News

Soleno Therapeutics plans to help accelerate research toward finding a genetic cure for Prader-Willi syndrome (PWS). “We will commit up to $5 million in grants in the next year, where we will find the most promising genetic approaches to treat PWS, and find the ones where further significant investment…

Aardvark Therapeutics has amended the protocol for the Phase 3 HERO clinical trial testing ARD-101, its investigational oral therapy for excessive hunger associated with Prader-Willi Syndrome (PWS), to include patients as young as 10 years old. “Expansion of the Phase 3 HERO trial to include children 10 years…

Tonix Pharmaceuticals is planning the launch of a Phase 2 clinical trial to test its TNX-2900 nasal spray — containing a specialized form of oxytocin — in children and teens with Prader-Willi syndrome (PWS). The company has already received clearance from the U.S. Food and Drug Administration (FDA) to…

A Phase 3 clinical trial testing ACP-101 for hyperphagia, or uncontrollable hunger, in Prader-Willi syndrome (PWS) patients failed to meet its primary and secondary goals. Top-line results from the trial of Acadia Pharmaceuticals’ experimental nasal spray, dubbed COMPASS PWS (NCT06173531), showed no significant differences between the ACP-101…

Targeting an enzyme called EHMT2 may help treat Prader-Willi syndrome (PWS) by reactivating the maternal PWS genes that are normally turned off, according to a new study. PWS is caused by the loss of certain genes in a region of the paternal chromosome 15 called the PWS locus.

Adults with Prader-Willi syndrome (PWS) caused by a genetic deletion have higher obesity rates and more severe obesity than those with PWS associated with other genetic abnormalities, a study found. “Given the higher risk of obesity observed in individuals with the deletion subtype, our results support the need for…

Hypogonadism, a condition in which the body produces few or no sex hormones, is common in girls and young women with Prader-Willi syndrome (PWS). But while hormone replacement therapy (HRT) is often prescribed, many patients stop using it because of bothersome side effects, a U.S. study found. “These findings…

An imbalance of fatty molecules in the blood, as assessed by the triglyceride-to-high-density lipoprotein cholesterol ratio (TG/HDL-C), can accurately detect metabolic syndrome in children and adolescents with Prader-Willi syndrome (PWS), a study has shown. Metabolic syndrome is a cluster of health conditions, including abdominal obesity, high blood pressure,…

Aardvark Therapeutics plans to expand its ongoing Phase 3 clinical trial of ARD-101, an oral therapy for easing excessive hunger in Prader-Willi syndrome (PWS), to include patients younger than 13. The decision, supported by the PWS community, is based on historical data indicating that younger PWS patients are…

Relmada Therapeutics is working toward the launch, next year, of a Phase 2 clinical trial to test sepranolone — which the company believes will be a first-in-class treatment for easing compulsive behaviors — in people with Prader-Willi syndrome (PWS). Preparations include engaging U.S. Food and Drug Administration officials…