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.
New results from a Phase 1 study of cemdisiran in combination with pozelimab in healthy volunteers, in collaboration with Regeneron, were presented at the 63rd American Society of Hematology (ASH) Annual Meeting, held December 11-14, 2021.
Findings from a 36-month analysis of the ENVISION Phase 3 study of GIVLAARI® (givosiran) in patients with acute hepatic porphyria (AHP) were presented during the 63rd American Society of Hematology (ASH) Annual Meeting & Exposition (Dec. 11-14). The data, which encompass the 6-month double-blind and 30-month open label extension (OLE) periods, further confirm that long-term dosing with GIVLAARI provides sustained and continuous benefit to patients with AHP, while maintaining an acceptable and consistent safety profile.
Long-term use of GIVLAARI during the OLE period demonstrated a durable response with efficacy across a wide range of clinical parameters, including sustained reductions in the annualized rate of composite porphyria attacks and hemin use, sustained lowering of ALA and PBG levels and improvements in physical functioning and QOL.
New results from the ATLAS Phase 3 program with fitusiran, an investigational RNAi therapeutic in development for the treatment of hemophilia A or B with and without inhibitors, in collaboration with Sanofi, were presented at the 63rd American Society of Hematology (ASH) Annual Meeting, held December 11-14, 2021.
Six-month results from the ongoing ILLUMINATE-B Phase 3 open-label pediatric study 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 published in Genetics in Medicine. The findings show that, in patients under the age of six, treatment with OXLUMO led to a 72% mean reduction in spot urinary oxalate:creatinine ratio from baseline to Month 6, averaged across Months 3 to 6. The efficacy results and safety profile of OXLUMO in these patients were similar to those observed in adults and children six years or older in the ILLUMINATE-A study.
On November 19, 2021, we hosted a virtual R&D Day event showcasing Alnylam’s late stage clinical efforts, next wave programs, and platform advances. The event included presentations from Alnylam senior leaders as well as guest speakers.
To view the webcast, click here.
To view the R&D Day presentation, click here.
Positive interim findings from the ongoing Phase 1 study of zilebesiran (formerly known as ALN-AGT) in adults with hypertension were presented during the American Heart Association (AHA) Scientific Sessions 2021 (Nov. 13-15).
Positive results from the six-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients with advanced primary hyperoxaluria type 1 (PH1), were presented as part of the late-breaking session at the American Society of Nephrology (ASN) Kidney Week 2021 virtual meeting held on November 4-7.
The POWER study (POrphyria Worldwide Patient Experience Research) surveyed 92 patients living with acute hepatic porphyria (AHP) around the world to bring to light the emotional, mental, and physical burden of AHP on the individual and their social circle. Survey participants reported substantial emotional burden associated with AHP, citing moderate to severe depression and anxiety. They also reported significant pain levels and work impairment. These data are being presented at the American College of Gastroenterology (ACG) Annual Scientific Meeting 2021. Encore 24-month interim data from the open-label extension period of the ENVISION Phase 3 study of GIVLAARI® (givosiran) in patients with AHP will also presented, along with additional findings on the burden of AHP.
Positive findings from a 24-month interim analysis of the ongoing open-label extension (OLE) period of the ENVISION Phase 3 study of GIVLAARI® (givosiran) in patients with acute hepatic porphyria (AHP) were presented during the United European Gastroenterology (UEG) Week Virtual 2021 (Oct. 3-5). The data further confirm that long-term dosing with GIVLAARI provides sustained and continuous benefit to patients with AHP while maintaining an acceptable safety profile. Findings on the burden of AHP from the ENVISION Phase 3 study and from Part B of the EXPLORE study were also presented.
On October 1, 2021, we hosted an online RNAi Roundtable to review the progress with our CNS and extrahepatic RNAi pipeline programs.
Access the replay
View the presentation
Vasant Jadhav, Ph.D.
Senior Vice President, Research
Kirk Brown, Ph.D.
Senior Director, CNS Program
Bret Bostwick, M.D.
Director, Clinical Research
