Alizé Pharma is preparing to advance the development of its treatment for Prader-Willi syndrome after the drug AZP-531 came through a Phase II study. In the 47-person clinical trial, participants with the genetic appetite-affecting disorder who received AZP-531 showed a significant improvement in food-related behavior.
Lyon, France-based Alizé tracked the food-related behavior of the participants using the Hyperphagia Questionnaire, a tool other developers of treatments for Prader-Willi such as Zafgen ($ZFGN) have used in their clinical trials. Participants in the AZP-531 arm reportedly performed significantly better than patients who received the placebo, particularly against the hyperphagic severity domain score of the questionnaire. Alizé has yet to release data to quantify the difference between the two arms in terms of the units measured by the hyperphagia scale.
The company has seen enough to commit to pushing ahead with development of AZP-531, though. “Based on the Phase II results in Prader-Willi syndrome, this first-in-class unacylated ghrelin peptide analog is ready to enter the next stage of development in a well-defined indication,” Alizé President Thierry Abribat said in a statement. “Throughout the four clinical trials performed to date, involving 159 healthy volunteers and patients, AZP-531 has been very well tolerated and has consistently shown positive metabolic effects.”
In the latest trial, Alizé saw no serious or severe adverse events or “clinically-significant changes with respect to safety laboratory tests” among the patients who received AZP-531. With safety concerns potentially presenting an obstacle to Zafgen’s ambitions of winning approval for its treatment for Prader-Willi syndrome--an indication in which regulators may have little appetite for risk--the data to date could give Alizé encouragement that it can succeed despite being further back in development than its rival.
To do so, Alizé will have to generate and share more than the limited data it has released to date. The Phase II trial didn’t detect significant changes in the body weights of participants who received AZP-531, a finding Alizé attributed to the short duration of the 14-day study. Alizé will need to run longer trials if it is to show that AZP-531 makes a meaningful difference to the lives of Prader-Willi patients, a challenge it now appears keen to take on.
- read the statement