Catabasis' CAT-2003 Demonstrates Significant Reduction of Fasting and Post-Prandial Triglycerides in Successful Phase 1 Trial

CAMBRIDGE, Mass., July 30, 2013 /PRNewswire/ -- Catabasis Pharmaceuticals, Inc., today announced that preliminary data from a Phase 1 trial of CAT-2003, an oral SMART Linker conjugate constructed using the company's proprietary technology, demonstrated a significant reduction in fasting and post-prandial triglyceride levels. CAT-2003 also had positive effects on other biomarkers, including reductions in apolipoprotein C-III (ApoC-III), a negative regulator of lipoprotein lipase, and apolipoprotein B (ApoB), the lipoprotein associated with VLDL and LDL cholesterol. In addition, reductions in plasma PCSK9 levels and LDL cholesterol were observed. There were no serious adverse events reported. Data also showed that the conjugated compound was absorbed following oral administration and metabolized into its active components intracellularly within target tissues.

"We believe these data support an enormous opportunity for CAT-2003 in treating patients with hypertriglyceridemia as well as a variety of other dyslipidemias," said Jill Milne, Ph.D., co-founder and chief executive officer of Catabasis. "CAT-2003 was safe and well tolerated at a therapeutic dose and produced reductions in both fasting and post-prandial triglycerides in subjects with triglycerides over 150 mg/dL. We are planning to evaluate CAT-2003 in additional trials in patients with severe hypertriglyceridemia as well as mixed dyslipidemias."

This randomized, double-blind, placebo-controlled Phase 1 study was conducted in two parts in 99 patients. In the first part, 41 healthy adults received a single ascending dose of CAT-2003 or placebo. In the second part, 58 healthy adults and patients with mildly elevated lipids received CAT-2003 or placebo daily for 14 days. The study evaluated safety, tolerability and pharmacokinetics. Triglycerides, LDL cholesterol and biomarkers related to the mechanism of action of CAT-2003 were also assessed. A Phase 2 study evaluating CAT-2003 in patients with hypertriglyceridemia is set for initiation this quarter.

About CAT-2003

CAT-2003 is a new chemical entity that is a SMART Linker conjugate. It is being investigated for the treatment of severe hypertriglyceridemia. In preclinical models of severe hypertriglyceridemia, a significant and dose-dependent reduction in plasma triglycerides was observed with CAT-2003. In preclinical models of dyslipidemia, CAT-2003 dramatically reduced LDL cholesterol. In combination with a statin, CAT-2003 synergistically lowered LDL cholesterol. 

About Hypertriglyceridemia

Severe hypertriglyceridemia refers to a condition involving levels of triglycerides equal to or above 500 mg/dL. Severe hypertriglyceridemia is associated with markedly increased risk for cardiovascular disease and pancreatitis, and recent studies have demonstrated that elevated triglyceride levels can be regarded as an independent risk factor for cardiovascular events such as myocardial infarction, ischemic heart disease and ischemic stroke.  The prevalence of hypertriglyceridemia is rapidly increasing in the United States and throughout the world.  It is estimated that over four million patients have severe hypertriglyceridemia in the United States. The National Cholesterol Education Program (NCEP) Expert Panel on Detection, Evaluation and Treatment of High Blood Cholesterol recommends that the first priority for the management of severe hypertriglyceridemia be triglyceride reduction to decrease the risk of pancreatitis. Current treatments include adhering to a low-fat, low-carbohydrate diet, exercise and alcohol abstinence. Medical therapies can include statins to normalize other lipid parameters, fibrates and omega-3 fatty acids.

About Catabasis

Catabasis is a clinical-stage company dedicated to the discovery and development of innovative, effective and safe medicines to treat inflammatory and metabolic diseases. The company's drug development programs are rooted in the principles of pathway pharmacology, the treatment of diseases by simultaneously modulating more than one target in a disease pathway. Using its proprietary SMART Linker technology, the company conjugates two drugs that act on different components of a disease pathway to produce new chemical entities with significantly enhanced efficacy and an improved safety and tolerability profile. The company has assembled a team of passionate and experienced scientists who are committed to improving the lives of patients. The company was founded in 2008 and is headquartered in Cambridge, Mass.

Please visit www.catabasis.com for more information.

SOURCE Catabasis Pharmaceuticals, Inc.