Capella

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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

Posted at 08:40h in Capella

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.

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

Posted at 05:30h in Capella

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.

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

Posted at 07:30h in Capella

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

Posted at 07:31h in Capella

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.

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”

Read the MDCO press release

02 Sep, 2019 ORION-11 Phase 3 Results for Inclisiran Presented at ESC

Posted at 03:02h in Capella

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 ALNY press release

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

Posted at 11:02h in Capella

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.

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, 2019 Final Positive Results from Phase 1/2 Study of Lumasiran, an Investigational Therapeutic for the Treatment of PH1

Posted at 18:55h in Capella

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.

21 Jun, 2019 New RNAi Platform Advances, Including Oral Route of Administration and CNS and Ocular Delivery

Posted at 11:08h in Capella

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.

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”

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

Posted at 10:51h in Capella

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.

Danese et al. – “The Importance of Evaluating Potential Underlying Causes of Kidney Stones: A Survey of Physician Experiences in Diagnosing Primary Hyperoxaluria Type 1”