ARO-C3 / Arrowhead - LARVOL DELTA

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease (clinicaltrials.gov) - P1/2 | N=62 | Active, not recruiting | Sponsor: Arrowhead Pharmaceuticals | Trial completion date: Jun 2025 ➔ Sep 2025

Trial completion date • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease

ARO-C3, an investigational RNAi Therapeutic Targeting Complement C3, Reduces Proteinuria, Hematuria, and Complement Activity in IgAN Patients (ERA 2025) - P1/2 | "ARO-C3 was well-tolerated in patients with IgAN, achieving robust and durable reductions in serum C3 levels and measures of complement function, while demonstrating meaningful improvements in proteinuria and hematuria through 24 weeks of treatment. The observed duration of effect supports quarterly dosing in later phase studies. To our knowledge, this represents the first and only data on the clinical efficacy of siRNA targeting hepatic complement C3 in patients with IgAN."

Clinical • Late-breaking abstract • Complement-mediated Rare Disorders • Cough • Glomerulonephritis • IgA Nephropathy • Infectious Disease • Pain • Renal Disease • Respiratory Diseases

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease (clinicaltrials.gov) - P1/2 | N=62 | Active, not recruiting | Sponsor: Arrowhead Pharmaceuticals | Phase classification: P1 ➔ P1/2

Phase classification • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease

Arrowhead Pharmaceuticals Announces Topline Results from Part 2 of Phase 1/2 Study of ARO-C3 in Patients with IgA Nephropathy (Businesswire) - P1/2a | N=62 | NCT05083364 | Sponsor: Arrowhead Pharmaceuticals | "Patients with IgA nephropathy (IgAN) (n=14) received subcutaneous doses of ARO-C3 (400 mg) on Days 1, 29, and 113 and were followed through Day 169....Effects on proteinuria: Mean reduction in spot UPCR of 41% and maximum individual reduction of 89% from baseline by week 24. Safety and Tolerability: ARO-C3 was generally well-tolerated in patients with IgAN; No serious or severe treatment emergent adverse events (TEAE) and no TEAEs that led to study or study drug discontinuation; The only TEAEs reported in more than 1 subject were headache, cough, and nasopharyngitis....'We look forward to sharing more data from the Phase 1/2 clinical study of ARO-C3 at an upcoming medical meeting in 2025'."

P1/2 data • IgA Nephropathy

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease (clinicaltrials.gov) - P1 | N=62 | Active, not recruiting | Sponsor: Arrowhead Pharmaceuticals | Recruiting ➔ Active, not recruiting | Trial primary completion date: Dec 2024 ➔ May 2025

Enrollment closed • Trial primary completion date • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease (clinicaltrials.gov) - P1 | N=60 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | Trial completion date: Dec 2024 ➔ Jun 2025

Trial completion date • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease (clinicaltrials.gov) - P1 | N=60 | Recruiting | Sponsor: Arrowhead Pharmaceuticals

Trial completion date • Trial primary completion date • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Complement Mediated Renal Disease (clinicaltrials.gov) - P1 | N=60 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | Phase classification: P1/2 ➔ P1

Phase classification • Complement-mediated Rare Disorders • Glomerulonephritis • IgA Nephropathy • Nephrology • Renal Disease

Gene targeting as a therapeutic avenue in diseases mediated by the complement alternative pathway. (PubMed, Immunol Rev) - "This review explains key concepts of RNA and DNA targeting and discusses assets in clinical development for the treatment of diseases driven by the alternative pathway, including the RNA-targeting therapeutics ALN-CC5, ARO-C3, and IONIS-FB-LRX, and the gene therapies GT005 and HMR59. These therapies are but the spearhead of potential drug candidates that might revolutionize the field in coming years."

Journal • Review • Age-related Macular Degeneration • Complement-mediated Rare Disorders • Gene Therapies • Hematological Disorders • Inflammation • Macular Degeneration • Ophthalmology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Retinal Disorders

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria and Complement-Mediated Renal Disease (clinicaltrials.gov) - P1/2 | N=84 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | N=62 ➔ 84

Enrollment change • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • IgA Nephropathy • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease

Study of ARO-C3 in Adult Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria and Complement-Mediated Renal Disease (clinicaltrials.gov) - P1/2 | N=62 | Recruiting | Sponsor: Arrowhead Pharmaceuticals | Not yet recruiting ➔ Recruiting

Enrollment open • Complement-mediated Rare Disorders • Glomerulonephritis • Hematological Disorders • IgA Nephropathy • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Renal Disease