(RTTNews.com) - Alnylam Pharmaceuticals Inc. ( ALNY ) announced new positive results from its Phase 1/2 study with lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase or GO for the treatment of Primary Hyperoxaluria Type 1 or PH1.
Updated interim data were from Part B of the Phase 1/2 study and were as of the data cut-off date of March 29, 2018. Part B is a randomized (3:1 drug:placebo), single-blind, placebo-controlled evaluation of lumasiran in patients with PH1. Cohorts 1 and 2 received three monthly doses of lumasiran at 1 mg/kg or 3 mg/kg, respectively; Cohort 3 received two quarterly doses at 3 mg/kg.
An additional eight patients received open-label lumasiran in expansions of each of the first two cohorts, totaling 20 patients enrolled. Patients randomized to the placebo group also received subsequent subcutaneous administration of lumasiran following administration of placebo. Patients had a mean age of 14.9 years (range: 6-43) and a mean estimated glomerular filtration rate (eGFR) of 77 mL/min/1.73m2 (range: 42-131).
Lumasiran demonstrated a mean maximal reduction in urinary oxalate of 64 percent in patients enrolled in Cohorts 1-3 (N=12). All lumasiran-treated patients experienced a lowering in urinary oxalate below 0.7 mmol/24 hrs/1.73m2, a threshold level associated with a reduced rate of progression to end-stage renal disease. On day 85, patients receiving lumasiran (N=9) maintained a mean reduction in urinary oxalate of 63 percent (range: 49-73 percent).
Alnylam believes the potent and durable reductions in urinary oxalate support a once quarterly, subcutaneous dose regimen. Further, these results continue to support the hypothesis that GO inhibition has the potential to reduce and possibly normalize levels of hepatic oxalate production, thus potentially halting PH1 disease progression. Dosing in Part B of the Phase 1/2 study is ongoing and eligible patients are transitioning into an open-label extension (OLE) study.
The company expects to present additional data from all cohorts as well as from the OLE study in late 2018.
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