Akari Therapeutics announces data on long-term self-administration of nomacopan

Akari Therapeutics announces new data on the efficacy and safety profile of long-term self-administration of nomacopan for treatment of patients with PNH. The data from 19 PNH patients2 treated for a median of 18.5 months are derived from the Phase II COBALT trial, the Phase II trial which specifically recruited eculizumab-resistant patients, the Phase III CAPSTONE trial, and from the long-term safety study CONSERVE, which accepted patients from the Phase II and Phase III studies. Sixteen of the 19 PNH patients were transfusion dependent prior to treatment with nomacopan, of whom 14 were treated with nomacopan for six months or more. The data show that long term self-administration of nomacopan by PNH patients: Reduced transfusion dependence by 79% in the 14 formerly transfusion dependent patients treated with nomacopan for at least six months, with a median time since last transfusion of 13.8 months. In the Phase III CAPSTONE trial, all patients dosed with nomacopan were transfusion independent for the six-month treatment period while all patients on placebo remained transfusion dependent, which was the primary end point of the study. The difference is statistically significant at the p=0.034 level. The active and placebo groups were very similar in terms of transfusion requirements prior to treatment. The number of units of packed red blood cells received per month declined in all 16 of the transfusion dependent PNH patients by 77% from a mean value of 0.91 U PRBC per month prior to treatment with nomacopan to 0.21 U PRBC per month on nomacopan. The three PNH patients who were transfusion independent prior to treatment with nomacopan remained transfusion independent on nomacopan. None of the 19 PNH patients treated with nomacopan for over 30 cumulative patient-years had a reported major adverse vascular event. There has only been a single reported serious adverse event in one patient that was considered possibly related to nomacopan. None of the patients has had a meningococcal infection reported. Terminal complement activity was below the lower limit of assay quantification in all patients at all time points measured during long-term treatment on nomacopan. Transfusion independence as used above is defined as a period of at least six months without transfusion. This duration is considered clinically important as it is the same duration of transfusion independence as used for the primary endpoint in CAPSTONE and also the pivotal studies of eculizumab and ravulizumab that led to approval of these drugs for the treatment of PNH. The 79% transfusion independence reported for the 14 PNH patients all formerly transfusion dependent who were treated with nomacopan for at least six months, compares favorably to the treatment of PNH patients on long-term eculizumab therapy where between approximately 50%-60% of transfusion dependent patients become transfusion independent in a 12-month period. Similar data has been reported in the more recent head to head Phase III comparative study of eculizumab and ravulizumab where transfusion independence in the two arms respectively increased from 21% to 66% of and 23% to 74% of patients over the first 6 months of treatment. The transfusion data from the CONSERVE long term study is considered particularly important because it provides "real world" data from a total of 15 patients on nomacopan treatment as a long-term self-administered therapy. In CONSERVE, clinicians were free to follow their normal transfusion practices and patients were only scheduled to visit the clinic at three to six monthly intervals, mimicking "real world" clinical practice. The Company's lead programs for nomacopan are focused on diseases with no approved treatments, including BP and pediatric HSCT-TMA. Data from the Company's PNH programs, while discontinued, support the Company's main clinical programs, all of which have complement dysregulation playing an important role.