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 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”

Positive 9-month results from the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, were presented in an oral session at the 2021 American Academy of Neurology (AAN) Virtual Annual Meeting held April 19, 2021 in a virtual event.

Read the press release

Adams, et al. – “HELIOS-A: 9-month Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy”

The presentation of HELIOS-A results in a conference call held April 19, 2021 includes additional HELIOS-A 9-month results from the Phase 3 study. Click here to access the conference call slides.

For additional presentations from Alnylam and collaborators presented at AAN, scroll to the bottom of this post.

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Positive results from the ongoing Phase 1 study of ALN-AGT, an investigational RNAi therapeutic for the treatment of hypertension, were presented during the 2021 Joint Meeting of the European Society of Hypertension (ESH) and the International Society of Hypertension (ISH). ALN-AGT treatment was associated with dose-dependent knockdown of angiotensinogen (AGT) and reductions in blood pressure (BP), with a durability that supports the potential for a once quarterly or biannual dosing regimen.

Read the press release

View the presentation

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During the National Kidney Foundation Virtual Spring Clinical Meetings, we presented post hoc subgroup analyses from the ongoing ILLUMINATE-A Phase 3 study of OXLUMO® (lumasiran) in patients with primary hyperoxaluria type 1, showing a lowering of 24-hour urinary oxalate (UOx) regardless of crystallization inhibitor use, hydration status, and genotype. The reduction in 24-hour UOx was sustained through Month 12.

Saland, et al. “Lumasiran lowered urinary oxalate in patients with primary hyperoxaluria type 1 irrespective of pyridoxine use, hydration status, and genotype in the Phase 3 clinical trial ILLUMINATE-A.”

Habtemariam, et al. “24hr and random spot urine collections were comparable in assessing oxalate excretion and response to treatment in primary hyperoxaluria type 1.”

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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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