Statistically significant improvement in hyperphagia-related behavior and other clinically relevant endpoints support therapeutic potential of AZP-531 in Prader-Willi syndrome
Lyon, France, July 18, 2016 - Alizé Pharma SAS, an Alizé Pharma group company specialized in the development of biopharmaceuticals to treat metabolic disorders and rare diseases, announces today that the results from its Phase II trial for AZP-531, its unacylated ghrelin analog, conducted in patients with Prader-Willi syndrome, will be presented at two upcoming scientific conferences:
• 9th Conference of the International Prader-Willi Syndrome Organization (www.ipwso.org), in Toronto, Canada, July 20-24, 2016. Presentation is scheduled on July 22, at 8:40 am (EST)
• 55th Annual Meeting of the European Society for Paediatric Endocrinology (www.espe2016.org), in Paris, France, September 10-12, 2016. Presentation during the session entitled Obesity: Management, scheduled on September 12, at 12:15 am (CET)
The presentations will be held by Prof. Maïthé Tauber, pediatric endocrinologist, Hospital of Toulouse, and coordinator of the Reference Center for Prader-Willi Syndrome in France. She was also the coordinating principal investigator of this study.
The top-line results of the Phase II trial were announced in April 2016. The randomized, double-blind, placebo-controlled study assessed the safety and efficacy of two-week administration of AZP-531 vs. placebo in a total of 47 patients with genetically diagnosed PWS and evidence of hyperphagia. The results showed a significant improvement in food-related behavior as assessed by the PWS Hyperphagia Questionnaire, supported by a reduction in patient-reported appetite. In addition, improvement was reported in glucose control along with a reduction in waist circumference and fat mass. AZP-531 was well tolerated with no serious or severe adverse events and no clinically significant changes were noticed with respect to safety laboratory tests.
About the AZP-531 program in Prader-Willi syndrome
The objective of the program is to develop AZP-531, a stabilized analog of human unacylated ghrelin (UAG), the first product of a new therapeutic class for the treatment of metabolic and cardiovascular disorders. The unique pharmacological profile of AZP-531 differentiates it from ghrelin antagonists and all existing therapeutic classes. Preclinical and clinical data indicate that UAG and AZP-531 counteract acylated ghrelin (AG)-induced food consumption, inhibit fat deposition and improve glucose control. By targeting and inhibiting the metabolic effects of abnormally high AG levels seen in Prader-Willi syndrome, AZP-531 might represent an attractive treatment option. As of today, AZP-531 has been tested in three Phase I trials that included 112 healthy volunteers, obese subjects and T2D patients, and in a Phase II trial conducted in 47 PWS patients. Alizé Pharma owns a portfolio of 46 pending and granted patents protecting UAG analogs and their therapeutic applications.
About Prader-Willi Syndrome (PWS)
PWS is a rare genetic disease and the most common form of genetic obesity with an estimated prevalence of 1 to 9 per 100,000. Hyperphagia is the most salient and constant feature of the syndrome. It begins early in childhood and is associated with a lack of satiety. Patients typically display abnormal eating behaviors including obsessive food seeking, food storage, foraging and hoarding, that represent a lifelong source of distress for them and their families. Patients live a dependent life requiring continuous care and supervision. In addition, hyperphagia is associated with significant morbidity and mortality. As a consequence of obesity, up to 25% of adult patients with PWS have type 2 diabetes. Obesity-related complications are the leading cause of death in this patient population. PWS is a unique clinical condition specifically associated with increased blood levels of acylated ghrelin (AG), a potent appetite-stimulating hormone that also induces insulin resistance and fat deposition. This abnormal regulation of ghrelin is thought to be at least in part responsible for the hyperphagia in PWS patients. There is currently no effective pharmacological treatment for hyperphagia and its associated abnormal eating behaviors.
About Alizé Pharma
Alizé Pharma is a group of companies specialized in the development of innovative biopharmaceutical drugs, proteins and peptides for the treatment of metabolic diseases and rare diseases. The group is managed by a team of drug development experts and by a board of directors with a breadth of international experience. Its business strategy is to advance programs based on medical innovation to the clinical stage and establish partnerships with pharmaceutical companies to secure both short and long-term revenue streams. Since its creation the group has raised over €15 million ($16.6M) from private and institutional investors and has acquired and implemented three programs in three distinct companies:
• Alizé Pharma SAS is developing AZP-531, a stabilized analog of unacylated ghrelin, a physiological gastrointestinal peptide. This program is in Phase II clinical development for the treatment of Prader-Willi syndrome
• Prior to its acquisition by a subsidiary of Jazz Pharmaceuticals plc in March 2016, Alizé Pharma II SAS focused on the development of pegcrisantaspase (ASPAREC®), a new pegylated recombinant L-asparaginase in onco-hematological rare diseases
• Alizé Pharma III SAS has acquired exclusive worldwide rights on a family of new peptides with bone anabolic properties, to be developed in osteoporosis and other bone diseases. This I-HBD1 program is at the lead optimization stage
