Capella

22 Jun, 2019 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 at the 2019 Oxalosis & Hyperoxaluria (OHF) International Workshop, held June 21-22 in Boston.

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21 Jun, 2019 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.

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14 Jun, 2019 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, 2019 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.

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12 Apr, 2019 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.

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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, 2019 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.

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06 Feb, 2019 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.”

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Read the paper in The New England Journal of Medicine

05 Dec, 2018 Alnylam R&D Day 2018

09 Nov, 2018 Updated Phase 1/2 OLE Results for Givosiran and EXPLORE Natural History Study Data at AASLD 2018

We presented updated results from the ongoing Phase 1/2 open-label extension (OLE) study of givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the treatment of acute hepatic porphyria (AHP), at The Liver Meeting® 2018 of the American Association for the Study of Liver Diseases (AASLD), held November 9-13, 2018 in San Francisco, CA.

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Anderson et al. – “Phase 1/2 and Open Label Extension Studies of Givosiran, an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Acute Intermittent Porphyria”

Bonkovsky et al. – “EXPLORE: A Prospective, Multinational, Natural History Study of Acute Hepatic Porphyrias (AHP) Patients with Recurrent Attacks”

25 Oct, 2018 New Clinical Results from Phase 1/2 and Phase 2 OLE Studies of Lumasiran at ASN 2018 Annual Meeting

We presented new results from the Phase 1/2 and Phase 2 open-label extension (OLE) studies of lumasiran at the American Society of Nephrology (ASN) 2018 Annual Meeting, held October 23-28, 2018 in San Diego, CA.

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Hulton et al. – “Safety and Efficacy Study of Lumasiran (ALN-GO1), an Investigational RNA Interference (RNAi) Therapeutic, in Patients with Primary Hyperoxaluria Type 1”