Alnylam's Lumasiran Substantially Reduces Plasma Oxalate Levels PH1 Patients With Severe Renal Impairment

Alnylam Pharmaceuticals Inc ALNY announced topline results from the ILLUMINATE-C Phase 3 open-label study of lumasiran in primary hyperoxaluria type 1 (PH1) associated with progressive decline in renal function. 

• Lumasiran is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1), the gene encoding glycolate oxidase (GO).
• Results of the primary analysis at six months demonstrated a substantial reduction in plasma oxalate from baseline in patients (N=21) with advanced disease, including those on hemodialysis. 
• Elevated plasma oxalate is directly related to the pathophysiology of oxalosis and results in systemic deposition of oxalate in extra-renal tissues. 
• The safety and tolerability profile is encouraging, with no drug-related serious adverse events and injection site reactions as the most common adverse event.
• The Company plans to submit a supplemental marketing application for lumasiran with the FDA and the European Medicines Agency in late 2021. 
• Price Action: ALNY shares are up 0.87% at $183.68 during the market session on the last check Thursday.