Capella

09 Nov, 2019 New Clinical Results for Lumasiran at ASN

We presented new positive efficacy results from the ongoing Phase 2 open-label extension (OLE) study of lumasiran, an investigational subcutaneously administered RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1 (PH1), at the American Society of Nephrology (ASN) 2019 Annual Meeting, held November 5-10 in Washington, DC.

Read our press release

View the Phase 2 OLE poster

07 Nov, 2019 EXPLORE Natural History Study Data Published in “Hepatology”

We published results from the EXPLORE natural history study of more than 100 acute hepatic porphyria (AHP) patients with recurrent attacks in Hepatology in a paper titled, “EXPLORE: A Prospective, Multinational, Natural History Study of Patients with Acute Hepatic Porphyria with Recurrent Attacks.”

Read the paper in Hepatology

20 Oct, 2019 Results from Pediatric Cohort of Phase 1/2 Study of Lumasiran in Patients with Primary Hyperoxaluria Type 1 (PH1) and Insights from a Survey of Physician Experiences with PH1

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

10 Oct, 2019 RNAi Roundtable: Lumasiran, in Development for the Treatment of Primary Hyperoxaluria Type 1

On October 10, 2019, we hosted an online RNAi Roundtable to review the progress with lumasiran, an RNAi therapeutic in development for the treatment of primary hyperoxaluria type 1.

Access the replay

View the presentation

Read the transcript

07 Oct, 2019 RNAi Roundtable: Givosiran, in Development for the Treatment of Acute Hepatic Porphyria

On October 7, 2019, we hosted an online RNAi Roundtable to review the progress with givosiran, an RNAi therapeutic in development for the treatment of acute hepatic porphyria.

Access the replay

View the presentation

Read the transcript

16 Sep, 2019 RNAi Roundtable: Patisiran & Vutrisiran, in Development for the Treatment of Transthyretin-Mediated Amyloidosis

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.

13 Sep, 2019 New Evidence Demonstrating Significant Association of V122I TTR Gene Mutation with Clinical Diagnosis of Polyneuropathy

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) 23^rd 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”

10 Sep, 2019 New Clinical Results for Givosiran at ICPP

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)”

06 Sep, 2019 Sixth Annual RNAi Roundtable Series

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.

03 Sep, 2019 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”