Soleno Therapeutics announced the awarding of a patent for diazoxide choline controlled-release (DCCR) tablets, the company’s investigational therapy for Prader-Willi syndrome (PWS) being evaluated in a new Phase 3 trial. The patent covers “pharmaceutical formulations of diazoxide and diazoxide choline” to treat patients with poor lean body mass and a lean body mass/fat mass ratio.
Low muscle mass is a major characteristic of patients with PWS, and it results in low lean body mass and weak muscles, a condition called hypotonia, which can limit patients’ ability to perform day-to-day activities and lead independent and reasonably active lives.
DCCR is an oral, extended-release, crystalline salt formulation of diazoxide — a potassium channel activator or opener — that has been shown to significantly decrease fat mass and increase lean body mass in preclinical and Phase 1/2 clinical (PC025, NCT02034071) studies.
The new patent, No. 10/058,557, was issued by the U.S. Patent and Trademark Office.
“This patent further strengthens Soleno’s intellectual property portfolio in the treatment of PWS,” Anish Bhatnagar, MD, chief executive officer of Soleno Therapeutics, said in a press release.
“Low lean body mass is a significant challenge for PWS patients, impacting both physical activity and metabolism. We hope that treatment with DCCR will result in increases in lean body mass and are currently evaluating this as an outcome measure in our Phase 3 trial,” Bhatnagar added.
The Phase 3 clinical trial (NCT03440814) is now recruiting about 105 children and adults with PWS to evaluate DCCR given once daily (dose range, 75 to 450 mg) against placebo. Its primary outcome is changes in hyperphagia (excessive eating)-related behavior based on a score drawn from an established questionnaire. Changes in body fat mass, and clinical and caregiver perceptions of those changes are secondary study endpoints.
Enrollment is underway at five U.S. sites. For more information about this trial and how to participate, please click here.
The U.S. Food and Drug Administration (FDA) granted DCCR Fast Track designation in July, a status designed to accelerate the development and review of new medicines to provide quicker access to them. It was previously designated an orphan drug in both the U.S. and Europe as a potential PWS treatment.
Diazoxide, as an oral suspension medication, is approved to treat hypoglycemia (low blood sugar) caused by certain cancers and other diseases or conditions.