On April 6, 2020, we announced topline results from our pivotal Phase 2b clinical trial of livoletide in Prader-Willi syndrome (PWS), also called ZEPHYR, that evaluated the safety and efficacy of livoletide for the treatment of hyperphagia and food-related behaviors in patients with PWS. Unfortunately, treatment with livoletide did not significantly improve these symptoms for the livoletide treated groups in our study. As a result, we have made the difficult decision to discontinue the livoletide PWS program and related ongoing studies.
The challenges associated with this condition are numerous and the unmet need significant, and we are disappointed that livoletide will not be able to help address them. We are deeply appreciative of all the support and participation from patients, caregivers and sites, including clinicians and staff, who made this study possible and who supported us on this journey.
Livoletide is an unacylated ghrelin analogue that was previously in late-stage clinical development for the treatment of hyperphagia in Prader-Willi syndrome (PWS). This rare genetic disease is characterized by hyperphagia, a chronic unrelenting hunger, that leads to obesity, metabolic dysfunction, reduced quality of life and early mortality.
In the ZEPHYR study, a randomized, double-blind, placebo-controlled pivotal Phase 2b clinical trial in 158 patients with PWS, administration of livoletide once daily for 12 weeks showed that livoletide did not result in a statistically significant improvement in hyperphagia and food-related behaviors.
Livoletide was well tolerated during the ZEPHYR study, with injection site reaction being the most frequently reported adverse event, as expected with an injectable drug, and mostly mild in severity. A total of 2 patients (1.3%) dropped out of the study during the 12-week core period. There were 4 serious adverse events reported during the 12-week period, with none being related to livoletide treatment.
Millendo has made the decision to stop all livoletide development efforts in PWS, including discontinuation of the 9-month extension and initiation of the Phase 3 ZEPHYR study.
For more information about Millendo’s pivotal study of livoletide (ZEPHYR) please visit www.clinicaltrials.gov (NCT03790865).