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Published on: September 5, 2016

Emerging therapies to lower lipoprotein(a) and the current clinical trials landscape: RNA-therapeutics and beyond

Sawye Raygani1, Michael J Wilkinson2

  • 1School of Medicine, University of California San Diego (UCSD), San Diego, United States.

Journal of Clinical Lipidology
|July 28, 2026
PubMed

Insights

Lipoprotein(a) [Lp(a)] is a key ASCVD risk factor. Emerging therapies show promise in lowering Lp(a) levels, with ongoing trials to determine if this reduces cardiovascular events.

Area of Science:

  • Cardiovascular Medicine
  • Pharmacology
  • Genetics

Background:

  • Lipoprotein(a) [Lp(a)] is an independent genetic risk factor for atherosclerotic cardiovascular disease (ASCVD) and calcific aortic valve disease.
  • Current FDA-approved Lp(a) reduction therapy is limited to lipoprotein apheresis.
  • Emerging targeted pharmacotherapies are demonstrating significant Lp(a) reductions in phase 2 trials.

Purpose of the Study:

  • To review the current landscape of Lp(a) lowering therapies.
  • To highlight the ongoing phase 3 clinical trials assessing cardiovascular outcomes.
  • To discuss the anticipated results regarding Lp(a) reduction and major adverse cardiovascular events (MACE).

Main Methods:

  • Review of data from PubMed and ClinicalTrials.gov.
  • Analysis of findings from phase 2 trials of emerging Lp(a) pharmacotherapies.
  • Identification of ongoing phase 3 cardiovascular outcomes trials.

Main Results:

  • Certain lipid-lowering drugs, including PCSK9 inhibitors and siRNA therapeutics, offer modest Lp(a) reduction.
  • Lp(a)-targeted therapies (ASOs, siRNA, small molecules) significantly reduced Lp(a) in phase 2 trials.
  • Multiple novel therapies are progressing to phase 3 cardiovascular outcomes trials.

Conclusions:

  • The development of Lp(a)-lowering therapies has advanced significantly, with several agents in phase 3 trials.
  • Anticipated results will clarify if substantial Lp(a) reduction effectively prevents MACE across diverse patient populations.
  • These trials are crucial for establishing new treatment paradigms for ASCVD prevention.
Abstract

Keywords:
Antisense oligonucleotidesAtherosclerotic cardiovascular diseaseCardiovascular outcomesLipoprotein(a)Preventive cardiologySmall interfering RNA

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