November 2019

On November 22nd, 2019, we will be hosting an R&D Day in New York City. The event will showcase Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The Company will also discuss its perspective on its transition toward achieving a self-sustainable financial profile. To view the webcast, click here. A replay of the webcast will be available at that link on November 23rd. To view the R&D Day Presentation, click here.

We presented results on the pediatric cohort of patients (N=16; age range 6-17) from the Phase 1/2 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) for the treatment of primary hyperoxaluria type 1 (PH1), at the International Pediatric Nephrology Association (IPNA) 2019 Annual Meeting, held October 17-21 in Venice, Italy. Frishberg et al. – Phase 1/2 Study of Lumasiran, Investigational RNAi Therapeutic, in Patients with Primary Hyperoxaluria Type 1 Danese et al. – Understanding the Burden of Primary Hyperoxaluria Type 1 (PH1): A Survey of Physician Experiences with PH1

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. Access the replay View the presentation Read the transcript 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. Read our press release Parker et al. – “The V122I Mutation in Hereditary Transthyretin-Mediated Amyloidosis is Significantly Associated with Polyneuropathy”

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. Read our press release Gouya 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. – “Overall Health, Daily Functioning, and Quality of Life in Patients with Acute Hepatic Porphyria: ENVISION, a Phase 3 Global, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial” Balwani et al. – “Disease Characteristics of Patients with Acute Hepatic Porphyria Patients: ENVISION, a Phase 3 Global, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial” Bonkovsky et al. – “A Phase 1/2 Open-Label Extension Study of Givosiran, an Investigational RNAi Therapeutic, in Patients with Acute Intermittent Porphyria” Anderson et al. – “Acute Hepatic Porphyria (AHP) Disease Manifestations and Daily Life Impacts in EXPLORE International Prospective, Natural History Study” Gill et al. – “Burden of Illness in Acute Hepatic Porphyria (AHP): Insights from Patient and Caregiver Members of the British Porphyria Association” Gill et al. – “The Evolving Diagnosis and Care of Patients with Acute Hepatic Porphyria (AHP) in the UK: from 2006 to 2018” Vassiliou et al. – “A Drug-Drug Interaction Study to Investigate the Effect of Givosiran on the Activity of 5 Major Drug Metabolizing CYP450 Enzymes in Subjects with Acute Intermittent Porphyria (AIP) who are Chronic High Excreters (CHE)”

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.


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