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.

On December 13, 2023, we hosted a virtual R&D Day event showcasing Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The event included presentations from Alnylam senior leaders as well as guest speakers.

To view the webcast, click here
To view the presentation, click here

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24-month results, including data on the odds of disease progression as assessed by NYHA class and disease stage, from an interim analysis of the open-label extension period of the APOLLO-B Phase 3 study of investigational patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were presented during the American Heart Association (AHA) Scientific Sessions 2023.

Hung, et al. “APOLLO-B, a Study of Patisiran in Patients with Transthyretin Cardiac Amyloidosis: Primary Long-term Results from the Open-Label Extension Period”

New results from the KARDIA-1 Phase 2 study of zilebesiran, an investigational RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of patients with hypertension and high cardiovascular risk, were presented at the American Heart Association (AHA) Scientific Sessions 2023.

Bakris, et al. “Sustained Blood Pressure Reduction with the RNA Interference Therapeutic, Zilebesiran: Primary Results from KARDIA-1, a Phase 2 Study in Patients with Hypertension”

New 24-month results from the ILLUMINATE-C Phase 3 study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1) were presented at the American Society of Nephrology (ASN) Kidney Week 2023 Meeting. Additional findings from a retrospective study of the natural history of advanced PH1 were also presented.

Lieske, et al. “Lumasiran for Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 24-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Lieske, et al. “Natural History of Advanced Primary Hyperoxaluria Type 1: A Retrospective Study”

We presented additional positive interim results for the ongoing single ascending dose portion of the Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein in development for the treatment of Alzheimer’s disease and cerebral amyloid angiopathy, in a late-breaker session at the 16th Clinical Trials on Alzheimer’s Disease conference.

Mummery, et al. “Phase 1 Safety, Tolerability, and Pharmacological Results of ALN-APP, the First Investigational RNA Interference Therapeutic in Development for Early-Onset Alzheimer’s Disease”

New 24-month results from an interim analysis of the open-label extension period of the APOLLO-B Phase 3 study of patisiran in patients with the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis were presented at the Heart Failure Society of America (HFSA) Annual Scientific Meeting (ASM) 2023. Findings from a post-hoc analysis of the APOLLO-B Phase 3 study that further characterize the impact of patisiran on health status and quality of life in patients with the cardiomyopathy of ATTR amyloidosis were also presented, as well as an overview of baseline characteristics of patients who enrolled in the expanded access program for patisiran.

Maurer, et al. “Primary Results from APOLLO-B Open-label Extension Study of Patisiran in Patients with Transthyretin Cardiac Amyloidosis”
Shah, et al. “Impact of Patisiran on Health Status and Quality of Life in Patients with Transthyretin Cardiac Amyloidosis”
Saha, et al. “Baseline Characteristics of Patients with Transthyretin Cardiac Amyloidosis Enrolled in the Patisiran Expanded Access Program”

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