Soleno to continue Phase III DESTINY PWS study as planned

3rd October 2019 (Last Updated December 23rd, 2019 12:08)

Soleno Therapeutics has received a positive recommendation from the Data Safety Monitoring Board (DSMB) following analysis of interim data from the Phase III DESTINY PWS clinical trial.

Soleno Therapeutics has received a positive recommendation from the Data Safety Monitoring Board (DSMB) following analysis of interim data from the Phase III DESTINY PWS clinical trial.

The trial is assessing a diazoxide choline controlled-release (DCCR) tablet for the treatment of Prader-Willi syndrome (PWS), a genetic disorder characterised by a chronic feeling of insatiable hunger known as hyperphagia.

After a review of results from more than half of participants, the DSMB said that the trial can be continued as planned without any changes.

The randomised, double-blind, placebo-controlled trial is comparing oral, once-daily DCCR with placebo in around 100 patients with a confirmed PWS diagnosis.

The trial’s primary endpoint is the change from baseline hyperphagia score at week 13. Patients completing this study may participate in the open-label safety extension study called C602.

Soleno Therapeutics CEO Dr Anish Bhatnagar said: “We are pleased to again receive the DSMB’s recommendation to continue the Phase III trial as planned and without modification, which provides ongoing support of DCCR’s safety profile. Enrolment in DESTINY PWS remains on track for the availability of top-line data in the first half of 2020.”

DCCR is an extended-release, crystalline salt formulation of diazoxide, used to treat certain rare diseases.

Soleno said that its DCCR development programme is backed by results from five completed Phase I trials conducted in healthy people and three completed Phase II trials, including one in PWS.

According to Phase II data, DCCR demonstrated the ability to address hyperphagia and symptoms such as aggressive/destructive behaviours, fat mass and abnormal lipid profiles in PWS patients.

In July last year, DCCR received fast track designation from the US Food and Drug Administration (FDA) for treating PWS.