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Paroxysmal nocturnal haemoglobinuria treatment with pegcetacoplan

Presented By
Prof. Peter Hillmen, St James's University Hospital, UK
EHA 2020
Phase 3, PEGASUS trial
The novel complement C3 inhibitor pegcetacoplan is significantly better at improving haemoglobin as well as other key clinical outcomes at week 16 versus the current standard of care, eculizumab, in patients with paroxysmal nocturnal haemoglobinuria (PNH). Prof. Peter Hillmen (St James's University Hospital, UK) presented the findings from the phase 3 PEGASUS trial [1]. Preliminary data from the phase 1b PHAROAH and PADDOCK trials provided proof-of-principle that pegcetacoplan could improve lactate dehydrogenase and haemoglobin levels in patients who had experienced a suboptimal response to eculizumab, and in eculizumab-naïve patients, respectively. In...

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