Apellis Pharmaceuticals Presents Update on Phase 1b PHAROAH Trial of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) at Annual Scientific Assembly of the International PNH Interest Group (IPIG)
December 8, 2017
Six patients were enrolled in the PHAROAH trial and two discontinued due to reasons unrelated to therapy. Four patients continue to be treated with daily doses of APL-2 270mg in combination with eculizumab for at least 12 months. Their average baseline hemoglobin level was 8.9 g/dL (normal range 12.0-15.0 g/dL) and the average number of transfusions in the 12 months preceding initiation of treatment was 5.25. Three of four patients were being treated with eculizumab at doses or frequencies in excess of 900mg/bi-weekly, while the remaining patient was being treated with 900mg/bi-weekly. The four patients had an average baseline reticulocyte count of 332 103 μL (normal range 39.0 – 123.0 103 μL) and average lactate dehydrogenase (LDH) levels of 210 U/L (normal range 110 -209 U/L).
All four patients remain transfusion independent with an average hemoglobin level of 11.6 g/dL (range 10.4 – 12.7 g/dL) at one year of treatment. Average reticulocyte count decreased to 56.02 103 μL by month one and has remained steady since that time. Average LDH level was 184.5 U/L at one year of treatment.
The three patients co-treated with high dose eculizumab have had their dose lowered to 900 mg bi-weekly during the course of the study, with no impact on hemoglobin, LDH or reticulocytes.
About Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, potentially life-threatening disease characterized by complement-mediated hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes and/or some degree of bone marrow dysfunction. A significant subset of patients treated with the current standard of care still suffer from debilitating anemia and transfusion dependence.
APL-2 is designed to inhibit the complement cascade centrally at C3, and may have the potential to treat a wide range of complement-mediated diseases more effectively than is possible with partial inhibitors of complement. APL-2 is a synthetic cyclic peptide conjugated to a polyethylene glycol (PEG) polymer that binds specifically to C3 and C3b, effectively blocking all three pathways of complement activation (classical, lectin, and alternative).
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Source: Apellis Pharmaceuticals, Inc.