JAN 2018 – Circulating levels of orexigenic Acylated Ghrelin (AG), the ‘hunger hormone’, are known to be elevated in PWS with a relative deficit of Unacylated Ghrelin (UAG). AZP-531 is an unacylated ghrelin (UAG) analog which had been shown to inhibit the orexigenic effect of AG in animals and to improve glycemic control and decrease body weight in humans. This phase 2 study investigated the safety and efficacy of AZP-531 in patients with PWS and observed significant improvements in hyperphagia-related behaviour.
There have been four clinical trials to date, including the PWS trial, involving 159 participants and AZP-531 has been very well tolerated with no serious or severe adverse events. The consistently positive metabolic effects ensure that AZP-531 will be developed further, and in particular, as a treatment for patients with PWS.
Alize Pharma planned to conduct longer-term clinical trials with treatment duration of up to 6 months. It was proposed that, over time, AZP-531 will also decrease weight and cause a reduction in the onset of type 2 diabetes as a result of the observed improvements in glucose control. In December 2017, Millendo Therapeutics announced their acquisition of Alize Pharma in a stocks based transaction. Millendo intend to continue the development of AZP-531, now known as livoletide, which has now been granted orphan drug designation in PWS by the U.S. Food and Drug Administration (FDA). Millendo plans to announce details of additional clinical development of livoletide in 2018.