The partnership builds upon the companies' existing collaboration, which centres around the development and commercialisation of pelacarsen
Ionis Pharmaceuticals’ headquarters building entrance. (Credit: Ionis Pharmaceuticals, Inc.)
Ionis Pharmaceuticals and Novartis have joined hands in a collaboration and license agreement aimed at discovering, developing, and bringing to market an innovative medicine designed specifically for patients suffering from lipoprotein(a) or Lp(a)-driven cardiovascular disease (CVD).
The partnership builds upon the companies’ existing collaboration, which centres around the development and commercialisation of pelacarsen. Presently, Novartis is evaluating pelacarsen in a Phase 3 cardiovascular outcome study.
The newly developed next-generation compound is envisioned to be a potential successor to pelacarsen, offering promising prospects for addressing Lp(a)-driven CVD more effectively.
Ionis Pharmaceuticals CEO Brett Monia said: “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.
“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.”
Under the collaboration and license agreement, Ionis Pharmaceuticals will receive an upfront payment of $60m from Novartis.
Additionally, Ionis stands to earn further payments based on development, regulatory, and commercial milestones, as well as tiered royalties.
Novartis will take full responsibility for the development, manufacturing, and potential commercialisation of the next-generation Lp(a) therapy.
The agreement is subject to meeting the requirements of the Hart-Scott-Rodino Antitrust Improvements Act of 1976.
Lp(a) is a lipoprotein particle formed in the liver, comprising a particle similar to low-density lipoprotein cholesterol (LDL-C) and apolipoprotein(a). The levels of Lp(a) in the bloodstream can significantly vary among individuals due to genetic differences, and they do not necessarily correlate with LDL-C levels.
Pelacarsen, discovered by Ionis and licensed to Novartis in 2019 for exclusive worldwide development, manufacturing, and commercialisation, is an investigational antisense medicine.
It is designed to reduce apolipoprotein(a) production in the liver, offering a direct approach to lowering circulating Lp(a) levels. It is estimated that over eight million people worldwide have elevated Lp(a) and CVD.
Currently, Pelacarsen is undergoing evaluation in the Lp(a) HORIZON study (NCT04023552), a global, multicenter, double-blind, placebo-controlled pivotal Phase 3 trial conducted by Novartis. Enrollment for the study has been completed with 8,323 participants, and data from the trial are expected to be available in 2025.