European Hematology Association - Pegcetacoplan Maintains a Durable Response in Patients with Paroxysmal Nocturnal Hemoglobinuria Through Week 48

THE HAGUE, Netherlands, June 12, 2021 /PRNewswire/ -- The PEGASUS phase-3 study previously demonstrated that pegcetacoplan, a drug controlling intra- and extravascular hemolysis, was superior to eculizumab in patients with paroxysmal nocturnal hemoglobinuria at the week 16 endpoint of the randomized controlled period (RCP). After week 16, patients entered an open-label period where all patients were placed on pegcetacoplan treatment. Here, we show the efficacy and safety of pegcetacoplan in 77 patients through 48 weeks of treatment.

Improvement of hemoglobin (Hb) level was maintained through Week 48 in all patients treated with pegcetacoplan monotherapy. Importantly, patients who switched from eculizumab to pegcetacoplan at week 16 displayed improved Hb levels at week 48 that was comparable to the pegcetacoplan group in the RCP. Similarly, these patients showed improved laboratory and clinical parameters and almost 75% remained transfusion-free, which were comparable to the pegcetacoplan arm in the RCP. Overall, 6% experienced serious adverse events that were possibly related to pegcetacoplanand 15% discontinued the study due to treatment-emergent adverse events. These safety parameters were consistent with previously reported data. In conclusion, patients with suboptimal response to eculizumab experienced durable treatment effect when switched to pegcetacoplan, suggesting that pegcetacoplan is an effective therapeutic option for patients with paroxysmal nocturnal hemoglobinuria.

Presenter:          Dr. Régis Peffault de Latour

Affiliation:          French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Assistance Publique – Hôpitaux de Paris, Université de Paris,  France

Abstract:              #S174  FORTY-EIGHT WEEK EFFICACY AND SAFETY OF PEGCETACOPLAN IN ADULT PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AND SUBOPTIMAL RESPONSE TO PRIOR ECULIZUMAB TREATMENT

About the EHA Annual Congress: Every June, EHA organizes its Annual Congress in a major European city. This year, due to the persisting COVID19 pandemic, EHA organizes a virtual Congress for the second time. The Congress is aimed at health professionals working in or interested in the field of hematology. The scientific program topics range from stem cell physiology and development to leukemia; lymphoma; diagnosis and treatment; red blood cells; white blood cells and platelet disorders; hemophilia and myeloma; thrombosis and bleeding disorders; as well as transfusion and stem cell transplantation.

Website: www.ehaweb.org

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