Part 2 of Prader-Willi Study to Begin Enrolling Adolescents

This morning, biotechnological company Saniona announced that it has obtained approval to initiate the second part of its Phase 2a trial for Tesomet in adolescents with Prader-Willi syndrome (PWS).

Tesomet is being developed specifically to provide a meaningful reduction in hyperphagia, or food cravings, in patients with PWS, characterized by an extreme and insatiable appetite. Historically, this has proven to be a major, debilitating problem for this patient population and its respective caregivers.

This exploratory randomized, double-blind, placebo-controlled Phase 2a study in patients with PWS was started in April 2016 and divided into 2 parts. The first part of the study, which included 9 adult patients, was completed in 2017, and results from this study concluded that Tesomet could potentially provide clinically significant weight loss and a substantial reduction in hyperphagia. Additionally, it was revealed in the study that patients with PWS should be administered lower doses of Tesomet in comparison to the dosages given to other patient groups.

“We are excited to continue advancing Tesomet’s clinical development for Prader-Willi syndrome following encouraging results from our exploratory Phase 2a study in adult patients and positive discussions with key opinion leaders in the field. By pursuing an orphan indication such as Prader-Willi syndrome, we may develop and commercialize our own product in the U.S. and Europe within a relative short time and at a limited investment in an indication where the commercial opportunities appear to be very large,” commented Jørgen Drejer, CEO of Saniona in a press release. “We believe that low dose Tesomet could prove to be a compelling treatment for adolescents with this currently untreatable disease. Patients suffering from Prader Willi syndrome currently face debilitating hyperphagia, which has severe consequences that also affect their families and carry a very high cost to payors and the society.”

The second part of this study could potentially include up to 10 adolescents with PWS. Based on the results from the first part of study, patients will receive either Tesomet (tesofensine 0.125 mg + metoprolol 25 mg daily) or placebo at a 3:2 randomization with the change in bodyweight over 12 weeks of treatment compared to placebo serving as the primary endpoint. Secondary objectives include an examination of eating behavior and hyperphagia, body composition, lipids and other metabolic parameters. The study also includes comprehensive evaluations of tolerability, safety and pharmacokinetic parameters in the indication.

“The data supporting Tesomet for Prader Willi patients has been impressive. During the first 3-month study, patients not only experienced a significant weight loss but also a remarkable reduction in cravings, measured by the hyperphagia questionnaire,” Dr. Roman Dvorak, Saniona Chief Medical Officer said.

Moving forward, the company intends to continue the study at a lower dose in adolescents with PWS.

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