December 2020

On December 15th and 16th, 2020, we hosted a virtual R&D Day event showcasing Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The Company also discussed its perspective on its transition toward achieving a self-sustainable financial profile. The event included presentations from Alnylam management and key opinion leaders. To view a replay of the webcast, click here. To view the R&D Day presentation for Day 1, click here. To view the R&D Day presentation for Day 2, click here.

During the 62nd American Society of Hematology Annual Meeting & Exposition, we presented new 18-month interim results from the ongoing open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI® (givosiran) in patients with acute hepatic porphyria (AHP), demonstrating sustained efficacy and safety with long-term dosing. Kuter, et al. Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the ENVISION Open-Label Extension

Positive results from the six-month primary analysis of the ongoing ILLUMINATE-B Phase 3 open-label pediatric study of lumasiran, an investigational RNAi therapeutic in development for the treatment of adults and children with Primary Hyperoxaluria Type 1 (PH1), were presented at the American Society of Nephrology (ASN) Kidney Week 2020, held October 22-25 as a virtual event. Additionally, new 12-month results from the ILLUMINATE-A pivotal Phase 3 study and the ongoing Phase 2 open-label extension (OLE) study were also presented. Read the press release View the ILLUMINATE-B presentation View the ILLUMINATE-A presentation View the Phase 2 OLE presentation View the Chart Review presentation

We presented progress on our RNAi therapeutics platform efforts at the Oligonucleotide Therapeutics Society (OTS) 2020 Annual Meeting. Akinc et al. – “ALN-COV: An Investigational RNAi Therapeutic for COVID-19” Brown et al. – “Evidence for an Intracellular Depot that Contributes to the Extended Duration of Activity of...

On September 3, 2020, we hosted an online RNAi Roundtable to review progress with patisiran and vutrisiran, RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis. Of note, we announced we have obtained clinical pharmacology data supporting the potential for a biannual subcutaneous dosing regimen option for vutrisiran, providing further product differentiation as a potential best-in-class agent, and discussed new clinical data presented at the European Society of Cardiology 2020 Congress providing further evidence that treatment with patisiran may lead to substantial reduction in cardiac amyloid burden in ATTR amyloidosis. Read the press release Access the replay View the presentation Read the transcript

During The Digital International Liver Congress™ 2020, we shared new interim data from the Phase 1/2 open-label extension (OLE) study of GIVLAARI® (givosiran) in acute hepatic porphyria (AHP), as well as encore 12-month interim data from the OLE period of the ENVISION Phase 3 study of GIVLAARI. Stein, et al. - “A Phase 1/2 Open-Label Extension Study of Givosiran, an Investigational RNAi Therapeutic, in Patients with Acute Intermittent Porphyria” View the 12-month ENVISION OLE results


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