Alizé Pharma has announced the launch of a Phase II clinical trial of AZP-531, its unacylated ghrelin analog, in patients with Prader-Willi syndrome.
The trial is a randomized, double blind and placebo controlled study. It aims to evaluate the safety and effects of a two-week treatment with AZP-531 on food-related behavior and on weight in up to 40 patients with Prader-Willi syndrome. The multicenter trial will be conducted in several European countries. As of today sites are open for recruitment in France and Spain.
Prader-Willi syndrome is a rare genetic metabolic syndrome, characterized by hyperphagia, an excessive eating behavior, and severe obesity. Unlike common obesity and type 2 diabetes, Prader-Willi syndrome is associated with elevated blood levels of acylated ghrelin, a hormone with strong stimulating effect on appetite.
As an unacylated ghrelin analog, AZP-531 is expected to improve hyperphagia by inhibiting the effects of increased acylated ghrelin blood levels in these patients. It is also expected to decrease weight and improve glucose control, a key additional benefit as 25% of adult Prader-Willi patients have type 2 diabetes.
“There is strong evidence for an abnormal regulation of the ghrelin system in Prader-Willi syndrome, associated with the development of severe hyperphagia and obesity that occur in these patients. AZP-531, an unacylated ghrelin analog, could constitute an attractive therapeutic approach by targeting a key underlying endocrine defect of the syndrome,” said Professor Maïthé Tauber, pediatric endocrinologist, Hospital of Toulouse, coordinator of the Reference Center for Prader-Willi in France and principal investigator of the trial.
This trial is the fourth AZP-531 clinical trial to be launched by Alizé Pharma. Two Phase I trials in healthy volunteers and obese subjects have been completed. The results indicate that AZP-531 was well tolerated, with improved glucose control and decreased weight in obese subjects over a two-week treatment period. A Phase Ib study in type 2 diabetes was initiated in 2014 with results expected in H2 2015.
“We are very pleased to announce the start of this Phase II trial of AZP-531 in Prader-Willi syndrome,” said Thierry Abribat, manager of TAB Consulting, president of Alizé Pharma. “We look forward to delivering key data in the near future on the therapeutic potential of AZP-531 from the two ongoing trials in type 2 diabetes and in Prader-Willi syndrome.”