Efficacy and safety of K-877, a novel selective peroxisome proliferator-activated receptor α modulator (SPPARMα), in combination with statin treatment: Two randomised, double-blind, placebo-controlled clinical trials in patients with dyslipidaemia

Atherosclerosis. 2017 Jun:261:144-152. doi: 10.1016/j.atherosclerosis.2017.03.032. Epub 2017 Mar 24.

Abstract

Background and aims: Substantial residual cardiovascular risks remain despite intensive statin treatment. Residual risks with high triglyceride and low high-density lipoprotein cholesterol are not the primary targets of statins. K-877 (pemafibrate) demonstrated robust efficacy on triglycerides and high-density lipoprotein cholesterol and a good safety profile as a monotherapy. The aim of these studies was to evaluate the efficacy and safety of K-877 add-on therapy to treat residual hypertriglyceridaemia during statin treatment.

Methods: The objectives were investigated in two, multicentre, randomised, double-blind, placebo-controlled, parallel group comparison clinical trials: (A) K-877 0.1, 0.2, and 0.4 mg/day in combination with pitavastatin for 12 weeks in 188 patients, (B) K-877 0.2 (fixed dose) and 0.2 (0.4) (conditional up-titration) mg/day in combination with any statin for 24 weeks in 423 patients.

Results: In both studies, we found a robust reduction in fasting triglyceride levels by approximately 50% in all combination therapy groups, which was significant compared to the statin-monotherapy (placebo) groups (p < 0.001). High-performance liquid chromatography analysis for lipoprotein subfractions revealed that atherogenic lipoprotein profiles were ameliorated by K-877 add-on therapy, i.e. small low-density lipoproteins decreased whereas larger ones increased, and larger high-density lipoproteins decreased whereas smaller ones increased. The incidence rates of adverse events and adverse drug reactions in K-877 combination therapy groups were comparable to those in statin-monotherapy groups without any noteworthy event in both studies.

Conclusions: These results strongly support the favourable benefit-to-risk ratio of K-877 add-on therapy in combination with statin treatment.

Keywords: Combination therapy; Dyslipidaemia; K-877; Pemafibrate; SPPARMα; Statin; Triglyceride.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Benzoxazoles / adverse effects
  • Benzoxazoles / therapeutic use*
  • Biomarkers / blood
  • Butyrates / adverse effects
  • Butyrates / therapeutic use*
  • Double-Blind Method
  • Drug Therapy, Combination
  • Female
  • Humans
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors / adverse effects
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors / therapeutic use*
  • Hypertriglyceridemia / blood
  • Hypertriglyceridemia / diagnosis
  • Hypertriglyceridemia / drug therapy*
  • Hypolipidemic Agents / adverse effects
  • Hypolipidemic Agents / therapeutic use*
  • Japan
  • Male
  • Middle Aged
  • PPAR alpha / agonists*
  • PPAR alpha / metabolism
  • Quinolines / adverse effects
  • Quinolines / therapeutic use*
  • Signal Transduction / drug effects
  • Time Factors
  • Treatment Outcome
  • Triglycerides / blood*
  • Young Adult

Substances

  • Benzoxazoles
  • Biomarkers
  • Butyrates
  • Hydroxymethylglutaryl-CoA Reductase Inhibitors
  • Hypolipidemic Agents
  • K-877 compound
  • PPAR alpha
  • Quinolines
  • Triglycerides
  • pitavastatin