Soleno Therapeutics Announces Peer-Reviewed Publication of Data Comparing DCCR Treatment to the Natural History of Prader-Willi Syndrome - Seite 2

“DCCR has the potential to substantially improve the quality of life for individuals with PWS and their families,” said PATH Study Principal Investigators, Jennifer Miller, M.D. and Shawn McCandless, M.D. “These data clearly demonstrate that long-term treatment with DCCR resulted in changes in hyperphagia and other behavioral complications of PWS that are meaningfully improved compared to the natural history of the disease. We believe these results support the significant potential of DCCR in PWS and are eager to offer a much-needed treatment option to patients in need, if approved.”

About the DCCR C601/602 Dataset

C602 an open-label extension study enrolled participants who completed DESTINY PWS (C601), an international, multi-center, randomized, double-blind, placebo-controlled study of DCCR in 127 PWS patients at 29 sites in the U.S. and UK.

About PATH for PWS

The PATH from PWS (PATH) study (https://www.clinicaltrials.gov/study/NCT03718416) is a recently completed study that was concurrently recruited with DESTINY PWS and sponsored by the Foundation for Prader-Willi Research (FPWR). The key objective of PATH is to advance the understanding of the natural history in individuals with PWS, particularly long-term behavioral changes in the syndrome.

About PWS
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 patients 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 morbidities (e.g., obesity, diabetes, cardiovascular disease) and mortality (e.g., stomach rupture, choking, accidental death due to food seeking behavior). In a global survey conducted by the Foundation for Prader-Willi Research, 96.5% of respondents (parent 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.