TXA127 for the treatment of muscular dystrophies

Dr Richard Franklin, Tarix Orphan LLC, USA

10th May 2014

Tarix Orphan LLC, Cambridge MA proposes to test its compound TXA127, a pharmaceutical formulation of the peptide angiotensin-(1-7), in a first clinical trial in children with neuromuscular disorders. TXA127 is involved in the alternative pathway of the renin-angiotensin system and has already been tested in phase II trials in hematological indications comprising over 100 adult patients without serious adverse events. Preclinical investigations in two mouse-models of neuromuscular diseases have shown positive effects on histology and function via multiple pathways. Thus, efficacy in several human neuromuscular diseases could be expected. TACT concluded that TXA127 is an interesting drug with possible activity in a broad range of neuromuscular diseases. TACT proposed further preclinical work addressing additional aspects of pathophysiology, interaction with steroids and other compounds used in neuromuscular disorders, toxicity in young animals, long-term toxicity and pharmacokinetics before progressing to clinical trials in children with Duchenne Muscular Dystrophy or Congenital Muscular Dystrophy. Furthermore, an oral preparation would be much preferred versus daily subcutaneous application in pediatric patients and should be tested preclinically. In addition, TACT made proposals for a first in children study which should address mainly dosage finding and tolerability and lay the ground for a later efficacy study by including some functional neuromuscular outcome measures.

12 Apr 2017