December 2021

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. Van Zyl, et al. “Open-Label, Ascending-Dose, Phase 1 Study of the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single Doses of the Subcutaneously Administered Human Monoclonal Antibody Pozelimab in Combination with Single Doses of the Subcutaneously Administered siRNA Cemdisiran in Healthy Volunteers”

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. Srivastava, et al. “Fitusiran, an Investigational siRNA Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: First Results from a Phase 3 Study to Evaluate Efficacy and Safety in People with Hemophilia a or B without Inhibitors (ATLAS-A/B)” Young, et al. “Efficacy and Safety of Fitusiran Prophylaxis, an siRNA Therapeutic, in a Multicenter Phase 3 Study (ATLAS-INH) in People with Hemophilia A or B, with Inhibitors (PwHI)”

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). Read the press release Huang, et al. “Durable Reductions in Circulating Angiotensinogen and Blood Pressure 6 Months after Single Doses of ALN-AGT, an RNA Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Hypertensive Patients” Huang, et al. “Safety and Tolerability of ALN-AGT, an RNA Interference Therapeutic Targeting Hepatic Angiotensinogen Synthesis, in Hypertensive Patients during Sodium Depletion or Irbesartan Coadministration”

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. Read the press release Michael, et al. - “ILLUMINATE-C, a single-arm, phase 3 study of lumasiran in patients with primary hyperoxaluria type 1 and CKD3b-5, including those on hemodialysis” Lieske, et al. - “Modeling the risk of progression to kidney failure in patients with primary hyperoxaluria type 1 treated with lumasiran relative to a natural history cohort not treated with lumasiran”

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...

SIGN UP FOR EMAIL UPDATES

Receive news and updates on the work at Alnylam that affects you most.