Essentialis, Inc., a pharmaceutical company, engages in the development of medicines for the treatment of cardiovascular and metabolic diseases. It offers DCCR, a once-a-day tablet formulation for patients with very high triglycerides. The company was founded in 2003 and is based in San Diego, California.
9393 Towne Centre Drive
San Diego, CA 92121
Founded in 2003
Essentialis Presents Results From the First Phase of Clinical Study PC025 Evaluating DCCR in the Treatment of Prader-Willi Syndrome at ACMG Annual Clinical Genetic Meeting
Mar 26 15
Essentialis announced that the principal investigator for clinical study PC025 will be presenting a summary of the results of the first phase of clinical study PC025 at the Annual Clinical Genetics Meeting of the American College of Medical Genetics and Genomics. Clinical study PC025 is designed to evaluate the safety and efficacy of DCCR in obese Prader-Willi syndrome (PWS) patients. PWS is a complex multi-system genetic disorder affecting 1:10,000 to 1:30,000 males and females and people of all ethnicities equally. Initial clinical features of PWS include hypotonia leading to poor suck, difficulty feeding and failure to thrive in infancy. Low muscle mass and low resting energy expenditure is present throughout life. The central neurological defect of the disease causes PWS patients to sense that they are starving and signals them to further conserve energy and to significantly increase their caloric intake resulting in hyperphagia, morbid obesity, and an increased incidence of diabetes. Developmental delay/mild intellectual disability, growth hormone deficiency, behavioral problems, including aggressive and threatening behaviors, mental illness and neuroendocrine abnormalities are frequently observed. Clinical study PC025, a randomized withdrawal study, enrolled 13 obese, genetically confirmed, PWS patients between the ages of 10 and 22. The first phase involved 10 weeks of open label treatment during which patients were titrated up on dose and then stably treated for the remainder of the 10 weeks. This is followed by a 4 week double-blind, placebo-controlled treatment phase. The endpoints in the first phase included changes in body composition (body fat, lean body mass, lean body mass/fat mass ratio), changes in hyperphagia, changes in behavior, and safety. Five female and 8 male patients were randomized in the study. On average, they were 16 years old, weighed 89 kg, had 52% body fat, and moderate hyperphagia. Six of the 13 patients in the study were treated with growth hormone at baseline and continued that treatment throughout the study. Nearly all were cared for in the home and there was no per protocol change in caloric intake or exercise.
Essentialis, Inc. Secures Foundation for Prader-Willi Research Grant to Help Advance DCCR in the Treatment of Prader-Willi Syndrome
Jul 23 14
Essentialis, Inc. announced the receipt of a grant from the Foundation for Prader-Willi Research to support clinical study PC025, which is designed to evaluate the safety and efficacy of diazoxide choline controlled release tablets in obese Prader-Willi syndrome patients. The clinical study is being conducted at the University of California, Irvine under the direction of Dr. Virginia Kimonis.