Millendo Therapeutics recently announced the initiation of their Pivotal Phase 2b/3 clinical study of Livoletide for the treatment of Prader-Willi syndrome. This study which is investigating the efficacy of livoletide on food-related behaviors in PWS, will be enrolling approximately 150 patients ages 8 to 65 across the US and Europe. Two sites are now open (Minneapolis, MN and Toulouse, France) with additional sites planned to open soon. Read below for their recent press release.
Millendo Therapeutics, Inc. (Nasdaq: MLND), a clinical-stage biopharmaceutical company developing novel treatments for orphan endocrine diseases, announced today that it has initiated its pivotal Phase 2b/3 clinical study investigating livoletide (AZP-531) in patients with Prader-Willi syndrome (PWS). The study, called ZEPHYR, will evaluate the safety and efficacy of livoletide on food-related behaviors in PWS patients. The primary endpoint is an assessment of livoletides impact on hyperphagia, the excessive hunger which is a hallmark symptom of the disease. ZEPHYR is one of the largest global PWS studies ever conducted and its Phase 2b portion aims to recruit 150 patients from up to 40 clinical sites in the United States and Europe.
Maithe Tauber, M.D., Professor of Pediatrics at the University of Toulouse and Chief of Endocrinology and Medical Genetics at the Childrens Hospital of Toulouse noted, Livoletide is an exciting investigational drug for Prader-Willi syndrome that has the potential to treat hyperphagia, the unrelenting hunger that often leads to excessive eating and is a root cause of morbidity and mortality in PWS patients. Current strategies to manage the disease present a heavy burden on caregivers, and do not address the underlying hyperphagia experienced by patients. Livoletide has the potential to be an important new treatment option that could positively impact the lives of patients and their caregivers.``
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 studys 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.
Pharis Mohideen, M.D., Chief Medical Officer at Millendo, further noted, ``The initiation of our pivotal trial for livoletide is an important step toward our mission to bring life-changing therapies to market for rare endocrine diseases with significant unmet medical needs, like Prader-Willi syndrome. We are pleased that multiple clinical sites are actively enrolling patients, and we expect to report topline results from the Phase 2b portion of ZEPHYR in the first half of 2020.