Ionis Pharmaceuticals, Inc. announced that it has entered a collaboration and license agreement with Novartis for the discovery, development and commercialization of a novel medicine for patients with lipoprotein(a), or Lp(a)-driven cardiovascular disease (CVD). This builds on the companies' existing collaboration focused on the development and commercialization of pelacarsen, which Novartis is currently evaluating in a Phase 3 cardiovascular outcome study. The next generation compound will be a potential follow-on to pelacarsen.
"We are pleased to expand our productive collaboration with Novartis aimed at delivering transformative therapies to patients with elevated Lp(a) who are at high risk of cardiovascular events," said Brett P. Monia, Ph.D., Ionis' chief executive officer. "This collaboration is designed to leverage Ionis' advancing RNA-targeting platform technologies to deliver a novel Lp(a)-targeting therapy that we expect will provide industry-leading efficacy and dosing frequency."
Ionis will receive a $60 million upfront payment from Novartis and is eligible to earn development, regulatory and commercial milestone payments and tiered royalties. Novartis will be solely responsible for the development, manufacturing and potential commercialization of the next generation Lp(a) therapy.
The agreement is subject to the satisfaction of requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
About Lp(a)
Lp(a) is a lipoprotein particle assembled in the liver that consists of a low-density lipoprotein cholesterol (LDL-C)-like particle and apolipoprotein(a). Lp(a) levels in the blood can vary greatly between individuals primarily due to genetic variations and do not correlate with LDL-C levels. Even patients with LDL-C lowered to target levels (<70 mg/dL) remain at high-risk of cardiovascular events if they have high levels of Lp(a). Elevated Lp(a) is a genetically determined condition that is not responsive to lifestyle changes, therefore patients are unable to adequately control their Lp(a) levels through improved diet or increased physical activity. Elevated Lp(a) is recognized as an independent, genetic cause of coronary artery disease, heart attack, stroke, peripheral arterial disease and aortic stenosis. Currently, there is no effective drug therapy to specifically and robustly lower elevated levels of Lp(a).
About Pelacarsen
Pelacarsen was discovered by Ionis and licensed to Novartis in 2019 for exclusive worldwide development, manufacturing and commercialization. It is an investigational antisense medicine designed to reduce apolipoprotein(a) production in the liver to offer a direct approach for reducing circulating Lp(a). It is estimated that more than eight million people worldwide have elevated Lp(a) and CVD.
Pelacarsen is currently being evaluated in Lp(a) HORIZON (NCT04023552), a global, multicenter, double-blind, placebo-controlled pivotal Phase 3 study conducted by Novartis. The study completed enrollment with 8,323 participants. Data are expected in 2025.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted therapy, pioneering new markets and changing standards of care. Ionis currently has four marketed medicines and a promising late-stage pipeline highlighted by cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision to become the leader in genetic medicine, utilizing a multi-platform approach to discover, develop and deliver life-transforming therapies.