RHEACELL GmbH & Co. KG has announced that the U.S. Food and Drug Administration (FDA) has approved its Phase 3 clinical trial (NCT06489028) for allo-APZ2-CVU in patients with refractory chronic venous ulcers (CVU). This pivotal, double-blind, multi-center study will evaluate the safety and efficacy of allo-APZ2-CVU in promoting wound healing for patients with therapy-resistant, non-healing CVU. The trial is set to enroll 250 patients across more than 100 sites internationally.
Allo-APZ2-CVU: A Novel Stem Cell Therapy
Allo-APZ2-CVU represents a first-in-class stem cell therapy that utilizes ABCB5-positive mesenchymal stem cells (MSCs) to address severe immune and inflammation-driven diseases. These MSCs possess unique immunomodulatory properties, including the ability to release interleukin-1 receptor antagonist (IL-1RA), which inhibits the release of IL-1β and dampens excessive immune responses. This mechanism promotes a shift from a pro-inflammatory M1 macrophage phenotype to a wound-healing M2 phenotype, facilitating the body's natural healing processes.
Clinical Trial Design and Endpoints
The Phase 3 trial is designed as a double-blind, placebo-controlled, multi-center, international study. The primary endpoint is wound closure, with secondary endpoints including time to wound closure, duration of wound closure, and quality of life improvements. The trial builds upon positive initial results from a Phase 2b study (NCT04971161), which demonstrated a favorable safety profile and preliminary efficacy of allo-APZ2-CVU in CVU patients refractory to standard of care. An interim analysis by an Independent Data Monitoring Committee (IDMC) recommended continuation of the study without modification, based on the potential for allo-APZ2-CVU to meet or exceed the presumed effect size compared to placebo (initial assumption: 15% wound closure in the placebo arm vs. 45% wound closure in the dose arm).
Addressing Unmet Needs in CVU Treatment
Chronic venous ulcers (CVUs) are a consequence of impaired venous drainage in the lower extremities, leading to microcirculatory disturbances and tissue breakdown. These ulcers often persist for years, significantly reducing patients' quality of life. According to RHEACELL, once patients become refractory to standard of care for CVU, there are no curative treatments available. The prevalence of chronic ulcers in Germany is reported to be 0.7% of the population. Allo-APZ2-CVU offers a potential new treatment option for these patients with limited alternatives.
RHEACELL's Focus on Innovative Stem Cell Therapies
RHEACELL is a clinical late-stage biopharmaceutical company based in Heidelberg, Germany, specializing in innovative stem cell therapies for severe inflammation-driven diseases with high unmet medical needs. Their investigational programs leverage ABCB5-positive mesenchymal stem cells isolated from the skin. The company believes that their ABCB5+ MSCs have the potential to significantly improve the lives of patients suffering from conditions like Epidermolysis Bullosa.
