Experimental Drug Proves Superior to Eculizumab as a Treatment for Paroxysmal Nocturnal Hemoglobinuria

According to a story from BioSpace, the biopharmaceutical company Apellis Pharmaceuticals, Inc., has recently announced the results of its phase 3 clinical trial, which was testing the company’s investigational drug pegcetacoplan as a treatment for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease. In this study, the drug was also compared side by side with the drug eculizumab, which is a standard therapy for the illness. In the comparison, pegcetacoplan displayed superior efficacy.

About Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare blood disease. This life-threatening illness is most characterized by the destruction of red blood cells by a part of the innate immune system called the complement system. Paroxysmal nocturnal hemoglobinuria is an acquired disease, meaning that it is not caused by genetic abnormalities. It can appear on its own or alongside other blood diseases, such as myelodysplastic syndromes or aplastic anemia. The disease occurs because of a defect in a protein that appears on the surface of red blood cells called DAF. Symptoms of the disease include red urine (especially in the morning), palpitations, blood clots, fatigue, shortness of breath, pain when swallowing, abdominal pain, and erectile dysfunction. Blood clots are a primary cause of death for patients with paroxysmal nocturnal hemoglobinuria. This disease is best treated with either eculizumab or, more recently, ravulizumab where it is available. To learn more about paroxysmal nocturnal hemoglobinuria, click here.

Trial Findings: Pegcetacoplan vs Eculizumab

This clinical trial included a total of 80 adult patients. All of these patients were receiving treatment with eculizumab when the study began. During the 16 week treatment phase, patients were randomized to either continue their current regimen of eculizumab or receive 1080 mg of pegcetacoplan two times a week. In the study, patients receiving the experimental drug saw the hemoglobin levels increase by 2.4 g/dL from a baseline of 8.7 g/dL. Meanwhile, patients who continued with eculizumab saw their hemoglobin drop by 1.5 g/dL. This makes pegcetacoplan the first investigational drug to display superiority in hemoglobin levels when compared to eculizumab.

The drug also displayed encouraging results in several secondary endpoints and had a comparable safety profile to eculizumab. Ultimately, the findings from the study bode well for the future development of the drug and the future of the treatment of patients with paroxysmal nocturnal hemoglobinuria.


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