Prader-Willi Treatment Enters Phase 3

Soleno Therapeutics, Inc. announced this morning that its multi-center Phase 3 clinical trial of Diazoxide Choline Controlled-Release (DCCR) tablets for the treatment of Prader-Willi Syndrome has been initiated.

The potential therapy is being evaluated at the Seattle Children’s Hospital, and Parisa Salehi, MD, is serving as the trial’s principal investigator at this first site.

The Phase 3 clinical trial is a multi-center, randomized, double-blind, placebo-controlled study that will treat an estimated 100 PWS patients at between 10 and 15 sites in the US. This trial is expected to take approximately 9-12 months to complete. DCCR has previously been orphan drug designation for the indication in both the US and in the European Union (EU).

“Prader-Willi Syndrome leads to hyperphagia that can cause life-threatening obesity if left uncontrolled,” said Dr Salehi in a press release. “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 III trial.”

DCCR tablets are made up of novel, proprietary extended-release, crystalline salt formulation of diazoxide, and they are intended to be taken daily by individuals with Prader-Willi syndrome. The parent molecule of the drug, diazoxide, has been used for decades in thousands of patients in a few rare diseases in neonates, infants, children and adults, but has never been approved in the US for this indication.

Soleno thought of and initiated widespread patent protection on the therapeutic use of diazoxide and DCCR in patients with Prader-Willi syndrome. The DCCR development program is supported by positive data from 5 completed Phase 1 clinical studies in an array of metabolic indications or in healthy volunteers and 3 completed Phase 2 clinical studies, one of which was in PWS patients.

In the PWS Phase 2 study, DCCR showed potential in addressing hyperphagia — a chronic feeling of insatiable hunger – the hallmark symptom of PWS.

“The initiation of the Phase III clinical trial of DCCR for the treatment of PWS represents a significant milestone for Soleno,” said Anish Bhatnagar, M.D., 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.”

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