Soleno Therapeutics recently released an update on the company’s achievements during 2017 and plans for its investigational therapeutic for Prader-Willi syndrome (PWS), a condition currently without an approved therapy.
One of the highlights for the next steps in PWS is the FDA’s agreement with Soleno’s for a Phase 3 clinical trial in PWS to continue to test the company’s leading therapeutic, diazoxide choline controlled-release (DCCR), after the positive results of a Phase 2 trial.
DCCR is an extended-release, crystalline salt formulation of diazoxide designed to be administered once-daily in the form of a tablet. Diazoxide is a channel agonist capable of crossing the brain’s blood-brain barrier and has been used for decades in certain rare diseases, but never in PWS.
The PWS Phase 2 study (PC025), enrolled 13 obese subjects with confirmed PWS between the ages of 11 and 21.
Treatment of PWS adolescents and adults with DCCR resulted in marked and sustained improvements in hyperphagia (abnormally increased appetite) and reductions in aggressive/threatening behavior. Moreover, researchers also detected improvements in cardiovascular risk factors and insulin sensitivity, as well as a reduction in body fat and increases in lean body mass.
The promising results earned DCCR the European Medicines Agency (EMA) orphan drug designation for PWS, which facilitates the development and authorization of medicines for rare diseases.
Moreover, the EMA indicated that results from a single pivotal trial would support an application for DCCR approval — the so-called Marketing Authorization Application.
The results also have led to two new patents in the U.S. for DCCR, and pharmaceutical formulations of diazoxide and its salts, such as diazoxide choline, as a potential therapy for PWS — one addressing specifically DCCR’s role in hyperphagia, and the other to reduce one or more aggressive behaviors.
“The past 12 months have been transformative for our company,” Anish Bhatnagar, MD, Soleno CEO, said in a press release. “We have advanced our lead product candidate [DCCR] … for the treatment of Prader-Willi Syndrome, towards a Phase 3 clinical trial, which we expect to initiate shortly. As a result of meetings with the U.S. Food and Drug Administration for DCCR in PWS, we now have alignment with the agency on key aspects of the planned Phase 3 clinical trial.”
“In addition, we have strengthened our intellectual property around DCCR, and our lead product candidate received designation as an orphan medicinal product for the treatment of PWS in the European Union,” Bhatnagar added. “We closed 2017 by securing a $15 million private placement led by leading healthcare-focused investors, and our DCCR clinical development program is now supported by a strong balance sheet.”