FDA Grants Fast Track Designation To Tarix Orphan LLC’s TXA127 as Promising Duchenne Muscular Dystrophy Treatment
Privately-held biopharmaceutical company, Tarix Orphan LLC, has just announced the US Food and Drug Administration has granted its lead candidate for Duchenne Muscular Dystrophy, TXA127 (angiotensin 1-7), Fast Track Designation for its potential in lessening damage and fibrotic activity in skeletal muscle. Under a previously filed Investigational New Drug (IND) application, the company also received clearance to proceed with a multi-site Phase II safety and efficacy study in early 2016 in the US and EU. TXA127 development is also currently operating under Orphan Drug Status in both major territories.
“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 upcoming 2016 Phase II study is designed to be a double-blind, randomized, placebo-controlled safety and efficacy study of TXA127. Tarix will aim to enroll 45 ambulant DMD patients, who will receive either a placebo or the drug for 48 weeks, before proceeding to a 96-week open-label extension study. The trial will be carried out in 3 sites in the US and another 3 in EU. Endpoints for the study will be evaluated based on muscle quality by MRI, ambulatory assessments including the 2-Minute Walk Test, and safety assessments.
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The Wellstone Muscular Dystrophy Cooperative Research Center (MDCRC), at the University of Iowa (UI), has won a $7.4 million grant from the National Institute of Neurological Disorders and Stroke (NINDS), a subdivision of the National Institutes of Health (NIH), renewing its funding for five years, through 2020. The center’s key goals are both muscular dystrophy research and education of future medical specialists.