ARWR - Arrowhead begins dosing in phase 1/2 study of ARO-C3 for blood disorders

Arrowhead Pharmaceuticals (NASDAQ:ARWR) dosed the first person in a phase 1/2 study of ARO-C3, an RNA interference (RNAi) therapeutic aimed at reducing the production of complement component 3 (C3) and be potential therapy for various complement mediated diseases. The study, dubbed AROC3-1001will evaluate the safety and tolerability 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 patients with PNH or complement-mediated renal disease will be enrolled to receive ARO-C3 on day 1 and day 85 at one of two dose levels to be determined in part 1. PNH is a rare disorder in which red blood cells break apart prematurely.

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Arrowhead begins dosing in phase 1/2 study of ARO-C3 for blood disorders