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news.Biopharmaceutical firm Tarix Orphan has received fast track status from the US Food and Drug Administration for its TXA127 (angiotensin 1-7) to treat patients with Duchenne Muscular Dystrophy (DMD).
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The approval has been granted for TXA127 to reduce skeletal muscle damage and fibrosis, allowing to improve muscle strength in DMD patients.
DMD is a genetic disorder that occurs primarily in boys and is characterized by rapidly progressive muscle degeneration and weakness. It is induced by a defective gene for dystrophin, a protein in muscle.
Earlier, Tarix obtained orphan drug status for TXA127 in the DMD indication in both the US and Europe.
Tarix Orphan CEO Rick Franklin said: "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.
The firm will also commence a multi-site Phase II safety and efficacy study in patients with DMD in early 2016 at both US and European trial sites, as it received FDA notice to proceed clinical testing of TXA127 under investigational new drug (IND) application.
The study is a double-blind, randomized, placebo-controlled safety and efficacy trial of TXA127 in 45 ambulant patients with DMD. It will be carried out for 48 weeks, followed by a 96-week open-label extension study.
Tarix will conduct the study at three sites in the US and three in Europe.
According to the firm, the trial’s endpoints will comprise evaluation of muscle quality by MRI, ambulatory assessments including the two minute walk test and safety assessments.