According to a story from PR-Inside, the biotechnology company Saniona recently announced that the second part of its Phase 2a clinical trial testing Tesomet as a treatment for Prader-Willi syndrome has achieved its initial recruitment goal. This trial was be testing the drug in adolescent patients, and recruitment will continue to remain open for a few weeks. Saniona’s developmental focus is centered on ion-channel research.
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.
The Trial So Far
The first portion of Saniona’s Phase 2 clinical trial resulted in encouraging results. The trial began in April of 2017. This portion was with adult patients only and Tesomet was able to reduce over-eating and resulted in significant weight loss. The trial with adolescent patients will be similar in overall configuration, but the dose that will be administered will be reduced slightly, as the initial trial also indicated that Prader-Willi syndrome patients can see meaningful benefit from a smaller dose. The trial will monitor changes in patients for a period of 12 weeks.
There is a strong need for new treatment options for Prader-Willi syndrome. Overall, the potential of Tesomet as a treatment for this rare syndrome seems good so far. Hopefully future research will continue to display the capability of this medication as a therapy for Prader-Willi syndrome.