Soleno Therapeutics Announces Presentation of Body Composition Results from DESTINY PWS, a Phase III Trial of DCCR in Prader-Willi Syndrome
REDWOOD CITY, Calif., Nov. 03, 2020 (GLOBE NEWSWIRE) — Soleno Therapeutics, Inc. (“Soleno”) (NASDAQ: SLNO), a clinical-stage biopharmaceutical company developing novel therapeutics for the treatment of rare diseases, today announced presentation of body composition data from the Company’s Phase III trial, DESTINY PWS (C601), evaluating once-daily Diazoxide Choline Controlled Release (DCCR) tablets for patients with Prader-Willi Syndrome (PWS). The results were presented today by Parisa Salehi, M.D., Clinical Director of the Prader-Willi Syndrome Clinic at Seattle Children’s Hospital, in a late-breaking oral presentation at The Obesity Society’s ObesityWeek® 2020 meeting. Dr. Salehi presented the data on behalf of the DESTINY PWS Investigators.
DESTINY PWS is a randomized, double-blind, placebo-controlled Phase III study of once daily oral administration of DCCR in 127 PWS patients conducted at 29 sites in the U.S. and U.K. The objective of the study was to assess the efficacy and safety of DCCR in subjects with genetically-confirmed PWS aged four years and older and weighing between 20 and 134 kg. Patients who completed the double-blind study enrolled in study C602, an ongoing open-label, extension study. Updated top-line results were previously announced in September 2020 that demonstrated DCCR’s beneficial impact on hyperphagia, the predominant symptom of PWS, other behaviors and body composition abnormalities typical of PWS.
Key results for effects on body composition and adipokines following DCCR treatment were presented at ObesityWeek 2020 as follows:
Fat mass and body mass:
Significant reductions in fat mass (p=0.0027) were observed after 13 weeks of double-blind treatment
– Greater mean reductions in fat mass were observed in those participants weighing more than 100 kg at baseline (placebo-adjusted difference -4.82 kg)
Linear exposure-response relationship for fat mass was significant with greater fat loss occurring at higher circulating drug concentrations (p<0.0001)
Trend towards increased lean body mass for DCCR compared to placebo (p=0.058)
Significant increase in lean body mass/fat mass ratio (p=0.001)
Significant reduction in leptin while it increased in placebo-treated subjects (p<0.0001)
Linear exposure-response relationship for leptin was significant with greater reductions in leptin occurring at higher circulating drug concentrations (p<0.0001)
Reductions in leptin were greater than would be predicted solely by the loss of body fat, suggesting an improvement in leptin sensitivity
Adiponectin, a cardioprotective hormone, increased in DCCR-treated subjects, but decreased in placebo-treated participants (p<0.0001)
“We are pleased to present further results from the DESTINY PWS study that demonstrate DCCR’s effect on body composition. In the DCCR group compared to placebo, we have observed a significant reduction in fat mass and leptin. Improvements in leptin sensitivity, as suggested by the results, may have an important impact on regulating hyperphagia,” said Dr. Salehi. “The data presented to date show that DCCR has the potential to manage both behavioral and co-morbid metabolic components of PWS.”
“These data represent additional means by which DCCR may address the significant unmet medical needs and the life-threatening comorbidities associated with PWS,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics. “We remain focused on advancing DCCR as a potential treatment