Three years after launching Leqvio in the U.S., Novartis is still steadily advancing its blockbuster ambitions for the cholesterol-lowering drug. Early Wednesday, the company touted phase 3 evidence that it believes could support wider use of the drug.
As a monotherapy, the twice-yearly drug delivered clinically meaningful and statistically significant reductions in low-density lipoprotein cholesterol (LDL-C) compared with both placebo and Merck’s Zetia in patients at low or moderate risk of developing atherosclerotic cardiovascular disease (ASCVD) who are not on a lipid-lowering therapy.
ASCVD accounts for 85% of all cardiovascular disease fatalities, according to Novartis. It's caused by plaque in the inner lining of the arteries that’s mainly composed of a build-up of LDL-C. Leqvio is currently approved as an adjunct to diet and statin therapy for patients already with ASCVD or heterozygous familial hypercholesterolemia who require additional lowering of LDL-C.
The phase 3 study, called V-MONO, is the first to weigh a small interfering RNA (siRNA) treatment as a monotherapy to lower LDL-C in patients at risk of developing ASCVD, the company said in its release. Novartis plans to present the results at an upcoming medical meeting and share them with regulatory agencies, including the FDA.
“We are proud that we continue to advance the scientific understanding of using siRNA therapy to tackle one of the world’s biggest healthcare challenges, as too many people still struggle to reach their cholesterol goals,” chief medical officer Shreeram Aradhye, M.D., noted in a statement. “This trial adds to the growing body of evidence for Leqvio across the full spectrum of ASCVD as we strive to help more patients in need.”
V-MONO is a part of Novartis’ VictORION program, which is “one of the largest clinical trial programs of its kind,” with over 60,000 patients enrolled in more than 50 countries across dozens of trials. Through the studies, the company is looking to evaluate Leqvio’s potential across various cardiovascular treatment settings. Additional readouts are expected through 2027.
Leqvio was always believed to be a multi-year project for the company. Soon after the drug's 2021 FDA approval for LDL-C reduction as an add-on to statin therapy, CEO Vas Narasimhan pointed out that the foundations were being established for the drug to become a “very significant brand” for Novartis. Still, the CEO noted that it would be a “long journey” to reach the drug's full potential.
The unique twice-yearly dosing of Leqvio offers an edge among other more frequently administered drugs, especially considering a typical lower compliance rate for asymptomatic disease treatment. Two years ago, that factor pushed GlobalData’s 2027 sales expectations for Leqvio to $2.5 billion.
More recently, Novartis scrapped a planned primary prevention study in partnership with NHS England, although it’s still studying Leqvio in that setting in a separate 14,000-patient trial.
The company picked up the med back in 2019 from its $9.7 billion acquisition of The Medicines Company. In 2023, Leqvio brought in about $400 million sales as Novartis worked on additional patient on-boarding, removing access hurdles and enhancing medical education.