Promising clinical trial results for therapy against rare „butterfly disease.“

A successfully completed clinical trial by RHEACELL, a company specializing in stem cell therapies, now promises significant progress in the treatment of this disorder. The multi-center international trial, approved by the U.S. Food and Drug Administration (FDA) and other relevant national and international regulatory authorities and granted orphan drug designation, involved patients from the United States, the UK, Austria, France, Italy and Germany.

The objective of this Phase IIa trial was to investigate the efficacy and safety of ABCB5-positive mesenchymal stem cells (ABCB5+ MSCs) in patients with recessive dystrophic epidermolysis bullosa (RDEB).

ABCB5+ stem cell therapy, classified by the European Medicines Agency (EMA) as an Advanced Therapy Medicinal Product (ATMP), was capable of measurably reducing disease activity based on its systemic anti-inflammatory mechanisms. Through promoting local wound healing, it improved the general health condition of treated patients.

Following demonstration of efficacy and safety of the ATMP in the now concluded Phase IIa trial, the data obtained will be used for further clinical development of the drug, with next phase clinical trials currently in preparation. Should interim results prove sufficiently convincing, RHEACELL would be positioned to potentially also apply for early market authorization by EMA.

The starting material for generating ABCB5+ MSCs in high numbers is allogeneic human donor skin. These stem cells are manufactured by TICEBA GmbH, Heidelberg, in a patented process. Using this method, highly purified stem cells can be manufactured in large numbers, reliably isolated and finally produced as a highly purified, homogeneous drug substance [highly functional manufactured stem cells (H.F.M stem cells)]. These ABCB5+ MSCs are classified as an ATMP and are manufactured under good manufacturing practice (GMP) in accordance with §13 paragraph 1 of the German Medicinal Products Act (AMG).