Updated Results Demonstrate Significant DCCR Exposure Response Relationship
Interim Analysis of Open-Label Extension Data Demonstrate Continued Efficacy
Improvements in Body Composition and Metabolic Measures Support Efficacy of DCCR in Treatment of PWS Symptoms
REDWOOD CITY, Calif., Sept. 30, 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 that updated top-line results 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), were highlighted in an oral presentation by Jennifer L. Miller, M.D., Professor in the Division of Pediatric Endocrinology at the University of Florida, at the Foundation for Prader-Willi Research Annual Research Symposium.
DESTINY PWS is a randomized, double-blind, placebo-controlled Phase III study of oncedaily 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 ages four years and older, with genetically-confirmed PWS. Patients who completed the double-blind study enrolled in study C602, an ongoing open-label, extension study.
Soleno previously announced initial top-line results from DESTINY PWS in June 2020. The study did not meet its primary endpoint of change from baseline in hyperphagia, measured by the total score of a Hyperphagia Questionnaire for Clinical Trials (HQ-CT, 036). However, significant changes were observed in two of three key secondary endpoints, improvement in physician assessed Clinical Global Impression of Improvement score and reduction of body fat mass, in subjects receiving DCCR as compared to placebo. An interim analysis of the subset of patients who completed 13 weeks of treatment on C602 showed a continued improvement in hyperphagia, as well as several other PWS related behaviors.
Key updated results:
- A significant exposure response relationship between DCCR plasma concentrations and change from baseline in HQ-CT score was observed.
- Analyses of treatment windows revealed continued efficacy of DCCR over time as compared to worsening effects for placebo.
- While there was a larger decrease from baseline to week 4 in placebo compared to DCCR subjects, from week 4 to the end of the study placebo subjects worsened, while DCCR subjects continued to improve (p=0.03).
- Significant improvements for DCCR compared to placebo were observed in leptin and adiponectin, adipokines that are differentially expressed in obesity and cardiovascular diseases. Leptin levels are elevated in obesity due to increased body fat mass and leptin resistance, while adiponectin levels are downregulated in obesity.
- DCCR led to a decrease in leptin levels, as compared to an increase for placebo (p