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.

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”

Complete results from the Phase 1/2 study of lumasiran for the treatment of primary hyperoxaluria type 1 (PH1) were published in the Clinical Journal of the American Society of Nephrology. The final results demonstrated an acceptable safety profile of OXLUMO in adult and pediatric patients. The majority of adverse events were mild or moderate; there were no serious adverse events considered to be drug-related and no adverse events led to death, study discontinuations or study withdrawals. After treatment with lumasiran, all 20 lumasiran-treated patients exhibited near-normal1 levels of 24-hour urinary oxalate excretion, with majority of such patients achieving levels within the normal2 range.

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Positive early results on clinical outcome measures from the 12-month analysis of ILLUMINATE-A Phase 3 study of OXLUMO® (lumasiran), an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the treatment of primary hyperoxaluria type 1 (PH1), were presented at the American Society of Pediatric Nephrology (ASPN)/Pediatric Academic Societies (PAS) virtual meeting held on April 30–May 4, 2021. The results showed that treatment with OXLUMO for 12 months was associated with evidence of improvements in nephrocalcinosis in one or both kidneys of PH1 patients, relative to baseline. Additional data were also presented regarding effects on estimated glomerulal filtration rate (eGFR) and kidney stone events.

Read our press release
Sas, et al. “Clinical outcomes in Phase 3 studies of lumasiran in pediatric and adult patients with primary hyperoxaluria type 1”

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