- Updated results from a Phase 1/2 clinical trial assessing Alnylam Pharmaceuticals' (NASDAQ:ALNY) lumasiran in patients with primary hyperoxaluria type 1 (PH1) continued to show a treatment effect. The data were presented at OxalEurope in Naples, Italy.
- All treated patients experienced a lowering of urinary oxalate below the threshold associated with a reduced rate of progression to end-stage renal disease. Nine patients receiving lumasiran maintained an average reduction in urinary oxalate of 63%.
- Lumasiran is an RNAi therapeutic that targets a liver enzyme called glycolate oxidase (GO). Reducing GO depletes the substrate for oxalate production which contributes to the pathology of PH1, a rare inherited disorder affecting the kidneys. Excess oxalate combines with calcium to form kidney and bladder stones (calcium oxalate).
- A pivotal Phase 3 study should launch shortly with topline results expected in 2019. If all goes well, a U.S. marketing application will be filed in early 2020.
- Previously: Interim data on Alnylam's lumasiran show positive effect in PH1 patients; shares ahead 2% (Nov. 3, 2017)
Alnylam's lumasiran shows positive effect in Phase 1/2 PH1 study
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