New Pivotal Data Demonstrate Superiority of Apellis’ Pegcetacoplan to Eculizumab in Improving Hemoglobin Levels, Independent of Prior Transfusions, in PNH - Seite 2

Data presented at EHA demonstrated that pegcetacoplan reduced transfusion requirements consistently across the study population. Overall, 85% of pegcetacoplan-treated patients were transfusion-free over 16 weeks vs. 15% of eculizumab-treated patients. In patients with low or no transfusion requirements, 85% of pegcetacoplan-treated patients were transfusion-free compared to 31% of eculizumab-treated patients. In patients with high transfusion requirements, 86% of pegcetacoplan-treated patients were transfusion-free compared to 4% of eculizumab-treated patients.

Pegcetacoplan also showed higher normalization rates in key markers of hemolysis and clinically meaningful improvements in FACIT-fatigue score compared to eculizumab at 16 weeks:

• 78% of pegcetacoplan-treated patients achieved reticulocyte normalization vs. 3% of eculizumab-treated patients.
• 71% of pegcetacoplan-treated patients achieved LDH normalization vs. 15% of eculizumab-treated patients.
• 73% of pegcetacoplan-treated patients achieved at least a three-point improvement in FACIT-fatigue score vs. 0% of eculizumab-treated patients. A three-point improvement in FACIT-fatigue score is generally considered to be clinically meaningful.²

“Even with current treatments, the anemia and relentless fatigue caused by C3-driven extravascular hemolysis continue to be a significant problem for people living with PNH,” said Peter Hillmen, M.B., Ch.B., Ph.D., Professor of Experimental Hematology at the University of Leeds and an investigator in the PEGASUS study. “These results demonstrate that pegcetacoplan controls both extravascular and intravascular hemolysis, and has the potential to become a new standard of care for PNH.”

As previously reported, the safety profile of pegcetacoplan was comparable to eculizumab in this study. Seven of 41 patients (17%) in the pegcetacoplan group experienced a serious adverse event (SAE), and 6 of 39 patients (15%) in the eculizumab group experienced SAEs. No cases of meningitis and no deaths were reported in either treatment group.

Apellis plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration and a Marketing Authorization Agreement (MAA) to the European Medicines Agency for pegcetacoplan for the treatment of PNH in the second half of 2020.

Conference Call and Webcast
Apellis will host a conference call and webcast with Dr. Peter Hillmen to discuss the results of the PEGASUS Phase 3 clinical study on June 12 at 8:30 a.m. ET. To access the conference call, please dial [phone] (local) or [phone] (international) at least 10 minutes prior to the start time and refer to conference ID 7291583. A live audio webcast of the event and accompanying slides may also be accessed through the “Events and Presentations” page of the “Investors and Media” section of the company’s website at http://investors.apellis.com/events-and-presentations. A replay of the webcast will be available for 30 days following the event.