Novartis licenses lipoprotein-lowering CVD drug AKCEA-APO(a)-LRx

Excited by strong phase II data, Novartis is exercising its option to license Akcea Therapeutics’ AKCEA-APO(a)-LRx, a treatment to reduce the risk of cardiovascular disease in people living with elevated levels of inherited lipoprotein(a). Now Novartis will guide the molecule to late-stage studies. Novartis licensed AKCEA-APO(a)-LRx, now called TQJ230 by Novartis. Akcea will receive $150 million in a milestone payment from Novartis for the therapy, half of which will go to Ionis Pharmaceuticals, Akcea’s parent company. 

Early Development

• 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 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.

Suggested mechanism of action

Lipoprotein(a), or Lp(a), is made up of apo(a) protein bound to LDL cholesterol and contains oxidized phospholipids. Elevated Lp(a) is recognized as an independent, genetic cause of cardiovascular disease present in approximately 20 to 30 percent of the population, Akcea said. Lp(a) levels are determined at birth and, therefore, lifestyle modifications, including diet and exercise, do not impact Lp(a) levels. There are currently no treatment options for these patients.