Cambridge, Mass.-based Tarix Orphan, a privately held biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases, has received the FDA’s Fast Track Designation for TXA127 (angiotensin 1-7) to reduce skeletal muscle damage and fibrosis and thereby improve muscle strength in Duchenne Muscular Dystrophy (DMD) patients.
Tarix Orphan was given 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 II safety and efficacy study in patients with DMD in early 2016 at both U.S. and European trial sites. Tarix previously has 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 Tarix Orphan CEO Rick Franklin. “We look forward to beginning our multi-site international phase II 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, and hope to initiate studies in 2016 in that indication, for which there are no current treatments.”
The planned phase II 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 three sites in the United States and three 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.