Extension Study for Prader-Willi Syndrome Initiated After Completion of Phase 2a Trial

Prader-Willi Syndrome (PWS) is caused by deleted genes on chromosome 15. This results in improper signaling of satiety, leading to obesity. Individuals with the condition can suffer from respiratory/cardiac disease, gastric rupture, pulmonary embolism, and infection. For those who are able to manage their extreme hunger and avoid obesity, life expectancy is normal, but for far too many it is shortened. There is currently no cure for the condition, which affects one in every 15,000 individuals.

But Saniona has just announced the completion of the treatment period of part two of their Phase 2a study examining Tesomet as a potential treatment for PWS. They hope to report data received from this trial within the first few months of 2019. They have also initiated an open-label extension study.

The Study

There were two parts of Saniona’s Phase 2a study- each one including nine patients. Part one of the trial was completed in 2018. It showed that Tesomet was efficacious when administered to adult patients. These patients were given a dose of .5mg. Results showed clinically meaningful weight loss in addition to a reduction in hyperphagia (overeating).

The second part of the study examined the effect of the drug for adolescent patients and utilized a lower dose (.125mg). This is because the half-life of the drug was determined by the first segment of the study to be longer than researchers had originally expected. This second segment of the study is extremely important because adolescents make up a great percentage of this population of patients.

Patients in part two were given either Tesomet or a placebo. They were randomized at a 3:2 ratio. The trials primary endpoint was change in bodyweight but secondary endpoints also included the patients food cravings, eating behavior, body composition, and various other metabolic parameters. This trial lasted 12 weeks.

Eight of the patients in the second part of the study agreed to move on to an extension study which will last 24 weeks.  This longer trial will provide researchers more information on the efficacy of this drug, ultimately helping to move this drug toward FDA approval.

“We remain confident that Tesomet holds the potential to treat debilitating hyperphagia and significantly reduce weight in this severely underserved population.”

Keep an eye out for data from part two of the Phase 2a study which should be coming soon, as well as future data from the extension study. Hopefully we will see Tesomet become a new treatment option for this population of patients.

You can read more about this Phase 2a study and the research being completed for PWS here.

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