Neurocrine to present Vykat XR and PWS research at FPWR conference

Neurocrine Biosciences (Nasdaq: NBIX) announced it will present eight studies and analyses related to Prader-Willi syndrome (PWS) and its treatment Vykat XR (diazoxide choline) at the Foundation for Prader-Willi Research 2026 Annual Research Symposium & Family Conference, taking place Oct. 7-10 in Philadelphia.

The presentations include baseline results from PWS-VISTA, an ongoing observational study collecting real-world data on patients initiating Vykat XR in routine clinical practice, focusing on long-term safety and treatment outcomes. A second observational study, BRAVE-PWS, is designed to capture caregiver-reported information on the experience and burden of caring for someone with PWS.

Additional presentations will include an integrated analysis of adverse events of special interest during Vykat XR treatment — specifically fluid retention, hyperglycemia, hirsutism and hypertrichosis — as well as an oral presentation examining real-world GLP-1 receptor agonist use and gastrointestinal complications in PWS patients.

Vykat XR received FDA approval on March 26, 2025, for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with PWS. The company describes it as the first FDA-approved treatment for this indication.

PWS is a rare genetic neurodevelopmental disorder estimated by the Prader-Willi Syndrome Association USA to occur in approximately one in every 15,000 live births. Its defining symptom, hyperphagia, is a chronic condition characterized by persistent hunger and food-seeking behavior that can lead to serious complications including obesity, diabetes, and cardiovascular disease.

The most common adverse reactions associated with Vykat XR, occurring in at least 10% of patients and at least 2% more than placebo, include hypertrichosis, edema, hyperglycemia, and rash. The drug is contraindicated in patients with known hypersensitivity to diazoxide or thiazides.

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