Novartis' $150M RNA-Targeted Therapy TQJ230 Granted Clinical Trial Approval in China for Cardiovascular Risk Reduction

By Baihuawen

On April 7, Novartis’ TQJ230 injection was approved for clinical trials in China for the reduction of cardiovascular risk. This is an antisense oligonucleotide (ASO) therapy; Novartis acquired the global development and commercialization rights to this drug in February 2019 for $150 million.

TQJ230 was discovered by Ionis Pharmaceuticals and is being co-developed with Akcea Therapeutics. Currently, this drug is in global Phase III clinical trials. If approved, it will become the first-in-class therapy specifically targeting elevated lipoprotein(a).

Lipoprotein(a) is a type of lipoprotein in the blood. High levels of lipoprotein(a) accumulate in the arteries, causing them to gradually narrow and restrict blood supply to the heart, brain, kidneys, and legs, thereby increasing the risk of coronary heart disease, atherosclerosis, thrombosis, and stroke.

Millions of people worldwide have elevated lipoprotein(a) levels, an independent risk factor for hereditary cardiovascular disease. It is estimated that 20–30% of patients with cardiovascular disease also have elevated lipoprotein(a) [Lp(a)] levels. Currently, there are no specific treatments to lower high lipoprotein(a) levels, and dietary or other lifestyle modifications cannot effectively reduce lipoprotein(a) concentrations.

Results from a Phase II study presented at the 2018 AHA Scientific Sessions demonstrated that TQJ230 significantly reduced lipoprotein(a) levels in patients with elevated lipoprotein(a) and established cardiovascular disease.

Novartis already has a blockbuster therapy in the cardiovascular disease field, Entresto (sacubitril/valsartan sodium), which was approved in China in July 2017 and included in the Class B list of the National Reimbursement Drug List in 2019. Financial reports show that Entresto generated $1.726 billion in sales revenue for Novartis in 2019.

In November 2019, Novartis acquired The Medicines Company for $7 billion, thereby securing the RNAi therapy inclisiran and further strengthening its portfolio in the cardiovascular field. Currently, this drug is under review by the FDA and is expected to be approved this year as the third RNAi therapy to reach the market.