One04 Therapeutics Will Present Phase 1 Trial Data
The biotech firm will share interim results for its C3 glomerulopathy treatment at ASN Kidney Week in October.
Updated on Oct. 6, 2026 in Biotech
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Should medical researchers prioritize public disclosure of interim clinical trial results for rare diseases?
One04 Therapeutics will present Phase 1 clinical trial data for its Factor H therapy, CPV-104, on October 22, 2026. The presentation will occur during the ASN Kidney Week conference in Denver.
Why it matters
This interim analysis provides critical insights into the safety and efficacy of CPV-104, which is currently being developed to address complement-mediated kidney and retinal diseases.
The study evaluates CPV-104 across two primary cohorts, including 21 healthy volunteers in the single-ascending dose arm and 18 patients with C3 glomerulopathy in the multiple-ascending dose study.
The players
One04 Therapeutics
This biotechnology firm based in Freiburg, Germany, specializes in the development of Factor H therapies for various diseases.
Prof. Dr. Michael Wiesener
He is the lead investigator scheduled to present the interim Phase 1 trial findings for CPV-104 during the upcoming medical conference.
The details
The interim analysis for CPV-104 focuses on safety, tolerability, and pharmacokinetics in patients with C3 glomerulopathy. No dose-limiting toxicities were observed in the initial group of healthy volunteers, and dosing is currently proceeding for the highest dose cohort in the multi-dose study.
Timeline
October 21-25, 2026: ASN Kidney Week conference occurs in Denver.
October 22, 2026: One04 Therapeutics presents interim data at the conference.
October 26, 2026: The company will host a virtual key opinion leader event.
The Tech Race
This development reflects the broader trend of biotech companies advancing specialized Factor H therapies to replace or augment traditional treatments for complement-mediated diseases. It positions One04 Therapeutics within an increasingly competitive field of developers seeking to address rare kidney and retinal conditions.
For patients suffering from C3 glomerulopathy, this study represents a potential advancement in therapeutic options as the company moves through clinical trials. Further results will determine the timeline for the eventual availability of this novel treatment for those impacted by kidney disease.
The takeaway
This data release marks a pivotal step in the validation of Factor H-based therapies for rare conditions. Stakeholders should monitor the upcoming virtual event for additional clarity on the future development timeline for these specialized treatments.
What happens next
One04 Therapeutics will host a virtual key opinion leader event on October 26, 2026, to discuss the clinical trial data and future development plans.
Further reading
For more on emerging developments in the sector, visit the Biotech section.
More information
To register for the discussion, visit the virtual KOL event registration portal.
Live Poll
Should medical researchers prioritize public disclosure of interim clinical trial results for rare diseases?
