September 2019

03 Sep New Clinical Research Findings for ONPATTRO® (patisiran) at EU-ATTR Amyloidosis Meeting

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. Read our press release Ticau et al. – “Neurofilament Light Chain (NfL) as a Potential Biomarker in Hereditary Transthyretin-Mediated (hATTR) Amyloidosis” Parker et al. – “The Transthyretin Stabilizing Mutation (T119M) Is Not Associated with Extended Lifespan or Protection Against Vascular Diseases: Analysis of the UK Biobank Cohort”

02 Sep ORION-11 Phase 3 Results for Inclisiran Presented at ESC

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. Read the MDCO press release Read the ALNY press release View the presentation

24 Jun New Clinical Results for ONPATTRO® (patisiran) at 2019 PNS Annual Meeting

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. Read our press release Polydefkis et al. – "Long-Term Safety and Efficacy of Patisiran in Patients with hATTR Amyloidosis: Global OLE Study" Lin et al. – "Efficacy of Patisiran in Patients with hATTR Amyloidosis and Prior Tafamidis Use: Analysis of APOLLO" Obici et al. – "Indirect Treatment Comparison of the Efficacy of Patisiran and Inotersen for hATTR Amyloidosis with Polyneuropathy"

22 Jun Final Positive Results from Phase 1/2 Study of Lumasiran, an Investigational Therapeutic for the Treatment of PH1

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...

21 Jun New RNAi Platform Advances, Including Oral Route of Administration and CNS and Ocular Delivery

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 3^rd 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. Read our press release View the presentation

14 Jun Case Study of a Healthy Human with Mutations in HAO1 Gene, a Validated Target for the Treatment of PH1, and Diagnostic Journey Research on PH1

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 56^th Congress of the European Renal Association (ERA) and European Dialysis and Transplant Association (EDTA) held on June 13-16 in Budapest, Hungary. Erbe et al. - "Identification and Phenotyping of a Healthy Human with Mutations in HAO1 Supports Glycolate Oxidase Knockdown as a Potential Approach to Therapy for Primary Hyperoxaluria Type 1" Danese et al. - "The Importance of Evaluating Potential Underlying Causes of Kidney Stones: A Survey of Physician Experiences in Diagnosing Primary Hyperoxaluria Type 1"

15 Apr New Positive Results from Phase 2 Open-Label Extension Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1

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. Read our press release View the presentation

12 Apr ENVISION Phase 3 Results for Givosiran Presented at EASL

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) 54^th Annual International Liver Congress™, held April 10 – 14, 2019 in Vienna, Austria. Read our press release Balwani et al. – “ENVISION, a Phase 3 Study to Evaluate the Efficacy and Safety of Givosiran, an Investigational RNAi Therapeutic Targeting Aminolevulinic Acid Synthase 1, in Acute Hepatic Porphyria Patients” Sardh et al. – “Management of Recurrent Acute Hepatic Porphyria (AHP) Attacks in Europe and the United States: EXPLORE International, Prospective, Natural History Study” Ventura et al. – “Disease Manifestations of Patients with Recurrent Acute Hepatic Porphyria (AHP) and Daily Life Impacts in EXPLORE International, Prospective, Natural History Study”

29 Mar Pre-Clinical Data on Delivery of RNAi Therapeutics to the CNS and Eye

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. View the presentation

06 Feb Phase 1 Results for Givosiran Published in “The New England Journal of Medicine”

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.” Read our press release Read the paper in The New England Journal of Medicine