One04 Therapeutics to present late-breaking interim Phase 1 results for CPV-104 in C3 glomerulopa...

Highlights

Press release from One04 Therapeutics

First clinical data for CPV-104 in patients, including interim safety, tolerability and pharmacokinetics, accepted as a late-breaking poster
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)
Registration now open for the virtual KOL event

Freiburg, Germany, October 6, 2026 – 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 announced the acceptance of a late-breaking poster presenting positive interim data from the ongoing Phase 1 study of CPV-104 at the American Society of Nephrology (ASN) Kidney Week, taking place October 21-25, 2026, in Denver, Colorado.

In the poster session, Prof. Dr. Michael Wiesener, Department of Nephrology and Hypertension, University Hospital Erlangen, will present an interim analysis of safety, tolerability and pharmacokinetics (PK) from the first two cohorts of patients with C3 glomerulopathy (C3G) in the multiple-ascending dose (MAD) portion of the study.

Following ASN Kidney Week, Dr. Wiesener will join One04 management and an expert panel in a virtual key opinion leader (KOL) event on October 26, 2026, to discuss the safety, tolerability and PK data and encouraging early signals of clinical activity in the ongoing Phase 1 trial in more detail. Management will also present future development plans for CPV-104 in complement-mediated kidney and retinal diseases.

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 10:00 AM to 12:00 PM MDT

Virtual KOL event details

Title: Discussion of positive interim Phase 1 data for CPV-104 in patients with C3G
Company presenters:
Dr. Björn Cochlovius, Chief Executive Officer
Dr. Erich Rajkovic, MBA, Chief Business Officer
KOL presenters:
Dr. Marc Hilhorst, Department of Nephrology and Renal Transplantation, Amsterdam University Medical Center
Prof. Dr. Bernd Jilma, Department of Clinical Pharmacology, Medical University of Vienna
Dr. Dinesh Khullar, Chairman, Department of Nephrology and Renal Transplant Medicine, Max Super Speciality Hospital, Saket
Prof. Dr. Michael Wiesener, Department of Nephrology and Hypertension, University Hospital Erlangen
Prof. Dr. Peter Zipfel, Koania Complement Analytics GmbH

Moderator:

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

The placebo-controlled single-ascending dose (SAD) study arm evaluated CPV-104 across four dose cohorts in 21 healthy volunteers, with no dose-limiting toxicities or treatment-related serious adverse events observed. The subsequent MAD portion of the Phase 1 trial includes three dose cohorts totaling 18 patients. Dosing of the highest dose cohort is currently ongoing.

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
[email protected]
Phone: +49 172 6565 227
Valency Communications
Mario Brkulj
[email protected]
Phone: +49 160 9352 9951

INVESTOR CONTACT

Cohesion Bureau
Giovanni Ca’ Zorzi
[email protected]
Phone: +33 7 84 67 07 27

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