Early-stage cholesterol drug offers new hope for patients resistant to statins

A study published in The Lancet found that participants in a Phase I clinical trial saw their level of low density lipoprotein (LDL) cholesterol, or “bad” cholesterol, fall by up to 57% as a result of taking ALN-PCS.

The drug, under development by USA-based Alnylam Pharmaceuticals (Nasdaq: ALNY), works by stopping the body's production of a protein known as PCSK9, which helps reduce cholesterol.

Specifically, ALN-PCS administration resulted in a rapid, dose-dependent reduction in plasma PCSK9 of up to 84% relative to baseline and placebo, with a corresponding reduction in serum levels of low-density lipoprotein cholesterol (LDL-C) – a clinically validated endpoint – of up to 57% relative to baseline and placebo. The knockdown of PCSK9 and lowering of LDL-C were also found to be durable, with effects lasting for weeks after a single dose. This new paper documents the first human proof of concept for an RNAi therapeutic impacting a clinically validated endpoint.