One04 Therapeutics launches on Phase 1 data for Factor H candidate
One04 Therapeutics GmbH has formally launched as a clinical-stage biopharmaceutical company, announcing encouraging interim Phase 1 results for CPV-104, its recombinant full-length human Factor H candidate. The Freiburg-based company is positioning CPV-104 as a potential disease-modifying therapy across a range of complement-mediated diseases, beginning with C3 glomerulopathy (C3G) and extending into dry age-related macular degeneration (AMD) and geographic atrophy (GA).
The company has received undisclosed funding from D11Z.Ventures, a European early-stage venture capital fund, and says it is fully funded to what it describes as key value inflection points. CPV-104 was originally discovered using Eleva Biologics' moss-based Bryotechnology production platform and was subsequently transferred to One04 to give both entities greater operational focus.
Phase 1 interim data
Interim results from the first two cohorts of the multiple-ascending dose (MAD) portion of the ongoing Phase 1 trial, which covers 18 patients across three dose cohorts, showed favourable safety, tolerability and pharmacokinetics, alongside early efficacy trends in patients with C3G. The placebo-controlled single-ascending dose (SAD) phase, completed in October 2025 across 21 healthy volunteers in four dose cohorts, generated no safety concerns. One04 will present detailed interim data via a public livestream on 26 October 2026, following its attendance at American Society of Nephrology Kidney Week.
Chief executive Björn Cochlovius described CPV-104 as "a pipeline-in-a-product that has matured into a highly promising clinical candidate for complement-mediated diseases." The company holds EU Orphan Designation for C3G, a rare kidney disease characterised by over-activation of the complement system's alternative pathway, which carries significant unmet need and no approved disease-modifying therapy.
Market context and competitive positioning
The complement inhibition space has grown considerably more competitive in recent years, largely driven by the commercial success of Alexion's eculizumab and its successor ravulizumab in related complement disorders. More recently, a number of companies have advanced targeted agents against specific nodes of the complement cascade, including C3, C5 and properdin. One04's stated differentiation is that CPV-104 does not inhibit a single component but instead aims to restore the system's own regulatory function via Factor H, theoretically preserving the immune homeostasis benefits of normal complement activity while damping pathological over-activation. Whether this mechanistic distinction translates into a clinical or commercial advantage will depend heavily on the Phase 2 readout, where absolute complement biomarker data and renal function endpoints will be closely scrutinised.
Dry AMD and geographic atrophy represent a substantially larger commercial opportunity. The complement pathway has long been implicated in AMD pathogenesis, and regulatory approvals in this space have been hard-won: Apellis's pegcetacoplan (targeting C3) secured FDA approval in 2023 for GA after a protracted clinical journey, illustrating both the opportunity and the difficulty of the indication. For One04, Phase 1 data in AMD from the trial planned to start in early 2027 will represent an early but important proof-of-concept step.
One04 plans to initiate an open-label Phase 2 study in up to 24 patients with C3G and selected other complement-mediated kidney diseases in early 2027, with eligible Phase 1 completers offered the option to roll over. A larger, comparator-controlled Phase 2 in kidney diseases is planned for late 2027, subject to additional financing. The funding caveat attached to that later study is the most significant variable for investors tracking the company's near-term prospects.