Capella

Capella—the Online Voice of Progress in RNAi

Welcome to Capella, Alnylam’s destination for updates on our work translating the breakthrough discovery of RNA interference (RNAi) into an innovative new class of medicines. We’ve been pioneering RNAi therapeutics since 2002 and are excited to share our ongoing scientific progress.

During the annual Heart Failure congress of the Heart Failure Association of the European Society of Cardiology, taking place May 21-24, 2022, we presented new 18-month results from the exploratory cardiac endpoints in the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis.

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Garcia-Pavia, et al. “HELIOS-A: 18-Month Exploratory Cardiac Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis”

New results from the HELIOS-A study of vutrisiran, an investigational RNAi therapeutic, in hereditary transthyretin-mediated (hATTR) amyloidosis patients with polyneuropathy, were presented during the Peripheral Nerve Society (PNS) 2022 Annual Meeting, taking place May 14-17, 2022.

Ajroud-Driss, et al. “HELIOS-A: Impact of Vutrisiran on Quality of Life and Functional Status in Hereditary Transthyretin-Mediated Amyloidosis”

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”

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”

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.

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 71st 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”

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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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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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 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.

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 63rd 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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