Soleno Therapeutics has published results from its pivotal Phase 3 clinical study of VYKAT™ XR (diazoxide choline extended-release tablets, DCCR) in The Journal of Clinical Endocrinology and Metabolism, a peer-reviewed journal for endocrine and metabolic research.
The publication reports findings related to the treatment of hyperphagia in individuals with Prader-Willi syndrome and provides an opportunity for clinicians, researchers, and the broader medical community to review and assess the data generated from this clinical program.
In March 2025, the U.S. Food and Drug Administration (FDA) approved VYKAT XR for the treatment of hyperphagia in individuals with PWS aged 4 years and older. The JCEM publication provides additional detail on the clinical evidence that supported this approval.
Overview of the Published VYKAT XR (DCCR) Withdrawal Study
The paper, titled “Diazoxide Choline Extended-Release Tablets in Prader-Willi Syndrome: A Randomized, Double-Blind, Placebo-Controlled, Withdrawal Period Study,” describes results from Soleno's 16-week randomized withdrawal study that included 77 children and adults with PWS who had previously participated in earlier Phase 3 trials of VYKAT XR (DCCR). Participants were randomly assigned to either continue treatment with VYKAT XR or to transition to placebo.
Key findings reported in the publication include:
- Hyperphagia worsened significantly when treatment with DCCR was withdrawn, compared to continued DCCR administration.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.
According to the study authors, the return of hyperphagia symptoms following treatment withdrawal suggests that continued therapy may be necessary to maintain observed effects.
Looking Ahead - Optimizing VYKAT Use Through the Global PWS Registry
To understand how VYKAT works in the real world, we will be collecting data on its use and impact through the Global PWS Registry. Collecting data through the Registry can help us better understand treatment experiences outside of clinical trials, including potential benefits, risks, and variability across individuals. By sharing your VYKAT experience in the registry, you will help us build an understanding of how to optimize the use of VYKAT in the PWS population. Visit the registry today at www.pwsregistry.org to enroll and complete your surveys!
