June 2021

07 Jun New Analyses from OXLUMO® (lumasiran) Clinical Development Program Presented at ERA-EDTA Congress

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...

14 May Complete Results from Phase 1/2 Study of Lumasiran (OXLUMO®) Published in Clinical Journal of the American Society of Nephrology

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.

03 May 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...

19 Apr 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. 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.

11 Apr 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. Read the press release View the presentation

10 Apr 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.”

19 Jan 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...