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. 2018 Oct 11;40(2):537–557. doi: 10.1210/er.2018-00184

Table 5.

Emerging Therapies for Hypertriglyceridemia

Mechanism of Action Trial Trial Description
SPPARM-α
 Pemafibrate (Kowa Research Institute) Selective PPAR-α modulator PROMINENT (NCT03071692) (159) RCT evaluating the efficacy of Pemafibrate vs placebo in reducing incidence of adverse CV events in ∼10,000 participants
Inclusion criteria: participants with T2DM and fasting TG ≥200 mg/dL and <500 mg/dL and HDL-C ≤40 mg/dL
OM3FAs
 VASCEPA (icosapent ethyl) also known as AMR101 (Amarin Pharma) Highly purified ethyl ester of EPA REDUCE-IT (NCT01492361) (159) RCT evaluating the efficacy of AMR101 vs placebo in reducing incident CV events in ∼8000 participants
  Treatment dose: 4 g/d Inclusion criteria: individuals with established CVD or at high risk with hypertriglyceridemia and receiving statin therapy
 Epanova (AstraZeneca) Omega-3 carboxylic acids STRENGTH (NCT02104817) (160) RCT evaluating the efficacy of Epanova vs corn oil in reducing MACE in ∼13,000 participants
Inclusion criteria: patients at high risk for future CV events on stable diet and statin therapy with LDL-C <100 mg/dL and TG level ≥180 mg/dL and <500 mg/dL and HDL-C <42 mg/dL for men or HDL-C <47 mg/dL for women
 Omacor Marine OM3FAs (465 mg of EPA and 375 mg of DHA) VITAL (NCT01169259) (161) RCT in 25,871 participants evaluating daily dietary supplementation of 2000 units of vitamin D3 or OM3FAs for reducing the risk of developing cancer, heart disease, and stroke
  Treatment dose: 1 capsule/d (840 mg of marine OM3FA)
 Icosabutate (162) Potent, synthetically modified EPA molecule No phase 3 outcomes trials underway N/A
ApoCIII inhibitor
 Volanesorsen Antisense apoCIII inhibitor COMPASS (NCT02300233) (160) RCT evaluating the efficacy of volanesorsen vs placebo given for 52 wk in reducing fasting TG levels
Inclusion criteria: fasting TG level ≥500 mg/dL
Antisense apoCIII inhibitor APPROACH (NCT02211209) (161) RCT evaluating the efficacy of volanesorsen vs placebo in reducing fasting TG levels in patients with FCS
Inclusion criteria: FCS with fasting TG level ≥750 mg/dL (8.4 mmol/L) at screening
ANGPTL3 inhibition
 Evinacumab (REGN1500; Regeneron) (124) Human monoclonal antibody against ANGPTL3 No ongoing CV outcomes trials N/A
 IONIS-ANGPTLRX; Ionis Pharmaceuticals (163) Antisense oligonucleotides targeting hepatic ANGPTL3 mRNA No ongoing CV outcomes trials N/A

Abbreviations: ANGPTL, angiopoietin-like proteins; APPROACH, Study of Volanesorsen (formerly IONIS-APOCIIIRx) in Patients With Familial Chylomicronemia Syndrome; COMPASS, Apolipoprotein C-III Inhibition With Volanesorsen in Patients With Hypertriglyceridemia; FCS, familial chylomicronemia syndrome; MACE, major adverse cardiac event; N/A, not applicable; PPAR, peroxisome proliferator–activated receptor; PROMINENT, Pemafibrate to Reduce Cardiovascular Outcomes by Reducing Triglycerides in Diabetic Patients; REDUCE-IT, Reduction of Cardiovascular Events With EPA–Intervention Trial; STRENGTH, Statin Residual Risk Reduction With Epanova in High CV Risk Patients With Hypertriglyceridemia; VITAL, Vitamin D and Omega-3 Trial.