Agent
Pelacarsen (TQJ230, AKCEA-APO(a)-LRx)
Status Phase 3; first phase 3 result July 2026 (apheresis trial); outcomes trial Lp(a)HORIZON awaited
A GalNAc3-conjugated second-generation antisense oligonucleotide against LPA mRNA, given as 80 mg subcutaneously every 4 weeks. Lowers Lp(a) by about 80 percent (NEJM 2020) and replaced weekly apheresis in 25 of 26 patients in Lp(a)FRONTIERS APHERESIS (EHJ 2026). Lp(a)HORIZON (n=8,323) is the first Lp(a) outcomes trial; readout guided to 2026, not yet announced.
Mechanism. Binds apo(a) mRNA in hepatocytes and recruits RNase H1 to degrade it; the N-acetylgalactosamine ligand delivers it to the asialoglycoprotein receptor, which is why the dose is a tenth of the unconjugated parent (IONIS-APO(a)Rx).
Milestones. Phase 1 (Tsimikas, Lancet 2015); phase 2 of the conjugated drug (Viney, Lancet 2016; Tsimikas, NEJM 2020); Lp(a)HORIZON started December 2019 and completed enrolment (8,323) in 2022; Lp(a)FRONTIERS APHERESIS positive (EHJ 2026); further US-population and inclisiran-background studies running.
Watch. Lp(a)HORIZON topline; ESC 2026 and AHA 2026.
antisensepelacarsenRNA therapeutics
Page updated 17 August 2026.