Essentialis Announces the Dosing of the First Patient in a Clinical Study of DCCR in Prader-Willi Syndrome - Applied Clinical Trials

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Essentialis Announces the Dosing of the First Patient in a Clinical Study of DCCR in Prader-Willi Syndrome


Essentialis Announces the Dosing of the First Patient in a Clinical Study of DCCR in Prader-Willi Syndrome

PR Newswire

CARLSBAD, Calif., June 27, 2014 /PRNewswire/ -- Essentialis announced today the dosing of the first patient in clinical study PC025, which is designed to evaluate the safety and efficacy of diazoxide choline controlled release tablets (DCCR) in obese Prader-Willi syndrome patients. 

"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.  The speed with which patients for this study are being recruited and qualified is a testament to the hard work of Dr. Virginia Kimonis' team and the staff of the ICTS at the University of California, Irvine, and the assistance and encouragement of the Prader-Willi California Foundation, The Foundation for Prader-Willi Research, Children's Hospital of Orange County and the commitment of parents and caregivers of PWS patients," said Dr. Neil M. Cowen, President and Chief Scientific Officer of Essentialis.  "We are looking forward to open label data from this study being available starting in July and double-blind, placebo-controlled results in Q4." 

Trial Design Clinical trial PC025 (www.clinicaltrials.gov, NCT02034071) is a single-center, randomized withdrawal study of DCCR in genetically confirmed, obese PWS patients.  Twelve PWS patients between 10 and 20 years old will be enrolled in the study.  Patients will be titrated through 5 doses of DCCR over 10 weeks of open label treatment followed by a 4 week double-blind, placebo-controlled, randomized withdrawal phase during which half of the patients in the study will be randomized to continue on DCCR and half will be randomized to placebo.  Endpoints include safety measures, and changes in hyperphagia, weight, fat mass and resting energy expenditure.  These endpoints will be measured both during the open label titration phase and the double-blind, placebo-controlled, randomized withdrawal phase. 

About DCCR DCCR is a proprietary crystalline salt of diazoxide formulated as a controlled-release, once-a-day tablet. It is in development for the treatment of Prader-Willi syndrome and hypothalamic obesity. DCCR is covered by multiple issued US and granted EU and Japanese patents, which provide composition of matter protection until 2028. Essentialis is globally prosecuting claims to the use of pharmaceutical formulations of KATP channel activators including diazoxide and DCCR in the treatment of Prader-Willi syndrome. Essentialis has evaluated DCCR in more than 200 subjects in multiple double-blind, placebo-controlled studies.

About Prader-Willi syndrome Prader–Willi syndrome afflicts about 1 in 15,000 to 1 in 25,000 individuals, with the US Prader-Willi syndrome population estimated between 12,500 and 21,000. There may be as many as 350,000 Prader–Willi syndrome patients globally. Prader–Willi syndrome is equally frequent in males and females. Clinical features of Prader–Willi syndrome include hypotonia and poor feeding in infancy. Low muscle mass and low resting energy expenditure is present throughout life. Obesity typically begins around age 2 years if the diet is not restricted. Ultimately, the central neurological defect associated with the condition causes Prader–Willi syndrome patients to sense that they are starving and signals them to further conserve energy and to significantly increase their caloric intake. This results in even lower resting energy expenditure, hyperphagia, morbid obesity, and a progression to diabetes. Mental retardation, growth hormone deficiency, behavioral problems and neuroendocrine abnormalities are also characteristic of Prader–Willi syndrome. The death rate among Prader–Willi syndrome patients is about twice that of the general population at all ages.

About Essentialis, Inc. Essentialis is a Carlsbad-based pharmaceutical company focused on the development of breakthrough medicines targeted to the ATP-sensitive potassium channel, a metabolically regulated membrane protein whose modulation has potential to treat and prevent a wide range of metabolic, CNS and cardiovascular diseases.

For more information visit. http://essentialistherapeutics.com/  

SOURCE Essentialis, Inc.

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