CAMBRIDGE, Mass., June 27, 2016 /PRNewswire/ -- Tarix Orphan LLC today announced that the company's lead compound, TXA127, has shown positive preclinical results in the C7-hypomorphic mouse model of recessive dystrophic epidermolysis bullosa (DEB), a rare and often severe genetic skin disorder.
Researchers in the laboratory of Dr. Leena Bruckner-Tuderman at the University of Freiburg, Germany showed that four weeks of daily subcutaneous administration of TXA127, a pharmaceutical formulation of the natural Angiotensin (1‐7) peptide, substantially reduced the fusion of digits, a symptom analogous to mitten deformity common in patients with DEB. In addition, immunohistology showed that TXA127 significantly reduced tenascin c, a marker of dermal fibrosis.
"These are very interesting results from two perspectives," said Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC. "First, DEB is a devastating disease with no currently approved therapies and treatment limited to supportive care. These results suggest that TXA127 may be able to affect the progression of the disease.
"The second important conclusion from this work," he continued, "is that it supports a mechanism of action for our compound that is involved in other serious orphan diseases. TXA127 interferes with the TGF beta pathway, which is involved in the pathophysiology of DEB, Marfan Syndrome, and muscular dystrophy."
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