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
Clinical study PC025 is a randomized withdrawal study designed to evaluate the safety and efficacy of DCCR in Prader-Willi syndrome (PWS) patients. The study will enroll male and female genetically-confirmed PWS patients between the ages of 5 and 20 years old. From an efficacy standpoint, the study will focus on the effects of DCCR treatment on hyperphagia, resting energy expenditure, and weight. Patients will be titrated through several doses of DCCR over 70 days of open-label treatment. If, at the end of the 70 days of open-label treatment, patients have responded to DCCR with a reduction in hyperphagia or an increase in resting energy expenditure, they will be eligible to participate in the double-blind, placebo-controlled, randomized withdrawal phase of the study.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