Results Contribute to Understanding of Mechanism of Action Relevant to a Number of Orphan Diseases Including Marfan Syndrome and Duchenne Muscular Dystrophy
CAMBRIDGE, MA, USA I June 27, 2016 I 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.”
Tarix Orphan intends to investigate TXA127 further in pre-clinical models of DEB with the ultimate goal of testing the drug in clinical trials.
About TXA127
TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide Angiotensin (1‐7) which Tarix Orphan is developing for the treatment of a number of orphan and genetic diseases, with an initial focus on Duchenne muscular dystrophy (DMD). TXA127 has orphan and Fast Track designations and is expected to enter Phase II in DMD in early 2017.
About Tarix Orphan
Tarix Orphan LLC is a private biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases. The company’s lead drug candidate is TXA127, a pharmaceutical formulation of the naturally occurring peptide Angiotensin (1-7), for the treatment of rare neuromuscular and connective tissue diseases. TX127 has shown therapeutic activity in animal models of Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophy (LGMD), congenital muscular dystrophy (MDC1A), and Marfan syndrome. Tarix Orphan has broad IP protection for TXA127, and Orphan Drug Designations (ODDs) have been granted for DMD and LGMD in the United States, and for DMD in Europe. For more information on Tarix Orphan, please visit our website at www.tarixorphan.com.
SOURCE: Tarix Orphan
Post Views: 27
Results Contribute to Understanding of Mechanism of Action Relevant to a Number of Orphan Diseases Including Marfan Syndrome and Duchenne Muscular Dystrophy
CAMBRIDGE, MA, USA I June 27, 2016 I 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.”
Tarix Orphan intends to investigate TXA127 further in pre-clinical models of DEB with the ultimate goal of testing the drug in clinical trials.
About TXA127
TXA127 is a pharmaceutical grade formulation of the naturally occurring peptide Angiotensin (1‐7) which Tarix Orphan is developing for the treatment of a number of orphan and genetic diseases, with an initial focus on Duchenne muscular dystrophy (DMD). TXA127 has orphan and Fast Track designations and is expected to enter Phase II in DMD in early 2017.
About Tarix Orphan
Tarix Orphan LLC is a private biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases. The company’s lead drug candidate is TXA127, a pharmaceutical formulation of the naturally occurring peptide Angiotensin (1-7), for the treatment of rare neuromuscular and connective tissue diseases. TX127 has shown therapeutic activity in animal models of Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophy (LGMD), congenital muscular dystrophy (MDC1A), and Marfan syndrome. Tarix Orphan has broad IP protection for TXA127, and Orphan Drug Designations (ODDs) have been granted for DMD and LGMD in the United States, and for DMD in Europe. For more information on Tarix Orphan, please visit our website at www.tarixorphan.com.
SOURCE: Tarix Orphan
Post Views: 27
