Vykat XR restart reduces insatiable hunger in PWS patients: Trial data
Behavioral improvements were also seen in all six areas that were measured
After discontinuing Vykat XR (diazoxide choline) for a few months as part of a clinical trial, Prader-Willi syndrome (PWS) patients who restarted the treatment showed sustained reductions in insatiable hunger, or hyperphagia, and behavioral problems.
The results were presented in a poster, titled “Resuming diazoxide choline extended-release (DCCR) after 16-week randomized withdrawal is associated with significant improvements in hyperphagia and behavioral symptoms in PWS (Study C614),” at this year’s Pediatric Endocrine Society meeting, held last week at the National Harbor in Maryland.
Hunger often leads to excess weight, obesity
Earlier this year, Vykat XR became the only medication approved in the U.S. to treat hyperphagia in adults and children, 4 years and older, with PWS. It is available on the market as extended-release tablets that are taken once daily.
“We are pleased to share the results from our ongoing clinical development program for Vykat XR,” Anish Bhatnagar, MD, chairman and CEO of Soleno Therapeutics, the therapy’s developer, said in a company press release. “These results support the effectiveness of Vykat XR in managing the critical symptoms of PWS and highlight the importance of long-term, continuous treatment.”
In early childhood, children with PWS start to experience an uncontrollable hunger, often leading to excess weight and even obesity. This hyperphagia can cause challenging behaviors as well, like anger when trying to obtain food or frustration when food is unavailable.
Newly presented data concerned PWS patients 4 and older, who had previously received Vykat XR or a placebo in the Phase 3 DESTINY PWS trial (NCT03440814), followed by three years of Vykat XR treatment in the open-label extension C602 study (NCT03714373).
The C602 study included a withdrawal period, where participants were randomly assigned to continue Vykat XR or switch to a placebo for four months.
Patients showed reduced hunger after 13 weeks of treatment
Previously reported data showed those on the placebo experienced worsening of hyperphagia, with an average 5-point score increase in the caregiver-reported Hyperphagia Questionnaire for Clinical Trials (HQ-CT) compared with those who stayed on Vykat XR. Higher scores reflect more severe hyperphagia.
After completing C602, participants could join another extension study, called C614 (NCT05701774), where all participants are receiving Vykat XR for a longer period.
The new data showed patients who were assigned the placebo upon C602’s withdrawal period and restarted Vykat XR as part of the ongoing C614 study experienced significant hyperphagia reductions after 13 weeks of treatment (about three months). These improvements were sustained over one year, when participants had a 6.3-point drop in their HQ-CT scores.
This meant a restart of Vykat XR treatment brought their HQ-CT scores back to where they were before discontinuing the therapy during the withdrawal period.
Participants also showed behavioral improvements in all six areas that were measured after a year of restarting Vykat XR, according to Soleno.
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