More than 150 patients aged eight to 65 years have been recruited at 38 sites in the US, Europe and Australia. The company is adding another cohort for patients aged four to seven years.
PWS is a rare genetic disease that causes chronic unrelenting hunger known as hyperphagia. Livoletide is an unacylated ghrelin analogue being developed to treat the disease.
The ZEPHYR study is a two-part, randomised, double-blind, placebo-controlled trial being conducted to assess the safety and efficacy of the drug on food-related behaviours in PWS patients.
The Phase IIb portion is a double-blind, placebo-controlled three-month period. During the period, participants will be administered with one of two doses of livoletide or placebo.
It will be followed by a nine-month extension period where all subjects will receive the drug.
The six-month double-blind, placebo-controlled Phase III portion of the study will enrol additional patients, who will be given livoletide or placebo. A six-month extension period will follow, with all patients receiving the drug.
Primary endpoint for both portions is the change in food-related behaviours, measured using the Hyperphagia Questionnaire for Clinical Trials (HQ-CT).
Top-line results from the trial are expected to be reported in the first half of next year. The company anticipates that Phase IIb data alone may support a new drug application (NDA).
Millendo Therapeutics president and CEO Julia Owens said: “Completing recruitment of over 150 patients in this pivotal study is an important step forward as we continue our efforts to advance livoletide for PWS patients and families who struggle with the life-threatening symptoms of the disease, especially hyperphagia.
“Ahead of sharing top-line results from the Phase IIb portion of ZEPHYR in the first half of next year, we are preparing for a build of our commercial organisation in the Boston area.”
In a previous Phase II trial in 47 PWS patients, treatment with livoletide once-daily for two weeks demonstrated a clinically meaningful improvement in hyperphagia and also decreased appetite.