05 Nov, 2022 12-Month Results from ILLUMINATE-C Phase 3 Study of OXLUMO® (lumasiran) and Phase 2 Study of Investigational Cemdisiran Presented at ASN Kidney Week

During the American Society of Nephrology (ASN) Kidney Week, we presented new 12-month results from the ILLUMINATE-C Phase 3 study of lumasiran in patients of all ages with primary hyperoxaluria type 1 (PH1) and advanced kidney disease. Additional research was presented on the development and validation of a novel assay to determine oxalate concentration in plasma.

Frishberg, et al. “Lumasiran for Patients with Primary Hyperoxaluria Type 1 and Impaired Kidney Function: 12-Month Analysis of the Phase 3 ILLUMINATE-C Trial”
Clausen, et al. “Quantification of Oxalate in Human Plasma by Novel Liquid Chromatography—Tandem Mass Spectrometry: Method Development, Validation, and Application in Lumasiran Clinical Trials”

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26 Oct, 2022 NfL Biomarker Findings for hATTR Amyloidosis with Polyneuropathy Presented at ANA 2022

New 18-month findings evaluating Neurofilament light chain (NfL) as a potential biomarker of treatment response in patients with hATTR amyloidosis with polyneuropathy were presented at the 147^th annual meeting of the American Neurological Association (ANA).

Aldinc, et al. “NfL Levels Significantly Decrease in Response to Treatment with Patisiran or Vutrisiran in hATTR Amyloidosis with Polyneuropathy”

05 Oct, 2022 Alnylam Presents at OTS 2022 Annual Meeting

We presented on platform and clinical results at the Oligonucleotide Therapeutics Society (OTS) 2022 18^th Annual Meeting held in Phoenix, Arizona on October 2-5, 2022.

Human Genetics as an Enabler of RNAi Therapeutics – Paul Nioi

Phase 3 study, HELIOS-A, in hATTR Patients Evaluating a Single Dose Regimen – Gabriel Robbie

Toxicology and DMPK Assessment of siRNA Therapeutics – Joe Dybowski

30 Sep, 2022 Results from APOLLO-B Phase 3 Study of Patisiran Presented as HFSA

At the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022, we presented results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

Read the press release
Maurer, et al. “Primary Results from APOLLO-B, a Phase 3 Study of Patisiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy”
Kale, et al. “Exploratory Analyses from APOLLO-B, a Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy”

22 Sep, 2022 New Analysis from ILLUMINATE-C Phase 3 Study of Lumasiran Presented at IPNA

Results from a post-hoc analysis of patients on hemodialysis in the ongoing ILLUMINATE-C Phase 3 open-label study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1) were presented at the International Pediatric Nephrology Association (IPNA) Congress (Sept. 7-11, 2022).

Michael, et al. “Relationship of Baseline Weight and Response to Lumasiran in Patients With Primary Hyperoxaluria Type 1 on Hemodialysis”

08 Sep, 2022 Results from APOLLO-B Phase 3 Study of Patisiran Presented at ISA

During a late-breaker session at the 18^th International Symposium on Amyloidosis, we presented results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

Read the press release
Maurer, et al. “Primary Results from APOLLO-B, a Phase 3 Study of Patisiran in Patients with Transthyretin-Mediated Amyloidosis with Cardiomyopathy”

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