Dosing of CPV-104 in healthy volunteers marks key milestone in rare kidney disease program

Eleva has administered the first dose of its recombinant Factor H candidate, CPV-104, in a Phase 1 clinical trial involving healthy volunteers.

The study is evaluating safety, tolerability, and pharmacokinetics of the drug, which is being developed to treat C3 Glomerulopathy (C3G) — a rare and progressive kidney disease with limited treatment options.

C3G is caused by uncontrolled activation of the alternative pathway of the complement cascade, part of the innate immune system.

This dysregulation leads to deposits of complement protein C3 in the glomeruli, triggering inflammation and renal damage. By mimicking the activity of natural Factor H, CPV-104 is designed to restore regulatory control over the complement system and help prevent disease progression.

“Today’s milestone reflects the continued progress of our Factor H platform and the growing interest it’s generating in the clinic and among potential partners,” said Eleva CEO Björn Cochlovius.

“We aim to expand its potential across multiple indications.”

The Phase 1 trial involves a single ascending dose design in healthy volunteers, providing foundational data for subsequent studies in patients. A second arm of the program is expected to assess multiple ascending doses and eventually transition into patient cohorts.

“We are pleased to move our lead molecule into human studies,” added Dr Martin Bauer, chief medical officer at Eleva.

“This is a significant step forward for our team and for patients who currently have limited treatment options.”

Preclinical results recently published in Frontiers in Immunology support the compound’s potential, showing CPV-104’s ability to normalize serum C3 levels and accelerate clearance of complement deposits in the kidneys. The molecule has been granted Orphan Drug Designation in the European Union for the treatment of C3G.

Eleva is also exploring the use of CPV-104 in dry age-related macular degeneration (AMD), another complement-mediated disease. An intravitreal formulation is currently in preclinical development. In parallel, the company is advancing RPV-001 (aGal), its enzyme replacement therapy for Fabry disease, which has completed a positive Phase 1b study.