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.
On July 17, 2020, we hosted an online RNAi Roundtable to review progress in our early stage RNAi therapeutics pipeline.
New results and analyses of study data for ONPATTRO® (patisiran) were presented at the 2020 Peripheral Nerve Society (PNS) Annual Meeting, held June 27-30 as a virtual event.
Lin, et al. – “Evaluation of Patisiran with Concomitant or Prior Use of Transthyretin Stabilizers”
Interim results from the open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI® (givosiran) in acute hepatic porphyria (AHP) were presented by study investigator Eliane Sardh, M.D., Ph.D. during a webinar hosted by Alnylam, demonstrating sustained efficacy and acceptable safety through 12 months of treatment, with evidence for potentially improved efficacy over time.
On June 23, 2020, we hosted an online RNAi Roundtable to review the progress with ALN-AGT, an RNAi therapeutic in development for the treatment of hypertension.
New positive results from an interim analysis of the Phase 2 open-label extension (OLE) study of fitusiran, in development for the treatment of hemophilia A or B, with or without inhibitors, were presented at the World Federation of Hemophilia (WFH) Virtual Summit, held June 14-19, 2020.
We are hosting a series of online “RNAi Roundtables” at which Alnylam scientists and program leaders, as well as clinical collaborators, will review recent progress in many of our pipeline programs and platform.
The New England Journal of Medicine (NEJM) published pivotal results from the ENVISION Phase 3 study of givosiran, our RNAi therapeutic targeting aminolevulinic acid synthase 1 for the treatment of acute hepatic porphyria, in a manuscript titled “Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.”
Positive results from the ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic in development for the treatment of Primary Hyperoxaluria Type 1 (PH1), were presented by study investigators at the European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) International Congress, held June 6-9, 2020 as a virtual event.
We presented new long-term results from the global Open-Label Extension (OLE) study of ONPATTRO® (patisiran) at the European Academy of Neurology (EAN) Virtual Congress, held May 23-26, 2020. In addition, results were presented from a Phase 3b trial evaluating treatment with patisiran in patients with hATTR amyloidosis with disease progression after receiving an orthotopic liver transplant (post-OLT). A post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI® (givosiran) was also presented.
View the patisiran global OLE poster
View the patisiran post-OLT poster
An abstract describing new 12-month interim data from the ongoing ENVISION open-label extension (OLE) study of givosiran in patients with acute hepatic porphyria (AHP) has been published online by the European Association for the Society of the Liver (EASL) on the International Liver Congress™ (ILC 2020) website. The data will be presented during the ILC 2020 (London, UK) which was rescheduled to August 25-28, 2020 due to the COVID-19 pandemic.
