STOCKHOLM - Swedish Orphan Biovitrum AB (publ) (Sobi) (STO:SOBI) and Apellis Pharmaceuticals, Inc. (Nasdaq: APLS) today announced that The New England Journal of Medicine (NEJM) published results from the phase 3 PEGASUS study, which demonstrated the superiority of pegcetacoplan, an investigational targeted C3 therapy, in improving haemoglobin levels and showed improvements in key clinical outcomes compared to eculizumab, a C5 inhibitor, in adults with paroxysmal nocturnal haemoglobinuria (PNH) at 16 weeks who had persistent anaemia following treatment with eculizumab.

'The data published in The New England Journal of Medicine underscore the potential of pegcetacoplan to be a significant advancement for people living with PNH,' said Peter Hillmen, M.B., Ch.B., Ph.D, professor of experimental haematology at Leeds Teaching Hospitals NHS Trust and PEGASUS study author. 'There is still a need for new treatments because many patients with PNH treated with C5 inhibitors today remain anemic, resulting in moderate to severe fatigue, with a proportion continuing to require transfusions.'

'The PEGASUS study results demonstrate that, if approved, pegcetacoplan has the potential to elevate the standard of care for PNH by providing more complete disease control,' said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. 'We are working to quickly bring this potential new treatment to PNH patients and to advance development of pegcetacoplan for many other serious, complement-driven diseases.'

'The NEJM publication of the PEGASUS study results reflect the significance of these data for both the medical and PNH patient communities,' said Ravi Rao, Head of Research & Development and Chief Medical Officer at Sobi. 'The results further advance our understanding of the role of C3 in PNH and our hope is to contribute to the improvement and care for PNH patients around the world'.

The results published in NEJM highlight that pegcetacoplan met the study's primary endpoint for efficacy, demonstrating superiority to eculizumab with a statistically significant improvement in adjusted means of 3.8 g/dL of haemoglobin at week 16 (p

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