[{"id":"peg-ep1","name":"Adjusted mean hemoglobin change at Week 16","type":"PRIMARY","unit":"g/dL","notes":"Difference: 3.84 g/dL favoring pegcetacoplan. Hb at Week 16: 11.54 vs ~7.2 g/dL.","p_value":"<0.001","results":[{"arm":"peg-pcc","label":"+2.4 g/dL (pegcetacoplan)","value":2.4},{"arm":"peg-ecu","label":"-1.5 g/dL (eculizumab)","value":-1.5}],"timepoint":"Week 16","description":"PNH patients on eculizumab (C5 inhibitor) still have residual anemia from extravascular hemolysis. Pegcetacoplan (C3 inhibitor) blocks upstream of C5, addressing BOTH intravascular and extravascular hemolysis."}]

Injection site reactions 37% (vs 3% eculizumab; mostly mild, none led to discontinuation). Infections 29% vs 26%. No meningococcal infections in either group. Breakthrough hemolysis 10% controlled period (24% all phases, 5 discontinuations). 48-week Hb maintained at 11.30 g/dL in continuers.

PEGASUS showed clear SUPERIORITY vs eculizumab. The 3.84 g/dL Hb difference is remarkable. Breakthrough hemolysis during C5-to-C3 transition is the main risk (5 discontinuations). For SOBI, this is an EU licensed product with orphan disease pricing. Market somewhat limited by small PNH population but high per-patient revenue.

C3 inhibitor vs C5 inhibitor. By blocking upstream at C3, pegcetacoplan prevents BOTH intravascular hemolysis (which eculizumab addresses) AND extravascular hemolysis (which eculizumab does NOT address). This explains the dramatic superiority.