Soleno Therapeutics Publishes Peer-Reviewed Analysis: DCCR's Impact on Prader-Willi Syndrome Compared to Natural History

A recent study has shed light on the effects of Diazoxide Choline (DCCR) Extended-Release tablets on patients suffering from Prader-Willi Syndrome (PWS). The research was conducted by Soleno Therapeutics, a clinical-stage biopharmaceutical firm, and involved a comparison between the outcomes of their Phase 3 study and the PATH for PWS natural history study. The findings were published in the Journal of Neurodevelopmental Disorders.

The Phase 3 study, known as DESTINY PWS (C601), was a randomized, double-blind, placebo-controlled international trial that included 127 PWS patients across 29 sites in the U.S. and UK. Following this, an open-label extension study (C602) was initiated, enrolling participants from the C601 study. The aim was to evaluate the long-term effects of DCCR on hyperphagia and PWS-related behaviors.

Hyperphagia, a chronic and intense hunger that characterizes PWS, was significantly reduced in participants treated with DCCR. This improvement was clinically meaningful and statistically significant at both 6 and 12 months post-treatment. Similarly, across all domains of the Prader-Willi Syndrome Profile (PWSP), which measures aggressive behaviors, anxiety, compulsivity, depression, disordered thinking, and rigidity-irritability, significant improvements were observed in DCCR-treated participants when compared to the PATH study group.

Dr. Theresa Strong, Director of Research Programs for FPWR, emphasized the importance of the PATH study in understanding the natural progression of PWS. She highlighted that the improvements observed with DCCR treatment are substantial and could greatly enhance the quality of life for PWS patients and their families.

PATH for PWS is a study that was conducted concurrently with DESTINY PWS and sponsored by the Foundation for Prader-Willi Research (FPWR). Its primary goal is to deepen the understanding of PWS's natural history, especially the long-term behavioral changes associated with the syndrome.

PWS is a rare genetic disorder estimated to occur in one of every 15,000 live births. The condition is marked by hyperphagia, which can lead to severe health complications such as obesity, diabetes, and cardiovascular disease. Additional symptoms include behavioral issues, cognitive impairments, and developmental delays. Currently, there are no approved treatments for the hyperphagia, metabolic issues, cognitive function, or behavioral aspects of PWS.

DCCR is a novel, once-daily oral tablet developed by Soleno Therapeutics. It contains a crystalline salt of diazoxide and has been granted Orphan Drug Designation for the treatment of PWS in the U.S. and E.U., along with Fast Track and Breakthrough Designations in the U.S.

Soleno Therapeutics is dedicated to developing and commercializing innovative treatments for rare diseases, with DCCR being their lead candidate for PWS. The company has recently completed the Phase 3 development program for DCCR and is preparing for a New Drug Application (NDA) submission.

The study's results are promising, suggesting that DCCR has the potential to significantly improve the lives of those living with PWS. The company looks forward to advancing through the regulatory process and potentially offering a much-needed treatment option for this rare and challenging condition.