NEW YORK (GenomeWeb) – Alnylam Pharmaceuticals last week released new preclinical data on ALN-CC5, its siRNA-based treatment for complement-mediated diseases, showing that the drug is effective in silencing its target and may offer therapeutic benefit in an associated kidney disorder.
In a study of non-human primates, ALN-CC5 was administered subcutaneously either monthly or twice a month. At 5 mg/kg, either dosing regimen led to as much as 98.7 percent clamped knockdown of serum levels of the C5 component of the complement pathway. It also inhibited complement activity by up to 91.3 percent.
Based on translational data of its second-generation GalNAc delivery conjugates, which will be used with the drug, Alnylam said that doses less than 1 mg/kg and 1 mL/injection are expected to have similar effects in humans.
Meanwhile, in a rat model of primary membranous nephropathy — a complement-mediated progressive disease of the kidney — ALN-CC5 treatment led to a greater than 70 percent reduction in proteinuria associated with complement-mediated renal damage.
The data were presented at the 25th International Complement Workshop in Brazil.
Alnylam said that it remains on track to file a regulatory application to begin testing ALN-CC5 in humans before the end of this year, with initial clinical data available by mid-2015.