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 October 7, 2019, we hosted an online RNAi Roundtable to review the progress with givosiran, an RNAi therapeutic in development for the treatment of acute hepatic porphyria.
On September 16, 2019, we hosted an online RNAi Roundtable to review the progress with patisiran and vutrisiran, RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis.
ONPATTRO® (patisiran) is approved in the U.S. and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults, in the EU for the treatment of hATTR amyloidosis in adults with stage 1 or stage 2 polyneuropathy, and in Japan for the treatment of transthyretin (TTR) type familial amyloidosis with polyneuropathy. Vutrisiran is an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis.
We presented new results from an analysis of the UK Biobank demonstrating a significant association of the V122I mutation, a highly prevalent mutation in the transthyretin (TTR) gene, with a clinical diagnosis of polyneuropathy. These results were presented at the Heart Failure Society of America (HFSA) 23rd Annual Scientific Meeting, held September 13-16 in Philadelphia, PA.
We presented additional results from the ENVISION Phase 3 study and the Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in development for the treatment of acute hepatic porphyria (AHP), at the 2019 International Congress on Porphyrins and Porphyrias (ICPP), held September 8-11 in Milan, Italy.
We are hosting a series of online “RNAi Roundtables” at which Alnylam scientists and program leaders, clinical collaborators, and patients or patient advocates review recent progress in many of our late-stage pipeline programs and discuss the related disease areas.
We presented results from a proteome-wide biomarker analysis of samples from the APOLLO Phase 3 study of ONPATTRO® (patisiran), as well as results from an analysis of the UK Biobank on clinical outcomes and medical history of individuals with the non-pathogenic transthyretin (TTR) ‘stabilizing’ T119M variant, at the Second European Meeting of ATTR Amyloidosis (EU-ATTR) for Doctors and Patients, held September 2-3 in Berlin, Germany.
Our partner The Medicines Company and collaborators presented positive complete results from the ORION-11 Phase 3 study of inclisiran, an RNAi therapeutic in development for the treatment of hypercholesterolemia, at the European Society of Cardiology’s ESC Congress 2019, held August 31 to September 4, 2019 in Paris, France.
We presented new 12-month interim results from the ongoing Global Open-Label Extension (OLE) study of ONPATTRO® (patisiran), demonstrating maintained reversal of disease progression and consistent safety profile with >4 years of patient experience and >6,000 doses administered. These results were presented at the 2019 Peripheral Nerve Society (PNS) Annual Meeting, held June 22-26 in Genoa, Italy.
We presented complete positive results from the Phase 1/2 clinical study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase for the treatment of primary hyperoxaluria type 1 (PH1) and reiterated positive results from our ongoing Phase 2 open-label extension (OLE) study. Results were presented at the 2019 Oxalosis & Hyperoxaluria (OHF) International Workshop, held June 21-22 in Boston.
We presented new advances in our RNAi therapeutics platform, including preclinical results demonstrating oral delivery of GalNAc-conjugated small interfering RNAs (siRNAs) directed to a liver target. The results were presented at the 3rd International Conference on the Long and the Short of Non-Coding RNAs, held June 18-23 in Crete, Greece. These new preclinical data are the first demonstration of functional delivery of GalNAc-siRNA conjugates via the oral route of administration, representing an important step forward in potentially advancing and expanding the clinical and commercial potential of RNAi therapeutics. We believe that this approach can be applied to existing and future liver-directed pipeline programs, potentially creating a relatively near-term opportunity for Alnylam.
