This site is intended for healthcare professionals

Apellis reports positive top-line results from phase III PEGASUS head-to-head study of pegcetacoplan compared to eculizumab for paroxysmal nocturnal hemoglobinuria.

Read time: 1 mins
Published:9th Jan 2020
Apellis Pharmaceuticals, Inc. announced positive results from the Phase III PEGASUS study evaluating pegcetacoplan (APL-2) in adults with paroxysmal nocturnal hemoglobinuria (PNH). Top-line data show that pegcetacoplan met the study’s primary efficacy endpoint, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of hemoglobin at week 16 (p<0.0001). at week 16, pegcetacoplan-treated patients (n="41)" had an adjusted mean hemoglobin increase of 2.4 g dl from a baseline of 8.7 g dl, compared to eculizumab-treated patients (n="39)" who had a change of -1.5 g dl from a baseline of 8.7 g dl. additionally, pegcetacoplan showed promising results in key secondary endpoints. pegcetacoplan met non-inferiority on transfusion avoidance and absolute reticulocyte count. pegcetacoplan also showed positive trends on lactate dehydrogenase (ldh) and fatigue as measured by the functional assessment of chronic illness therapy (facit)-fatigue score. in this study, the safety profile of pegcetacoplan was comparable to eculizumab. seven of 41 patients (17.1%) in the pegcetacoplan group experienced a serious adverse event (sae), and 6 of 39 patients (15.4%) 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 (36.6% vs. 2.6%), diarrhea (22.0% vs. 0%), headache (7.3% vs. 20.5%) and fatigue (4.9% vs. 15.4%). another common adverse event was hemolysis, which was reported in four patients in the pegcetacoplan group (9.8%) and nine patients in the eculizumab group (23.1%). this led to the three discontinuations in pegcetacoplan group. all patients who completed the randomization period in both groups (77 80) entered the 32-week open-label pegcetacoplan treatment period.>
Condition: Paroxysmal Nocturnal Haemoglobinuria
Type: drug

Learning Zones

The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.

Related news and insights