Millendo Therapeutics Initiates Pivotal Phase 2b/3 Clinical Study of Livoletide for the Treatment of Prader-Willi Syndrome
Top-line Phase 2b results are expected in 1H 2020 and may support an NDA submission for livoletide
Maithé Tauber, M.D., Professor of Pediatrics at the
The ZEPHYR study is a two-part, randomized, double-blind, placebo-controlled pivotal Phase 2b/3 study. The study will begin with a Phase 2b portion that includes a three-month double-blind, placebo-controlled core period in which patients receive one of two doses of livoletide or placebo followed by a nine-month extension period in which all patients receive livoletide. The second part is a Phase 3 study that will consist of a six-month double-blind, placebo-controlled core period in which patients will receive livoletide or placebo followed by a six-month extension period in which all patients receive livoletide. The study’s primary endpoint measures the change in food-related behaviors using the validated Hyperphagia Questionnaire for Clinical Trials (HQ-CT). ZEPHYR is a pivotal study and the results of the Phase 2b portion of the Phase 2b/3 study may support a new drug application (NDA) for livoletide.
Millendo’s lead asset, livoletide, is an unacylated ghrelin analogue in late-stage clinical development for the treatment of Prader-Willi syndrome (PWS), a rare genetic disease characterized by hyperphagia, a chronic unrelenting hunger that leads to obesity, metabolic dysfunction, reduced quality of life and early mortality. In a randomized, double-blind, placebo-controlled Phase 2 clinical trial in 47 patients with PWS, administration of livoletide once daily was associated with a clinically meaningful improvement in hyperphagia, as well as a reduction in appetite. Millendo has received orphan drug designation for livoletide from the
About Prader-Willi Syndrome
Prader-Willi syndrome (PWS) is a genetic disease caused by the lack of expression of several genes on chromosome 15, which leads to intellectual disability, short stature, incomplete sexual development and hyperphagia, among other symptoms. PWS patients are at risk of premature mortality, usually by the age of 30-40, mainly from obesity related conditions such as cardiovascular disease, respiratory distress and from accidents. The incidence of PWS is approximately 1 in 15,000 births. The prevalence of PWS is estimated between 8,000-11,000 patients in
Millendo Therapeutics is a late-stage biopharmaceutical company focused on developing novel treatments for orphan endocrine diseases where current therapies do not exist or are insufficient. As a leading orphan endocrine company, Millendo creates distinct and transformative treatments where there is a significant unmet medical need. The company is currently advancing livoletide for the treatment of Prader-Willi syndrome and nevanimibe for the treatment of classic congenital adrenal hyperplasia and endogenous Cushing’s syndrome. For more information, please visit www.millendo.com.
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