CAT-2054 is an oral small molecule that modulates the SREBP pathway. SREBP is a master regulator of lipid metabolism that impacts LDL, triglycerides and glucose with the potential to impact liver fat and hepatocellular carcinoma.
The CAT-2054 Phase 2a trial is a 4-week randomized, double-blind, placebo-controlled trial in patients with hypercholesterolemia. Catabasis plans to enroll approximately 150 patients that will receive one of four doses of CAT-2054 or placebo, both in addition to a stable dose of a high intensity statin, atorvastatin 40 mg per day.
The primary efficacy endpoint for this trial will be percent reduction in LDL cholesterol. Catabasis will also explore the activity of CAT-2054 on other metabolic parameters such as triglycerides and glucose.
Louisville Metabolic and Atherosclerosis Research Center medical director / president Harold Bays said: "Compared with currently approved cholesterol drugs, CAT-2054 represents a novel pharmacologic approach to treating dyslipidemia.
"Based on the encouraging Phase 1 study results, and the continued unmet need in treating patients with abnormal lipid levels, we look forward to participating as a research site in evaluating this agent."
Catabasis Pharmaceuticals chief medical officer Joanne Donovan said: "CAT-2054, via its modulation of SREBP activity, has the potential to impact LDL cholesterol, triglycerides and glucose metabolism.
"The initiation of the Phase 2a trial is an important milestone in the development of CAT-2054 and signifies that we are one step closer to potentially providing a novel oral treatment option to patients with hypercholesterolemia."