Arrowhead Pharmaceuticals initiates study of ARO-C3

AROC3-1001 is a phase 1/2, placebo controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-C3

Arrowhead Pharmaceuticals announced that it has dosed the first subjects in AROC3-1001, a phase 1/2 clinical study of ARO-C3, the company’s investigational RNA interference (RNAi) therapeutic designed to reduce production of complement component 3 (C3) as a potential therapy for various complement mediated diseases.

James Hamilton, Senior Vice President of Discovery and Translational Medicine at Arrowhead, said: “We believe a C3 targeted drug has the potential to address multiple complement mediated and complement associated diseases, where significant unmet medical need exists. These include IgA nephropathy, C3 glomerulopathy, paroxysmal nocturnal hemoglobinuria, and additional renal and hematologic diseases that we intend to evaluate in the future. ARO-C3 has progressed rapidly, and our preclinical data have been very encouraging. We are eager to continue this progress as we evaluate ARO-C3 in clinical studies.”

AROC3-1001 is a phase 1/2, placebo controlled, dose-escalating study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ARO-C3 in up to 24 adult healthy volunteers, up to 24 adult patients with paroxysmal nocturnal hemoglobinuria (PNH), and up to 14 adult patients with complement-mediated renal disease.

In Part 1 in healthy volunteers, four cohorts with four escalating dose levels of ARO-C3 will be evaluated. In Part 2, eligible subjects with PNH or complement-mediated renal disease will be enrolled to receive open-label ARO-C3 on day 1 and day 85 at one of two dose levels to be determined in Part 1.

The primary objective of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single doses of ARO-C3 in normal healthy volunteers and to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of multiple doses of ARO-C3 in subjects with PNH and in subjects with complement-mediated renal disease.