Study of NM8074 in Patients With aHUS With Evidence of Ongoing Thrombotic Microangiopathy

Inclusion Criteria

• •Patients ≥ 18 years at the time of consent
• •Patients with evidence of resistant or relapsed complement-mediated aHUS with symptoms of Thrombocytopenia, hemolysis, ongoing Thrombotic Microangiopathy and acute kidney injury.
• •Evidence of ongoing Thrombotic Microangiopathy which includes Haptoglobin \<LLN or undetectable and/or presence of schistocytes
• •Acute kidney injury (proteinuria/creatinuria \> ULN and/or reduced eGFR)
• •Platelets less than 150,000 per microliter (Thrombocytopenia)
• •Anemia (Hemoglobin ≤10 g/dL) due to hemolysis
• •Lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (xULN) during Screening
• •All patients must be vaccinated prior to dosing with MenACWY Menactra® polysaccharide diphtheria toxoid conjugate vaccination against Neisseria meningitidis serogroups A, C, Y, and W-135 and MenB meningococcal serogroup B vaccine (Bexsero®). If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics
• •Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and comply with the study visit schedule.
• •Male patients and partners of child-bearing potential must agree to use contraceptives and male patients must agree to refrain from donating sperm for the duration of the study.
• •Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for 1 month after stopping the investigational drug.