Alizé Pharma reports positive results from its Phase II clinical trial of AZP-531 in Prader-Willi Syndrome
Alizé Pharma SAS, an Alizé Pharma group company specialized in the development of biopharmaceuticals to treat metabolic disorders and rare diseases, announces today the top-line results of a Phase II clinical trial of AZP-531, its unacylated ghrelin analog, in patients with Prader-Willi Syndrome (PWS).
This randomized, double-blind, placebo-controlled, European multicenter study was aimed at evaluating the safety, tolerability and efficacy of AZP-531 administered daily subcutaneously for 14 days on food-related behavior, versus placebo. The trial was conducted across seven centers in France, Spain and Italy. It enrolled a total of 47 patients with genetically diagnosed PWS and evidence of hyperphagia, with a mean age of 27 years (range 13-46) and mean BMI of 38 kg/m2 (range 21-67).
The results showed a significant improvement in food-related behavior in patients treated with AZP-531 (p<0.05 versus placebo), as assessed by the Hyperphagia Questionnaire (HQ), the most widely used tool for assessing efficacy in clinical trials in PWS. The results showed a particular improvement in the Hyperphagic Severity domain score of the HQ (p<0.05 versus placebo). These findings were supported by a reduction in appetite following breakfast for patients treated with AZP-531, as assessed by a newly developed patient-reported outcome scale (p<0.001 versus baseline; not significant versus baseline for the placebo group).
Glucose control improved with AZP-531 treatment, with a greater effect observed in patients with higher fasting or post-prandial glucose levels at baseline. Body weight did not change significantly in either group, which is not unexpected following short-term treatment in a study population with a highly variable weight (range of 53-161 kg) and BMI at baseline. However, a significant reduction in waist circumference was noted in the AZP-531 group (p<0.05 versus baseline), which was not observed in the placebo group (not significant versus baseline).
AZP-531 was well tolerated with no serious or severe adverse events and no clinically significant changes with respect to safety laboratory tests.
Results will be presented at the IPWSO Conference, July 20-24, 2016, Toronto, Canada.
“Hyperphagia is a devastating feature of Prader-Willi Syndrome as it dramatically impairs the quality of life of the patients and their families and may also lead to morbid obesity and related cardiovascular complications. In this regard, the impact of AZP-531 on food-related behavior in this trial is clinically relevant, highly promising and calls for the implementation of longer-term clinical trials,” said Prof Maïthé Tauber, pediatric endocrinologist, Hospital of Toulouse, and coordinator of the Reference Center for Prader-Willi Syndrome in France. Prof Maïthé Tauber was also the coordinating principal investigator of this study.