Positive statistically significant top-line results from randomised withdrawal period of study C602 of DCCR for Prader-Willi syndrome.

“We are delighted with the highly statistically significant results from the randomized withdrawal phase of Study C602,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno. “These results will support our planned submission of a New Drug Application (NDA) to the FDA mid-year of next year. We would like to thank the patients, families, investigators, study site personnel and the advocacy community involved in this study, as well as the entire Soleno team for their support of the DCCR development program. We remain committed to the goal of delivering DCCR, if approved, as an effective and safe therapy to individuals with PWS as expeditiously as possible.”

The FDA previously acknowledged that data from this study have the potential to support an NDA submission for DCCR, which has Orphan Drug designation for the treatment of PWS in the U.S. and E.U. and Fast Track designation from the FDA.

C602 Randomized Withdrawal Period Design: The multi-center, randomized, double-blind, placebo-controlled randomized withdrawal period enrolled 77 patients previously enrolled in Study C602 who had been on open-label treatment with DCCR for between two and four years. Participants were randomized 1:1 to receive either DCCR (n=38) or placebo (n=39) for a period of four months. The primary endpoint was the change from baseline in hyperphagia-related behaviors as assessed by the hyperphagia questionnaire for clinical trials (HQ-CT), a caregiver-completed nine item validated questionnaire for assessing hyperphagia in PWS. Secondary endpoints included investigator assessments of participants’ overall severity of illness and change in condition, as measured by Clinical Global Impression of Severity (CGI-S) and Clinical Global Impression of Improvement (CGI-I) ratings, respectively.

Key Top-line Results: i. Hyperphagia-related behaviors markedly worsened in the placebo group compared to DCCR, represented by a highly statistically significant, clinically meaningful difference in mean change from baseline in the HQ-CT total score of 5.0 at week 16 (p=0.0022). ii. Secondary endpoints of CGI-S and CGI-I both showed strong trends towards worsening in the placebo group compared to DCCR over the course of the randomized withdrawal period (p=0.08 and 0.09), respectively. iii. DCCR continued to be generally well-tolerated in the randomized withdrawal period with no new or unexpected safety signals, including no serious adverse events or discontinuations due to adverse events occurring in any participants in the DCCR group.