Bildnachweis: Topadur/ ReadOn Therapeutics.
DEBRA Research gGmbH has invested in Swiss biopharmaceutical company Topadur Pharma AG to support the preclinical development of small-molecule therapies for dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. The programme will investigate Topadur’s clinical-stage compound TOP-N53 and other molecules from its proprietary DualTOP® platform, targeting impaired wound healing and fibrosis.
The studies will be conducted in collaboration with the Medical Center – University of Freiburg in Germany and EB Research Institute GmbH in Salzburg, Austria. DEB is characterised by extremely fragile skin, chronic wounds and progressive scarring, which can cause severe functional impairment. “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.”
Wound-healing activity in preclinical models
TOP-N53 has demonstrated wound-healing activity in preclinical models and increased local skin perfusion in a first-in-human Phase 1 study, in which it was well tolerated. The compound is currently in Phase 2a development for digital ulcers associated with systemic sclerosis and has received Orphan Drug Designation from both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Other compounds in the DualTOP® platform have shown anti-fibrotic activity in preclinical models. The new programme will investigate these complementary properties in models relevant to DEB. “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,” said PD Dr. Matthias Schäfer, CEO of Topadur Pharma AG.
