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Results from the suggest that the targeted C3 inhibitor pegcetacoplan improved hemoglobin levels better than eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were still anemic despite at least three months of eculizumab therapy. The data were presented at December's American Society of Hematology virtual meeting.

In this exclusive 番茄社区 video, study co-author , of Keck School of Medicine at the University of Southern California, discusses the trial's findings.

Following is a transcript of her remarks:

The PEGASUS study was a study designed to look at the effect of C3 inhibition versus C5 inhibition in patients with PNH who were already on C5 inhibition, but had a suboptimal response. Most of these patients were transfusion dependent and had hemoglobin <10.5 g/dL.

So, the patients received overlap therapy and then they were stratified to either receive a pegcetacoplan, the C3 inhibitor, versus standard eculizumab. Ravu [ravulizumab] was not considered in this cohort. And the patients were assessed at 16 weeks, and the 16 week data, which was presented originally at the European hematology meetings, and then ASH, show that there was an almost four gram improvement in hemoglobin in these patients. And there was an improvement in symptomatology as well.

So it does appear that as monotherapy, which is given subcutaneously two to three times a week, this was effective in stopping hemolysis in PNH patients who were intolerant to a C5 inhibitor.

It gives another option for patients who are not having the best response to a C5 inhibitor. There are still questions out there. Does it continue to suppress thrombosis the way a C5 inhibitor does? Strategies for breakthrough need to be assessed. And what are the best patients for this treatment? So we don't have any information on patients who have a suboptimal response to C5, but don't have significant anemia or aren't transfusion dependent. And we know that all PNH patients on a C5 inhibitor seem to have some component of extravascular clearance.

So those are some outstanding questions that need to be addressed. We did not see a significant number of infections, which you would have expected with a C3 inhibitor. But all the patients are vaccinated for everything and they were all on penicillin prophylaxis.

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