Soleno's drug misses key goal in Prader-Willi trial

Soleno Therapeutics has unveiled new data showing that its diazoxide choline controlled release (DCCR) tablets failed to reduce hyperphagia – or an abnormal desire for food – in patients with Prader-Willi syndrome.

The change was measured by the total score of a Hyperphagia Questionnaire for Clinical Trials (HQ-CT, 0 36), with an improvement being represented by a decrease in the score.

The mean change from baseline was -5.94 and for placebo -4.27, a difference that did not meet statistical significance.

However, significant changes were observed in two of three key secondary endpoints from baseline to week 13 in subjects receiving DCCR as compared to placebo:

• Improvement in Clinical Global Impression of Improvement (CGI-I) score as assessed by the investigator (p=0.029)

• Reduction of body fat mass measured by DXA scan (p=0.025)

Also, in a prespecified subgroup of subjects with more severe hyperphagia, the mean change from baseline for DCCR was significant (-9.67 versus -4.26, respectively).

In addition to the reduction in total body fat mass, other body composition changes in DCCR compared to placebo included significant decreases in trunk fat mass, and improvement in lean body mass to fat mass ratio. Also, Fat mass changes were most pronounced in subjects in the highest weight band.

“Hyperphagia, the predominant symptom of PWS, is an unrelenting hunger that can cause life-threatening co-morbidities, including obesity, and is a condition for which no treatments are available,” said Jennifer Miller, Professor in the Division of Pediatric Endocrinology at the University of Florida and a Principal Investigator in the Soleno study.

“PWS also leads to significant quality-of-life challenges for patients and families. These data show that DCCR therapy results in meaningful improvements in hyperphagia in severe patients, as well as various other positive impacts in behaviors and body composition, and if approved, could offer a safe and effective treatment to PWS patients struggling to manage their symptoms.”