03 May, 2021 Early Results on Clinical Outcome Measures Presented for OXLUMO® (lumasiran) at ASPN/PAS 2021 Annual Congress

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.

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

19 Apr, 2021 HELIOS-A Phase 3 Results for Vutrisiran Presented at AAN

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.

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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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11 Apr, 2021 New Results for ALN-AGT at the ESH-ISH 2021 Joint Meeting

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.

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10 Apr, 2021 New Analyses of OXLUMO® (lumasiran) ILLUMINATE-A Results at the National Kidney Foundation Virtual Spring Clinical Meetings

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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19 Jan, 2021 New Biomarker Research for Hereditary Transthyretin-Mediated Amyloidosis Published in “Neurology”

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.

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05 Dec, 2020 New Long-Term Results for GIVLAARI® (givosiran) at the 62nd American Society of Hematology Annual Meeting & Exposition

During the 62^nd 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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13 Nov, 2020 New Results for ALN-AGT at AHA Scientific Sessions 2020

Positive interim results from the ongoing Phase 1 study of ALN-AGT, an investigational RNAi therapeutic for the treatment of hypertension, were presented during the American Heart Association (AHA) Scientific Sessions 2020, taking place virtually from November 13 – 17, 2020.

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27 Oct, 2020 New Results for GIVLAARI® (givosiran) at the Thrombosis & Hemostasis Summit of North America 2020 Virtual Conference

During the Thrombosis & Hemostasis Summit of North America 2020 Virtual Conference, we presented new data from a post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI^® (givosiran), evaluating hemin use in patients with acute hepatic porphyria. We also presented encore data evaluating healthcare resource utilization among patients with acute intermittent porphyria.

Kuter, et al. “Hemin Use in Patients with Acute Hepatic Porphyria Treated with Givosiran: A Post Hoc Analysis of the Phase 3 ENVISION Study”

Silver, et al. “Disease Burden and Healthcare Utilization Among Patients with Acute Intermittent Porphyria Experiencing Chronic Pain: Analyses from a National Healthcare Database”

26 Oct, 2020 New Results for GIVLAARI® (givosiran) at the American College of Gastroenterology 2020 Virtual Annual Scientific Meeting

New data from a post-hoc analysis of the ENVISION Phase 3 study of GIVLAARI^® (givosiran) were presented during the American College of Gastroenterology 2020 Virtual Annual Scientific Meeting, evaluating outcomes in patients with acute hepatic porphyria with or without prior hemin prophylaxis. Findings on healthcare resource utilization among patients with acute intermittent porphyria were also presented, along with encore 12-month interim data from the open-label extension period of the ENVISION Phase 3 study.

Bonkovsky, et al. “Clinical Outcomes in Patients with Acute Hepatic Porphyria Treated with Givosiran Who Stopped Hemin Prophylaxis at Study Entry: A Post-hoc Analysis of Data From the Phase 3 ENVISION Study Through Month 12”

Silver, et al. “Disease Burden and Healthcare Utilization Among Patients with Acute Intermittent Porphyria Experiencing Chronic Pain: Analyses from a National Healthcare Database”

Sardh, et al. “Twelve-Month Interim Analysis of Efficacy and Safety of Givosiran, an Investigational RNAi Therapeutic for Acute Hepatic Porphyria, in the ENVISION Open-Label Extension”