Apellis presents data from ongoing Phase 2 PLAUDIT study of APL-2
Apellis Pharmaceuticals announced updated data from its Phase 2 PLAUDIT study of APL-2 in patients with autoimmune hemolytic anemia, including cold agglutinin disease and warm antibody autoimmune hemolytic anemia. Data from the PLAUDIT trial will be presented in an oral presentation at the 24th Annual Congress of the European Hematology Association, held in Amsterdam, the Netherlands. In the ongoing PLAUDIT study, 13 patients with CAD have been enrolled to receive subcutaneous APL-2 treatment, of which 10 patients have been on APL-2 for at least 168 days. The trial has also enrolled 11 patients with wAIHA, 8 of which were Direct Antiglobulin Test C3+; 5 of the C3+ wAIHA patients have been on APL-2 for at least 168 days. Of the 10 patients who reached Day 168: 70% showed a Hb increase of greater than or equal to2 g/dL, 40% had normalized Hb and 80% had Hb greater than or equal to11.0 g/dL at Day 168. Mean Hb increased from 8.9 g/dL at baseline to 11.2 g/dL at Day 168, a 2.4 g/dL increase. Mean Functional Assessment of Chronic Illness Therapy Fatigue Score increased from 29.4 at baseline to 39.1 at Day 168, an improvement of 9.7 points, where a clinically significant increase is 3 or more points. Mean absolute reticulocyte count decreased from 159 X 10/L at baseline to 64 X 10/L at Day 168. Mean indirect bilirubin decreased from 1.6 mg/dL at baseline to 0.4 mg/dL at Day 168. Mean LDH decreased from 500 U/L at baseline to 183 U/L at Day 168. Two heavily transfusion dependent patients did not respond to APL-2 and left the study at Day 56 and 108 respectively. The remaining transfusion dependent patients did not require any transfusions during maintenance treatment with APL-2. One previously non-transfusion dependent patient received an on-study transfusion prior to APL-2 steady-state. One patient is still participating in the study and has not yet reached Day 168. Of the 5 patients with C3+ wAIHA who reached Day 168: Mean Hb increased from 9.0 g/dL at baseline to 11.0 g/dL at Day 168, a 2.0 g/dL increase. Mean FACIT Fatigue Score increased from 38.4 at baseline to 40.8 at Day 168, an improvement of 2.4 points. Mean ARC decreased from 213 X 10/L at baseline to 92 X 10/L at Day 168. Mean indirect bilirubin decreased from 0.8 mg/dL at baseline to 0.3 mg/dL at Day 168. Mean LDH decreased from 241 U/L at baseline to 142 U/L at Day 168. Two of the eight enrolled C3+ wAIHA patients left the study due to lack of response, and one of the eight enrolled C3+ wAIHA subjects is still participating in the study and has not yet reached Day 168. The three enrolled patients who were not DAT C3+ did not show a meaningful response and two have left the study. In both the CAD and C3+ wAIHA populations, APL-2 was generally well tolerated; no serious adverse events related to APL-2 were reported in the PLAUDIT trial. In both the CAD and C3+ wAIHA populations, APL-2 was generally well tolerated; no serious adverse events related to APL-2 were reported in the PLAUDIT trial.