CPV-104, a Recombinant Variant of Human Complement Factor H Produced in Moss, to Be Studied in a Phase 1/2 Clinical Trial in Patients with C3 Glomerulopathy (C3G)

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Background: Human complement factor H (CFH) is the main regulator of the alternative pathway (AP). CFH deficiency is associated with several complement-mediated diseases, such as C3 Glomerulopathy (C3G) or atypical hemolytic uremic syndrome (aHUS). C3G is caused by an uncontrolled overactivation of AP in fluid phase and on cell surfaces with overlapping clinical and pathophysiological features. AP dysregulation leads to predominant C3 fragment deposition within the glomeruli. C3G appears with proteinuria, microhematuria, acute kidney injury, and chronic kidney failure. About 50% of patients develop end-stage renal disease within 10 years. Recurrence of C3G after kidney transplantation is common. Currently there is no established disease-modifying treatment available. CPV-104 is produced in the suspension culture of moss Physcomitrium patens in 500L single-use bioreactors. Methods: The current clinical program aims to develop CPV-104 for treatment of C3G. Results: Preclinical in vivo (FH (-/-) knockout mice) and in vitro studies with CPV-104 demonstrated comparable or superior efficacy to that achieved with serum-derived CFH. Conclusions: CPV-104 is a promising candidate for the 1st disease modifying treatment option in C3G. In preclinical studies CPV-104 demonstrated a strong potential to rebalance the dysregulated complement pathway, simultaneously maintaining its natural defense potential against infectious agents and pathogens. Following successful phase 1 and a proof-of-concept clinical trial in C3G, CPV-104 may also become a new treatment option for a broader spectrum of complement mediated diseases. Funding: Commercial Support - eleva GmbHClinical Trial Design

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