Prader-Willi syndrome treatment trial begins enrollment

A phase 2a trial for a novel combination drug to treat Prader-Willi syndrome has initiated enrollment, according to a press release from Saniona, a research company focused on the field of ion channels.

The trial will assess the safety and efficacy of a combination of the serotonin–noradrenaline–dopamine reuptake inhibitor tesofensine and the beta blocker metoprolol (Tesomet, Saniona). According to the Saniona website, tesofensine has shown promise in treating obesity but may increase heart rate at therapeutic doses. Researchers aim to enroll 10 to 15 adults with Prader Willi syndrome in Czech Republic and Hungary.

"We are proud to start testing Tesomet for this orphan indication,” Jørgen Drejer, CEO of Saniona, said in the press release. “[Prader-Willi syndrome] is a severe hereditary disease for which there is no treatment today. Due to its mode of action, Tesomet could provide substantial benefits to the patients as well as their families.”

The two-part study is a double blind and placebo-controlled trial that will randomize participants 3:2 to either Tesomet or a matching placebo. After 12 weeks of treatment, participants will be assessed for change in body weight and secondary outcomes of eating behavior, food craving, body composition and metabolic parameters.

After safety is established in adults, the second part may go on to examine 10 to 15 adolescents with Prader-Willi syndrome.