Capella

Capella—the Online Voice of Progress in RNAi

Welcome to Capella, Alnylam’s destination for updates on our work translating the breakthrough discovery of RNA interference (RNAi) into an innovative new class of medicines. We’ve been pioneering RNAi therapeutics since 2002 and are excited to share our ongoing scientific progress.

We published new research describing neurofilament light chain (NfL) as a biomarker of hereditary transthyretin-mediated amyloidosis in Neurology. Findings from the observational, case-controlled study which compared proteomes of patients with hATTR amyloidosis and healthy controls suggest NfL levels may serve as a biomarker of nerve damage and polyneuropathy in hATTR amyloidosis enable earlier diagnosis of patients and facilitate monitoring of disease progression.

Read the paper in Neurology

On December 15th and 16th, 2020, we hosted a virtual R&D Day event showcasing Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The Company also discussed its perspective on its transition toward achieving a self-sustainable financial profile. The event included presentations from Alnylam management and key opinion leaders.

To view a replay of the webcast, click here.

To view the R&D Day presentation for Day 1, click here.

To view the R&D Day presentation for Day 2, click here.

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During the 62nd American Society of Hematology Annual Meeting & Exposition, we presented new 18-month interim results from the ongoing open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI® (givosiran) in patients with acute hepatic porphyria (AHP), demonstrating sustained efficacy and safety with long-term dosing.

Kuter, et al. Eighteen-Month Interim Analysis of Efficacy and Safety of Givosiran, an RNAi Therapeutic for Acute Hepatic Porphyria, in the ENVISION Open-Label Extension

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During the Thrombosis & Hemostasis Summit of North America 2020 Virtual Conference, we presented new data from a post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI® (givosiran), evaluating hemin use in patients with acute hepatic porphyria. We also presented encore data evaluating healthcare resource utilization among patients with acute intermittent porphyria.

Kuter, et al. “Hemin Use in Patients with Acute Hepatic Porphyria Treated with Givosiran: A Post Hoc Analysis of the Phase 3 ENVISION Study”

Silver, et al. “Disease Burden and Healthcare Utilization Among Patients with Acute Intermittent Porphyria Experiencing Chronic Pain: Analyses from a National Healthcare Database”

New data from a post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI® (givosiran) were presented during the American College of Gastroenterology 2020 Virtual Annual Scientific Meeting, evaluating outcomes in patients with acute hepatic porphyria with or without prior hemin prophylaxis. Findings on healthcare resource utilization among patients with acute intermittent porphyria were also presented, along with encore 12-month interim data from the open-label extension period of the ENVISION Phase 3 study.

Bonkovsky, et al. “Clinical Outcomes in Patients with Acute Hepatic Porphyria Treated with Givosiran Who Stopped Hemin Prophylaxis at Study Entry: A Post-hoc Analysis of Data From the Phase 3 ENVISION Study Through Month 12”

Silver, et al. “Disease Burden and Healthcare Utilization Among Patients with Acute Intermittent Porphyria Experiencing Chronic Pain: Analyses from a National Healthcare Database”

Sardh, et al. “Twelve-Month Interim Analysis of Efficacy and Safety of Givosiran, an Investigational RNAi Therapeutic for Acute Hepatic Porphyria, in the ENVISION Open-Label Extension”

Positive results from the six-month primary analysis of the ongoing ILLUMINATE-B Phase 3 open-label pediatric study of lumasiran, an investigational RNAi therapeutic in development for the treatment of adults and children with Primary Hyperoxaluria Type 1 (PH1), were presented at the American Society of Nephrology (ASN) Kidney Week 2020, held October 22-25 as a virtual event. Additionally, new 12-month results from the ILLUMINATE-A pivotal Phase 3 study and the ongoing Phase 2 open-label extension (OLE) study were also presented.

Read the press release
View the ILLUMINATE-B presentation
View the ILLUMINATE-A presentation
View the Phase 2 OLE presentation
View the Chart Review presentation

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We presented progress on our RNAi therapeutics platform efforts at the Oligonucleotide Therapeutics Society (OTS) 2020 Annual Meeting.

Akinc et al. – “ALN-COV: An Investigational RNAi Therapeutic for COVID-19”
Brown et al. – “Evidence for an Intracellular Depot that Contributes to the Extended Duration of Activity of GalNAc-siRNA Conjugates”
Jadhav et al. – “Human Translation of GalNAc-siRNA Conjugates with Improved Specificity”
Janas et al. – “Advancing the Safety and Reach of RNAi Therapeutics”
MacLauchlin et al. – “The ADME of siRNA GalNAc Conjugates”

On September 3, 2020, we hosted an online RNAi Roundtable to review progress with patisiran and vutrisiran, RNAi therapeutics in development for the treatment of transthyretin-mediated amyloidosis. Of note, we announced we have obtained clinical pharmacology data supporting the potential for a biannual subcutaneous dosing regimen option for vutrisiran, providing further product differentiation as a potential best-in-class agent, and discussed new clinical data presented at the European Society of Cardiology 2020 Congress providing further evidence that treatment with patisiran may lead to substantial reduction in cardiac amyloid burden in ATTR amyloidosis.

Read the press release
Access the replay
View the presentation
Read the transcript

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