Two single injections of inclisiran, a small interfering RNA (siRNA) that targets PCSK9 mRNA, induce a 50% reduction in LDL-cholesterol (LDL-C) levels in 6 months, according to findings from the phase II ORION-1 trial presented at ACC.17. A total of 501 patients at high risk of cardiovascular disease and with high LDL-C levels were randomly assigned to receive one or two injections (at days 1 and 90) of inclisiran at three different doses or placebo. All inclisiran regimens induced a significant reduction in LDL-C levels at 180 days, but two injections of 300 mg inclisiran produced the greatest effect, with mean reductions from baseline of 69.1% in PCSK9 levels and 52.6% in LDL-C levels (P <0.001). PCSK9 and LDL-C levels remained lower than baseline at day 240 with all doses of inclisiran.
References
Ray, K. K. et al. Inclisiran in patients at high cardiovascular risk with elevated LDL cholesterol. N. Engl. J. Med. http://dx.doi.org/10.1056/NEJMoa1615758 (2017)
Rights and permissions
About this article
Cite this article
Fernández-Ruiz, I. Promising results with siRNA against PCSK9. Nat Rev Cardiol 14, 252 (2017). https://doi.org/10.1038/nrcardio.2017.43
Published:
Issue Date:
DOI: https://doi.org/10.1038/nrcardio.2017.43