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.
The company’s lead product targets the ATP-sensitive potassium channel, a metabolically-regulated membrane protein whose modulation has the potential to impact a wide range of rare metabolic, cardiovascular and CNS diseases.
The company has tested Diazoxide Choline Controlled-Release Tablet (DCCR), in 7 clinical studies and has recently advanced DCCR into a pilot study in Prader-Willi syndrome, a complex metabolic/neurobehavioral disorder.More
DCCR is a novel, crystalline patent-protected salt of diazoxide formulated as a controlled release once-a-day tablet. Diazoxide free base is approved as a three-times-a-day oral suspension (Proglycem®) that has been used safely for decades in tens of thousands of patients. Diazoxide is first line therapy in a range of orphan indications in neonates, children and adults. Over the last 40 years, there have been more than 3500 peer reviewed publications covering in-vitro, animal model, and clinical results with diazoxide.More
Essentialis recently completed clinical study PC025. It was a randomized withdrawal study designed to evaluate the safety and efficacy of DCCR in Prader-Willi syndrome (PWS) patients. The study enrolled 13 male and female genetically-confirmed PWS subjects between the ages of 10 and 22 years old. Subjects were titrated through several doses of DCCR over 70 days (10 weeks) of open-label treatment. If, at the end of the 10 weeks of open-label treatment, subjects responded to DCCR with a reduction in hyperphagia or an increase in resting energy expenditure, they were eligible to participate in the double-blind, placebo-controlled, randomized withdrawal phase of the study. In that phase, half of the patients were randomized to continue treatment on active drug and half were randomized to receive placebo. The duration of the randomized withdrawal phase was 28 days. Across both phases of the study, subjects were treated for a total of 98 days, or more than 3 months. The study was conducted at a single site, the University of California, Irvine under the direction of Dr. Virginia Kimonis, a PWS expert.More
Prader–Willi syndrome (PWS) is a complex neurobehavioral/metabolic disorder characterized by hypotonia and poor feeding in infancy, followed by a period of excess weight gain, eventually progressing to hyperphagia and morbid obesity.
When the hypothalamus is damaged, a syndrome of intractable weight gain can ensue, termed hypothalamic obesity. Hypothalamic obesity can occur due to the presence of a tumor in or near the hypothalamus, associated with surgery to resect it or due to subsequent radiation therapy.More