Alnylam Announces Positive Early Results on Clinical Outcome Measures from ILLUMINATE-A Phase 3 Study of OXLUMO (lumasiran)

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive early results on clinical outcome measures from the 12-month analysis of ILLUMINATE-A Phase 3 study of OXLUMO (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1). These data were presented at the American Society of Pediatric Nephrology (ASPN)/Pediatric Academic Societies (PAS) virtual meeting being held on April 30–May 4, 2021.

As previously reported, treatment with OXLUMO significantly reduced urinary oxalate levels in infants¹, children^1,2 and adults² with PH1 in the ILLUMINATE-A and ILLUMINATE-B studies. OXLUMO also demonstrated an acceptable safety profile across age groups, with injection site reactions as the most common drug-related adverse reaction. New results from ILLUMINATE-A showed that treatment with OXLUMO for 12 months was associated with evidence of improvements in nephrocalcinosis in one or both kidneys, relative to baseline.

“Nephrocalcinosis is a key indicator of disease severity in PH1,” said Jeffrey M. Saland, M.D., Professor and Chief, Pediatric Nephrology and Hypertension, Jack and Lucy Clark Department of Pediatrics, Mount Sinai Kravis Children’s Hospital, New York City and Investigator on the ILLUMINATE-A trial. “Thus, unilateral and bilateral improvements in nephrocalcinosis in some patients treated with OXLUMO are a welcome observation and are consistent with our expectation of the potential clinical impact of a sustained and substantial reduction in urinary oxalate levels.”

“We are thrilled to be presenting positive nephrocalcinosis data from our ILLUMINATE-A study,” said Pushkal Garg, M.D., Chief Medical Officer at Alnylam. “These early data provide emerging evidence demonstrating improvements in nephrocalcinosis in some patients. Notably, the majority of patients with baseline nephrocalcinosis and available ultrasounds experienced improvement, often in both kidneys, after 12 months of treatment – an encouraging sign indicating the potential of OXLUMO to improve renal outcome measures in patients with PH1. We look forward to reporting additional data later in the year.”