10 Jun, 2020 ENVISION Phase 3 Results for Givosiran Published in the “New England Journal of Medicine”

The New England Journal of Medicine (NEJM) published pivotal results from the ENVISION Phase 3 study of givosiran, our RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria, in a manuscript titled “Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.”

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07 Jun, 2020 ILLUMINATE-A Phase 3 Results for Lumasiran Presented at ERA-EDTA

Positive results from the ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic in development for the treatment of Primary Hyperoxaluria Type 1 (PH1), were presented by study investigators at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress, held June 6-9, 2020 as a virtual event.

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22 May, 2020 New Results for Patisiran and Givosiran at EAN

We presented new long-term results from the global Open-Label Extension (OLE) study of ONPATTRO^® (patisiran) at the European Academy of Neurology (EAN) Virtual Congress, held May 23-26, 2020. In addition, results were presented from a Phase 3b trial evaluating treatment with patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (post-OLT). A post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) was also presented.

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View the patisiran global OLE poster

View the patisiran post-OLT poster

View the ENVISION post-hoc analysis

View the presentation on disease burden and healthcare utilization among patients with acute intermittent porphyria

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14 Apr, 2020 New Clinical Results for Givosiran at EASL

An abstract describing new 12-month interim data from the ongoing ENVISION open-label extension (OLE) study of givosiran in patients with acute hepatic porphyria (AHP) has been published online by the European Association for the Society of the Liver (EASL) on the International Liver Congress™ (ILC 2020) website.  The data will be presented during the ILC 2020 (London, UK) which was rescheduled to August 25-28, 2020 due to the COVID-19 pandemic.

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09 Nov, 2019 New Clinical Results for Lumasiran at ASN

We presented new positive efficacy results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, an investigational subcutaneously administered RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), at the American Society of Nephrology (ASN) 2019 Annual Meeting, held November 5-10 in Washington, DC.

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07 Nov, 2019 EXPLORE Natural History Study Data Published in “Hepatology”

We published results from the EXPLORE natural history study of more than 100 acute hepatic porphyria (AHP) patients with recurrent attacks in Hepatology in a paper titled, “EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.”

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20 Oct, 2019 Results from Pediatric Cohort of Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 (PH1) and Insights from a Survey of Physician Experiences with PH1

We presented results on the pediatric cohort of patients (N=16; age range 6-17) from the Phase 1/2 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the International Pediatric Nephrology Association (IPNA) 2019 Annual Meeting, held October 17-21 in Venice, Italy.

Frishberg et al. – Phase 1/2 Study of Lumasiran, Investigational RNAi Therapeutic, in Patients with Primary Hyperoxaluria Type 1

Danese et al. – Understanding the Burden of Primary Hyperoxaluria Type 1 (PH1): A Survey of Physician Experiences with PH1

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