Additional Relevant Details
The CAT-2054 Phase 2a trial was a randomized, double-blind, placebo-controlled trial conducted at 31 clinical trial sites that enrolled 153 patients with hypercholesterolemia on statins. After a 4-week run-in on atorvastatin 40 mg, patients were randomized into one of four treatment groups (CAT-2054 250 mg or 400 mg once daily or twice daily) or placebo. The patients in this trial had hypercholesterolemia on statins with LDL cholesterol levels over 70 and less than 190 mg/dL. At baseline, mean LDL levels were 103 mg/dL, 30% of the patients had type 2 diabetes and 60% had metabolic syndrome. After four weeks of dosing with CAT-2054, the maximum LDL-C decrease observed was 7.1% compared to 4.5% with placebo. Additional secondary lipid end points included triglycerides and non-HDL cholesterol and these did not change significantly with treatment. In one pre-specified analysis, in a subset of the patients more likely to have NASH as determined by FIB-4 (Fibrosis-4, a non-invasive scoring system based on several laboratory tests that estimate the amount of scarring in the liver), a potentially favorable pattern in LFTs was seen. In this subset of patients (N=51), from a mean baseline ALT of 26 UL/mL there was a mean reduction in ALT of 3.0 IU/mL across all dosing arms of CAT-2054 compared to a reduction of 0.2 IU/mL with placebo and from a mean baseline AST of 25 IU/mL there was a mean reduction in AST of 2.8 IU/mL for those treated with CAT-2054 compared to an increase of 0.2 IU/mL with placebo.