Date: 2015-07-13
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: amyotrophic lateral sclerosis (ALS)
Therapeutic area: Neurodegenerative diseases - Rare diseases
Country:
Trial details:
Latest
news: * On July 13, 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 SOD1 model of amyotrophic lateral sclerosis (ALS). Researchers showed that ALS model mice treated daily for up to 8 weeks via osmotic pump with TXA127, a pharmaceutical formulation of the natural Ang 1?7 peptide, exhibited a statistically significant increase in survival compared to control mice who received only the vehicle solution (164 days versus 134 days; p ≤ 0.0001). The TXA127?treated animals also exhibited a strong benefit in quality of life/disease progression scores for 128.33 days versus 102.67 days for control animals, as measured by the number of animals with scores ≤ 2 (animals ambulatory but dragging hind foot).
