10 Sep, 2021 New Results from the OXLUMO® (lumasiran) Clinical Program Presented at AUA Annual Meeting

New kidney stone data from ongoing clinical trials of OXLUMO^® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase – for the treatment of primary hyperoxaluria type 1 (PH1), were presented at the American Urological Association (AUA) Annual Meeting, held virtually September 10-13, 2021. Results from the Phase 1/2 clinical trial of lumasiran and its Phase 2 open-label extension (OLE) period showed that treatment with lumasiran led to an apparent reduction in kidney stone related adverse events in pediatric and adult patients with PH1. A reduction in kidney stone event (KSE) rates – an exploratory endpoint in ILLUMINATE-A and ILLUMINATE-B Phase 3 trials – was reported in ILLUMINATE-A at Month 12, with KSE rates remaining stable through Month 6 in ILLUMINATE-B.

Lieske, et al. – “Effect of lumasiran on kidney stones and nephrocalcinosis in patients with primary hyperoxaluria type 1”

07 Sep, 2021 New Results for Vutrisiran at EU-ATTR Amyloidosis Meeting

Additional positive 9-month results from subgroup analyses and exploratory endpoints of the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with polyneuropathy, were presented at the 3^rd EU-ATTR Amyloidosis Meeting. Subgroup analyses and exploratory endpoints demonstrated that vutrisiran improved important areas of patient health and function compared with placebo at 9 months. Additional analyses showed similar improvements in progression of neuropathy and quality of life measures with vutrisiran compared with placebo, regardless of prior TTR stabilizer use.

Gillmore, et al. – “HELIOS-A: 9-Month Subgroup Analyses and Exploratory Efficacy Results From the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

Berk, et al. – “Reason for Stopping Transthyretin Stabilizers Prior to HELIOS-A and the Impact of Prior Stabilizer Use on the Efficacy of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

07 Jun, 2021 New Results for Patisiran at PNS 2021

Positive results from a Phase 3b open-label study of patisiran in hereditary ATTR amyloidosis patients with polyneuropathy progression after receiving an orthotopic liver transplant were presented at the Peripheral Nerve Society’s 2021 Annual Meeting.

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07 Jun, 2021 New Analyses from OXLUMO® (lumasiran) Clinical Development Program Presented at ERA-EDTA Congress

Results from pooled, post-hoc analyses from ongoing clinical trials of OXLUMO^® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase – for the treatment of primary hyperoxaluria type 1 (PH1), were presented at the 58^th European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) virtual congress, held June 5-8, 2021. The analyses found that treatment with lumasiran led to substantial and clinically meaningful reductions in urinary and plasma oxalate that were similar in pediatric and adult patients with PH1 in the ILLUMINATE-A and ILLUMINATE-B Phase 3 trials, with lumasiran demonstrating an acceptable safety profile in both patient populations. In additional pooled, post-hoc analyses, PH1 patients 12 months and older with a baseline estimated glomerular filtration rate (eGFR) of ≥30 mL/min/1.73m², treated with lumasiran through Month 12, demonstrated stable eGFR levels, irrespective of baseline kidney function – an encouraging observation, given the progressive decline in kidney function characteristic of PH1.

Shasha-Lavsky, et al. “Lumasiran Demonstrated Comparable Oxalate Reduction and Safety in Children and Adults with Primary Hyperoxaluria Type 1”

Hayes, et al. “Stable eGFR in Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran, Regardless of Kidney Function at Start of Treatment”

14 May, 2021 Complete Results from Phase 1/2 Study of Lumasiran (OXLUMO®) Published in Clinical Journal of the American Society of Nephrology

Complete results from the Phase 1/2 study of lumasiran for the treatment of primary hyperoxaluria type 1 (PH1) were published in the Clinical Journal of the American Society of Nephrology. The final results demonstrated an acceptable safety profile of OXLUMO in adult and pediatric patients. The majority of adverse events were mild or moderate; there were no serious adverse events considered to be drug-related and no adverse events led to death, study discontinuations or study withdrawals. After treatment with lumasiran, all 20 lumasiran-treated patients exhibited near-normal¹ levels of 24-hour urinary oxalate excretion, with majority of such patients achieving levels within the normal² range.

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