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Tarix Orphan

Tarix Orphan, LLC Announces Patent Issuance for TXA127 in Treatment of Marfan Syndrome

TXA127 Shows Positive Activity in Fibrilin Model of Marfan Syndrome

Cambridge, MA (August 18, 2015): Tarix Orphan LLC, a privately held biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases, today announced that the U.S. Patent and Trademark Office (USPTO) has issued a patent covering the use of TXA127 in the treatment of Marfan Syndrome. The patent, No. 9,107,870, encompasses the use of “Angiotensin Peptides in Treating Marfan Syndrome and Related Disorders.” 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.

“The issuance of this U.S. patent strengthens our IP position around TXA127 as an important candidate for the treatment of a number of neuromuscular and connective tissue diseases where there remains significant, unmet medical need,” said Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC. “TXA127 has recently shown activity in preclinical research using the fibrillin‐1 model of Marfan Syndrome, where the peptide significantly reduced aortic growth in Marfan mice compared to control. We look forward to continuing our development of TXA127 in Marfan Syndrome and other indications.”

Marfan Syndrome is a systemic connective tissue disorder caused by mutations in the gene encoding fibrillin‐1 (FBN1), which result in an increase in TGF‐beta that affects connective tissue throughout the body. The major cause of mortality in Marfan Syndrome is aortic enlargement, dissection and rupture. Previous research has shown that reducing TGF‐beta or inhibiting the angiotension II type 1 receptor (AT1R) can prevent increased aortic root growth in Marfan mice. TXA127 is part of an alternative pathway of the reninangiotensin system, which directly opposes the action of the ATR1, and the peptide is also known to protect chronically damaged tissues from the harmful effects of ATR1 through mediation of TGF‐beta signaling. Tarix research suggests that TXA127 achieves its rescue effects on aortic root growth in Marfan mice by selective upregulation of the known endogenous TGF‐beta inhibitor, SKIL.

About Tarix Orphan
Tarix Orphan LLC is a private biopharmaceutical company focused on the development of 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), Marfan syndrome, and ALS. 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.

Contact:
Elizabeth Wagner, Vice President
Tarix Orphan
elizabeth.wagner@tarixpharma.com

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