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Novel therapy for Prader-Willi syndrome advances to phase 3 trial

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Two presentations at the 2018 Foundation for Prader-Willi Research Annual Conference in Las Vegas detailed research updates on a diazoxide choline controlled-release tablet which could potentially mediate the hallmark symptoms of Prader-Willi syndrome, according to a press release from Soleno Therapeutics. The diazoxide choline controlled-release (DCCR) tablet, developed by Soleno Therapeutics, is a proprietary extended-release, crystalline salt formulation of diazoxide, administered once-daily. The DCCR development program is supported by five phase 1 trials and three phase 2 trials. Soleno is now preparing for a phase 3 trial based on findings from these studies, some of which were presented during the conference.

Neil Cowen, PhD, senior vice president of drug development for Soleno, discussed the underlying neural mechanism in hyperphagia and how excessive secretion of neurotransmitters NPY and AgRP may lead to the condition. According to Cowen, DCCR lowered these secretions and reduced hyperphagia in animals. Similar results were observed in a phase 2 trial, he said.

Jennifer Abuzzahab, MD, of the Children’s Hospital and Clinics of Minnesota, department of diabetes and endocrinology, examined DCCR’s dosing in comparison with diazoxide. She noted that DCCR produced a similar frequency of adverse events as diazoxide and that dosing for more than 10 days improved safety.

“The data presented on DCCR at this year’s [Foundation for Prader-Willi Research Conference] continue to support the design of our ongoing phase 3 trial,” Anish Bhatnagar, MD, CEO of Soleno Therapeutics, said in a press release. “These results indicate that DCCR targets the underlying neural mechanisms of hyperphagia in [Prader-Willi syndrome]. Published preclinical and phase 2 clinical data provide evidence-backed rationale for DCCR treating hyperphagia in this patient population.”

Bhatnagar added that the safety and dosing findings from eight clinical studies of DCCR further demonstrate the therapy’s safety profile and the benefit of dose titration, which was utilized in the phase 2 study and is incorporated into the phase 3 trial.

Editor’s note: This article was updated on Oct. 23 to correct the spelling of Anish Bhatnagar, MD.