VSA012 / Arrowhead - LARVOL DELTA

VSA012, a CFB-targeted siRNA, demonstrates a favorable safety profile and sustained efficacy in PNH patients: Interim Analysis from a Phase Ib Study (ASH 2025) - P | "The preliminary data of interim analysis demonstrated a favorable safety profile andsustained clinical benefits of such a siRNA drug, VSA012 in PNH patients. In VSA012 60 mg group, 6 out of8 PNH patients (75%) achieved an Hb increase≥2 g/dL within 56 days. Further investigations with longerfollow-up duration are ongoing to confirm these preliminary findings of VSA012."

Clinical • P1 data • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • CFB

VSA012-1001: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of of VSA012 in Subjects with Paroxysmal Nocturnal Hemoglobinuria(PNH) (clinicaltrials.gov) - P=N/A | N=16 | Not yet recruiting | Sponsor: Visirna Therapeutics HK Limited

New trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases