RHEACELL, a biopharmaceutical company specializing in stem cell therapies, has received approval from the European Medicines Agency (EMA) to proceed with a Phase 3 clinical trial evaluating allo-APZ2-CVU for the treatment of chronic venous ulcers (CVU). This decision follows a similar approval from the FDA and is based on promising results from a Phase 2b study, marking a significant step forward in addressing this challenging condition.
The pivotal, double-blind, multicenter, international Phase III clinical trial (NCT06489028) is designed to assess the efficacy and safety of allo-APZ2-CVU in promoting wound healing in patients with therapy-resistant, non-healing CVU. The trial is set to enroll 250 patients across more than 100 sites, with enrollment expected to begin in Europe in the first quarter of 2025.
Trial Design and Endpoints
The primary endpoint of the study is wound closure. Secondary endpoints include time to wound closure, duration of wound closure, and improvements in patient quality of life. This comprehensive approach aims to provide a thorough evaluation of the treatment's benefits.
allo-APZ2-CVU Mechanism of Action
allo-APZ2-CVU utilizes ABCB5-positive mesenchymal stem cells, which possess unique immunomodulatory properties. These cells can release interleukin (IL)-1RA, a molecule that inhibits the release of IL-1β by binding to the IL-1 receptor. This action dampens the excessive immune response, shifting the macrophage phenotype from a pro-inflammatory M1 to a wound-healing M2 phenotype, thereby supporting the body's natural healing processes.
The Burden of Chronic Venous Ulcers
Chronic venous ulcers (CVUs) arise from impaired venous drainage in the lower extremities, often due to venous reflux or leg vein obstructions. This leads to increased venous pressure, microcirculatory disturbances, and ultimately, tissue breakdown and painful wounds. In chronic wounds, the healing process is hindered by a persistent inflammatory state. In Germany, the prevalence of chronic ulcers is approximately 0.7% of the population. CVUs significantly diminish patients' quality of life due to costly and repeated wound treatments and associated pain. Currently, there are limited effective treatment options, particularly for patients with refractory disease.
RHEACELL's Vision
"Our goal is to provide innovative therapeutic options for hard-to-treat immune and inflammatory diseases such as refractory CVU and EB," stated Dr. Christoph Ganss, founder and CEO of RHEACELL. "The positive decision of the EMA follows the FDA and is a further validation of the potential of our drug allo-APZ2-CVU. It allows us to initiate our study early next year with the goal of quickly reaching the market and providing meaningful help to patients in need."
