Still-recruiting ARD-101 trial for PWS lowers US enrollment age to 7
Aardvark oral therapy aims to ease excessive hunger in children and adults
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An ongoing, global clinical trial testing Aardvark Therapeutics’ ARD-101 for hyperphagia, or excessive hunger, in people with Prader-Willi syndrome (PWS) has lowered the minimum enrollment age in the U.S. to allow children as young as 7 to take part, according to a study update.
The Phase 3 HERO (NCT06828861) trial is testing ARD-101 in as many as 90 children and adults with PWS-related hyperphagia. In addition to the U.S., participants are being enrolled at sites in Australia, Canada, South Korea, and the U.K. The minimum age in all countries outside the U.S. is 13, except in Australia, where patients as young as 10 are being enrolled.
This protocol change was agreed upon by the U.S. Food and Drug Administration’s Institutional Review Board, and follows an eligibility expansion last year that reduced the minimum age of trial participants in the U.S. from 13 to 10 years.
“Hyperphagia may begin early in life for some individuals with PWS, and it presents a persistent burden for patients and caregivers,” Tien Lee, MD, Aardvark’s founder and CEO, said in a company press release announcing the change in age.
“Expanding eligibility reflects our commitment to addressing the urgent need in the PWS community for a differentiated therapy and we aim to ensure broad and equitable access,” Lee said.
According to the company, the trial remains on track to allow the announcement of top-line data between July and September of this year.
“We believe these data will further inform our understanding of ARD-101’s potential role as a differentiated therapeutic approach for the treatment of hyperphagia associated with PWS,” Lee said.
ARD-101 aims to let the brain know the body is full
PWS is caused by the loss of function of genes located in a region of chromosome 15 called the PWS locus. Hyperphagia, or a persistent sensation of hunger, is a hallmark PWS symptom, which typically starts at approximately 8 years of age. It can lead to obesity and extreme food-seeking behaviors.
An oral small molecule, ARD-101 activates gut-based bitter taste receptor proteins to stimulate hormones that tell the brain the body is full. By mimicking this natural signal, the experimental therapy aims to lower appetite and help patients manage the urge to constantly seek out food.
The medication received orphan drug and rare pediatric disease designations for PWS in the U.S. These statuses are meant to help incentivize and accelerate the development and regulatory review of drugs meant to treat rare conditions, defined in the U.S. as those affecting fewer than 200,000 people.
In a Phase 1 clinical trial involving healthy adults, ARD-101 showed a favorable safety profile. Data from a Phase 2 study (NCT05153434) enrolling adolescents and adults with PWS showed that about a month of ARD-101 treatment led to meaningful reductions in hyperphagia and favorable changes in body composition, including reduced body fat and increased lean muscle, according to the company.
HERO trial is testing therapy against a placebo
Now, HERO participants are being randomly assigned to take either ARD-101 or a placebo twice daily for about three months. For those on the therapy, the dosage will increase from 200 mg in the first week to 400 mg in the second week, and to 800 mg thereafter.
The study’s main goal is to assess changes in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), a widely used measure of food-seeking behaviors in PWS. Secondary measures include changes in caregiver- and clinician-reported measures of hyperphagia severity.
According to Lee, “enrollment in HERO is progressing steadily.”
Participants who complete the trial may enter a long-term open-label extension study (NCT07197034), where all will receive ARD-101 for up to a year.
