pp-213 Solution for injection

Indication: Paroxysmal nocturnal haemoglobinuria (PNH) and IgA nephropathy (IgAN) Clinical stage: Phase 2 — first patients dosed April 2026 (Phase 2 for PNH/IgAN) Modality: Biological (GalNAc-conjugated siRNA targeting complement factor B — NBE) Mechanism / Target: First-in-class siRNA silencing complement factor B (CFB), rate-limiting enzyme of the alternative complement pathway convertase; monthly SC dosing via GalNAc conjugation; reduces alternative-pathway-driven haemolysis (PNH) and IgA-immune-complex deposition (IgAN) Route / form: Subcutaneous injection (monthly) Differentiation: First CFB-targeting siRNA for complement-mediated haematological/renal disease; addresses PNH patients with extravascular haemolysis not covered by eculizumab/ravulizumab; Phase 2 first patients April 2026;