According to a story from GlobeNewswire, the biopharmaceutical company Soleno Therapeutics, Inc. recently hosted a Key Opinion Leader (KOL) webinar, which was focused primarily on the latest updates to its ongoing phase 3 trial program. This program is investigating Diazoxide Choline Controlled Release (DCCR) tablets as treatment for Prader-Willi syndrome, a rare genetic disorder. Soleno is primarily focused on the development of novel therapies for rare diseases.
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.
Study Findings Amid COVID-19
The webinar discussed the data from its phase 3 clinical trial, which took place during the ongoing COVID-19 pandemic. As the event disrupted the trial process, Soleno specifically focused on the data generated before the official national emergency declaration was made in March of last year. In this data set, treatment with DCCR was able to generate a statistically significant improvement in the primary study endpoint, which was change in baseline hyperphagia (over-eating) based on the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) score. There were also improvements in secondary endpoints that weren’t found in the topline analysis, which was concluded in June 2020.
Theresa Strong, Ph.D., co-founder of the Foundation for Prader-Willi Research and Director of Research Programs, also presented the results from an Impact of COVID survey conducted by the Global PWS registry. This included feedback from 300 patients and families; caregivers reported 85 percent more stress, citing disruptions to social interaction, in-person therapy, and other structure and routines.
A recording of the webinar can be found in the investor section at Soleno’s website.