CARLSBAD, Calif., Aug. 11 Essentialis, Inc., a clinical stage pharmaceutical company, announced today its intention to file a Special Protocol Assessment (SPA) with the FDA for the pivotal study to support the NDA filing on its lead drug, DCCR, for the treatment of very high triglycerides. The proposed study would include 280 randomized subjects with the primary efficacy endpoint assessed at 12 weeks. This decision followed a productive Type C meeting with the FDA on July 30th, where the overall development plan for DCCR and the non-clinical package supporting the NDA were reviewed and discussed. Commenting on this development, Essentialis President and CEO Iain Dukes stated, "The submission of the SPA will bring us one step closer towards filing for approval of DCCR for the treatment of patients with very high triglycerides. This development, along with positive Phase 2 study results and the recent issuance of a US patent providing extensive protection to DCCR, underscores the tremendous clinical and commercial potential of this drug worldwide."
DCCR, diazoxide choline controlled release tablet, is an ATP-sensitive potassium channel agonist which has potential therapeutic utility in several cardiovascular and metabolic diseases. Extensive Phase 1 testing has demonstrated the drug's potential to lower triglycerides (TG), non-HDL cholesterol and LDL-cholesterol (LDL-C) while raising HDL-cholesterol. In addition, weight loss has been demonstrated in 2 investigator sponsored studies. A Phase 2, 8-week, placebo-controlled, double-blind study involving 90 patients, which was powered to be pivotal, demonstrated statistically significant reductions in triglycerides, without impacting LDL-C. In subjects with elevated LDL-C at baseline, DCCR reduced LDL-C levels. DCCR was generally well tolerated, and most AEs were mild to moderate, resolving without sequelae. US patent 7,572,789, containing claims to DCCR, was issued on August 11, 2009. Additional applications filed in the US and globally will further enhance the patent portfolio. Phase 2 and pivotal studies of DCCR in combination with a statin for the treatment of high triglycerides (TG between 200 and 500 mg/dL) are planned, and study synopses have been provided to the FDA.
Very high triglycerides (TG greater than or equal to 500mg/dL) are a risk factor for developing pancreatitis. There are approximately 3.8 million patients in the US with very high triglycerides. The leading products for treating very high triglycerides are fenofibrate and omega-3 fatty acids. While effective, these compounds cause significant elevations in LDL-C. High triglycerides (TG between 200 and 500 mg/dL) are a risk factor for coronary heart disease even after LDL-C targets have been achieved.
About Essentialis, Inc.
Essentialis, Inc. is a pharmaceutical company based in Carlsbad, CA. The Company's products are targeted at the ATP-sensitive potassium channel, a metabolically-regulated membrane protein whose modulation has the potential to impact a wide range of cardiovascular and metabolic diseases. The Company's lead product, DCCR, is set to commence pivotal studies in subjects with very high triglycerides. Essentialis is actively exploring its partnering options for DCCR.
SOURCE Essentialis, Inc.