Essentialis Announces the Dosing of the First Prader-Willi Syndrome Patients in a Long-Term Extension to Clinical Trial PC025

Sep 09, 2015, 06:00 ET from Essentialis, Inc.

CARLSBAD, Calif., Sept. 9, 2015 /PRNewswire/ -- Essentialis announced today the dosing of the first Prader-Willi syndrome (PWS) patients in the long-term extension to clinical study PC025.  Patients who completed the first two phases of clinical study PC025 are currently being enrolled in a 6-month open label extension.  The objective of the extension is to document the long-term therapeutic benefits of DCCR, Essentialis' lead drug, on hyperphagia, body fat, lean body mass, aggressive, threatening and destructive behaviors and on stamina.  The 6-month extension is being run by Dr. Virginia Kimonis' team and the staff of the ICTS at the University of California, Irvine.

"Given that we observed highly significant and clinically relevant reductions in hyperphagia, reductions in body fat, increases in lean body mass, reductions in aggressive, threatening and destructive behaviors and reductions in cardiovascular risk factors, with a safety profile that was well matched against these benefits, it is not surprising that nearly every patient who completed the first two phases of the study wanted to continue on drug," said Dr. Neil M. Cowen, President and Chief Scientific Officer of Essentialis.  "We are looking forward to data from this long-term extension being available in Q1 of 2016." 

About DCCR DCCR is a controlled-release, once-a-day tablet. It is in development for the treatment of PWS. DCCR is covered by multiple issued US and granted EU and Japanese patents, which provide composition of matter protection until 2028. The company has patent applications which could extend protection to 2035. Essentialis has evaluated DCCR in more than 200 subjects in multiple double-blind, placebo-controlled studies.

About Prader-Willi syndrome PWS afflicts about 1 in 15,000 to 1 in 25,000 individuals, with the US PWS population estimated between 12,500 and 21,000. There may be as many as 350,000 PWS patients globally. Clinical features of PWS include low muscle mass and hypotonia throughout life. The accumulation of excess body fat begins around age 2 years and continues into adulthood. Ultimately, the central neurological defect associated with the condition causes PWS patients to sense that they are starving and signals them to significantly increase their caloric intake. This results in hyperphagia, difficult to manage food-related behaviors and an acceleration of the accumulation of excess body fat. Mental retardation, growth hormone deficiency, behavioral problems and neuroendocrine abnormalities are also characteristic of PWS. The death rate among PWS patients is about twice that of the general population at all ages.

About Essentialis, Inc. Essentialis is a clinical stage biotechnology company focused on the development of breakthrough medicines for the treatment of rare metabolic diseases where there is increased mortality and risk of cardiovascular and endocrine complications.

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SOURCE Essentialis, Inc.