Press release
Alnylam Presents New Results from the ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1
– Lumasiran Demonstrated Evidence of Improvements in Exploratory Endpoints of Systemic Oxalosis, Including Echocardiographic Structure and Function,
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– Lumasiran Demonstrated Evidence of Improvements in Exploratory Endpoints of Systemic Oxalosis, Including Echocardiographic Structure and Function, Nephrocalcinosis, and Kidney Stone Events –\n\n– Expands Upon Previously Reported 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, today announced new positive results from the six-month primary analysis period 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). As previously reported, treatment with lumasiran resulted in substantial reductions in plasma oxalate (POx) at six months. Results from new exploratory analyses provide evidence that lumasiran treatment resulted in improvements in cardiac measures, nephrocalcinosis and kidney stone events. In addition, some patients treated with lumasiran experienced improvements in their most burdensome symptoms (as assessed by the investigator), including fatigue, nausea, and bone pain, with no patients reporting worsening of those symptoms. These data were presented at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress being held both in Paris, France, and as a virtual event on May 19-22.\n\n“Patients with compromised renal function due to advanced PH1 tend to have elevated levels of plasma oxalate, which can lead to systemic spread of oxalate to organs beyond the kidneys, including the heart,” said Professor Jaap Groothoff, M.D., Ph.D., Head of the Department of Pediatric Nephrology, Emma Children’s Hospital, Amsterdam UMC, University of Amsterdam, The Netherlands. “The ILLUMINATE-C study of lumasiran shows impressive plasma oxalate lowering in patients with severely impaired kidney function or who are on dialysis. These data, along with previous findings from ILLUMINATE-A and ILLUMINATE-B Phase 3 studies, provide evidence supporting the potential of lumasiran to benefit not only these patients, but those across the full spectrum of PH1 severity.”\n\n“We are thrilled to be presenting preliminary data o...