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.
We believe that ALN-PCSsc, a first-in-class PCSK9 synthesis inhibitor, has a highly competitive profile as compared with anti-PCSK9 monoclonal antibodies that are labeled for twice-monthly or monthly dosing. The potential for management of hypercholesterolemia with two injections per year could be a transformative option for this massive, at-risk, often non-adherent patient population. We look forward to the continued advancement of ALN-PCSsc for the treatment of hypercholesterolemia.