Phase 2b Prader-Willi Syndrome Trial Ends in Disappointment

According to a story from Malvern Daily Record, the biopharmaceutical company Millendo Therapeutics, Inc. recently released data from its phase 2b clinical trial. This study was testing the company’s experimental therapy livoletide as a treatment for Prader-Willi syndrome (PWS), a rare disorder that causes overeating and excessive weight gain. Unfortunately, livoletide failed to have a meaningful impact when compared to placebo. As a result, Millendo has announced that it will halt all efforts to develop livoletide for this disorder.

About Prader-Willi Syndrome

Prader-Willi syndrome is a genetic disorder which is most characterized by childhood obesity that results from an abnormal, insatiable appetite. This obesity often continues into adulthood. In most cases, the syndrome is caused by the deletion of a certain section of chromosome 15. In about a quarter of cases, the patient receives two copies of chromosome 15 from the mother but gets none from the father. This syndrome is not considered heritable, as the genetic changes occur during gestation. Symptoms of Prader-Willi syndrome include slow development, poor feeding, muscle weakness, obesity, over-eating, abnormal flexibility, scoliosis, sleeping excessively, speech delays, intellectual disability, poor muscle tone, delayed puberty, and infertility. Excessive eating also leads to elevated risk of diabetes. Management may include physical, occupational, and speech therapy, limiting access to food, and injections of growth hormone (in child patients only). To learn more about Prader-Willi syndrome, click here.

About the Trial

The clinical trial’s primary endpoint was improvement in hyperphagia and other behaviors related to food as assessed with the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). Unfortunately, the differences in scores between the placebo group and the two dose cohorts was not large enough to be considered significant. No impacts on secondary endpoints, such as waist circumference, body weight, or fat mass, were observed.

Shifting Focus

With Millendo’s decision to abandon its development of livoletide, the company will now shift focus to its experimental product candidates MLE-301 and nevanimibe, which are being developed for menopausal vasomotor symptoms and congenital adrenal hyperplasia respectively. Millendo is primarily focused on the development of novel therapies for endocrine system diseases.


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