One04 Therapeutics Launches on Encouraging CPV-104 Phase 1 Interim Data in C3 Glomerulopathy
核心洞察
One04 Therapeutics (搜索) launched as a clinical-stage biopharmaceutical company based on encouraging Phase 1 interim results for CPV-104, a first-in-class recombinant human Factor H (搜索) candidate.
Interim data from the multiple-ascending dose portion showed favorable safety, tolerability and pharmacokinetics alongside early efficacy trends in patients with C3 glomerulopathy (搜索).
The company plans an open-label Phase 2 trial in up to 24 patients with C3G and other complement-mediated kidney diseases, plus a Phase 1 study in dry AMD/GA, both starting in early 2027.
One04 Therapeutics (搜索) GmbH & Co. KG announced its launch as a clinical-stage biopharmaceutical company on September 15, 2026, in Freiburg, Germany, on the strength of encouraging interim Phase 1 results for CPV-104, a first-in-class, full-length recombinant human Factor H (搜索) candidate with disease-modifying potential across a broad range of complement-mediated diseases.
The company said top-line interim results from the first two cohorts of the multiple-ascending dose (MAD) portion of the ongoing Phase 1 trial show favorable safety, tolerability and pharmacokinetics for CPV-104, alongside early efficacy trends in patients with C3 glomerulopathy (搜索) (C3G). The MAD portion includes three dose cohorts totaling 18 patients, one of which is ongoing.
"The encouraging interim Phase 1 data underscore the potential of CPV-104 as a differentiated therapeutic candidate in C3G, an indication with significant unmet need," said Erich Rajkovic, Ph.D., MBA, Chief Business Officer of One04 Therapeutics (搜索). "We believe this potential extends to a broader range of complement-mediated diseases of the kidney and eye, supported by CPV-104's differentiated approach to restoring endogenous complement regulation from pro-inflammatory toward immune homeostasis."
A Differentiated Mechanism: Restoring Rather Than Inhibiting Complement Regulation
According to the company, CPV-104 differs from current approaches that inhibit individual components of the complement cascade. Instead, the candidate is designed to restore the system's endogenous regulation through Factor H (搜索), an approach intended to control pathological complement overactivation while preserving broader complement function important for homeostasis, host protection and immune defense. One04 stated that preclinical and early interim clinical findings support this therapeutic rationale, which will be further investigated in continued clinical development.
Factor H (搜索) has been widely researched and has broad potential to treat complement-mediated diseases of the kidney and eye, the company noted, adding that Factor H is known to restore endogenous complement regulation from pro-inflammatory toward immune homeostasis, suggesting CPV-104 has disease-modifying potential.
Phase 1 Design and Upcoming Data Disclosure
The placebo-controlled single-ascending dose (SAD) portion of the Phase 1 program was completed in October 2025 and evaluated CPV-104 across four dose cohorts in 21 healthy volunteers, with no safety concerns observed.
Detailed interim Phase 1 data in patients with C3G will be presented in greater detail via livestream on October 26, 2026, following One04 Therapeutics (搜索)' attendance at the American Society of Nephrology (ASN) Kidney Week conference. CPV-104 has EU Orphan Designation for C3G.
Development Plans Spanning Kidney and Eye Indications
One04 Therapeutics (搜索) will initiate an open-label Phase 2 study in early 2027 to evaluate CPV-104 in up to 24 patients with C3G and selected other complement-mediated kidney diseases. Eligible patients who completed the Phase 1 C3G study may be offered the opportunity to continue treatment in the Phase 2 study.
The company also plans to initiate an open-label Phase 1 study of CPV-104 in dry age-related macular degeneration (搜索) (AMD) and its advanced form, geographic atrophy (搜索) (GA), in early 2027. A subsequent, larger, open-label, comparator-controlled Phase 2 study in complement-mediated kidney diseases is planned for late 2027, subject to additional financing.
"CPV-104 is a pipeline-in-a-product that has matured into a highly promising clinical candidate for complement-mediated diseases," said Björn Cochlovius, Ph.D., Chief Executive Officer of One04 Therapeutics (搜索). "The formation of One04 provides the dedicated structure and funding needed to accelerate its development. Our current funding enables us to reach multiple value inflection points, including an initial Phase 2 readout in complement-mediated kidney disease and Phase 1 data in the much larger indication of dry AMD/GA."
Funding and Origins of the Asset
One04 Therapeutics (搜索) has received undisclosed funding from D11Z.Ventures (搜索), a European venture capital fund specializing in early-stage investments. The company said it is fully funded to key value inflection points, with data from two CPV-104 clinical trials both starting in early 2027.
CPV-104 was discovered and developed using Eleva Biologics (搜索)' moss-based Bryotechnology production platform. The asset was transferred to One04 Therapeutics (搜索) to facilitate optimal operational focus and strategic independence for both companies.