Phase III C601 and C602 trials show DCCR demonstrates positive impact in Prader-Willi syndrome.- Soleno Therapeutics
Soleno Therapeutics presented new positive clinical data from its ongoing late-stage DCCR development program for the treatment of Prader-Willi syndrome (PWS) at ENDO 2022. An oral presentation titled, “Hormonal and Cardiometabolic Changes Associated with Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome,” presented by Dr. Eric Felner, Emory University School of Medicine, summarized data demonstrating positive hormonal, cardiometabolic and body composition changes in PWS patients following one year of treatment with DCCR.
The patients were treated in C601, a Phase III randomized, double-blind, placebo-controlled study and C602, its ongoing open-label extension.
Patients (n=82) experienced improvements in body composition, as evidenced by statistically significant changes in lean body mass (p<0.0001) and the ratio of lean body mass to fat mass (p="0.0005)," based on dxa scanning. these changes were accompanied by reduced levels of leptin (p><0.0001), fasting insulin (p="0.0004)" and an improvement in insulin sensitivity (homa ir p="0.0033)," likely related to a combination of reduced fat mass and improved leptin resistance. a statistically significant increase of adiponectin (p><0.0001), a cardioprotective marker, was also observed. similar effects were observed in obese participants (n="40)" enrolled in these studies. in addition a poster, titled, “long-term safety of diazoxide choline extended-release (dccr) tablets in patients with prader-willi syndrome,” highlighted the ongoing safety of dccr, with the most common adverse events (hypertrichosis, hyperglycaemia, and peripheral edema) being as expected based on prior experience with dccr.></0.0001),></0.0001),></0.0001)>
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