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.

New kidney stone data from ongoing clinical trials of OXLUMO® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase – for the treatment of primary hyperoxaluria type 1 (PH1), were presented at the American Urological Association (AUA) Annual Meeting, held virtually September 10-13, 2021. Results from the Phase 1/2 clinical trial of lumasiran and its Phase 2 open-label extension (OLE) period showed that treatment with lumasiran led to an apparent reduction in kidney stone related adverse events in pediatric and adult patients with PH1. A reduction in kidney stone event (KSE) rates – an exploratory endpoint in ILLUMINATE-A and ILLUMINATE-B Phase 3 trials – was reported in ILLUMINATE-A at Month 12, with KSE rates remaining stable through Month 6 in ILLUMINATE-B.

Lieske, et al. – “Effect of lumasiran on kidney stones and nephrocalcinosis in patients with primary hyperoxaluria type 1”

Additional positive 9-month results from subgroup analyses and exploratory endpoints of the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with polyneuropathy, were presented at the 3rd EU-ATTR Amyloidosis Meeting. Subgroup analyses and exploratory endpoints demonstrated that vutrisiran improved important areas of patient health and function compared with placebo at 9 months. Additional analyses showed similar improvements in progression of neuropathy and quality of life measures with vutrisiran compared with placebo, regardless of prior TTR stabilizer use.

Gillmore, et al. – “HELIOS-A: 9-Month Subgroup Analyses and Exploratory Efficacy Results From the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

Berk, et al. – “Reason for Stopping Transthyretin Stabilizers Prior to HELIOS-A and the Impact of Prior Stabilizer Use on the Efficacy of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

We are hosting a series of online “RNAi Roundtables” at which Alnylam scientists and program leaders, as well as medical thought leaders, will review recent progress in many of our pipeline programs and platform, and provide perspectives on clinical developments and unmet needs in various therapeutic areas.

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Results from pooled, post-hoc analyses from ongoing clinical trials of OXLUMO® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 – the gene encoding glycolate oxidase – for the treatment of primary hyperoxaluria type 1 (PH1), were presented at the 58th European Renal Association – European Dialysis and Transplant Association (ERA-EDTA) virtual congress, held June 5-8, 2021. The analyses found that treatment with lumasiran led to substantial and clinically meaningful reductions in urinary and plasma oxalate that were similar in pediatric and adult patients with PH1 in the ILLUMINATE-A and ILLUMINATE-B Phase 3 trials, with lumasiran demonstrating an acceptable safety profile in both patient populations. In additional pooled, post-hoc analyses, PH1 patients 12 months and older with a baseline estimated glomerular filtration rate (eGFR) of ≥30 mL/min/1.73m2, treated with lumasiran through Month 12, demonstrated stable eGFR levels, irrespective of baseline kidney function – an encouraging observation, given the progressive decline in kidney function characteristic of PH1.

Shasha-Lavsky, et al. “Lumasiran Demonstrated Comparable Oxalate Reduction and Safety in Children and Adults with Primary Hyperoxaluria Type 1”

Hayes, et al. “Stable eGFR in Patients With Primary Hyperoxaluria Type 1 Treated With Lumasiran, Regardless of Kidney Function at Start of Treatment”

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