CAMBRIDGE, Mass., Dec. 5, 2013 /PRNewswire/ -- Catabasis Pharmaceuticals, Inc., today announced the initiation of a Phase 2 clinical trial investigating the safety and efficacy of CAT-2003 in patients with severe hypertriglyceridemia, defined as serum triglyceride levels of 500 mg/dL or greater. CAT-2003 is a conjugate of niacin and the omega 3 fatty acid, eicosapentaenoic acid (EPA), employing Catabasis' proprietary SMART Linker technology.
In this second Phase 2 trial of CAT-2003, patients with severe hypertriglyceridemia, either naive or refractory to current therapy, will be treated for 28 days. The primary endpoint of the trial is change from baseline in fasting triglycerides. Secondary endpoints include changes from baseline in postprandial total and chylomicron triglyceride levels. Catabasis expects top-line results from the trial to be available in mid-2014.
"Patients with treatment-refractory severe hypertriglyceridemia are at a significantly-elevated risk for pancreatitis, a dangerous and difficult-to-manage condition that is often driven by bursts of postprandial triglyceride levels," said Jill Milne, Ph.D., co-founder and chief executive officer of Catabasis. "We believe that CAT-2003 may effectively reduce both fasting and postprandial triglyceride levels in these patients, potentially offering a differentiated efficacy profile."
The Phase 2 clinical trial program for CAT-2003 includes an ongoing Phase 2 trial in patients with hypertriglyceridemia and in combination with statins in patients with hypercholesterolemia (News Release – Aug. 21, 2013).
For more information on the CAT-2003 Phase 2 program, please visit clinicaltrials.gov.
CAT-2003 is a new chemical entity that is a SMART Linker conjugate of niacin and the omega 3 fatty acid EPA. CAT-2003 is being investigated for the treatment of severe hypertriglyceridemia and hypercholesterolemia. In animal models of severe hypertriglyceridemia, significant and dose-dependent reductions in plasma triglycerides were observed with CAT-2003. In animal models of dyslipidemia, CAT-2003 dramatically reduced LDL cholesterol. In a Phase 1 trial, CAT-2003 demonstrated significant reductions in fasting and post-prandial triglyceride levels and had positive effects on lipid biomarkers including reductions in LDL, apolipoprotein C-III (ApoC-III), ApoB and PCSK9.
Catabasis is a clinical-stage company dedicated to the discovery and development of innovative 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 with the goal of producing new chemical entities with significantly enhanced efficacy and improved safety and tolerability profiles. 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.