Alizé Pharma launches Phase II clinical trial of AZP-531 in Prader-Willi syndrome

Alizé Pharma launches Phase II clinical trial of AZP-531 in Prader-Willi syndrome

Lyon, France, March 2, 2015 - Alizé Pharma, a company specialized in the development of drugs for the treatment of metabolic disorders and rare diseases, today announces 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 opened for recruitment in France and Spain. Results are expected to be available by mid-2016.

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, an 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 2015 H2.

"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."

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. It is a complex clinical syndrome, characterized by life-threatening obesity, short stature, hypogonadism, cognitive, behavioral and psychiatric disturbances. As a consequence of obesity, up to 25% of adult patients with Prader-Willi syndrome have type 2 diabetes. 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 family. Patients live a dependent life requiring continuous care and supervision. In addition, hyperphagia is associated with significant morbidity and mortality. Obesity-related complications are the leading cause of death in this patient population. Prader-Willi syndrome is a unique clinical condition specifically associated with increased levels of acylated ghrelin, a potent orexigenic hormone. There is currently no effective pharmacological treatment for hyperphagia and associated abnormal eating behaviors.

About the UAG (UnAcylated Ghrelin) program

The objective of the UAG program is to develop AZP-531, an analog of unacylated ghrelin, the first product of a new therapeutic class for the treatment of metabolic and cardiovascular disorders. Since 2008 Alizé Pharma has collaborated on this program with the Erasmus Medical Center in Rotterdam and the University of Turin. Preclinical and clinical data suggest that unacylated ghrelin and its analogs have the therapeutic potential to address unmet medical needs in the treatment of type 2 diabetes, Prader-Willi syndrome and some ischemia-related conditions. Alizé Pharma owns a portfolio of 37 pending and granted patents protecting UAG analogs and their therapeutic applications.

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 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 in 2007 the group has raised €13.3 million ($16.6 million) from private and institutional investors. It has advanced two programs to the clinical phase and has also signed its first industrial partnership.

The first group entity, Alizé Pharma SAS, is working on a peptide derived from unacylated ghrelin (AZP-531). This program is in Phase II clinical development for the treatment of Prader-Willi syndrome and in Phase Ib for type 2 diabetes.

The second entity, Alizé Pharma II SAS, is focusing on the development of pegcrisantaspase (ASPAREC® / JZP416), a new pegylated recombinant L-asparaginase for the treatment of acute lymphoblastic leukemia (ALL). It is partnered with Jazz Pharmaceuticals (Nasdaq: JAZZ) and is currently in Phase II/III clinical development.

Founded in 2014, Alizé Pharma III SAS has acquired exclusive worldwide rights to develop and commercialize a family of new peptides with bone anabolic properties.

For further information, please contact:
Andrew Lloyd & Associates
Hillary Rock-Archer / Sandra Régnavaque
[email protected] / [email protected]
Tel: +44 1273 675 100
US: +1 617 202 4491
Alizé Pharma
Thierry Abribat
[email protected]