Soleno Therapeutics Announces Publication of Results from Pivotal Study of VYKAT™ XR in the Journal of Clinical Endocrinology and Metabolism
The randomized withdrawal period study was a key part of the comprehensive Phase 3 clinical program that established the efficacy and safety of VYKAT XR and supported its approval by the
“This important study provided additional controlled data confirming the safety and efficacy of VYKAT XR in people with PWS, and we are pleased that the positive results have been accepted for publication in JCEM, the world’s leading peer-reviewed journal focused on cutting-edge endocrine and metabolic research,” said Dr.
Dr.
The 16-week, randomized withdrawal period study included 77 individuals who previously completed 13-week placebo-controlled and long-term open-label Phase 3 studies. Participants were randomized 1:1 to either VYKAT XR (n=38) or placebo (n=39). The primary endpoint was Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score change from baseline to week 16. Secondary endpoints included Clinical Global Impression of Severity (CGI-S) and Improvement (CGI-I); exploratory endpoints included weight and body mass index (BMI) z-score.
Key highlights from the publication:
- Hyperphagia worsened significantly when treatment with DCCR was withdrawn, compared to continued DCCR administration. Statistically significant increases (worsening) in HQ-CT from baseline to week 16 were observed with placebo versus DCCR (P=0.0022).
- CGI-S and CGI-I scores favored DCCR and approached, but did not reach, statistical significance.
- Consistent with the hyperphagia response, the placebo cohort gained more weight and increased their BMI z-score more than the DCCR cohort (LS mean weight difference (95% confidence interval) -1.6 kg (-3.1, -0.1); LS mean z-score difference -0.09 (-0.17, -0.01).
- Adverse events were similar with both arms, with no serious adverse events in the DCCR treatment arm. No participant experienced an adverse event leading to study drug discontinuation.
About PWS
Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in the gene expression on chromosome 15.
About VYKAT XR
VYKAT XR was approved by the U.S. Food and Drug Administration (FDA) on March 26, 2025, and is now commercially available to U.S. patients.
VYKAT XR is indicated for the treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS).
IMPORTANT SAFETY INFORMATION
Contraindications
Use of VYKAT XR is contraindicated in patients who have a known hypersensitivity to diazoxide, other components of VYKAT XR, or to thiazides.
Warnings and Precautions
Hyperglycemia
Hyperglycemia, including diabetic ketoacidosis, has been reported. Before initiating VYKAT XR, test fasting plasma glucose (FPG) and HbA1c; optimize blood glucose in patients who have hyperglycemia. During treatment, regularly monitor fasting glucose (FPG or fasting blood glucose) and HbA1c. Monitor fasting glucose more frequently during the first few weeks of treatment in patients with risk factors for hyperglycemia.
Risk of Fluid Overload
Edema, including severe reactions associated with fluid overload, has been reported. Monitor for signs or symptoms of edema or fluid overload. VYKAT XR has not been studied in patients with compromised cardiac reserve and should be used with caution in these patients.
Adverse Reactions
The most common adverse reactions (incidence ≥10% and at least 2% greater than placebo) included hypertrichosis, edema, hyperglycemia, and rash.
Please see the full Prescribing Information, including Medication Guide.
About Soleno Therapeutics, Inc.
Soleno is focused on the development and commercialization of novel therapeutics for the treatment of rare diseases. The company’s first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, formerly known as DCCR, is a once-daily oral treatment for hyperphagia in adults and children 4 years of age and older with Prader-Willi syndrome and was approved by the
Forward-Looking Statements
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Corporate Contact:
212-915-2578
Media Contact:
media@soleno.life
Source: Soleno Therapeutics