- Alnylam Pharmaceuticals (NASDAQ:ALNY) earns a $10M milestone payment from The Medicines Company (MDCO) by initiating a Phase 1 trial evaluating the safety and tolerability of Alnylam's cholesterol-lowering product candidate ALN-PCSsc in up to 76 volunteers with elevated baseline LDL-C. The U.K.-based study will be conducted in two stages: a single ascending dose and multiple doses. Initial data from the trial should be available by mid-2015.
- ALN-PCSsc is a PCSK9 inhibitor that is delivered via subcutaneous injection. Based on preclinical studies, it has the potential for a once-monthly or once-quarterly dose regimen. In non-human primate studies, the administration of ALN-PCSsc resulted in PCSK9 knockdown of up to 92% and lowering of LDL-C up to 77% without the co-administration of statins.