The double-blind, placebo-controlled study randomized 153 patients into four treatment arms. All participants were on statin therapy and had LDL-C levels between 70 and 190 mg/dL.
At baseline, mean LDL-C levels were 103 mg/dL. 30% of patients had type 2 diabetes and 60% had metabolic syndrome.
Following four weeks of treatment, the maximum LDL-C decrease observed was 7.1% compared to 4.5% for placebo. Triglyceride and non-HDL cholesterol levels did not change significantly.
Additional pre-specified analyses included changes in liver function tests to assess potential activity in nonalcoholic steatohepatitis (NASH).
Catabasis Pharmaceuticals CEO Jill Milne said: "While CAT-2054 lowered LDL-C in normal healthy volunteers in its Phase 1 clinical trial, this effect was not observed in this Phase 2a trial in patients with hypercholesterolemia who were also on high-intensity statins.
"The trial was well designed and efficiently run, enabling us to quickly determine that we do not expect to invest further in CAT-2054 for hypercholesterolemia."
The company intends to complete further analysis of the data and identify the best path forward for CAT-2054 in NASH.
CAT-2054 is an oral small molecule that inhibits a protein called Sterol Regulatory Element-Binding Protein, a master regulator of lipid metabolism in the body.