Ionis Pharmaceuticals, Inc. has said that the AstraZeneca has commenced a Phase 2b clinical trial of ION449 (AZD8233).
It is an investigational antisense medicine designed to bring down blood cholesterol levels in patients with dyslipidemia by targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), an important regulator of low-density lipoprotein cholesterol (LDL-C).
An enzyme, PCSK9 controls the number of LDL receptors on the surface of cells. People with genetic variations that reduce PCSK9 function have lower LDL-C levels in the blood and a lower risk for major cardiovascular events.
ION449 is a LIgand Conjugated Antisense (LICA) medicine being developed by AstraZeneca as part of its collaboration with Ionis.
Sotirios “Sam” Tsimikas, M.D., senior vice president, clinical development and cardiovascular franchise leader at Ionis, said, “Results from the Phase 1 study showed that ION449 potently reduces PCSK9 and LDL cholesterol. ION449 demonstrated best-in-class potential for PCSK9 inhibition and LDL-C reduction, supporting larger clinical trials that are now underway to further evaluate efficacy and safety.”
He added: “”The growing evidence supporting Ionis’ advanced LICA technology in cardiovascular disease holds promise for more effective approaches to lower LDL-C and to address cardiovascular disease, the leading cause of death worldwide.”
The randomized, double-blind, placebo-controlled Phase 2b trial will enroll approximately 108 participants, aged 18-75, who have LDL-C levels between 70 and 190 mg/dL and are receiving moderate- or high-intensity statin therapy as defined by the American College of Cardiology/American Heart Association (ACC/AHA) guidelines on blood cholesterol management.
The main objective is to assess the effect of different doses of ION449 on LDL-C compared to placebo at Week 12 in patients taking baseline statin therapy.
The research will analyse three dose levels of ION449 versus placebo, all administered once a month by subcutaneous injection. Safety and tolerability will be evaluated along with a number of secondary endpoints.
(With inputs from The OnLook News Research Bureau)