Targets apolipoprotein(a) messenger RNA, reducing apolipoprotein(a) production. Source
Established cardiovascular disease and elevated Lp(a)
Study contextCurrent approval status is not summarized in this therapy record. A trial phase or a brand name does not establish approval for this use.
Status & indicationsLp(a): 80% reduction within arm · Week 27, the six-month endpoint for weekly dosing
AKCEA-APO(a)-LRx phase 2 · Phase 2 · Peer-reviewed trial · 20 mg weekly · Mean within-arm change in Lp(a); placebo decreased 6%.
Evidence & limitationsInjection-site reactions were most common; no significant platelet, liver or kidney differences versus placebo.
Safety contextDate not established · Full pelacarsen results at an undated congress; other programs retain separate outcome-trial windows.
Unknown timing
Milestone detailsStudy results apply to their own population, dose and analysis. They do not establish a ranking between therapies.
Each result belongs to its own study population, dose and analysis. Open a result for its chart, comparator, safety findings and limitations.
Lp(a): 80% reduction within arm · Week 27, the six-month endpoint for weekly dosing
20 mg weekly
Established cardiovascular disease and elevated Lp(a)
Mean within-arm change in Lp(a); placebo decreased 6%.
Injection-site reactions were most common; no significant platelet, liver or kidney differences versus placebo.
Limitations: This dose-finding biomarker result is separate from the later 80 mg-monthly HORIZON outcomes regimen.
Status in this atlas: Current approval status is not summarized in this therapy record. A trial phase or a brand name does not establish approval for this use.
Targets apolipoprotein(a) messenger RNA, reducing apolipoprotein(a) production.
Mechanism sourceUses, doses and availability depend on the formulation, indication and country. Study doses below describe research; they are not prescribing instructions.
Hepatocyte-targeted antisense oligonucleotide with a three-arm GalNAc conjugate.
Shown: GalNAc-conjugated antisense oligonucleotide (TQJ230; IONIS-APO(a)-LRx).
This is the GalNAc-conjugated pelacarsen compound, distinct from its earlier unconjugated predecessor. No exact whole-drug atomic model has been verified for this bundle.
DOI 10.1111/cts.70344Pelacarsen · compound history across indications
Selected sourced milestones, not a complete development history. Dates distinguish study starts, publications, announcements and approvals; the first listed date is not necessarily when development began.
Akcea announced Lancet publication of the first human study of the liver-targeted compound later named pelacarsen. Multiple doses lowered Lp(a); the publication also covered its earlier, unconjugated predecessor, which is a distinct compound.
Akcea announces Lancet clinical results for IONIS-APO(a)-LRx (2016)The registry records the actual start of the phase 3 trial testing whether pelacarsen reduces cardiovascular events in patients with established cardiovascular disease and elevated Lp(a).
ClinicalTrials.gov: Lp(a)HORIZON, NCT04023552The plotted dose-ranging trial in 286 patients with cardiovascular disease reported dose-dependent Lp(a) lowering, reaching a mean 80% reduction at the highest tested regimen. This was a biomarker study, not an outcomes trial.
Lipoprotein(a) Reduction in Persons with Cardiovascular Disease — NEJM (2020)Novartis announced that the phase 3 cardiovascular composite was not met despite lower Lp(a). The announcement did not provide a hazard ratio or confidence interval; full results were pending.
Novartis: Lp(a)HORIZON phase 3 topline results (September 4, 2026)Selected sources checked Sep 14, 2026. This is not an exhaustive history.