Saniona has just announced that their Phase 2b clinical trial examining Tesomet as a treatment for Prader-Willi syndrome (PWS) will be beginning soon.

This treatment is a combination therapy which researchers believe could minimize hyperphagia, the uncontrollable hunger patients with this disease face. The treatment is already being examined in a Phase 2b trial for hypothalamic obesity.

Researchers expect results from the new Phase 2b trial for PWS by the first half of next year, 2023.

PWS

PWS is a rare disease which leads to a wide array of symptoms including cognitive deficits, social deficits, emotional deficits, hypotonia, abnormal growth/body composition, and hyperphagia.

Hyperphagia is extreme, insatiable hunger and many caregivers have said it’s the most challenging aspect of the disease. Caregivers often must lock up food in the home in effort to minimize consumption.

It can lead to obesity, a shorter life span, choking, pulmonary embolisms, gastric ruptures, cardiac failure, and more.

Unfortunately, there has yet to be an approved treatment for this debilitating symptom. Additionally, PWS itself has yet to have a cure.

The Trial

This Phase 2b clinical investigation was double-blind, randomized, and placebo-controlled. It will include 16 weeks of treatment and then a 36 week open-label extension where patients will be allowed to continue the treatment if they wish, or begin it if they were first receiving placebo. During the extension, the dose of the treatment given to patients will be the highest dose tolerated during the treatment period. The treatment period will test three different doses of Tesomet and placebo.

The trial will enroll around 12o PWS patients who are between 18 and 65 years old. In the future, the researchers hope to add adolescent patients (between 13 and 17 years old). The trial will be conducted at numerous sites in the US, Australia, New Zealand, Spain, Italy, Sweden, the UK, and more.

Its primary endpoint will be the measured change in hyperphagia after 16 weeks of treatment. This measurement will be taken from a caregiver survey which includes questions surrounding food seeking behavior including time spent mentioning food and time spent sneaking food. Also documented will be changes in body weight, disease severity, and general clinical status. 

Tesomet has already been given orphan drug designation by the FDA.

You can read more about this treatment and the upcoming clinical trial here.