21 Jan, 2022 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran Presented at SFNP

Positive 18-month results from the HELIOS-A Phase 3 study evaluating vutrisiran, an investigational RNAi therapeutic, in hereditary transthyretin-mediated (hATTR) amyloidosis patients with polyneuropathy, were presented in an oral session at the Société Francophone du Nerf Périphérique (SFNP) Annual Meeting, held January 21-22, 2022.

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Adams et al. – “HELIOS-A: Study of Vutrisiran in Patients with hATTR Amyloidosis”

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13 Dec, 2021 New Results from Phase 1 Study of Cemdisiran + Pozelimab Presented at ASH Annual Meeting 2021

New results from a Phase 1 study of cemdisiran in combination with pozelimab in healthy volunteers, in collaboration with Regeneron, were presented at the 63rd American Society of Hematology (ASH) Annual Meeting, held December 11-14, 2021.

Van Zyl, et al. “Open-Label, Ascending-Dose, Phase 1 Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Doses of the Subcutaneously Administered Human Monoclonal Antibody Pozelimab in Combination with Single Doses of the Subcutaneously Administered siRNA Cemdisiran in Healthy Volunteers”

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13 Dec, 2021 New Long-Term Results for GIVLAARI® (givosiran) Presented at ASH Annual Meeting & Exposition

Findings from a 36-month analysis of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) in patients with acute hepatic porphyria (AHP) were presented during the 63^rd American Society of Hematology (ASH) Annual Meeting & Exposition (Dec. 11-14). The data, which encompass the 6-month double-blind and 30-month open label extension (OLE) periods, further confirm that long-term dosing with GIVLAARI provides sustained and continuous benefit to patients with AHP, while maintaining an acceptable and consistent safety profile.

Kuter, et al. “Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyria: 36-Month Results of the Phase 3 ENVISION Randomized Clinical Trial”

Long-term use of GIVLAARI during the OLE period demonstrated a durable response with efficacy across a wide range of clinical parameters, including sustained reductions in the annualized rate of composite porphyria attacks and hemin use, sustained lowering of ALA and PBG levels and improvements in physical functioning and QOL.

12 Dec, 2021 New Results from ATLAS Phase 3 Program with Fitusiran Presented at ASH Annual Meeting 2021

New results from the ATLAS Phase 3 program with fitusiran, an investigational RNAi therapeutic in development for the treatment of hemophilia A or B with and without inhibitors, in collaboration with Sanofi, were presented at the 63^rd American Society of Hematology (ASH) Annual Meeting, held December 11-14, 2021.

Srivastava, et al. “Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)”

Young, et al. “Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)”

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09 Dec, 2021 ILLUMINATE-B Phase 3 Results of OXLUMO® (lumasiran) Published in Genetics in Medicine

Six-month results from the ongoing ILLUMINATE-B Phase 3 open-label pediatric study 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 published in Genetics in Medicine. The findings show that, in patients under the age of six, treatment with OXLUMO led to a 72% mean reduction in spot urinary oxalate:creatinine ratio from baseline to Month 6, averaged across Months 3 to 6. The efficacy results and safety profile of OXLUMO in these patients were similar to those observed in adults and children six years or older in the ILLUMINATE-A study.

Read the paper in Genetics in Medicine

13 Nov, 2021 New Results for Zilebesiran Presented at AHA Scientific Sessions 2021

Positive interim findings from the ongoing Phase 1 study of zilebesiran (formerly known as ALN-AGT) in adults with hypertension were presented during the American Heart Association (AHA) Scientific Sessions 2021 (Nov. 13-15).

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Huang, et al. “Durable Reductions in Circulating Angiotensinogen and Blood Pressure 6 Months after Single Doses of ALN-AGT, an RNA Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Hypertensive Patients”

Huang, et al. “Safety and Tolerability of ALN-AGT, an RNA Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Hypertensive Patients during Sodium Depletion or Irbesartan Coadministration”

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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”