According to a story from Financial Buzz, the biopharmaceutical company Soleno Therapeutics recently provided an update on the progress of the Company’s Phase 3 clinical trial. This trial is evaluating the Soleno’s Diazoxide Choline Controlled-Release (DCCR) tablets for the treatment of Prader-Willi syndrome, a rare genetic disorder that causes overeating and severe childhood obesity. These tablets are administered once per day. The increased interest of patients in the open label extension study suggests that the drug is having a positive impact, which has also been demonstrated in prior clinical trials.
About Prader-Willi Syndrome (PWS)
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.
Trial Progress
This update is from July 24th, 2019. So far the trial has reached half of its total participant enrollment goal. In addition, 90 percent of these patients have either completed treatment successfully or are continuing to receive treatment. 90 percent of patients that have finished the study have chosen to continue into a nine month long open label extension study that will evaluate the long term safety effects of DCCR tablets. 95 percent of patients who have participated in the extension are continuing to do so. There have also been no unexpected, severe adverse events recorded so far.
Enrollment is staging at 20 different sites in both Europe and the US. Soleno plans to extend the extension study from nine months to a year due to strong interest from patients in the trial.
Soleno is working with Casimir, Inc., a rare disease research group that is innovating the design of clinical trial outcome measurements to more accurately reflect the quality of life impacts of rare disease drugs on patients.
