Akari Therapeutics presents case study from Phase 3 Part A trial of nomacopan

Akari Therapeutics announced the late-breaker presentation of a patient case study from the Phase 3 Part A clinical trial of nomacopan in pediatric hematopoietic stem cell transplant-related thrombotic microangiopathy. The case study, Clinical Response to Nomacopan in the Pediatric HSCT-TMA Setting, was presented on Thursday February 16, at 5:45 pm ET at the Transplantation & Cellular Therapy Tandem Meetings in Orlando, Florida. Nomacopan is an investigational bispecific inhibitor of both complement C5 and leukotriene B4. A 6-year-old male patient at Royal Manchester Children’s Hospital, Manchester University NHS Foundation Trust in Manchester, United Kingdom received a 6/8 HLA-mismatched unrelated cord blood HSCT conditioned with fludarabine, treosulfan and thiotepa, for relapsed refractory acute myelogenous leukemia. The patient received 7 granulocyte infusions peri-transplant as part of an experimental protocol to augment the graft-versus-leukemia effect. His immediate post-transplant course was complicated by engraftment syndrome, acute gut graft-versus-host disease grade 3 and cytomegalovirus viraemia. At day +66 after transplant, the patient developed features consistent with TMA, was enrolled in the Phase 3 Part A clinical trial, and began treatment with nomacopan on day +74. A single-age and weight-based ablating dose was followed by maintenance dosing for 21 days. The patient was treated by investigator Professor Rob Wynn, M.D. After initial pharmacodynamic analysis at day 14 of treatment, the patient was found to have pre-dose terminal complement activity slightly higher than the LLOQ. Although his TCA had been reduced by 95% from a high baseline CH50 of 299.6U Eq/ml and sC5b9 had normalized, dose was increased in line with the study protocol. A few days later the patient developed neurological symptoms following a period of hypertension and was diagnosed with posterior reversible encephalopathy syndrome. Nomacopan was stopped for 3 days and restarted after the diagnosis was deemed to be unrelated to nomacopan treatment. Treatment continued for 46 days until the patient’s urine protein creatinine ratio was corrected for greater than or equal to28 days. Gut pathology and thrombocytopenia were resolved. The patient was discharged from the hospital and remains well and in remission. No adverse events related to nomacopan were experienced during the 72-day treatment period.