Novartis invests in next generation therapies to reduce cardiovascular risk in patients with underlying lipid disorders

  • Novartis enters into an exclusive option agreement with Ionis and Akcea to license two investigational treatments expected to significantly reduce cardiovascular risk in patients living with elevated levels of lipoprotein Lp(a) or ApoCIII, which is a potent regulator of triglycerides
  • Investment in biomarker-based therapies bolsters Novartis’ cardiovascular specialty pipeline and commitment to address unmet medical need of high-risk atherosclerosis/dyslipidemia patients
  • Atherosclerosis, commonly called the "silent killer", is a major cause of death globally and no options exist today to effectively treat patients whose disease is driven by Lp(a) and ApoCIII

- Novartis announced today a collaboration and option agreement with Ionis Pharmaceuticals, Inc. and its affiliate Akcea Therapeutics, Inc., to license two novel treatments with the potential to significantly reduce cardiovascular risk in patients suffering from high levels of lipoproteins known as Lp(a) and ApoCIII. The two investigational antisense therapies developed by Ionis-called AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx-have the potential to lower both lipoproteins up to 90% and significantly reduce cardiovascular risk in high-risk patient populations. In addition Novartis entered into a stock purchase agreement with Ionis.

"Novartis is building a robust cardiovascular portfolio of targeted therapies to address unmet medical need of high-risk patients," said Vasant Narasimhan, Global Head, Drug Development and Chief Medical Officer, Novartis. "Lp(a) and ApoCIII are potent, genetically validated cardiovascular risk reduction targets. The importance of predictive biomarkers in achieving successful cardiovascular outcomes will also be essential in the future payer environment. We look forward to working with Ionis and Akcea to develop both treatments."

Novartis will be able to exercise its options to license and commercialize AKCEA-APO(a)-LRx and AKCEA-APOCIII-LRx following the achievement of specified development milestones and prior to the initiation of Phase 3 studies for each program. Upon in-licensing Novartis will be responsible for worldwide development and commercialization of both assets.

Ionis’ antisense technology is currently the most effective way to inhibit synthesis of both lipoproteins in the liver, according to data published in the Lancet. The GalNAc3-conjugated antisense oligonucleotide technology is 30 times more potent than the parent antisense oligonucleotide, leading to a lower dose and the potential for highly effective therapeutic targeting and much improved tolerability.

The deal is subject to customary closing conditions and regulatory approvals.

About Atherosclerosis
Atherosclerosis is a disease in which plaque builds up inside the arteries leading to gradual narrowing and hardening of the arteries and increased risk of blood clots, heart attack and stroke. Atherosclerosis that affects the arteries of the heart is commonly known as coronary heart disease.

About Dyslipidemia
Dyslipidemia is the elevation of plasma cholesterol, triglycerides, or both, or a low high-density lipoprotein level that contributes to the development of atherosclerosis.

About Lipoprotein(a) (Lp(a))
Lp(a) is a lipoprotein that travels through the blood. Elevated levels of Lp(a) collect in the arteries, gradually narrowing the arteries and limiting blood supply to the heart, brain, kidneys and legs. This leads to increased risk of coronary heart disease, atherosclerosis, thrombosis and stroke. Additional information is available through Lipoprotein (a) Foundation at

About Apolipoprotein-CIII (ApoCIII)
ApoCIII is a protein produced in the liver that plays a central role in the regulation of triglycerides, a type of fat found in the blood. People with elevated levels of ApoCIII have high triglycerides which are associated with multiple metabolic abnormalities such as insulin resistance and/or metabolic syndrome. People who do not produce ApoCIII have lower levels of triglycerides and lower instances of cardiovascular disease.