Vykat XR may help PWS patients with restricted food access, diabetes
'Statistically significant, clinically meaningful benefit from treatment'
Vykat XR (diazoxide choline) can safely reduce hyperphagia — a feeling of insatiable hunger that is a hallmark of Prader-Willi syndrome (PWS) — in patients whose access to food has been restricted and in those with pre-diabetes or diabetes.
These data were presented in two poster presentations at the 2025 United In Hope: International Prader-Willi Syndrome Conference, held June 24-28 in Phoenix, which was anticipated to be the largest conference focused on the disease.
“We believe that these data reveal critical insights on the potential benefit of VYKAT XR to important subpopulations of individuals living with PWS,” Anish Bhatnagar, MD, CEO and chairman of the board of Soleno Therapeutics, which markets Vykat XR, said in a company press release.
In infancy, symptoms of PWS include weak muscle tone and slow growth. Later in childhood, hyperphagia typically begins, often leading to excessive weight gain and obesity. This hunger can also trigger challenging behaviors, such as irritability when denied food, sneaking food, foraging through trash, or waking at night to search for food.
Vykat XR approved in US for hyperphagia in PWS patients, 4 and older
Since earlier this year, Vykat XR has been the only medication approved in the U.S. for hyperphagia in people 4 years and older with PWS. It is available as a once-daily extended-release tablet that works by regulating neural pathways that help control appetite. It also acts on protein channels in the pancreas to block the release of insulin, a hormone that helps move glucose from the blood into the body’s cells, as a way to increase the feeling of satiety and reduce the buildup of fat.
Patients treated with Vykat XR as part of a Phase 3 clinical study called DESTINY PWS (NCT03440814) and its open-label extension (NCT03714373) showed significant reductions in hyperphagia after one year. Improvements were even greater in those who entered the study with more severe symptoms of hyperphagia.
Now, Vykat XR may be effective even in patients with highly restricted access to food, according to a poster titled “Diazoxide Choline Extended-Release Tablets Significantly Reduce Hyperphagia in Patients with PWS Who Are Managed with Strict Food Controls.”
These patients improved as much, or even more, than those with less-strict food rules after being treated with Vykat XR for up to three years. Improvements were measured using the Hyperphagia Questionnaire for Clinical Trials, where higher scores indicate more severe symptoms of hyperphagia.
“For those individuals who are managed with strict food controls, these data suggest that they had more hyperphagia symptoms at baseline [the start of the study] yet still saw a statistically significant, clinically meaningful benefit from treatment with VYKAT XR,” Bhatnagar said.
In another poster, titled “Safety and Efficacy of Diazoxide Choline Extended-Release in Patients with PWS who Have Pre-Diabetes or Diabetes,” researchers focused on patients with pre-diabetes or diabetes. Pre-diabetes refers to higher-than-normal blood sugar levels that are not yet in the diabetes range.
After three years on Vykat XR, patients with pre-diabetes or diabetes and those with normal blood sugar showed less severe hyperphagia. Side effects of hyperglycemia (high blood sugar) were more common in participants with pre-diabetes or diabetes (42.7% vs. 24%), but these were usually manageable.
Few patients stopped taking Vykat XR due to side effects related to treatment, suggesting “VYKAT XR can be safely administered to individuals with PWS who have pre-diabetes or diabetes, providing a benefit despite this co-morbidity [coexisting condition],” Bhatnagar said.
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