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Tarix Orphan

Tarix Orphan, LLC Announces Positive Results for TXA127 in Model of Limb Girdle Muscular Dystrophy

Cambridge, MA (October 27, 2014): Tarix Orphan LLC, a privately held biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases, today announced that the company’s lead product candidate, TXA127, has achieved positive results in an established mouse model of Limb Girdle Muscular Dystrophy.

SGCD‐/‐ (sarcoglycan delta deficient) mice demonstrate reduced locomotor activity and a dystrophic phenotype in skeletal muscles at a young age. The Tarix researchers showed that such mice treated daily for eight weeks with TXA127 via infusion by osmotic minipump exhibited a marked increase in activity compared to control animals. Moreover quadriceps muscles of the treated SGCD‐/‐ mice exhibit markedly reduced fibrosis in their skeletal muscle. In addition, older mice that began treatment at age 45‐50 weeks showed improvements in cardiac function, compared to controls.

“These results further support TXA127’s potential as a treatment for many forms of muscular dystrophy, as well as other neuromuscular and connective tissue diseases for which few therapies exist today,” said Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC. “We look forward to continuing the development of this product candidate in muscular dystrophy.”

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 DMD. Additional diseases which may benefit from treatment with TXA127 include congenital muscular dystrophies, Marfan Syndrome, and amyotrophic lateral sclerosis (ALS). TXA127 is part of the “alternative renin angiotensin system (RAS)” and counteracts the “classical” RAS, which promotes hypertension, fibrosis, hypertrophy and inflammation.

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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