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– FDA approved blinded, multi-centric Phase-3 trial (NCT06489028) to investigate the safety and efficacy of allo-APZ2-CVU in patients with CVU
– The study will be conducted at more than 100 sites and is expected to enroll 250 patients
– Allo-APZ2-CVU is a first-in-class stem cell therapy leveraging ABCB5-positive mesenchymal stem cells to treat patients with severe immune and inflammation-driven diseases with high unmet medical need
Heidelberg, Germany, 24.09.2024 RHEACELL today announced that the U.S. Food and Drug Administration (FDA) approved the companies blinded, multi-centric Phase-3 trial (NCT06489028) to investigate allo-APZ2-CVU in patients with chronic venous ulcers (CVU). Allo-APZ2-CVU is a first-in-class stem cell therapy leveraging ABCB5-positive mesenchymal stem cells (MSCs) to treat patients with severe immune and inflammation-driven diseases with high unmet medical need, including CVU and Epidermolysis bullosa.
The FDA approval is based on initial positive results from a Phase 2b study (NCT04971161) evaluating allo-APZ2-CVU in patients with CVU refractory to standard of care and a national approval in Germany by the Paul Ehrlich Institute (PEI) for treatment-refractory CVU. The Phase 2 study demonstrated a favorable safety profile and preliminary efficacy of allo-APZ2-CVU. At a planned interim analysis, an unblinded Independent Data Monitoring Committee (IDMC) recommended continuation of the study without modification, indicating that the selected dose group for allo-APZ2-CVU was able 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).
“Once patients become refractory to standard of care for CVU, there are no curative treatments available, and wounds often persist for years, considerably reducing patients’ quality of life. The approval of our Phase 3 trial with allo-APZ2-CVU is an important step forward in providing these patients with urgently needed treatment options,” said Dr. Christoph Ganss, founder and CEO of RHEACELL. “We believe that our first-in-class technology has the potential to address several serious immune and inflammatory diseases, and that this approval further highlights the potential of our ABCB5+ MSCs.”
The pivotal, double-blind, multicenter, international Phase III clinical trial is designed to evaluate the efficacy and safety of allo-APZ2-CVU in wound healing of therapy-resistant non-healing CVU. The study will be conducted at more than 100 sites and is expected to enroll 250 patients. The primary endpoint of the study is wound closure. Secondary endpoints include among others time to wound closure, duration of wound closure and quality of life.
About allo-APZ2-CVU-III
The pivotal, double-blind, placebo-controlled, multicenter, international Phase III clinical trial, allo-APZ2-CVU-III (NCT06489028), aims to investigate the efficacy and safety of allo-APZ2-CVU in wound healing of therapy-resistant non-healing CVU. The trial follows initial positive results and a favorable safety profile from the previous Phase 2b study, allo-APZ2-CVU-II (NCT04971161), with allo-APZ2-CVU in CVU. The trial will be conducted at more than 100 sites in 12 countries, including the US, Europe and the UK.
About ABCB5+ mesenchymal stem cells
RHEACELL’s investigational programs leverage ABCB5-positive mesenchymal stem cells isolated from the skin to treat immune- and inflammation-driven diseases.