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

This is a Phase II, open-label study designed to determine if intravenously administered NM8074 results in remission from TMA in treatment-naïve aHUS patients.

Inclusion Criteria:Patients ≥ 18 years at the time of consentPatients 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 schistocytesAcute kidney injury (proteinuria/creatinuria > ULN and/or reduced eGFR)Platelets less than 150,000 per microliter (Thrombocytopenia)Anemia (Hemoglobin ≤10 g/dL) due to hemolysisLactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (xULN) during ScreeningAll 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 antibioticsWilling 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.Exclusion Criteria:History of bone marrow, hematopoietic stem cell, or solid organ transplantationTreatment with complement blockersPatients with infectionsHUS due to ADAMTS-13 deficiency (<5%)Kidney disease other than aHUSChronic dialysis (hemo or peritoneal)Liver disease or other major autoimmune diseasesTypical HUS (Shiga toxin +)Known Systemic Lupus Erythematosus (SLE), Systemic Sclerosis, or antiphospholipid antibody positivity or syndromeHistory of currently active primary or secondary immunodeficiencyCurrently active systemic infection or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infectionsHas a currently active or known history of meningococcal disease or N. meningitidis infectionSevere concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (CKD stage 4, chronic dialysis)Females who have a positive pregnancy test result at Screening or on Day 1.Pregnant, planning to become pregnant, or nursing female subjects.