Phase 2a Prader-Willi Syndrome Trial Enters Open-Label Extension

According to a story from finanznachrichten.de, the biotechnology company Saniona recently announced that its treatment period for adolescent patients in its Phase 2a clinical trial testing Tesomet as a treatment for Prader-Willi syndrome has been completed. This portion of the study featured nine patients. Eight of them will continue to receive treatment in an open label extension study. The company expects to release data from this trial early in 2019. Saniona is dedicated to the development of therapies for central nervous system and eating disorders. 

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 Study

This extension study is intended to last for a period of 24 weeks. The first portion of the study, which assessed adult patients, showed that Tesomet had a meaningful effect on this patient group in reducing weight and excessive eating (hyperphagia). The primary endpoint for these studies was to measure changes in body weight for patients over a period of 12 weeks in comparison to a placebo control group. Secondary endpoints included changes in eating behavior and overall body composition.

The earlier study of nine adult patients, which was completed last year, also revealed that Tesomet had a longer half-life than originally projected. For this reason, adolescents received a lower dose (.125mg versus .5mg) than the adult patient group did. Hopefully, the data from the adolescent patient group will be consistent with the positive results experienced in the adult group.

 


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