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Ionis announces enrollment completion of Phase 3 Lp(a) HORIZON cardiovascular outcomes study of pelacarsen

Published: 2022-07-20 11:05:00 ET
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  • Pelacarsen is a potentially first-in-class treatment specifically targeting elevated lipoprotein(a) (Lp(a)), an independent, inherited and causal risk factor for cardiovascular disease
  • There are currently no approved pharmacological therapies to effectively lower Lp(a), which cannot be effectively addressed by diet and other lifestyle changes
  • Topline results expected 2025

CARLSBAD, Calif., July 20, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that Novartis has completed patient enrollment of the pivotal Phase 3 Lp(a) HORIZON cardiovascular outcomes study of pelacarsen, with 8,325 study participants. Lp(a) HORIZON is evaluating the safety and efficacy of pelacarsen in reducing cardiovascular events in patients with cardiovascular disease and elevated levels of Lp(a). Topline data from the study are expected in 2025. Novartis licensed pelacarsen from Ionis in 2019.

(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

Pelacarsen, formerly IONIS-APO(a)-LRx, is an investigational antisense medicine designed to inhibit the production of apolipoprotein(a) in the liver to reduce elevated lipoprotein(a) or Lp(a) levels, an independent, inherited and causal risk factor for cardiovascular disease (CVD) and calcific aortic valve stenosis. High Lp(a) levels, which are associated with significant risk of coronary heart disease, cannot be reduced with lifestyle modifications or with existing lipid-lowering therapies. Pelacarsen uses Ionis' Ligand-Conjugated Antisense (LICA) technology platform. 

"Completing enrollment of this pivotal outcomes study brings us closer to the day when this potentially transformative treatment is available to patients," said Sotirios "Sam" Tsimikas, M.D., senior vice president, clinical development and cardiovascular franchise leader at Ionis, who specializes in Lp(a). "There are no approved pharmacological therapies to effectively lower Lp(a) for the more than eight million patients living with cardiovascular disease and elevated levels of Lp(a) worldwide. We are grateful to the patients, investigators and site staff who are participating to make the Phase 3 Lp(a) HORIZON study possible."

Data from a Phase 2 study showed pelacarsen reduced Lp(a) levels below the recommended threshold of risk for CVD events (