Soleno Therapeutics Publishes Pivotal Study Results for VYKAT™ XR in JCEM
REDWOOD CITY, Calif., January 05, 2026 (GLOBE NEWSWIRE) — Soleno Therapeutics, Inc. (NASDAQ: SLNO), a biopharmaceutical firm dedicated to developing innovative treatments for rare diseases, has announced the publication of significant findings in the peer-reviewed Journal of Clinical Endocrinology and Metabolism (JCEM). The study, titled “Diazoxide Choline Extended-Release Tablets in Prader-Willi Syndrome: A Randomized, Double-Blind, Placebo-Controlled, Withdrawal Period Study,” discusses a comprehensive 16-week study of VYKAT™ XR (diazoxide choline extended-release tablets, or DCCR), aimed at understanding its effects on hyperphagia in Prader-Willi Syndrome (PWS).
Study Overview and Key Findings
The randomized withdrawal study (C602-RWP) included 77 participants aged four years and older, all diagnosed with hyperphagia due to PWS. The study is a vital component of Soleno’s Phase 3 clinical program that showcased the safety and effectiveness of VYKAT™ XR, which has since received approval from the U.S. Food and Drug Administration (FDA) as the first treatment for hyperphagia in PWS.
Dr. Anish Bhatnagar, CEO of Soleno Therapeutics, remarked, “This important study provided additional controlled data confirming the safety and efficacy of VYKAT XR in people with PWS. We are pleased that the positive results have been accepted for publication in JCEM, the world's leading journal focused on endocrine and metabolic research.”
Clinical Implications of VYKAT XR
According to Dr. Jennifer Miller, the lead author and Professor of Pediatric Endocrinology at the University of Florida, “The compelling results further reinforce the meaningful and sustained benefit of VYKAT XR. When participants were switched to placebo, we noted a significant worsening in hyperphagia in contrast to those who remained on VYKAT XR.”
- Primary Endpoint: Hyperphagia Questionnaire for Clinical Trials (HQ-CT) total score change from baseline to week 16.
- Secondary Endpoints: Clinical Global Impression of Severity (CGI-S) and Improvement (CGI-I).
- Exploratory Endpoints: Weight and body mass index (BMI) z-score.
Significant Results from the Study
The study yielded several noteworthy findings:
- Patients experienced a statistically significant worsening of hyperphagia when switched to placebo (P=0.0022).
- The CGI-S and CGI-I scores showed a tendency favoring VYKAT XR, although they did not achieve statistical significance.
- Participants in the placebo group gained more weight and had an increased BMI z-score compared to those who continued on VYKAT XR.
Adverse events were similarly reported in both treatment groups, with no serious adverse events noted in the VYKAT XR cohort. Importantly, no subjects discontinued participation due to adverse effects.
Understanding Prader-Willi Syndrome (PWS)
PWS is a rare genetic disorder stemming from an abnormality on chromosome 15, affecting approximately one in every 15,000 live births. Individuals with PWS often suffer from hyperphagia, a condition that can pose severe health risks, such as obesity, diabetes, and other comorbidities.
About VYKAT XR
VYKAT XR received FDA approval on March 26, 2025, and is currently accessible to patients in the U.S. It represents a breakthrough in addressing hyperphagia in patients aged four years and older suffering from PWS.
Important Safety Information
VYKAT XR is contraindicated in patients with known hypersensitivity to diazoxide or any of its components. Potential side effects include hyperglycemia and edema, necessitating regular monitoring for these conditions.
About Soleno Therapeutics, Inc.
Soleno Therapeutics is committed to developing and commercializing innovative treatments for rare diseases. Their flagship product, VYKAT™ XR, signifies a major advance in the management of hyperphagia associated with PWS. More information can be found on their website, www.soleno.life.
For further details on the study and its implications for the treatment of PWS, please refer to the full publication in JCEM.