ALN-PCSsc – an RNAi investigational agent that inhibits PCSK9 – may be safe and is "generally well tolerated" in patients with high low-density lipoprotein cholesterol (LCL-C), according to results of initial ALN-PCSsc phase 1 study results, presented Nov. 11 during AHA 2015 in Orlando.

The trial looked at 69 patients with LDL-C >100 mg/dl, on or off statins, who were randomized to a single-ascending dose and multiple dose of ALN-PCSsc. Results showed that overall, ALN-PCSsc was "generally well tolerated," and no serious adverse events occurred. Further, a single injection of ALN-PCSsc reduced PCSK9 levels and lowered LDL-C for more than 140 days.

The investigators note that ALN-PCSsc may be a "promising innovative therapeutic approach to managing hypercholesterolemia." They add that a phase 2 study – ORION-1 – will begin in quarter four of 2015.

Keywords: Cholesterol, Dyslipidemias, Hydroxymethylglutaryl-CoA Reductase Inhibitors, Hypercholesterolemia, Lipoproteins, LDL, RNA Interference, AHA Annual Scientific Sessions

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