Arrowhead finds RNAi kidney disease candidate is well tolerated in phase 1/2 IgAN trial cohort
Based on the search results, here are the key details about Arrowhead's RNAi kidney disease candidate ARO-C3 in the phase 1/2 trial for IgA nephropathy (IgAN):
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ARO-C3 is an investigational RNA interference (RNAi) therapeutic designed to reduce liver production of complement component 3 (C3) as a potential treatment for complement-mediated renal diseases like IgAN.
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In Part 2 of the Phase 1/2 study, 14 patients with IgAN received subcutaneous doses of 400 mg ARO-C3 on Days 1, 29, and 113 and were followed through Day 169.
- Key efficacy results:
- Mean sustained reductions of ≥87% in C3, ≥76% in AH50, and ≥89% in Wieslab AP through week 24
- Mean reduction in spot urine protein-to-creatinine ratio (UPCR) of 41% by week 24, with maximum individual reduction of 89%
- Safety and tolerability:
- ARO-C3 was generally well-tolerated in IgAN patients
- No serious or severe treatment-emergent adverse events
- No adverse events led to study or drug discontinuation
- Most common adverse events were headache, cough, and nasopharyngitis
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No infections with encapsulated organisms reported
- The duration of effect supports quarterly or less frequent subcutaneous dosing in future studies.
In summary, ARO-C3 demonstrated promising efficacy in reducing complement activity and proteinuria while being well-tolerated in this cohort of IgAN patients in the phase 1/2 trial. The results support further development of ARO-C3 for complement-mediated kidney diseases.