AstraZeneca’s small molecule lowered levels of “bad” cholesterol by more than half in patients who had never received treatment for their condition, according to results from an early-stage trial.
The U.K. pharma presented the results today at the European Atherosclerosis Society in France, according to a release.
AZD0780 is an oral small molecule PCSK9 inhibitor in development for dyslipidaemia, a condition characterized by elevated cholesterol in the blood that can progress to clogged arteries, also called atherosclerosis.
The phase 1 trial is focused on this disease, however, an additional arm is testing the therapy in 35 patients with hypercholesterolaemia, or high cholesterol. High LDL-C, also known as "bad" cholesterol, is associated with a risk of cardiovascular disease. The main goal of the test was the safety, tolerability, pharmacokinetics and pharmacodynamics of AZD0780 in lowering LDL-C.
In the hypercholesterolaemia arm presented at the conference, patients who took AZD0780 as well as a statin had a statistically significant 52% reduction in low-density lipoprotein cholesterol (LDL-C), with a 78% total reduction from baseline.
The trial moved on to phase 2 in patients with dyslipidaemia earlier in the year, according to AstraZeneca.
“AZD0780 inhibits PCSK9 via a novel, previously unexplored, mode of action that is compatible with traditional oral small molecule drug discovery,” said AstraZeneca’s Sharon Barr, executive vice president of biopharmaceuticals R&D, in a statement. “We are progressing development of AZD0780 as an innovative, convenient oral option for patients who are currently unable to meet their LDL-C targets with statins alone to reduce their risk of CV events.”
AstraZeneca is significantly trailing Merck & Co. in the hypercholesterolaemia space. The rival’s oral PCSK9 inhibitor MK-0616 is already in three phase 3 trials to examine its ability to lower LDL-C and lower the risk of cardiovascular events. The primary completion date for the initial two studies in the trio is September 2025. The third trial is looking at cardiovascular outcomes and isn’t expected to wrap up until late 2029, after six years of watching patients.
Both entrants are trying to replace Amgen’s Repatha and Sanofi-Regeneron’s Praluent, which are approved injectables for the same condition.