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• Met predefined primary and secondary objectives for the interim analysis
• CPV-104 was generally well tolerated in healthy volunteers and patients with C3 glomerulopathy (C3G)
• Exploratory analyses in patients with C3G showed encouraging early signals of clinical activity
• Expert panel to join One04 management in a virtual key opinion leader (KOL) event to discuss the data on October 26, 2026, at 5pm CET (4pm GMT, 12pm ET)

FREIBURG, Germany, Oct. 22, 2026 (GLOBE NEWSWIRE) -- One04 Therapeutics GmbH & Co. KG, a clinical-stage biopharmaceutical company developing CPV-104, a first-in-class, recombinant, full-length human Factor H therapy, today reported positive interim data from the ongoing Phase 1 study of CPV-104 in patients with C3 glomerulopathy (C3G). The data are being presented in a late-breaking poster at the American Society of Nephrology (ASN) Kidney Week 2026, taking place October 21-25 in Denver, Colorado.

The Phase 1 study evaluated single ascending doses (SAD) of CPV-104 in healthy volunteers and continues to evaluate multiple ascending doses (MAD) in patients with C3G. The interim analysis, after two of the planned three MAD cohorts, met the primary safety objective and secondary objectives of tolerability, pharmacokinetics (PK) and immunogenicity. Exploratory endpoints in the MAD portion showed early signals of biological activity, supporting continued evaluation.

“I am encouraged by the safety and tolerability observations to date and by the early findings on kidney function in patients with C3G,” said Prof. Michael Wiesener, MD, Senior Physician at University Hospital Erlangen and presenting author of the poster. “This is an early Phase 1 study with a small number of patients and a short treatment period, but these results support continued clinical evaluation of CPV-104.”

“Meeting primary and secondary study objectives and seeing an early signal of biological activity marks an important milestone for CPV-104,” said Björn Cochlovius, PhD, Chief Executive Officer of One04 Therapeutics. “We are excited to discuss the findings and next steps, and we invite clinical experts, investors and potential partners to join our virtual KOL event on Monday, October 26.”

Phase 1 Study Interim Results

Interim data presented at ASN Kidney Week showed CPV-104 was generally well tolerated at doses up to 15 mg/kg in the SAD portion and 7.5 mg/kg dosed weekly for four weeks in the MAD portion. No serious adverse drug reactions (ADRs), serious treatment-emergent adverse events (TEAEs), deaths or treatment discontinuations were reported. Dosing in the highest MAD cohort (15 mg/kg) is ongoing.

No anti-drug antibodies (ADAs) were detected in evaluable samples. Exposure to CPV-104 increased approximately in proportion to dose. The observed pharmacokinetic profile, including a terminal half-life of three to four days and modest accumulation with weekly dosing, supports further evaluation of a once-weekly regimen.

Exploratory analyses in the MAD portion showed signals of reduction in proteinuria at 7.5 mg/kg with stable estimated glomerular filtration rate (eGFR). These preliminary findings clearly encourage continued evaluation of CPV-104 as a potentially disease-modifying therapy for C3G.

ASN Kidney Week presentation details
Title: First-in-human clinical trial of CPV-104, a recombinant version of human complement Factor H: safety, tolerability, and pharmacokinetics for C3 glomerulopathy
Presenting author: Prof. Dr. Michael Wiesener, Department of Nephrology and Hypertension, University Hospital Erlangen, Germany
Poster number: TH-PO1261
Date and time: October 22, 2026, from 10:00am to 12:00pm MDT

Virtual KOL event details
Title: CPV-104 Key Data Release
Company presenters:
Björn Cochlovius, PhD, Chief Executive Officer
Erich Rajkovic, PhD, Chief Business Officer
KOL presenters:
Marc Hilhorst, MD, PhD, Department of Nephrology and Renal Transplantation, Amsterdam University Medical Center
Prof. Dr. Bernd Jilma, MD, Department of Clinical Pharmacology, Medical University of Vienna
Dinesh Khullar, MD, Chairman, Department of Nephrology and Renal Transplant Medicine, Max Super Speciality Hospital, Saket
Prof. Michael Wiesener, MD, Department of Nephrology and Hypertension, University Hospital Erlangen
Prof. Dr. Peter Zipfel, PhD, Koania Complement Analytics GmbH

Moderator:
Dr. Sarah Jarvis, GP and Visiting Professor of General Practice, University of Huddersfield
Date and time: October 26, 2026, at 5pm CET (4pm GMT, 12pm ET)
Registration: https://www.q2q.co.uk/CPV-104-KeyData-Release/access

About the Phase 1 study of CPV-104
CPV-104 is being evaluated in a two-part Phase 1 study conducted at nine sites across nine European countries. The SAD part assessed single intravenous doses in 21 healthy volunteers across four dose cohorts (cohorts 1–4: 1, 3.75, 7.5 and 15 mg/kg); the first cohort was open-label, and the subsequent three cohorts were randomized, double-blind and placebo-controlled (4:2, CPV-104 to placebo). The MAD part is an open-label, single-arm evaluation of four weekly intravenous doses in patients with C3G, comprising three dose cohorts totaling 18 patients (cohorts 5–7: 3.75, 7.5 and 15 mg/kg); dosing of the highest dose cohort (cohort 7, 15 mg/kg) is currently ongoing. An independent safety review committee reviewed the data before each dose escalation and, in the MAD part, before the third and fourth doses.

ABOUT ONE04 THERAPEUTICS
One04 Therapeutics is a clinical-stage biopharmaceutical company developing CPV-104, a first-in-class, recombinant, full-length human Factor H therapy. Factor H has been widely researched and has broad potential to treat complement-mediated diseases of the kidney and eye. Early CPV-104 Phase 1 clinical data showed good initial safety, tolerability and early signals of biological activity in patients with a complement-mediated kidney disease. Factor H is known to restore endogenous complement regulation from pro-inflammatory toward immune homeostasis, suggesting CPV-104 has disease-modifying potential and should work well in combination with marketed therapies.

For regular updates about One04 Therapeutics, visit www.one04.com or follow us on LinkedIn.

MEDIA CONTACTS
Fabienne Zeitter
Director Corporate Communications
pr@one04.com
Phone: +49 172 6565 227

Valency Communications
Mario Brkulj
mbrkulj@valencycomms.eu
Phone: +49 160 9352 9951

INVESTOR CONTACT

Cohesion Bureau Giovanni Ca’ Zorzi giovanni.cazorzi@cohesionbureau.com Phone: +33 7 84 67 07 27

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