therapies to treat the hyperphagia/appetite, metabolic, cognitive function, or behavioral aspects of the disorder. DCCR is a novel, proprietary extended-release dosage form containing diazoxide choline, the crystalline salt of diazoxide and is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of individuals in a few rare diseases in neonates, infants, children and adults, but is not approved for use in PWS. Soleno conceived of and established extensive patent protection for the therapeutic use of diazoxide, diazoxide choline and DCCR in individuals with PWS. The DCCR development program is supported by data from five completed Phase 1 clinical studies in healthy volunteers and three completed Phase 2 clinical studies, one of which was in individuals with PWS. In the PWS Phase 3 clinical development program, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and other metabolic parameters. Diazoxide choline has received Orphan Drug Designation for the treatment of PWS in the U.S. and E.U., and Fast Track and Breakthrough Designations in the U.S. Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company recently submitted an NDA to the FDA, supported by its Phase 3 development program, for its lead candidate, DCCR (diazoxide choline) extended-release tablets, a once-daily oral tablet for the treatment of Prader-Willi syndrome (PWS). For more information, please visit www.soleno.life. The content above comes from the network. if any infringement, please contact us to modify.

AUSTIN, Texas, May 20, 2024 (GLOBE NEWSWIRE) -- Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical stage biopharmaceutical company focused on therapeutics for rare diseases, announced today that new analyses of data from its Phase 2 OraGrowtH210 and OraGrowtH212 clinical trials have been accepted for presentation in two posters at the 2024 Annual Meeting of the Endocrine Society (ENDO), to be held in Boston, MA June 1-4, 2024. Poster Session P069 – Monday June 3, 12:00-1:30 PM ET Poster MON-111: Oral LUM-201 Restores Pulsatile Growth Hormone Secretion and Growth Response in Moderate Pediatric Growth Hormone Deficiency (PGHD): Key Discoveries from Phase 2 of OraGrowtH212 Trial (Fernando Cassorla, MD, Chief of Pediatric Endocrinology, University of Chile, et al) Late-Breaking Poster Session P108 – Monday June 3, 12:00-1:30PM ET Poster MON-704: Growth Response to Oral Growth Hormone Secretagogue LUM-201 in Children with Moderate GH Deficiency (GHD) is dependent on the Pattern of Pulsatile GH Secretion Stimulated by LUM-201 (Adam Stevens, MSc, PhD, Lecturer, University of Manchester, et al) About Lumos Pharma Lumos Pharma, Inc. is a clinical stage biopharmaceutical company focused on the development and commercialization of therapeutics for rare diseases. The Company was founded and is led by a management team with longstanding experience in rare disease drug development. Lumos Pharma’s lead therapeutic candidate, LUM-201, is a novel, oral growth hormone (GH) secretagogue, seeking to transform the ~$4.7B global GH market from injectable to oral therapy. LUM-201 is currently being evaluated in multiple Phase 2 clinical studies in Pediatric Growth Hormone Deficiency (PGHD) and has received Orphan Drug Designation in both the US and EU. For more information, please visit . Investor & Media Contact: Lisa Miller Lumos Pharma Investor Relations 512-792-5454 ir@lumos-pharma.com Source: Lumos Pharma, Inc.