OBI-1 Clinical Study OBI-1 - 201 (in progress):
An Open-Label Study of the Hemostatic Activity, Pharmacokinetics and Safety of OBI-1 (B-Domain Deleted Recombinant Porcine fVIII), When Administered by Intravenous Injection, to Control Non-Life and Non-Limb Threatening Bleeding Episodes in Congenital Hemophilia A Patients with an Inhibitor to Human factor VIII (fVIII)

Eligible Study Population:
Abbreviated Inclusion Criteria:

• Adults and children 12 years and older at the time of consent
• Clinical diagnosis of hemophilia A, with an human FVIII inhibitor antibody of any titer
• Current treatment plan for an acute bleeding episode requires the use of inhibitor bypassing treatment, EITHER because the patient currently has an anti-human fVIII inhibitor antibody, OR because the patient has had an anti-human fVIII inhibitor antibody in the past and is known to have developed an anti-human fVIII inhibitor antibody anamnestic response to the administration of human fVIII
• Uncomplicated joint or soft tissue bleed, or other non-life threatening or non-limb threatening bleeding episode
• A current low (≤ 20 Bu) cross-reacting antibody to OBI-1 at Screening
• Written informed consent obtained

Abbreviated Exclusion Criteria:

Patients will be ineligible to participate if any of the following are present:

• Known bleeding disorder in addition to, or in place of, congenital hemophilia A.
• Current treatment plan for any acute bleeding episode incorporating the use of human fVIII (recombinant or plasma-derived).
• Presence of any of the following life- or limb-threatening bleeds:
  • Intracranial hemorrhage or severe head trauma
  • Penetrating wound of the abdomen or thorax
  • Acute intra-abdominal or gastrointestinal hemorrhage
  • Acute intra-thoracic hemorrhage
  • Acute iliopsoas hemorrhage
  • Acute throat or neck hemorrhage or any condition potentially compromising vital functions
• Received any human fVIII or prothrombin complex concentrate (PCC), within 7 days prior to Screening, OR received any PCC within 7 days prior to treatment with OBI-1.
• Received recombinant human fVIIa within 3 days prior to Screening OR within 3 days prior to treatment with OBI-1.
• Significant liver disease (esophageal varices, splenomegaly or alanine aminotransferase level more than 5 times the upper limit of normal for age and gender).
• Significant renal disease (serum creatinine >2.5 mg/dL).
• Pregnant or breastfeeding, OR female sexually active and not taking approved and effective contraceptive measures
• Unwillingness or inability to comply with the requirements of the protocol.

Planned Additional Studies:

• Octagen anticipates that in subsequent years we will conduct additional studies to establish the safety and efficacy of OBI-1 in patients with hemophilia A, an inhibitor antibody to human factor VIII, and with a low titer inhibitor antibody to porcine FVIII. Studies in this population are expected to assess:

1. treatment of episodic bleeding episodes
2. prophylactic coverage during required surgical procedures

• Octagen anticipates performing additional studies to establish safety and efficacy of OBI-1 in those non-hemophilia patients who have acquired FVIII inhibitor antibodies to human FVIII, and with a low titer inhibitor antibody to porcine FVIII. Studies in this population are expected to assess:

1. treatment of episodic bleeding episodes
2. prophylactic coverage during required surgical procedures.