VSA012 / Arrowhead Pharmaceuticals - LARVOL DELTA

A Multicenter, Randomized, Open-Label, Positive Controlled Phase III Study to Evaluate the Efficacy and Safety of VSA012 Injection in Participants With Paroxysmal Nocturnal Hemoglobinuria Who Are Naive to Complement Inhibitor Therapy (clinicaltrials.gov) - P3 | N=70 | Not yet recruiting | Sponsor: Bisirna Therapeutics (Suzhou) Co., Ltd.

New P3 trial • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

VSA012-1001: Phase Ib Clinical Study to Evaluate the Safety and Tolerability of VSA012 Injection in Paroxysmal Nocturnal Hemoglobinuria (clinicaltrials.gov) - P1 | N=50 | Active, not recruiting | Sponsor: Bisirna Therapeutics Pte. Ltd. | Not yet recruiting ➔ Active, not recruiting | Phase classification: P ➔ P1 | N=16 ➔ 50 | Trial completion date: Apr 2026 ➔ Aug 2027 | Trial primary completion date: Apr 2026 ➔ Feb 2027

Enrollment change • Enrollment closed • Phase classification • Trial completion date • Trial primary completion date • Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

A Phase I Clinical Study of VSA012 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=40 | Active, not recruiting | Sponsor: Bisirna Therapeutics (Suzhou) Co., Ltd. | Not yet recruiting ➔ Active, not recruiting | Trial completion date: Mar 2026 ➔ Jun 2027 | Trial primary completion date: Dec 2025 ➔ Aug 2026

Enrollment closed • Trial completion date • Trial primary completion date

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