Alnylam Pharmaceuticals (ALNY -0.2%) initiates an open-label Phase 3 clinical trial, ILLUMINATE-B, evaluating lumasiran in infants and young children with primary hyperoxaluria type 1 (PH1), a rare inherited disorder characterized by kidney and bladder stones caused by the buildup a compound called oxalate in the kidneys and urinary tract.
The primary endpoint of the eight-subject study is the percent reduction in urinary oxalate from baseline at month 6. The estimated primary completion date is March 2020.
Lumasiran is an RNAi therapeutic designed to lower oxalate by reducing the levels of an enzyme called glycolate oxidase in the liver which depletes the substrate necessary for oxalate production. It has accelerated review status in the U.S. and Europe.
Updated results from a Phase 1/2 study showed a 72% average maximum reduction from baseline in urinary oxalate excretion. The average reduction from baseline at day 85 was 69%.
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