Rudolf Baumgartner, MD, Chief Medical Officer and Head of Clinical Development at Saniona, gives an overview of Prader-Willi syndrome (PWS) and tesomet, a drug combination under investigation for the treatment of PWS.
As Dr. Baumgartner explains, PWS is a rare genetic endocrine condition that causes hypotonia and hyperphagia. It is caused by genetic abnormalities in the proximal long arm of chromosome 15. PWS is usually detected in childhood due to hypotonic and hyperphagic features of this disorder. While the obsession with food is the overwhelming symptom of PWS, the children have several other symptoms, including numerous cognitive and behavioral problems.
Unfortunately, there is currently no treatment approved for this condition. However, efforts by organizations such as the Foundation for Prader-Willi Research (FPWR), the Prader Willi Syndrome Association USA (PWSAUSA), and the International Prader-Willi Syndrome Organisation (IPWSO) are helping to raise awareness and funds to find therapies for this rare condition.
In the second part of the interview, Dr. Baumgartner describes tesomet – a combination of tesofensine, a serotonin-noradrenaline-dopamine reuptake inhibitor, and metoprolol, a selective beta blocker – which has been shown to be beneficial for PWS patients. A phase 2a trial for tesomet has been completed and Saniona is currently preparing for a phase 2b trial.
To learn more about PWS and other rare endocrine disorders, visit checkrare.com/diseases/endocrine-disorders/