Novartis Plans Leqvio Expansion After Phase III Cholesterol Success

Novartis is making strides in the treatment of atherosclerotic cardiovascular disease with its siRNA therapy, Leqvio, following promising results from the Phase III V-MONO study. Released recently, the findings suggest significant reductions in low-density lipoprotein cholesterol (LDL-C) levels, often referred to as "bad" cholesterol.

The V-MONO study, a randomized and double-blinded trial, evaluated Leqvio against a placebo and the lipid-lowering drug ezetimibe. The study enrolled 350 patients who were at low or moderate risk of developing atherosclerotic cardiovascular disease (ASCVD) and were not on any lipid-lowering therapies. The primary endpoint was the percentage decrease in LDL-C after 150 days. According to Novartis, Leqvio achieved a “clinically meaningful and statistically significant” reduction in LDL-C levels compared to both the placebo and ezetimibe. While the company did not disclose specific data points, it plans to reveal more detailed results at an upcoming scientific congress.

Novartis also intends to share these findings with the U.S. Food and Drug Administration (FDA) in hopes of expanding Leqvio’s label to include preventive use for a broader population. This could potentially allow the therapy to be used as a preventive measure against ASCVD.

Chief Medical Officer Shreeram Aradhye highlighted the importance of the trial’s results, noting that they add to the growing body of evidence supporting Leqvio’s efficacy across the spectrum of ASCVD. Aradhye emphasized Novartis’ commitment to advancing scientific understanding and helping patients who struggle to meet their cholesterol targets.

Leqvio is designed for subcutaneous administration and employs siRNA technology to target liver cells. Once inside the hepatocytes, Leqvio targets and degrades the mRNA that encodes for PCSK9, a protein crucial in regulating the number of cholesterol receptors on liver cells. By reducing PCSK9 levels, Leqvio increases the number of LDL-C receptors, thereby lowering circulating LDL-C levels.

Initially approved by the FDA in December 2021, Leqvio was authorized for lowering LDL-C in conjunction with diet and maximal statin therapy in patients with ASCVD or heterozygous familial hypercholesterolemia. The therapy requires an initial dose followed by maintenance doses administered twice a year. In July 2023, the FDA broadened Leqvio's indication to include patients with elevated LDL-C levels due to primary hyperlipidemia who are at risk of heart disease.

Leqvio was originally developed by Alnylam Pharmaceuticals before Novartis acquired the licensing rights in January 2020. This acquisition has allowed Novartis to further develop and expand the use of Leqvio as an innovative treatment option for managing cholesterol levels and preventing cardiovascular diseases.

The latest findings from the V-MONO study underscore Leqvio’s potential as a leading therapy for patients needing more effective cholesterol management, especially those who have not achieved their goals with current treatments. As Novartis continues to gather and present more detailed data, the medical community eagerly anticipates the expanded application of this promising therapy in the fight against cardiovascular disease.

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