According to a story from BioPortfolio, the biotechnology company Saniona has released an update in regards to the company’s Phase 2a clinical trial of Tesomet, which is being tested as a treatment for Prader-Willi syndrome. The goal of this study is to determine the optimal effective dose of the drug that will provide relief of symptoms for adolescent patients. In earlier trials, a dose of .5mg per day was enough to provide positive effects for adult Prader-Willi syndrome patients.
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.
Finding The Sweet Spot
As Prader-Willi syndrome is a disorder that begins to cause problematic symptoms early in life, it is critical that the minimum effective dose be established for younger, adolescent patients. The previous success in earlier trials of the drug mean that there is a good chance that the drug will be effective regardless of the patient’s age.
So far in the study, the dose level for patients has been low at just .125mg per day. This dosage has not been able to produce the plasma levels that are associated with weight loss and the reduction of hyperphagia (overeating). The earlier results with this drug suggest that the effective dose will probably be smaller for Prader-Willi syndrome patients than for other forms of genetic obesity because the half life of the drug is considerably longer in Prader-Willi syndrome patients.
Saniona has applied to continue the study for three more months using a stronger dose of .25mg per day. The first patient is expected to be dosed in March of 2019.
