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”

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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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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”

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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”

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11 Oct, 2018 New Clinical Results from APOLLO OLE Study at 2018 AANEM Annual Meeting

We presented new 12-month efficacy and safety data with patisiran from the APOLLO Global Open Label Extension (OLE) Study at the 2018 American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting, held October 10-13, 2018 in Washington, DC.

Gonzalez-Duarte et al. – “Long-Term Use of Patisiran, an Investigational RNAi Therpaeutic, in Patients with Hereditary Transthyretin-Mediated Amyloidosis: 12 Month Efficacy and Safety Data from Global Open Label Extension (OLE) Study”

08 Oct, 2018 New Research on Acute Hepatic Porphyrias at ACG 2018 Annual Meeting

We presented new research on the diagnostic patterns, clinical experience and healthcare utilization of patients with acute hepatic porphyrias at the American College of Gastroenterology 2018 Annual Scientific Meeting, held October 5-10, 2018 in Phildelphia, PA.

Ko et al. – “Real-World Analysis of Symptoms, Diagnostic Patterns, and Provider Perspective on Acute Hepatic Porphyrias”

Rudnick et al. – “The Patient Odyssey to Confirmed Acute Hepatic Porphyria Diagnosis: Clinical Characteristics and Healthcare Utilization of Patients Preceding Diagnosis of Acute Hepatic Porphyria”

04 Oct, 2018 Updated Results from Phase 1/2 Study of Lumasiran at 2018 ESPN Annual Meeting

We presented updated positive results from the Phase 1/2 clinical study of lumasiran, an investigational, subcutaneously administered RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the 2018 European Society for Paediatric Nephrology (ESPN) Annual Meeting, held October 3-6, 2018 in Antalya, Turkey.

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02 Oct, 2018 New Platform Innovations for RNAi Therapeutics at OTS 2018 Annual Meeting

We presented further progress on our platform efforts in extrahepatic delivery of novel siRNA conjugates, including CNS and ocular delivery in rat and non-human primates, at the Oligonucleotide Therapeutics Society (OTS) 2018 Annual Meeting, held September 30 to October 3 in Seattle, WA.

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Maier et al. – “ESC+ Design Minimizes the Off-Target Potential and Further Maximizes the Therapeutic Index of GalNAc-siRNA Conjugates”

Janas et al. – “Safety Evaluation of 2′-Deoxy-2′-Fluoro-Modified Nucleotides in GalNAc-siRNA Conjugates”

Milstein et al. – “Robust and Durable Target Silencing in the CNS with siRNA Conjugates”

Agarwal et al. – “Mechanisms of Rat Hepatotoxicity of GalNAc-siRNA Conjugates”

Theile et al. – “Bis-RNAi™ Conjugates for Simultaneous Silencing of Two Different Gene Transcripts”

Nair et al. – “Efficient and Durable Ocular Gene Silencing of TTR After Single Intravitreal Administration of siRNA Conjugates”

Rocca et al. – “The Influence of GalNAc Valency on the Pharmacokinetic and Pharmacodynamic Parameters of siRNA in Rats”