Soleno therapeutics recently completed enrollment of their ongoing Phase III Clinical trial, DESTINY PWS, and is expecting to announce top-line data the first half of 2020. The study is evaluating once-daily Diazoxide Choline Controlled-Release (DCCR) tablets for patients with Prader Willi Syndrome (PWS).
"We remain on track to announce top-line data during the first half of this year and would like to thank the patients, families and investigators involved in this study, as well as the Foundation for Prader-Willi Research (FPWR) and Prader-Willi Syndrome Association (PWSA) USA and UK for their support of DESTINY PWS,” says Anish Bhatnagar, M.D., Chief Executive Officer of Soleno Therapeutics.
127 participants are enrolled across 29 sites in the US and UK. This final sample size is powered to detect a difference in change from baseline in HQ-CT score between DCCR and placebo of 4 points. The HQ-CT is a validated questionnaire to measure hyperphagia, the primary outcome measure for the Phase III study. The range of scores is 0-36, with scores above 13 typically considered to be moderate to severe hyperphagia.
There is good news for participants currently enrolled in the study: the open-label extension has been increased from 12 months to up to 36 months. To date, all participants who have completed 12 months of open-label treatment have elected to continue to receive DCCR.
No new safety signals associated with DCCR have been identified to date and no serious, unexpected adverse events related to DCCR have been reported.
About the DESTINY PWS Trial
DESTINY PWS is a randomized, double-blind, placebo-controlled study of once-daily oral administration of DCCR versus placebo in 127 randomized subjects. Patients who complete DESTINY PWS have the option to enroll into an open-label extension study (C602) and continue treatment with DCCR. For further information about DESTINY PWS (NCT03440814), please visit: www.clinicaltrials.gov.
About Diazoxide Choline Controlled-Release (DCCR) Tablet
Diazoxide Choline Controlled-Release tablet is a novel, proprietary extended-release, crystalline salt formulation of diazoxide, which is administered once-daily. The parent molecule, diazoxide, has been used for decades in thousands of patients in a few rare diseases in neonates, infants, children and adults, but has not been approved for use in PWS. Soleno conceived of and established extensive patent protection on the therapeutic use of diazoxide and DCCR in patients with PWS. The DCCR development program is supported by data from five completed Phase I clinical studies in healthy volunteers and three completed Phase II clinical studies, one of which was in PWS patients. In the PWS Phase II study, DCCR showed promise in addressing hyperphagia, the hallmark symptom of PWS, as well as several other symptoms such as aggressive/destructive behaviors, fat mass and abnormal lipid profiles.
