Novartis licenses lipoprotein-lowering CVD drug

Excited by strong phase II data, Novartis is exercising its option to license Akcea Therapeutics’ TQJ230, a treatment to reduce the risk of cardiovascular disease in people living with elevated levels of inherited lipoprotein(a).

TQJ230, previously known as AKCEA-APO(a)-LRx, was discovered by Ionis Pharmaceuticals and has been co-developed to date by Akcea and Ionis.

In a deal announced in January 2017, Novartis said it would be working with Ionis to license and develop TQJ230 and another antisense therapy, AKCEA-APOCIII-LRX.

The deal involved an upfront payment of $75 million and near-term payments of $225 million including a $100 million investment by Novartis in Akcea.

The licensing follows phase II results announced in November demonstrating that of patients on the highest dose of TQJ230 of 20 mg once-weekly, 98% saw Lp(a) levels drop below the recommended risk threshold.

Novartis is now responsible for worldwide development and commercialisation of the asset.

The firm already markets recently-approved Entresto (sacubitril+valsartan) to treat heart failure, and diabetes drug Galvus (vildagliptin) through its cardio-metabolic division, and is looking to expand its offering in this area.

Lp(a) is a lipoprotein that travels through the blood. Elevated levels of Lp(a) collect in the arteries, gradually narrowing them and limiting blood supply to the heart, brain, kidneys and legs. This can lead to increased risk of coronary heart disease, atherosclerosis, thrombosis and stroke.

It is estimated that 20-30% of people who suffer from CVD have elevated Lp(a). Currently no treatment exists that specifically targets elevated Lp(a), and diet and other lifestyle changes are not effective at reducing elevated levels.

Novartis said it now plans to conduct a phase 3 cardiovascular outcomes trial with the potential of addressing the Lp(a) patient community's unmet need for effective treatment.

“No treatments are currently available to substantially lower Lp(a),” said John Tsai, head of global drug development and chief medical officer at Novartis.

“People with this inherited risk factor are facing cardiovascular risks that cannot be addressed effectively with lifestyle changes.

“We're excited about the novel, RNA-targeting approach that could be a game-changer for people with elevated Lp(a). If our phase 3 trial succeeds, we expect that TQJ230 will become the leading treatment option and another pillar of our longstanding commitment to re-imagining cardiovascular medicine.”

According to Ionis’ head of cardiovascular development Dr Sotirios Tsimikas – who is also director of vascular medicine at the University of California San Diego (UCSD) – a drug that reduces Lp(a) could be “a paradigm shift for the cardiovascular community”.