Apellis provides update on blood disorder med trial

Apellis announced an update on its US-based Phase 1b PHAROAH trial for patients with paroxysmal nocturnal hemoglobinuria, or PNH. The ongoing PHAROAH trial is evaluating treatment with APL-2, a novel inhibitor of complement factor C3, in patients on treatment with eculizumab who are severely anemic and transfusion-dependent. Patients enrolled in the PHAROAH trial were initially co-treated with APL-2 and eculizumab, with the potential for discontinuation of eculizumab at the discretion of the treating physician. Two of the six patients were removed from treatment with APL-2 due to pregnancy and BMI-associated comorbidities. Four of the six patients continued in the study for more than 32 weeks. In these four patients, co-treatment with APL-2 resulted in an improvement of hemoglobin levels and other markers for anemia and none of the four patients required a transfusion during the co-treatment period with eculizumab, which ranged from 17 to 20 months. Earlier this year Apellis announced that three of the four patients discontinued treatment with eculizumab and were continuing to receive APL-2. The fourth patient has now discontinued treatment with eculizumab and is continuing to receive APL-2. All four patients have improved hemoglobin and reticulocytes as compared to the baseline established with eculizumab monotherapy and have achieved lactate dehydrogenase levels below the upper limit of normal.

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