Date: 2016-04-26
Type of information: Results
phase: 2
Announcement: results
Company: Alize Pharma (France)
Product: AZP-531 (unacylated ghrelin analog)
Action
mechanism: peptide. 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.
Disease: Prader-Willi syndrome
Therapeutic area: Rare diseases - Genetic diseases
Country: France, Italy, Spain
Trial details:
Latest
news: * On April 26, 2016, Alizé Pharma, an Alizé Pharma group company specialized in the development of biopharmaceuticals to treat metabolic disorders and rare diseases, announced results of a Phase II clinical trial of AZP-531, its unacylated ghrelin analog, in patients with Prader-Willi Syndrome. 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 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. Based on these results, Alizé Pharma intends to enter the next stage of developement. * On March 2, 2015, Alizé Pharma 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. Results are expected to be available by mid-2016. Professor Maïthé Tauber, pediatric endocrinologist, Hospital of Toulouse, coordinator of the Reference Center for Prader-Willi in France is the 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.
