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.

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.

(more…)

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.

Read the press release

View the presentation

(more…)

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.”

(more…)

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.

Read the paper in Neurology

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

(more…)

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.

Read the press release

View the presentation

(more…)

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”

22 Oct, 2020 ILLUMINATE-B Phase 3 Results for Lumasiran Presented at ASN

Positive results from the six-month primary analysis of the ongoing ILLUMINATE-B Phase 3 open-label pediatric study of lumasiran, an investigational RNAi therapeutic in development for the treatment of adults and children with Primary Hyperoxaluria Type 1 (PH1), were presented at the American Society of Nephrology (ASN) Kidney Week 2020, held October 22-25 as a virtual event. Additionally, new 12-month results from the ILLUMINATE-A pivotal Phase 3 study and the ongoing Phase 2 open-label extension (OLE) study were also presented.

Read the press release
View the ILLUMINATE-B presentation
View the ILLUMINATE-A presentation
View the Phase 2 OLE presentation
View the Chart Review presentation

(more…)