Prader-Willi Syndrome Investigational Therapy DCCR Enters Phase 3 Trial

A multicenter, Phase 3 clinical trial assessing diazoxide choline controlled-release (DCCR) for the treatment of Prader-Willi syndrome (PWS) recently was initiated, the trial’s sponsor, biopharmaceutical company Soleno Therapeutics, announced.

Soleno is currently developing DCCR as a once-daily oral tablet for the treatment of PWS. The tablet is made of a new, extended-release, crystalline salt formulation of diazoxide. Diazoxide has been used in thousands of patients for a few rare diseases but has not yet been approved for PWS.

According to Soleno, DCCR can reduce the levels of appetite-stimulating neuropeptides, thereby reducing hyperphagia, or excessive eating. The company says DCCR also helps limit aggressive and other behavioral complications associated with PWS.

DCCR has received orphan drug designation for the treatment of PWS in the United States and in Europe.

Soleno is now advancing its DCCR development program into a Phase 3 trial, based on positive data from five completed Phase 1 clinical studies for various metabolic indications and in healthy volunteers, and from three completed Phase 2 studies, including one with PWS patients.

In the Phase 2 trial with PWS patients, DCCR showed promise in addressing hyperphagia (overeating), the hallmark of PWS.

“Prader-Willi Syndrome leads to hyperphagia that can cause life-threatening obesity if left uncontrolled,” Parisa Salehi, MD, the principal investigator leading the trial at Seattle Children’s Hospital, said in a press release. The Seattle hospital will be the first site to launch the trial.

“This excessive hunger can cause significant harm to the lives of these individuals and their families. There is a lack of effective medical therapy targeting hunger in this population, and such a drug would be life-altering. Based on the data generated to date, DCCR has the potential to address this treatment void. We look forward to further evaluating DCCR in this important Phase 3 trial,” Salehi added.

The Phase 3 clinical trial (NCT03440814), which is now recruiting, is a randomized, double-blind, placebo-controlled study to evaluate DCCR’s effects in children 8 and older and adults with PWS.

The study will enroll approximately 100 PWS patients at 10-15 sites in the United States for an estimated period of nine to 12 months.

“The initiation of the Phase 3 clinical trial of DCCR for the treatment of PWS represents a significant milestone for Soleno,” said Anish Bhatnagar, MD, chief executive officer of Soleno. “Importantly, following meetings with the U.S. Food and Drug Administration, we have alignment with the agency on the key aspects of the Phase 3 clinical trial. We look forward to working with our clinical trial sites and the PWS community to successfully complete the trial.”

Soleno met with the FDA last February after successfully completing the Phase 2 trial in PWS. According to the company, both the FDA and Soleno are aligned on key aspects of the company’s Phase 3 clinical trial for DCCR.