Benzinga | Mar 17, 2021 05:53PM EDT

Apellis and Sobi Announce The Publication To The New England Journal Of Medicine Of Their Phase 3 PEGASUS Study Results Comparing Pegcetacoplan To Eculizumab For PNH

* Pegcetacoplan, an investigational targeted C3 therapy for serious, complement-driven diseases, demonstrated superiority to eculizumab with a statistically significant improvement in hemoglobin levels and showed improvements in key clinical outcomes

* Marketing applications for pegcetacoplan are under Priority Review with the FDA and under review with the EMA

WALTHAM, Mass. and STOCKHOLM, March 17, 2021 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi(tm)) (STO:SOBI) today announced that the New England Journal of Medicine (NEJM) published results from the Phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving hemoglobin levels and showed improvements in key clinical outcomes compared to eculizumab, a C5 inhibitor, in adults with paroxysmal nocturnal hemoglobinuria (PNH) at 16 weeks who had persistent anemia following treatment with eculizumab.

"The data published in the New England Journal of Medicine underscore the potential of pegcetacoplan to be a significant advancement for people living with PNH," said Peter Hillmen, M.B., Ch.B., Ph.D, professor of experimental haematology at Leeds Teaching Hospitals NHS Trust and PEGASUS study author. "There is still a need for new treatments because many patients with PNH treated with C5 inhibitors today remain anemic, resulting in moderate to severe fatigue, with a proportion continuing to require transfusions."

"The PEGASUS study results demonstrate that, if approved, pegcetacoplan has the potential to elevate the standard of care for PNH by providing more complete disease control," said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. "We are working to quickly bring this potential new treatment to PNH patients and to advance development of pegcetacoplan for many other serious, complement-driven diseases."

"The NEJM publication of the PEGASUS study results reflect the significance of these data for both the medical and PNH patient communities," said Ravi Rao, head of research and development and chief medical officer at Sobi. "The results further advance our understanding of the role of C3 in PNH and our hope is to contribute to the improvement and care for PNH patients around the world."

The results published in NEJM highlight that pegcetacoplan met the study's primary endpoint for efficacy, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.001). Additionally, 85% of pegcetacoplan-treated patients were transfusion free over 16 weeks versus 15% of eculizumab-treated patients. There were meaningful improvements across key markers of disease such as absolute reticulocyte count, lactate dehydrogenase (LDH), and fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score.

Safety was comparable between pegcetacoplan and eculizumab in this study. Seven of 41 patients (17%) in the pegcetacoplan group experienced a 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.

The most common adverse events reported during the 16-week, randomized, controlled treatment period in the pegcetacoplan and eculizumab groups, respectively, were injection site reactions (37% vs. 3%), diarrhea (22% vs. 3%), breakthrough hemolysis (10% vs. 23%), headache (7% vs. 23%), and fatigue (5% vs. 15%).

Top-line results from the PEGASUS study at 48 weeks were recently announced, which showed sustained improvements in key markers of disease as well as a consistent safety profile with previously reported data.

Marketing applications for pegcetacoplan for the treatment of PNH are under review by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). The FDA granted the application Priority Review designation and set a target action date of May 14, 2021. An opinion from the Committee for Medicinal Products for Human Use (CHMP) is expected in 2021.