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Apellis therapy outflanks Alexion's Soliris in head-to-head paroxysmal nocturnal haemoglobinuria

Published on 08/01/20 at 12:00pm

Apellis Pharmaceuticals has revealed new Phase 3 demonstrating that its targeted C3 inhibitor pegcetacoplan met its primary endpoint and outclassed Alexion’s Soliris (eculizumab) in the treatment of paroxysmal nocturnal haemoglobinuria (PNH) in adult patients.

Specifically, it was found that patients treated with Apellis’ therapy experienced a “statistically significant improvement” in adjusted means of 3.8 g/dL of haemoglobin. After 16 weeks of treatment, these patients saw an increase of 2.4 g/dL in adjusted mean haemoglobin from a baseline of 8.7 g/dL, compared to a change of -1.5 g/dL with Soliris.

The therapy was also shown to be non-inferior to Alexion’s drug in terms of transfusion avoidance and absolute reticulocyte count, and presented positive trends on lactate dehydrogenase (LDH) and fatigue – encouraging results in the trials secondary endpoints.

“Going into the study, our most optimistic expectation was to see a 2 g/dL or more change in haemoglobin and a trend on the key secondary endpoints. Needless to say, we are thrilled with these results,” explained Dr Cedric Francois, Co-Founder and Chief Executive Officer of Apellis. “These data give us strong confidence in the further development of pegcetacoplan as a targeted C3 inhibitor in geographic atrophy and other serious complement-driven diseases.”

Dr Federico Grossi, Chief Medical Officer of Apellis, added: “Pegcetacoplan is the first and only investigational therapy to demonstrate superiority compared to eculizumab on haemoglobin levels. We are also excited to see that 85% of patients treated with pegcetacoplan were transfusion free. At Apellis, we are focused on developing groundbreaking therapies, and these results show that pegcetacoplan has the potential to transform the lives of people with PNH. We look forward to meeting with regulators in the first half of the year to discuss next steps.”

Matt Fellows

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