Alnylam Completes Enrollment In ILLUMINATE-C Phase 3 Study Of RNAi Therapeutic, Lumasiran To Treat Advanced Primary Hyperoxaluria Type 1

Alnylam Pharmaceuticals that it has achieved full patient enrollment in its ILLUMINATE-C phase 3 study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of adults and children with advanced primary hyperoxaluria type 1 (PH1). Enrollment was completed with 21 PH1 patients across 13 sites in 10 countries.“We are pleased to have completed enrollment in the ILLUMINATE-C phase 3 study,” said Pritesh J. Gandhi, PharmD., vice president and general manager, Lumasiran Program at Alnylam. “This is an important milestone to help evaluate the efficacy and safety of lumasiran in patients with advanced PH1 including those experiencing systemic oxalosis and undergoing hemodialysis. We believe lumasiran has the potential to address the full spectrum of PH1 disease severity and look forward to reporting topline results from this important study in mid-2021.”Lumasiran was recently approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients. It was also recently approved by the EMA for the treatment of PH1 in all age groups. Lumasiran is marketed in the US and EU as Oxlumo.ILLUMINATE-C is a single arm, open-label, multinational phase 3 study to evaluate the safety and efficacy of lumasiran in PH1 patients of all ages and advanced renal disease (eGFR = 45 mL/min/1.73m2 or elevated serum creatinine for patients <12 months of age). Cohort A enrolled 6 patients with advanced PH1 who do not yet require dialysis and Cohort B enrolled 15 patients who are dialysis-dependent. The dosing regimen is based on weight with three monthly starting doses followed by ongoing monthly or quarterly doses. The primary endpoint of the study is the percent change in plasma oxalate from baseline to month 6. Key secondary endpoints are designed to evaluate additional measures of plasma oxalate and changes in urinary oxalate, and quality of life assessments. Renal function, frequency and mode of dialysis, frequency of renal stone events, and measures of systemic oxalosis will also be evaluated in the extension period of the study.Lumasiran is a subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. Lumasiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. Lumasiran has received regulatory approvals from the US Food and Drug Administration (FDA) for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and from the European Medicines Agency (EMA) for the treatment of PH1 in all age groups under the brand name Oxlumo.PH1 is an ultra-rare disease in which excessive oxalate production results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones and nephrocalcinosis. Renal damage is caused by a combination of tubular toxicity from oxalate, calcium oxalate deposition in the kidneys, and urinary obstruction by calcium oxalate stones.

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