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Prader-Willi Syndrome

Positive results for AZP-531 Prader-Willi Syndrome study

Results show significant improvements in hyperphagia-related behaviour, a key therapeutic objective in Prader-Willi Syndrome

Alizé Pharma SAS has announced that top-line results of a Phase II clinical trial of AZP-531, its unacylated ghrelin analogue, in patients with Prader-Willi Syndrome (PWS), 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.

This randomised, double-blind, placebo-controlled, European multi-centre study was aimed at evaluating the safety, tolerability and efficacy of AZP-531 administered daily subcutaneously for 14 days on food-related behaviour, versus placebo. The trial was conducted across seven centres 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 BMI38 (range 21-67).

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).

“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.

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.

“We are very pleased with these Phase II results and wish to thank all the investigators and their teams, as well as the patients and their families, who actively participated in this trial,” said Soraya Allas, medical director at Alizé Pharma. “We look forward to pursuing the development of AZP-531 with the goal of applying a therapeutic approach to this severe medical condition for which no treatment is currently available.”

“The great news provided by these results paves the way for further development of AZP-531. Throughout the four clinical trials performed to date, involving 159 healthy volunteers and patients, AZP-531 has been very well tolerated and has consistently shown positive metabolic effects. Based on the Phase II results in Prader-Willi Syndrome, this first-in-class unacylated ghrelin peptide analog is ready to enter the next stage of development in a well-defined indication,” said Thierry Abribat, TAB Consulting, president of Alizé Pharma.

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