A Global, Open-label, Adaptive Design Study to Investigate the Efficacy and Safety of SerpinPC in Subjects With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B (PRESent-2)

Prior treatment

• Known severe thrombophilia, previous deep vein thrombosis (excluding line-associated thrombosis), pulmonary embolism, myocardial infarction, stroke, uncontrolled hypertension (>160/100 mmHg) or has active cancer and/or requires therapy for cancer, except for basal cell carcinoma
• Subject with previous factor VIII or factor IX inhibitor who responded to immune tolerance induction and remains on prophylactic factor concentrate
• Use of emicizumab in the 24 weeks before Baseline (Day 0) / Ongoing, or planned treatment with gene therapy for hemophilia / Current or planned treatment with anticoagulant or antiplatelet drugs

Other

• Body weight < 30 kg OR >150 kg OR body mass index >40 kg/m2

• Male subjects ≥12 and ≤65 years of age with severe HemA, with or without inhibitors, or moderately severe to severe HemB, without high titer inhibitor (high titer inhibitor defined as ≥5 Bethesda Units [BU]/mL) and not requiring current treatment with bypass agents.
• Subject is currently included in a prophylaxis program OR subject is undergoing an on-demand treatment regimen; if the latter, must have had ≥6 documented acute bleeding episodes that required treatment during the 6 months before Screening
• At least 12 (Part 1) or 24 (Part 2) weeks of prospective documentation of bleeding episodes in the AP-0105 noninterventional study before dosing, or willing to complete the observation period in AP-0102
• No bleeding in the 7 days before Baseline (observation period can be extended if actively bleeding)
• Adequate hematologic, hepatic, and renal function, and D-dimer of ≤750 μg/L