13 Dec, 2021 New Results from Phase 1 Study of Cemdisiran + Pozelimab Presented at ASH Annual Meeting 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”
The combined subcutaneous administration of pozelimab and cemdisiran was generally safe and well tolerated, permitting further development. Dose-dependent PD effects were discernible across cohorts in terms of timing, extent, and duration of complement inhibition. The observed data from this phase 1 study are consistent with model-based predictions and support advancement of the combination approach in an ongoing phase 2 study of patients with PNH switching from eculizumab.