ANN ARBOR—Millendo Therapeutics Inc., a biopharmaceutical company primarily focused on developing novel treatments for endocrine diseases, announced today that it is discontinuing the development of livoletide as a potential treatment for Prader-Willi syndrome (PWS).
The decision to discontinue the PWS program was based on topline data from the pivotal Phase 2b ZEPHYR study which showed that treatment with livoletide did not result in a statistically significant improvement in hyperphagia, or insatiable hunger, and food-related behaviors as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) compared to placebo.
“Unfortunately, treatment with livoletide did not significantly improve hyperphagia and food-related behaviors in our ZEPHYR study. While we are disappointed in these results, I want to recognize our team’s hard work and commitment in executing this robust study that informed the difficult decision to discontinue the livoletide PWS program,” said Julia C. Owens, president and CEO of Millendo. “We are deeply grateful to the patients, caregivers and researchers who made the ZEPHYR study possible. 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. Moving forward, we will shift our development focus to compelling portfolio programs nevanimibe for congenital adrenal hyperplasia (CAH) and MLE-301 for menopausal vasomotor symptoms. 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.”
The ZEPHYR study was a two-part, randomized, double-blind, placebo-controlled pivotal Phase 2b/3 study. The pivotal Phase 2b study included a three-month double-blind, placebo-controlled period in which 158 patients were randomized to either 60 µg/kg or 120 µg/kg of livoletide, or placebo. The Phase 2b data showed improvements from baseline in HQ-CT scores of -4.7 (p = 0.13) and -3.8 (p = 0.45) for the livoletide treated groups (60 µg/kg or 120 µg/kg, respectively) at 12 weeks compared to -2.8 for placebo. The average HQ-CT baseline score was 20.2. No positive trends were observed for any of the secondary endpoints of fat mass, body weight or waist circumference.
Livoletide was well tolerated during the ZEPHYR study, with injection site reaction being the most frequently reported adverse event, as expected with an injectable drug, and mostly mild in severity. A total of 2 patients (1.3%) dropped out of the study during the 12-week core period. There were 4 serious adverse events reported during the 12-week period, with none being related to livoletide treatment.
Millendo has made the decision to stop all livoletide development efforts in PWS, including the 9-month extension study and initiation of the Phase 3 ZEPHYR study.
To listen to a recording of a phone call discussing these results, visit http://investors.millendo.com.