- Alnylam Pharmaceuticals Inc ALNY has announced that trial results from the ILLUMINATE-A Phase 3 study evaluating lumasiran for the treatment of primary hyperoxaluria type 1 (PH1). The results were published online in The New England Journal of Medicine.
- Lumasiran an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO)
- In November last year, Oxlumo (lumasiran) was approved by the FDA to treat PH1 in pediatric and adult patients and received marketing authorization from the European Commission for the treatment of PH1 in all age groups.
- The data reported in the publication demonstrated that lumasiran led to substantial and sustained reductions in urinary oxalate, the toxic metabolite responsible for the clinical manifestations of PH1.
- As compared to placebo, treatment with lumasiran resulted in a clinically significant (53.5%) reduction in 24-hour urinary oxalate excretion from baseline to month 6.
- Improvements were also observed in many secondary endpoints, including the proportion of patients achieving normal or near-normal urinary oxalate levels, with 84% of lumasiran-treated patients compared with no patients on placebo.
- Patients treated with lumasiran also experienced favorable effects on exploratory endpoints related to nephrocalcinosis and the rate of renal stone events compared with placebo.
- Lumasiran was associated with a favorable safety and tolerability profile, with no serious or severe adverse events.
- Price Action: ALNY shares are trading 0.96% higher at $142.55 in the premarket session on the last check Thursday.
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