Millendo’s Livoletide Fails in Pivotal Trial for Prader-Willi Syndrome

Rare Daily Staff

Millendo Therapeutics reported that it will discontinue clinical development of the experimental treatment livoletide in patients with Prader-Willi syndrome after topline results from a pivotal trial showed no significant improvement in key symptoms compared to placebo, the primary endpoints of the study.

Shares of the biotech fell 70 percent on the news.

Prader-Willi syndrome, or PWS, is a complex neurodevelopmental genetic disorder, and the most common orphan genetic cause of life-threatening childhood obesity. It is characterized by hyperphagia, a chronic unrelenting hunger, which leads to obesity and metabolic dysfunction, reduced quality of life, and early mortality.

“Unfortunately, treatment with livoletide did not significantly improve hyperphagia and food-related behaviors in our ZEPHYR study,” said Julia Owens, president and CEO of Millendo. “We are committed to understanding the totality of the phase 2b results and we intend to report the data at a future scientific meeting or publication when they are available.”

In a randomized, double-blind, placebo-controlled pivotal phase 2b clinical trial in 158 patients with PWS, administration of livoletide once daily for 12 weeks showed that livoletide did not result in a statistically significant improvement in hyperphagia and food-related behaviors.

Millendo said it will shift its development focus to other portfolio programs, including its experimental therapeutic nevanimibe for congenital adrenal hyperplasia (CAH), a rare chronic monogenetic disease, being studied in a mid-stage trial, and MLE-301 for the hot flashes and night sweats of menopause.

“With the rapidly evolving COVID-19 global pandemic and the extraordinary burden it has put on hospitals and healthcare providers, we are monitoring the potential impact of the situation on these programs and will provide an update when we have more clarity on expected timelines,” said Owens.

Millendo has received Orphan Drug designation for nevanimibe for the treatment of congenital adrenal hyperplasia from the U.S. Food and Drug Administration and the European Medicines Agency.

Photo: Julia Owens, president and CEO of Millendo