December 2021

13 Dec 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”

13 Dec 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 63rd American Society of Hematology (ASH) Annual Meeting & Exposition (Dec. 11-14). The data, which encompass the 6-month double-blind and...

12 Dec 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)”

09 Dec 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...

13 Nov 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). Read the press release 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”

05 Nov 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. Read the press release 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”

26 Oct 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...

04 Oct 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...