Press release
Alnylam Presents Positive Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1
- Lumasiran Resulted in Substantial Reductions in Plasma Oxalate in PH1 Patients with Severe Renal Impairment - CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Lumasiran Resulted in Substantial Reductions in Plasma Oxalate in PH1 Patients with Severe Renal Impairment -\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today positive results from the 6-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – that is being investigated for the treatment of patients of all ages with advanced primary hyperoxaluria type 1 (PH1). The clinical data were presented at a late-breaking session at the American Society of Nephrology (ASN) Kidney Week being held as a virtual event on November 4-7. Lumasiran resulted in substantial reductions in plasma oxalate (POx) in PH1 patients with chronic kidney disease (CKD) stage 3b-5, with or without dialysis. Elevated plasma oxalate is directly correlated with the pathophysiology of systemic oxalosis, a life-threatening complication of PH1. Lumasiran demonstrated an acceptable safety profile through Month 6, with mild injection site reactions (ISRs) as the most common drug-related adverse event.\n\n“Elevated plasma oxalate levels are responsible for much of the morbidity associated with advanced PH1 and can result in oxalosis, leading to bone fractures, cardiomyopathy, vision loss, skin ulcers and other serious manifestations,” said Mini Michael, M.D., Associate Professor, Renal Section, Department of Pediatrics, Baylor College of Medicine, Houston, and Pediatric Nephrologist, Texas Children’s Hospital. “Changes in plasma oxalate of the magnitude seen in this study are encouraging and may positively impact long-term clinical outcomes, including those related to systemic oxalosis, which will be further evaluated in the extension period of this study.”\n\n“PH1 patients progressing to or being diagnosed with end-stage kidney disease often have substantial elevations in plasma oxalate despite intensive hemodialysis. The ILLUMINATE-C results underscore the potential of lumasiran to achieve meaningful reductions in plasma oxalate in patients with impaired kidney function, including those on hemodialysis, who are at risk of systemic oxalosis and multiorgan damage,” said Jeroen Valkenburg, General Manager, Lumasiran program at Alnylam. “We look forwa...