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Tarix Orphan Receives FDA Fast Track Designation for TXA127 as Potential Treatment for Duchenne Muscular Dystrophy
Date:10/6/2015

CAMBRIDGE, Mass., Oct. 6, 2015 /PRNewswire/ -- 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. Food and Drug Administration has granted Fast Track Designation to TXA127 (angiotensin 1-7) to reduce skeletal muscle damage and fibrosis and thereby improve muscle strength in Duchenne Muscular Dystrophy (DMD) patients. Tarix Orphan has received notice that clinical testing under the company's Investigational New Drug (IND) application for TXA127 may proceed, and the company expects to initiate a multi-site Phase 2 safety and efficacy study in patients with DMD in early 2016 at both U.S. and European trial sites. Tarix has previously received Orphan Drug Status for TXA127 in the DMD indication in both the United States and Europe.

"Studies with TXA127 have shown significant development potential in preclinical models of Duchenne Muscular Dystrophy, Limb Girdle Muscular Dystrophy, and congenital muscular dystrophy, MDC1A and other conditions associated with the TGF-beta pathway. This peptide has several biological actions and has shown positive effects in animals including reductions in muscle fibrosis, increases in muscle strength and ambulation in affected animals, as well as normalization of cardiac dysfunction," said Rick Franklin, Tarix Orphan Chief Executive Officer. "We look forward to beginning our multi-site international Phase 2 study in DMD patients in early 2016. We are additionally preparing a clinical protocol for a study of TXA127 in children with congenital muscular dystrophy (MDC1A), and hope to initiate studies in 2016 in that indication, for which there are no current treatments."

The planned Phase 2 trial will be a double-blind, randomized, placebo-controlled safety and efficacy study of TXA127 in 45 ambulant patients with DMD, conducted for 48 weeks, followed by a 96-week open-label extension study. The study will be conducted at 3 sites in the United States and 3 in Europe. Endpoints for the study will include assessment of muscle quality by MRI, ambulatory assessments including the 2-Minute Walk Test, and safety assessments.

About the U.S. FDA's Fast Track Designation
Fast Track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and address unmet medical needs. Companies that receive Fast Track designation are allowed to submit New Drug Applications (NDA) or Biologics License Applications (BLA) on a rolling basis, expediting the FDA review process and benefiting from more frequent communication with the agency to discuss aspects of clinical development. Additionally, drugs that receive Fast Track designation are eligible for accelerated approval and priority review.

About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a genetic disorder occurring primarily in boys that is characterized by rapidly progressive muscle degeneration and weakness. It is caused by a defective gene for dystrophin, a protein in muscle. Duchenne muscular dystrophy affects approximately 1 in 3500 male births worldwide, with symptoms usually appearing before age 6. Because DMD is an inherited disorder, risks include a family history of Duchenne muscular dystrophy. There is no cure for DMD. Current treatments are aimed at managing the symptoms and slowing the course of the disease.

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. TXA127 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 http://www.tarixorphan.com/.

Contact:

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

For Media:

Joan Kureczka
Kureczka/Martin Associates
Joan@kureczka-martin.com
Phone: 415-821-2413

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