11 Nov, 2015 New Clinical Data from Phase 1 Trial with ALN-PCSsc Confirms Potential for Bi-Annual Dosing
Alnylam and The Medicines Company reported updated positive results from the ongoing Phase 1 clinical trial with ALN-PCSsc, an investigational RNAi therapeutic targeting PCSK9 for the treatment of hypercholesterolemia. We had previously reported that subcutaneous administration of ALN-PCSsc resulted in an up to 83 percent lowering of LDL-C, with an up to 64 ± 5 percent mean maximum reduction. In new results, the effects of ALN-PCSsc were also found to be highly durable, with clinically significant and clamped reductions in LDL-C that now confirm the potential for a bi-annual subcutaneous dose regimen. An up to 53 percent maximal and 47 percent least squares mean reduction in LDL-C was achieved at day 180 after a single, low volume injection. In addition, ALN-PCSsc was shown to reduce a number of atherogenic lipids, including lipoprotein (a), and total cholesterol, which are associated with increased risk of cardiovascular disease. Importantly, ALN-PCSsc was generally well tolerated with no clinically significant drug-related adverse events to date.