Essentialis Kickstarts DCCR Clinical Study In Prader-Willi Syndrome

Essentialis reported that it has started the clinical study PC025 of diazoxide choline controlled release tablets (DCCR) in obese Prader-Willi syndrome patients. The first patient received treatment started last Friday.

DCCR is a once a day controlled-release oral tablet medication, composed of crystalline salt of diazoxide. The drug is being developed for the treatment of Prader-Willi syndrome and hypothalamic obesity. The company currently holds multiple patents in the U.S., EU, and Japan protecting DCCR’s composition of matter until 2028.

The single-center, randomized withdrawal study PC025 will evaluate DCCR versus placebo in genetically confirmed, obese PWS patients. Trial endpoints include safety measures, hyperphagia changes, weight, fat mass and resting energy expenditure. The endpoints will be assessed during the open label titration phase and the randomized withdrawal phase of the trial.

Dr. Neil M. Cowen, President and CSO of Essentialis, said, “Although we are announcing today the dosing of the first patient, fully one third of the patients for the study have already been qualified and are scheduled for dosing and another third are currently scheduled for screening… We are looking forward to open label data from this study being available starting in July and double-blind, placebo-controlled results in Q4.” Dr. Cowen also thanked the company’s partners, which include the University of California, Irvine, the Prader-Willi California Foundation, The Foundation for Prader-Willi Research, and the Children's Hospital of Orange County.

Prader-Willi syndrome affects about one in 15,000 to one in 25,000 individuals. The disease is characterized by poor feeding in infancy, hypotonia, low muscle mass, and low resting energy expenditure throughout the patient’s life. The central neurological defect associated with the condition also causes mental retardation, growth hormone deficiency, behavioral problems, and neuroendocrine abnormalities. The disease may affect as many as 350,000 around the world, whose death rate is about twice that of the normal population.