A Phase 3 trial of Levo Therapeutics’ LV-101 intranasal carbetocin in children with Prader-Willi Syndrome (PWS) failed to meet its primary endpoint, the company said. Enrollment in the study, which began in 2018, was stopped earlier than anticipated due to the COVID-19 pandemic, winding up with 119 patients in the primary analysis set.
The CARE-PWS trial compared two doses of LV-101 (9.6 mg and 3.2 mg) to placebo over 8 weeks. Neither dose resulted in statistically significant change in obsessive-compulsive behavior as measured by the Children’s Yale-Brown Obsessive Compulsive Scale, and only the lower dose demonstrated a statistically significant effect on extreme hunger as measured by the Hyperphagia Questionnaire for Clinical Trials.
According to Levo, “Consistency in benefit/response was observed in the 3.2 mg dose arm across other key secondary endpoints, including clinical global impression of change (CGI-C; p=0.027) and anxiety and distress behaviors, as evaluated by the PWS Anxiety and Distress Behaviors Questionnaire (PADQ; p=0.027).” Nearly all the patients enrolled continued in the follow-up study and will continue in the extension study on the lower dose.
LV-101 received Fast Track designation for the treatment of PWS in November 2019. Levo had acquired the intranasal carbetocin from Ferring Pharmaceuticals in 2017 and announced in mid-2018 that it would restart the clinical development program by the end of that year. Ferring had completed Phase 2 development of the nasal spray for PWS in 2014.
Levo Therapeutics CEO Sara Cotter said, “This is a long-awaited step towards addressing the substantial needs of individuals living with PWS. We are excited by these important results that were achieved after decades of interest in addressing the oxytocin deficiency in PWS. We are also pleased that our efforts to develop new tools for clinical evaluation of this rare, neurodevelopmental disorder have enhanced our understanding of the real-world impact LV-101 has on anxiety and distress behaviors. . . .We look forward to working closely with regulatory authorities in the United States and abroad to bring this promising therapeutic to patients as quickly as possible.”
Read the Levo Therapeutics press release.
