Date: 2015-08-03
Type of information: Results
phase: preclinical
Announcement: results
Company: Tarix Orphan (USA - MA)
Product: TXA127
Action
mechanism: peptide. 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.
Disease: congenital muscular dystrophy (MDC1A)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Country:
Trial details:
Latest
news: * On August 3, 2015, Tarix Orphan, a privately held biopharmaceutical company focused on the treatment of rare neuromuscular disorders and connective tissue diseases, announced that the company’s lead compound, TXA127, has shown positive results in the preclinical DyW model of congenital muscular dystrophy (MDC1A). Researchers showed that two doses of TXA127, a pharmaceutical formulation of the natural Ang 1?7 peptide, remarkably reduced inflammatory and fibrotic pathology, and also induced weight/muscle gain and increased muscle function. MDC1A is a hereditary muscle disorder associated with a genetic change in the LAMA2 gene which is responsible for the production of a key muscle protein, laminin alpha?2. MDC1A primarily affects the muscles used for movement and is one of the most common types of congenital muscular dystrophy (CMD). In addition to MDC1A’s effects on movement and motor skills, children with MDC1A may have weak respiratory muscles, leading to frequent chest infections and poor breathing at night. They may also frequently experience feeding difficulties, leading to weight loss and failure to thrive. Despite extensive research into major drivers of MDC1A, there is no cure or treatment for the condition at this time.
