DEBRA Research gGmbH, a global non-profit organization dedicated to advancing research and drug development for Epidermolysis bullosa (EB), and Topadur Pharma AG, a Swiss clinical-stage biopharmaceutical company, today announced an investment by DEBRA Research in Topadur to support a preclinical program exploring a new treatment approach for dystrophic Epidermolysis bullosa (DEB). DEB is one of the more severe forms of EB, characterized by extremely fragile skin and progressive, debilitating complications.
The program will evaluate Topadur’s clinical-stage compound TOP-N53 together with additional selected small molecules from its proprietary DualTOP® platform. The compounds will be investigated in in vitro and in vivo models addressing two major and interconnected challenges in DEB: impaired wound healing and fibrosis. The studies will be conducted in collaboration with the Medical Center – University of Freiburg, Germany, and EB Research Institute GmbH in Salzburg, Austria.
“People living with dystrophic EB face severe complications that extend far beyond chronic wounds,” said Prof. Hubert Trübel, MD, MBA, Managing Director of ReadOn Therapeutics, a DEBRA Research spinout. “Through our investment in Topadur, we are supporting the development of locally acting therapies that target several interconnected aspects of the disease. We see this as an important step toward exploring new approaches that go beyond wound management alone.”
DEBRA Research brings deep expertise in EB and a strong understanding of the needs of people living with this devastating disease. Their support as an investor and partner in this program is an important endorsement of our approach and underscores the broader potential of our DualTOP® platform in indications with significant unmet medical needs.”
PD Dr. Matthias Schäfer, CEO, Topadur Pharma AG
EB comprises a group of rare genetic conditions characterized by extremely fragile skin that is prone to blistering and wounds, even from minor friction or trauma. In DEB, mutations in the COL7A1 gene impair collagen VII, resulting in recurrent skin lesions within the dermis. Repeated cycles of injury and repair can lead to chronic wounds, progressive fibrosis, scarring, and severe functional impairment. Despite recent advances in wound treatment, there is still a significant need for therapies addressing these interconnected disease processes.
Topadur’s DualTOP® platform comprises locally acting small molecules designed to modulate complementary signaling pathways involved in tissue repair and disease progression. TOP-N53, Topadur’s lead asset, has demonstrated wound-healing activity in preclinical models and increased local skin perfusion in a first-in-human Phase 1 study, where it was well tolerated. It is currently in Phase 2a clinical development for digital ulcers in systemic sclerosis and has received Orphan Drug Designation from both the FDA and EMA. Across the broader DualTOP® platform, Topadur compounds have also demonstrated anti-fibrotic activity in preclinical models. The DEB program will investigate these complementary properties in disease-relevant models.
