29 Sep, 2021 RNAi Therapeutics Platform Progress at OTS 2021 Annual Meeting

We presented progress on our RNAi therapeutics platform efforts at the Oligonucleotide Therapeutics Society (OTS) 2021 Annual Meeting.

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Harnessing RNAi for a New Class of Medicines – John Maraganore

Expanding the Reach of RNAi Therapeutics – Kirk Brown

Development of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 – Pushkal Garg

Dose-Related and Prolonged Reductions in Blood Pressure with a RNAi Therapeutic Targeting Angiotensinogen in Hypertensive Patients: Interim Results from a Phase 1 Study with Zilebesiran (ALN-AGT01) – Stephen Huang

Progress of RNAi in Extrahepatic Tissues – Haiyan Peng

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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