Drugs / Pelacarsen

Trials 14

PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 16 trials · 1 met primary
Phase 1 NCT05026996 Nov 2021 → Oct 2022 — Novartis Pharmaceuticals Completed No outcome recorded
Phase 1 NCT05337878 Oct 2018 → Dec 2019 — Ionis Pharmaceuticals, Inc. Completed No outcome recorded
Phase 1 NCT03506854 Mar → Oct 2018 impaired renal function disease Akcea Therapeutics Completed No outcome recorded
Phase 1 NCT03392051 Dec 2017 → Mar 2018 cardiovascular disorder, hyperlipoproteinemia Akcea Therapeutics Completed No outcome recorded
Phase 1 NCT03426033 Dec 2017 → Feb 2018 cardiovascular disorder, hyperlipoproteinemia Akcea Therapeutics Completed No outcome recorded
Phase 1 NCT02414594 Apr 2015 → Feb 2016 hyperlipoproteinemia Ionis Pharmaceuticals, Inc. Completed Met primary
Phase 22 trials · 1 met primary
Phase 2 NCT05646381 Mar 2024 → Mar 2030 expected aortic valve stenosis Novartis Pharmaceuticals Recruiting No outcome recorded
Phase 2 NCT03070782 Mar 2017 → Jul 2018 cardiovascular disorder, hyperlipoproteinemia Akcea Therapeutics Completed Met primary
Phase 35 trials · 1 met primary
Phase 3 NCT07625306 Aug 2026 → Dec 2028 expected acute coronary syndrome Novartis Pharmaceuticals Not yet recruiting No outcome recorded
Phase 3 NCT07517263 May 2026 → Jan 2030 expected — Novartis Pharmaceuticals Recruiting No outcome recorded
Phase 3 NCT06875973 May 2025 → Dec 2030 expected atherosclerotic cardiovascular disease Novartis Pharmaceuticals Recruiting No outcome recorded
Phase 3 NCT05900141 Sep 2023 → Aug 2028 expected hyperlipoproteinemia Novartis Pharmaceuticals Active not recruiting No outcome recorded
Phase 3 NCT05305664 Aug 2022 → Jan 2025 hyperlipoproteinemia Novartis Pharmaceuticals Completed Met primary
Phase not applicable1 trial
— NCT04993664 Oct 2021 → Jun 2022 acute coronary syndrome, hyperlipoproteinemia University Medical Centre Ljubljana Withdrawn No outcome recorded
Comparator or background therapy2 trials
Phase 3 NCT06813911 Comparator Apr 2025 → Feb 2028 expected atherosclerotic cardiovascular disease Novartis Pharmaceuticals Active not recruiting No outcome recorded
— NCT07000695 Background — atherosclerotic cardiovascular disease Novartis Pharmaceuticals Available No outcome recorded

News releases announcing trial results or a regulatory action · 4

DateIssuerRelease
2026-09-04 Novartis Pharmaceuticals Results Novartis announces Lp(a)HORIZON Phase III topline results for pelacarsen in patients with elevated Lp(a) and established cardiovascular disease (CVD) novartis.com ↗
Although lower Lp(a) levels were observed with pelacarsen, the findings did not demonstrate that this translated into reduced cardiovascular risk in the overall study population.
2026-09-04 Ionis Pharmaceuticals Results Ionis partner Novartis announces Lp(a)HORIZON Phase 3 topline results for pelacarsen in patients with elevated Lp(a) and established cardiovascular disease (CVD) ionis.com ↗
2020-01-02 Ionis Pharmaceuticals Results New England Journal of Medicine Publishes Results from Phase 2 Study of AKCEA-APO(a)-LRx in Patients with Lp(a)-driven Cardiovascular Disease ionis.com ↗
Akcea Therapeutics, Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced that results from a Phase 2 study evaluating AKCEA-APO(a)-LRx, also known as TQJ230, in patients with established cardiovascular disease (CVD) and elevated levels of lipoprotein(a), or Lp(a), were published today in The New England Journal of Medicine (NEJM).
2016-09-22 Ionis Pharmaceuticals Results Akcea Announces Publication in The Lancet of Clinical Results with Lp(a)-Lowering Drugs Designed to Treat Cardiovascular Disease ionis.com ↗
In these studies, substantial Lp(a) reductions of up to 99% were noted, regardless of starting Lp(a) levels.

Evidence & citations 8 cited values

Every value below carries the sentence it was read from. 17 sources stand behind the page.

FieldValueCited text
Known as Pelacarsen ClinicalTrials.gov intervention name — accepted as the source's own label NCT06813911 ↗
10

NCT05900141 ↗

NCT06875973 ↗

NCT05646381 ↗

NCT05026996 ↗

NCT05337878 ↗

NCT04993664 ↗

NCT07000695 ↗

NCT07517263 ↗

NCT05305664 ↗

NCT07625306 ↗

Known as IONIS-APO(a)-LRx ClinicalTrials.gov intervention name — accepted as the source's own label NCT02414594 ↗
Known as ISIS 681257 ClinicalTrials.gov intervention name — accepted as the source's own label NCT03506854 ↗
3

NCT03426033 ↗

NCT03070782 ↗

NCT03392051 ↗

Known as TQJ230 “Pelacarsen (TQJ230) 80 mg s.c.” NCT05305664 ↗
2

“Pelacarsen (TQJ230) 80mg” NCT05646381 ↗

“Pelacarsen (TQJ230)” NCT04993664 ↗

Action Inhibit “Pelacarsen by using an ASO directed against the messenger ribonucleic acid (mRNA) of apolipoprotein (a), reduces the production of apolipoprotein (a) in the liver and thus, the...” NCT04993664 ↗
Modality Antisense oligonucleotide “IONIS-APO(a)-LRx, a ligand-conjugated antisense oligonucleotide designed to be highly and selectively taken up by hepatocytes” PMID 27665230 ↗ Sep 2016
Route Subcutaneous “IONIS-APO(a)-LRx by subcutaneous (SC) injection” NCT02414594 ↗
Target LPA “The gene encoding apo (a); LPA, is located on the long arm of chromosome 6 (6q2,6-2,7) and most variants in Lp (a) can be explained by genetic diversity in LPA. To date, the...” NCT04993664 ↗