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Tarix Orphan

Tarix Orphan, LLC Announces Positive Results with TXA127 in Animal Model of ALS

Noted ALS Researcher, Robert H. Brown, Jr., Ph.D., M.D., Joins Tarix Orphan Strategic Advisory Board

strong>Cambridge, MA (July 13, 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 company’s lead compound, TXA127, has shown positive results in the preclinical SOD1 model of amyotrophic lateral sclerosis (ALS). Researchers showed that ALS model mice treated daily for up to 8 weeks via osmotic pump with TXA127, a pharmaceutical formulation of the natural Ang 1‐7 peptide, exhibited a statistically significant increase in survival compared to control mice who received only the vehicle solution (164 days versus 134 days; p ≤ 0.0001). The TXA127‐treated animals also exhibited a strong benefit in quality of life/disease progression scores for 128.33 days versus 102.67 days for control animals, as measured by the number of animals with scores ≤ 2 (animals ambulatory but dragging hind foot).

Tarix Orphan also announced the appointment of Robert H. Brown, Jr., Ph.D., M.D. to the company’s Strategic Advisory Board. Dr. Brown, Chair of the Department of Neurology at the University of Massachusetts Medical School, is an expert on paralytic neuromuscular disorders, whose primary research focus since 1980 has been ALS. He is also the director and organizer of the ALS Therapy Alliance.

“We are encouraged by the activity that TXA127 continues to show in a variety of preclinical models of serious neuromuscular disorders including ALS, where current treatments are limited or on‐existing,” said Richard Franklin, President and Chief Executive Officer of Tarix Orphan LLC. “We are also very pleased that Dr. Brown, an internationally noted researcher in this field, has agreed to join our SAB and we look forward to benefiting from his insights and advice as we move forward with TXA127 development.”

About ALS
ALS is a progressive, neurodegenerative disease that affects nerve cells in the brain and spinal cord. The disease causes nerve cells to gradually break down and die, resulting in muscle weakness and decreased physical function over time. Eventually, ALS can affect the patient’s ability to control the muscles needed to move, speak, eat and breathe. The cause of ALS is mostly unknown, although 5‐10 percent of cases are inherited. There is currently no cure for the disease. Current treatments focus on slowing the progression of symptoms, preventing unnecessary complications and making patients more comfortable and independent.

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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