11 May, 2022 Preclinical Results on Alnylam’s Novel C16 Conjugate Technology for Delivery of siRNAs to the Central Nervous System Presented at TIDES USA 2022 Meeting

New results on Alnylam’s platform advancements enabling delivery of therapeutic short interfering RNAs (siRNAs) to the central nervous system (CNS) were presented at the TIDES USA, Oligonucleotide & Peptide Therapeutics 2022 Meeting being held in Boston on May 9-12. Additional preclinical data were presented on Alnylam’s GEMINI platform for simultaneous silencing of two transcripts within the CNS with a single siRNA entity.

Thiel CS., “Recent Advancements for the Delivery of siRNAs to the Central Nervous System”

25 Apr, 2022 New Results from 12M Analysis of ILLUMINATE-B Phase 3 Study of OXLUMO® (lumasiran) Presented at PAS 2022

New results from the 12-month analysis of the ongoing ILLUMINATE-B Phase 3 open-label pediatric study of OXLUMO^® (lumasiran) in patients less than six years of age with primary hyperoxaluria type 1 (PH1) were presented at the Pediatric Academic Societies (PAS) 2022 Meeting (April 21-25, 2022).

Michael, et al. “Efficacy and Safety of Lumasiran for Infants and Young Children With Primary Hyperoxaluria Type 1: 12-Month Analysis of the Phase 3 ILLUMINATE-B Trial”

07 Apr, 2022 New Results from the ILLUMINATE-A Study of OXLUMO® (lumasiran) Presented at NKF Spring Meetings

New findings from a 24-month analysis of the ongoing ILLUMINATE-A Phase 3 study of OXLUMO^® (lumasiran) in patients with primary hyperoxaluria type 1 (PH1) were presented during the National Kidney Foundation (NKF) Spring Clinical Meetings (April 6-10, 2022).

Lieske, et al. “Efficacy and Safety of Lumasiran in Patients with Primary Hyperoxaluria Type 1: 24-Month Analysis of the ILLUMINATE-A Trial”

Sas, et al. “Impact of Baseline Urinary Oxalate on Response to Lumasiran in Patients with Primary Hyperoxaluria Type 1”

Additional findings from a post hoc analysis of the impact of baseline urinary oxalate on response to lumasiran in patients with PH1 in the ILLUMINATE-A study were presented.

04 Apr, 2022 New Results from HELIOS-A Phase 3 Study of Vutrisiran Presented at ACC

New results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic, in hereditary transthyretin-mediated (hATTR) amyloidosis patients with polyneuropathy, were presented during a poster presentation at the 71^st Annual Meeting of the American College of Cardiology (ACC) held April 2–4, 2022. The data help further our understanding of the safety and efficacy of vutrisiran in patients with hATTR amyloidosis with polyneuropathy who have variants that have previously been associated with predominant cardiomyopathy.

González-Duarte, et al. “HELIOS-A: Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

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.

Read the press release

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