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.
We presented a case study of a healthy human with mutations in the HAO1 gene, a validated target for the treatment of primary hyperoxaluria type 1 (PH1), as well as results from research on the diagnostic journey of PH1 at the 56th Congress of the European Renal Association (ERA) and European Dialysis and Transplant Association (EDTA) held on June 13-16 in Budapest, Hungary.
We presented new results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1) at the International Society of Nephrology (ISN) 2019 Annual Meeting held on April 13-16 in Melbourne, Australia.
We presented positive complete results from the ENVISION Phase 3 study of givosiran, an investigational RNAi therapeutic in development for the treatment of acute hepatic porphyria (AHP), at the European Association for the Study of the Liver (EASL) 54th Annual International Liver Congress™, held April 10 – 14, 2019 in Vienna, Austria.
We presented new pre-clinical data on our efforts in CNS and ocular delivery of novel siRNA conjugates in rats and non-human primates at the sixth annual Cold Spring Harbor Conference on RNA and Oligonucleotide Therapeutics, held March 27 to 30 in Cold Spring Harbor, NY.
We published results from the Phase 1 study of givosiran, our investigational RNAi therapeutic for the treatment of acute hepatic porphyria (AHP), in The New England Journal of Medicine (NEJM) in a paper titled “Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria.”
On December 6th, 2018, we will be hosting an R&D Day in New York City where Alnylam management and key opinion leaders will discuss our late stage clinical efforts, in addition to next wave programs and platform advances. Scroll down to see the R&D Day agenda and links to the presentations. Learn more about our science and the science of RNAi. Click here to see our pipeline of RNAi therapeutics.
To view the webcast, click here. A replay of the webcast will be available at that link on December 7th.
To view the R&D Day Presentation, click here.
