05 Nov, 2021 Results from ILLUMINATE-C Phase 3 Study of Lumasiran Presented at ASN Kidney Week 2021

Positive results from the six-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1), were presented as part of the late-breaking session at the American Society of Nephrology (ASN) Kidney Week 2021 virtual meeting held on November 4-7.

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Michael, et al. – “ILLUMINATE-C, a single-arm, phase 3 study of lumasiran in patients with primary hyperoxaluria type 1 and CKD3b-5, including those on hemodialysis”

Lieske, et al. – “Modeling the risk of progression to kidney failure in patients with primary hyperoxaluria type 1 treated with lumasiran relative to a natural history cohort not treated with lumasiran”

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26 Oct, 2021 New International Survey Presented at ACG 2021 Sheds Light on AHP Burden

The POWER study (POrphyria Worldwide Patient Experience Research) surveyed 92 patients living with acute hepatic porphyria (AHP) around the world to bring to light the emotional, mental, and physical burden of AHP on the individual and their social circle. Survey participants reported substantial emotional burden associated with AHP, citing moderate to severe depression and anxiety. They also reported significant pain levels and work impairment. These data are being presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting 2021. Encore 24-month interim data from the open-label extension period of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) in patients with AHP will also presented, along with additional findings on the burden of AHP.

Dickey, et al. “POWER (Porphyria Worldwide Patient Experience Research) Study: Impact of Acute Hepatic Porphyria Assessed Through an International Patient Survey”

Bonkovsky, et al. “Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyria: 24-Month Interim Analysis of the Phase 3 ENVISION Randomized Clinical Trial”

Wang, et al. “Disease Burden in Patients with Acute Hepatic Porphyria: Experience from the Phase 3 ENVISION Study”

Cassiman, et al. “EXPLORE Part B: A Prospective, International, Long-term Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Symptoms”

04 Oct, 2021 New Long-Term Results for GIVLAARI® (givosiran) Presented at UEG Week Virtual 2021

Positive findings from a 24-month interim analysis of the ongoing open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) in patients with acute hepatic porphyria (AHP) were presented during the United European Gastroenterology (UEG) Week Virtual 2021 (Oct. 3-5). The data further confirm that long-term dosing with GIVLAARI provides sustained and continuous benefit to patients with AHP while maintaining an acceptable safety profile. Findings on the burden of AHP from the ENVISION Phase 3 study and from Part B of the EXPLORE study were also presented.

Bonkovsky, et al. “Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyria: 24-Month Interim Analysis of the Phase 3 ENVISION Randomized Clinical Trial”

Wang, et al. “Disease Burden in Patients with Acute Hepatic Porphyria: Experience from the Phase 3 ENVISION Study”

Cassiman, et al. “EXPLORE Part B: A Prospective, International, Long-term Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Symptoms”

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”