Soleno to Present on DCCR Tablets for Prader-Willi at ENDO 2024

Soleno Therapeutics, Inc. (NASDAQ: SLNO), a clinical-stage biopharmaceutical enterprise dedicated to creating innovative treatments for rare diseases, has announced that findings from the randomized withdrawal phase of Study C602 on DCCR (diazoxide choline) extended-release tablets for Prader-Willi Syndrome (PWS) will be presented at the Annual Meeting of the Endocrine Society (ENDO 2024), from June 1-4, 2024, in Boston, Massachusetts. The oral presentation is scheduled for June 2, 2024, between 2:45 – 3:00 pm ET, and will be delivered by Evelien Gevers, M.D., Ph.D., of Barts Health NHS Trust and Queen Mary University London.

Prader-Willi Syndrome is a rare genetic disorder occurring in approximately one in every 15,000 live births in the United States, according to the Prader-Willi Syndrome Association USA. The primary symptom of PWS is hyperphagia—an insatiable and life-threatening hunger that leads to a constant preoccupation with food. This symptom severely impacts the quality of life for individuals with PWS and their families. Other characteristics of PWS include behavioral issues, cognitive impairments, low muscle tone, short stature (without growth hormone treatment), excessive body fat, developmental delays, and incomplete sexual development. Hyperphagia can cause severe health complications such as obesity, diabetes, cardiovascular diseases, and even mortality due to stomach rupture or choking from food-seeking behaviors.

A global survey by the Foundation for Prader-Willi Research revealed that 96.5% of parents and caregivers rated hyperphagia, and 92.9% rated body composition issues as the most crucial symptoms needing treatment in PWS patients. Currently, there are no approved therapies that effectively address the hyperphagia, metabolic dysfunction, cognitive deficits, or behavioral challenges associated with PWS.

DCCR is an innovative extended-release formulation containing the crystalline salt of diazoxide, designed for once-daily oral administration. Diazoxide has historically been used in treating rare diseases across several age groups but has not yet received approval for PWS. Soleno Therapeutics has developed and secured extensive patent protection for the use of diazoxide, diazoxide choline, and DCCR in treating PWS. The development program for DCCR is bolstered by data from five Phase 1 clinical studies in healthy volunteers and three Phase 2 clinical studies, including one focusing on PWS patients. The Phase 3 clinical program demonstrated that DCCR could significantly address hyperphagia, the primary symptom of PWS, and improve other symptoms such as aggressive and destructive behaviors, fat mass, and metabolic parameters. DCCR has received Orphan Drug Designation for PWS treatment in both the U.S. and E.U., alongside Fast Track and Breakthrough Designations in the U.S.

Soleno Therapeutics remains committed to advancing and commercializing novel therapeutics for rare diseases. Their lead candidate, DCCR (diazoxide choline) extended-release tablets, is positioned as a promising once-daily oral treatment for PWS, having recently completed its Phase 3 clinical development program. The company plans to submit a New Drug Application (NDA) to further progress its approval and availability for PWS patients.

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