Date: 2014-10-27
Type of information: Results
phase: preclinical
Announcement: results
Company: Tarix Orphan (USA - MA)
Product: TXA127 (angiotensin)
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. Marfan Syndrome is a systemic connective tissue disorder caused by mutations in the gene encoding fibrillin-1 (FBN1), which result in an increase in TGFbeta that affects connective tissue throughout the body. The major cause of mortality in Marfan Syndrome is aortic enlargement, dissection and rupture. Previous research has shown that reducing TGFbeta or inhibiting the angiotension II type 1 receptor (AT1R) can prevent increased aortic root growth in Marfan mice. TXA127 is part of an alternative pathway of the renin angiotensin system, which directly opposes the action of the ATR1, and the peptide is also known to protect chronically damaged tissues from the harmful effects of ATR1 through mediation of TGFbeta signaling. Tarix research suggests that TXA127 achieves its rescue effects on aortic root growth in Marfan mice by selective upregulation of the known endogenous TGFbeta inhibitor, SKIL.
Disease: Limb Girdle Muscular Dystrophy
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Country:
Trial details:
Latest
news: * On October 27, 2014, 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 product candidate, TXA127, has achieved positive results in an established mouse model of Limb Girdle Muscular Dystrophy. SGCD?/? (sarcoglycan delta deficient) mice demonstrate reduced locomotor activity and a dystrophic phenotype in skeletal muscles at a young age. The Tarix researchers showed that such mice treated daily for eight weeks with TXA127 via infusion by osmotic minipump exhibited a marked increase in activity compared to control animals. Moreover quadriceps muscles of the treated SGCD?/? mice exhibit markedly reduced fibrosis in their skeletal muscle. In addition, older mice that began treatment at age 45?50 weeks showed improvements in cardiac function, compared to controls.
