Theravia licenses experimental molecule with potential to treat EB
DCN-tCRK led to significant improvements in survival in mouse study
France-based pharma company Theravia has obtained rights to an experimental molecule that may be used to treat epidermolysis bullosa (EB).
The molecule, known as DCN-tCRK, was discovered by scientists at Tampere University, in Finland. Under the new licensing agreement, Theravia will be in charge of further drug development and conducting clinical trials to test the investigational treatment.
Potential EB treatment result of 20 years of research
“I am proud that the molecule we discovered has been licensed,” Tero Järvinen, MD, PhD, a professor at Tampere University who helped develop the molecule, said in a press release. “This is the result of 20 years of systematic research efforts. We hope the product will eventually help treat EB, which can be fatal in infancy. Without Theravia, we could not have progressed any further in the drug development using only our academic resources.”
Theravia was one of several companies vying to license the new molecule. Ultimately, it was chosen as the company with the best chances of developing the therapy through to potential commercialization.
“We are eager to collaborate with academics who share our dedication to transforming the landscape of rare diseases,” said Franck Hamalian, CEO of Theravia, “and we are deeply grateful to Tampere University for entrusting us on this project. By leveraging our expertise, we aim to make this new treatment widely accessible to EB patients.”
EB encompasses a group of disorders that cause the skin to be unusually fragile, making it prone to wounds and blisters. A signaling molecule called TGF-beta (transforming growth factor-beta) contributes to inflammation and scarring that drives skin damage in EB.
DCN-tCRK combines a homing peptide — a small chain of amino acids, the building blocks of proteins, that directs the molecule to travel to the skin — with a protein called decorin, which acts as a natural inhibitor of TGF-beta. The therapy essentially works to block the activity of TGF-beta in the skin to reduce inflammation and promote wound healing.
In a preclinical study, treatment with DCN-tCRK led to significant improvements in survival in a mouse model of recessive dystrophic epidermolysis bullosa, one of the more severe forms of the disease.
“Pre-clinical studies have shown that our drug molecule is more effective than any previously tested molecule for this rare skin disorder,” Järvinen said.
Based on the promising preclinical data, both the U.S. Food and Drug Administration and the European Medicines Agency have granted orphan drug and rare pediatric disease designations to DCN-tCRK. These designations provide extra incentives to companies that are developing medicines to treat rare diseases.
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