FDA Confirms Soleno's Plan to Initiate Phase III Clinical Study to Treat PWS

Soleno Therapeutics has completed its End-of-Phase II meeting with the FDA regarding the biopharmaceutical company’s drug, Diazoxide Choline Controlled-Release (DCCR), a therapy used to treat patients with Prader-Willi syndrome (PWS).

The genetic condition begins during infancy, causing patients to have severe hypotonia, feeding difficulties, and slow growth. As patients mature into children, they will begin to have feelings of insatiable hunger and eat excessively, a dangerous characteristic defined as hyperphagia. This can cause a series of complications including diabetes, obesity, cardiovascular disease, and the stomach ruptures.

Approximately 12,000 to 15,000 people in the United States are diagnosed with PWS.

PWS patients are characterized as having short stature, hypogonadism, developmental delays, cognitive impairment, and distinctive behavioral characteristics. Children will have frequent temper tantrums, show extreme stubbornness, and obsessive-compulsive tendencies.

It is uncommon for PWS to be inherited, as the mutation of the gene is due to events during the formation of egg or sperm cells. The condition is caused by a loss of active genes in chromosome 15, causing patients to have sleep abnormalities, intellectual disability, small hands and feet, and distinctive facial features.

There are no available therapies on the market that can treat appetite, cognitive function, or behavioral aspects in PWS patients.

DCCR tablet is a novel crystalline salt formulation of diazoxide which is to be administered to patients once daily. Diazoxide has been used in the past as an oral suspension to treat other rare diseases in infants, children and adults. This is the first time diazoxide has been tested to treat patients with PWS.

DCCR has already received Orphan Drug Designation indicated to treat PWS in the United States and the European Union.

Soleno has completed 5 Phase I clinical trials for metabolic indications and 3 Phase II clinical trials, in which one evaluated the effect of the drug in PWS patients. Researchers found that DCCR showed potential success in addressing the main symptoms of PWS, specifically hyperphagia.

“Alignment with the FDA on our Phase III clinical trial design of DCCR in PWS allows us to confidently initiate this study,” said Anish Bhatnagar, M.D., Chief Executive Officer of Soleno in a press release. “If approved, we believe DCCR has the potential to be a safe and effective treatment for PWS, a rare and life-threatening disease with significant unmet needs.”

The meeting between the FDA and Soleno confirmed that the Phase III clinical study of DCCR in treating symptoms of PWS will be initiated in the near future.