Sobi and Apellis collaborate on the development and commercialisation of pegcetacoplan.

Swedish Orphan Biovitrum AB (publ) (Sobi) and Apellis Pharmaceuticals Inc. announced a strategic collaboration to accelerate the advancement of systemic pegcetacoplan, a targeted C3 therapy, for the treatment of multiple rare diseases with high unmet need that impact more than 275,000 patients globally. Sobi will receive global co-development and exclusive ex-US commercialisation rights for systemic pegcetacoplan. Apellis retains US commercialisation rights for systemic pegcetacoplan and worldwide commercial rights for ophthalmological pegcetacoplan, which is being evaluated by Apellis in two fully enrolled phase III studies in geographic atrophy (GA). Pegcetacoplan targets excessive activation of C3 in the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. Apellis and Sobi plan to jointly advance the clinical development of systemic pegcetacoplan in five parallel registrational programmes across haematology, nephrology and neurology. These include new registrational programmes in cold agglutinin disease (CAD) and haematopoietic stem cell transplantation-associated thrombotic microangiopathy (HSCT-TMA), both of which are expected to start in 2021. By controlling complement activation centrally, pegcetacoplan offers the potential to become a transformative new therapy in several rare diseases where patients have few or no treatment options today. As part of the collaboration, Apellis and Sobi will co-develop systemic pegcetacoplan in the following rare diseases: Haematology - Paroxysmal nocturnal haemoglobinuria (PNH), CAD, and HSCT-TMA. PNH represents the first potential indication to market for systemic pegcetacoplan. Marketing applications for pegcetacoplan for the treatment of PNH were submitted to the FDA and the European Medicines Agency (EMA) based on positive results from the Phase III PEGASUS study. Top-line results from the phase III PRINCE study, which is evaluating pegcetacoplan in treatment-naïve patients with PNH, are expected in the first half of 2021. Sobi will lead development activities for the phase iII study in CAD and a potential registrational phase II study in HSCT-TMA, both planned to start in 2021. Nephrology - Immune complex membranoproliferative glomerulonephritis (IC-MPGN) and C3 glomerulopathy (C3G) Apellis has initiated and will continue to lead a registrational programme in IC-MPGN and C3G which includes phase II and phase III studies. Neurology - Amyotrophic lateral sclerosis (ALS)- Apellis has initiated and will continue to lead a potential registrational phase II study in ALS. Multiple other neurological conditions are under consideration for future clinical development. About the transaction : Sobi will make an upfront payment of $ 250 million to Apellis and up to $ 915 million in other regulatory and commercial milestone payments, and will contribute $ 80 million in reimbursement payments over a four-year period for research and development to support the initial development plan, which includes ongoing studies in PNH, IC-MPGN/C3G, and ALS and new studies in CAD and HSCT-TMA. Apellis will also be eligible for tiered double-digit royalties on sales ranging from high teens to high twenties. Sobi intends to finance these payments with available funds. Sobi will receive reimbursement payments for the costs incurred by Sobi in connection with the CAD and HSCT-TMA trials that Sobi will conduct. The parties have agreed to split costs 50/50 for any future global studies beyond the initial development plan.