Catabasis Pharmaceuticals has begun a PATHWAYS I, Phase II trial of CAT-2003, a new chemical entity that is a SMART Linker conjugate, in patients with hypertriglyceridemia and hypercholesterolemia.
The trial will assess the safety, tolerability and efficacy of CAT-2003 in patients with hypertriglyceridemia and in combination with statins in patients with hypercholesterolemia.
Catabasis chief medical officer Dr Joanne Donovan said that the positive Phase I data showed that CAT-2003 significantly reduced fasting and post-prandial triglyceride levels in healthy individuals and produced an improvement in lipid biomarkers such as LDL-C, ApoB and PCSK9.
“Because of these promising results, we are advancing CAT-2003 into Phase 2 to evaluate the drug’s effects in patients with hypertriglyceridemia and hypercholesterolemia,” Donovan added.
A total of 72 patients with moderate hypertriglyceridemia will be randomised with one of three doses of CAT-2003 or placebo for a period four weeks in the first arm of the double-blind, placebo-controlled Phase II study.
Fasting triglycerides and non-HDL-C in the patients will be measured and compared to baseline.
A total of 24 patients with hypercholesterolemia on statins will be administered with either a specified dose of CAT-2003 or placebo once per day for four weeks in the second arm, in which fasting LDL-C levels will be assessed.
Catabasis co-founder and chief scientific officer Dr Michael Jirousek noted that CAT-2003 acts through the intracellular targeting of multiple nodes in the lipid synthesis pathway.
“One arm of this study will focus on evaluating the effect of CAT-2003 on lipid levels in combination with statins in patients with hypercholesterolemia,” Jirousek added.
“As a result of the inhibition of PCSK9 production we have observed with CAT-2003, we may see a synergistic effect with statins in lowering lipids such as LDL.”
The company expects top line data in the fourth quarter of 2013.