Benzinga | Aug 2, 2021 09:33AM EDT

Ionis Pharmaceuticals Says Lp HORIZON Achieves 50% Enrollment In Trial To Assess Safety, Efficacy Of Pelacarsen In Reducing Recurrent Cardiovascular Events

Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today announced that pelacarsen, formerly known as AKCEA-APO(a)-LRx, and licensed by Novartis (NYSE:NVS) for exclusive worldwide development and commercialization, continues to advance in the clinic as the potential first-in-class treatment for lipoprotein(a) or Lp(a)-driven cardiovascular disease. The Novartis pivotal Phase 3 cardiovascular outcomes study of pelacarsen, Lp(a) HORIZON (NCT04023552), has reached 50% enrollment with a target goal of 7,680 trial participants.

Pelacarsen is an investigational antisense medicine that uses Ionis' proprietary Ligand Conjugated Antisense (LICA) technology platform. It is designed to inhibit the production of apolipoprotein(a) in the liver to target elevated Lp(a) levels, an independent genetic risk factor for cardiovascular diseases (CVD), that are determined at birth and cannot be controlled with diet or exercise. High Lp(a) levels are associated with significant risk of cardiovascular disease, including heart attacks and strokes. There are no approved pharmacological therapies to effectively lower Lp(a).

"We are pleased by Novartis progress in advancing the Lp(a) HORIZON study and enrolling nearly 4,000 study participants around the world. Pelacarsen represents a potential first-in-class treatment to address a significant unmet need with the potential to become the new standard of care for Lp(a)-driven cardiovascular disease," said Sotirios "Sam" Tsimikas, M.D., senior vice president, clinical development and cardiovascular franchise leader at Ionis, who specializes in Lp(a). "Ionis' vision is that pelacarsen emerges as an effective therapy to improve cardiovascular outcomes through normalizing Lp(a) levels, as there are an estimated eight million people globally living with elevated Lp(a) and cardiovascular disease."

Data from a Phase 2 study published in the New England Journal of Medicine showed pelacarsen provided potent dose-dependent reductions of Lp(a) compared to placebo, with a favorable safety and tolerability profile in patients who had elevated Lp(a) levels and established CVD. These data support the potential of antisense-mediated reduction of Lp(a) with pelacarsen.

Ionis earned a $25 million milestone payment from Novartis for achieving 50% enrollment in the pivotal Phase 3 study. In 2017, Novartis entered a collaboration agreement with Ionis for pelacarsen. In February 2019, Novartis exercised an option to license the rights to develop and commercialize pelacarsen for targeted cardiovascular therapy for $150 million. Under the terms of the agreement, Novartis is exclusively responsible for worldwide development and commercialization of pelacarsen and Ionis is eligible to receive up to $675 million in regulatory and sales milestones. Ionis is also eligible to receive tiered royalties in the mid-teens to low 20% range on net sales of pelacarsen.

Additional information about Lp(a) HORIZON may be found at www.ClinicalTrials.gov (NCT04023552).