CAMBRIDGE, Mass., Sept. 7, 2016 /PRNewswire/ -- Tarix Orphan LLC today announced that the company has received Orphan Drug Status from the U.S. Food and Drug Administration for TXA127 as a potential treatment for the rare genetic skin disorder Recessive Dystrophic Epidermolysis Bullosa (RDEB). The company also announced with DEBRA Austria a research collaboration aimed at furthering the development of TXA127 as a potential treatment for RDEB. Under the agreement, DEBRA will fund a preclinical study of TXA127 that is designed to definitively show the efficacy of TXA127 in a mouse model of RDEB.
TXA127, a pharmaceutical formulation of the natural Angiotensin (1-7) peptide, interferes with the TGF beta pathway, which is involved in the pathophysiology of DEB as well as several other serious orphan diseases including Marfan Syndrome and muscular dystrophy. Previous preclinical studies with TXA127 have shown its ability to reduce the fusion of digits, a symptom in RDEB mice that is analogous to mitten deformity common in patients with severe RDEB.
Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC, said, "We are very pleased to have the support of DEBRA Austria, a member of DEBRA International, the worldwide network of groups working on behalf of patients with all forms of EB to further understand and provide preclinical proof of concept for TXA127 as a useful treatment for this condition. We are also very pleased that FDA has recognized the potential of TXA127 in this indication by its granting of Orphan Drug Status for RDEB."
"RDEB is a devastating disease with no currently approved therapies and treatment limited to supportive care," said Dr Rainer Riedl, CEO DEBRA Austria. "We are pleased to support this work with Tarix Orphan in hopes of demonstrating the potential of TXA127 as a means of slowing or halting the debilitating tissue damage that accrues with progression of RDEB. Our commitment to research is to develop not only treatments that address the underlying genetic causes of EB but, equally, treatments that address the incapacitating consequences: reducing scarring and contractures that result from chronic wounds would greatly improve quality of life for people with RDEB. This study to be undertaken by the research group led by Prof Leena Bruckner-Tuderman at the University of Freiburg will continue our support for their valuable work combatting fibrosis in EB, and we are delighted to do so."
Following completion of this DEBRA-funded preclinical study, Tarix Orphan's goal will be to test the drug in clinical trials of RDEB.
Recessive dystrophic epidermolysis bullosa (RDEB) is one of the more severe subtypes of EB, a group of rare genetic connective tissue-fragility disorders, characterized by extremely fragile skin and mucosa, and recurrent blister formation, resulting from minor mechanical friction or trauma.
The skin has two layers; the outer layer is called the epidermis and the inner layer the dermis. Normally, there are protein 'anchors' between the two layers that prevent them from separating from one another. In people with RDEB, the two skin layers lack the anchors that hold them together, and any action that creates friction between the layers (like rubbing or pressure) will create blisters and painful sores. People with severe RDEB have compared the sores to third-degree burns.
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 Orphan Drug Status
Orphan status is granted by the FDA to promote the development of products that demonstrate promise for the treatment of rare diseases affecting fewer than 200,000 Americans annually. Orphan drug designation entitles Tarix Orphan to a seven-year period of marketing exclusivity in the United States for TXA127 if it is approved by the FDA for the treatment of RDEB or any of the several other indications where FDA has granted this designation to TXA127, and it enables the company to apply for research funding, tax credits for certain research expenses, and a waiver from the FDA's application user fee.
About DEBRA Austria and DEBRA International
DEBRA Austria is a member of DEBRA International, the worldwide alliance of national DEBRA Epidermolysis Bullosa (EB) patient support groups, working in over 50 countries and growing. Our aims are to support people with EB and their organizations to work together to maximize quality of life, and to promote the development of effective treatments for the condition as quickly as possible through research. For more information about EB, and DEBRA's research support, please visit http://www.debra-international.org
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). 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, LGMD, and RDEB in the United States, and for DMD in Europe. For more information on Tarix Orphan, please visit our website at www.tarixorphan.com.
Elizabeth Wagner, COO
SOURCE Tarix Orphan LLC