ALIZE PHARMA AZP-531/UAG program
|Company||Alize Pharma SAS|
|Program||AZP-531, stabilized peptide analog of unacylated ghrelin|
|Stage of development||
Phase II in Prader-Willi syndrome
Phase I in other rare metabolic diseases
Preclinical in ischemia-related diseases
Sham Innovation Santé
The aim of the UAG program is to develop AZP-531, a stabilized peptide analog of unacylated ghrelin, the first product of a new therapeutic class for the treatment of Prader-Willi syndrome and other rare metabolic diseases.
The launch of this clinical program follows five years of collaborative research between Alizé Pharma and its academic partners at the Erasmus Medical Center in Rotterdam, in the Netherlands, and the University of Turin, in Italy. This research has led to the identification of unacylated ghrelin, a gastrointestinal peptide hormone, as a new therapeutic class, and to the design of AZP-531, a potent and stabilized analog. The unique pharmacological profile of AZP- 531 differentiates it from ghrelin antagonists and all existing therapeutic classes. Preclinical and clinical data suggest that unacylated ghrelin and its analogs have the therapeutic potential to address unmet medical needs in the treatment of Prader-Willi syndrome, as well as other metabolic and ischemia-related diseases, via a novel and unique mechanism of action.
AZP-531 is currently in Phase II clinical development for the treatment of Prader-Willi syndrome, a rare genetic disease with an estimated prevalence of 4 per 100,000. Hyperphagia, an abnormal and extreme food-related behavior, is the most salient and constant feature of the syndrome. It represents a lifelong source of distress for the patients and their families, and is associated with significant morbidity and mortality.
Preclinical and clinical data indicate that UAG and AZP-531 counteract the increased food consumption induced by acylated ghrelin (AG), inhibit fat deposition and improve glucose control. AZP-531 represents an attractive treatment option for patients with PWS as the syndrome is associated with high levels of acylated ghrelin and relatively low levels of unacylated ghrelin, which contribute to the pathophysiology of hyperphagia. Effects of AZP-531 on fat mass and glucose control provide additional potential benefits to these patients.
As of today, AZP-531 has been tested in randomized, double blind and placebo-controlled trials including three Phase I trials conducted in 112 healthy volunteers, obese subjects and T2D patients and in a Phase II trial conducted in 47 PWS patients. Results from this Phase II trial 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.
Alizé Pharma owns a portfolio of 46 pending and granted patents protecting UAG analogs and their therapeutic applications.
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