LAMA2 Muscular Dystrophy Drug Candidate TXA127 Is Granted Orphan Drug Status

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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LAMA2 muscular dystrophy drug candidate

Tarix Orphan LLC recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TXA127, the company’s lead candidate for the treatment of laminin-deficient congenital muscular dystrophy (LAMA2 MD).

Early-onset LAMA2-related muscular dystrophy (MD) occurs in nearly one in every 30,000 people and is responsible for 30 percent to 40 percent of all cases of congenital MD. LAMA2 MD is apparent at birth or within the first few months and is a part of a class of muscle disorders called congenital muscular dystrophies.

Infants affected by the condition suffer from severe muscle weakness, lack of muscle tone (hypotonia), little spontaneous movement, and joint deformities (contractures). The generalized weakened muscles also affect the entire digestive system, which can lead to feeding difficulties and an inability to grow at a healthy pace. Patients with this type of MD are also prone to lung infections.

TXA127 counteracts the classical rennin angiotensin system (RAS), which promotes hypertension, fibrosis, and inflammation, among others.  TXA127 is a pharmaceutical formulation of Angiotensin (17), which is a naturally existing peptide. Tarix Orphan, based in Cambridge, Massachusetts, is advancing the drug for the treatment of several genetic and orphan diseases, including congenital MD. Additional disesases that might benefit from the compound include Marfan Syndrome and amyotrophic lateral sclerosis (ALS).

“This is the third U.S. Orphan Drug designation granted to TXA127 for muscular dystrophies, with previously received designations for Duchenne muscular dystrophy (DMD), our lead indication, and limb girdle muscular dystrophy (LGMD),” said Tarix Orphan LLC’s president and CEO, Richard Franklin, in a press release. “We sought these Orphan Drug designations based on very positive pre-clinical data from mdx, sgcd -/- and DyW models, which correspond to DMD, LGMD and congenital muscular dystrophy (MDC1A) respectively. We now look forward to initiating the clinical evaluation of TXA127 in each of these indications, starting with our previously announced Phase 2 study in DMD, which we expect to begin later this year.”

The FDA grants Orphan Drug status to accelerate the development of promising products for the treatment of rare (orphan) diseases that affect fewer than 200,000 individuals a year in the U.S. If the FDA approves the compound for the treatment of congenital muscular dystrophy (MDC1A) and allows the company to apply for research funding and tax credits for sub-sequential research expenses, the Orphan Drug designation will entitle Tarix Orphan to seven years of marketing exclusivity in the U.S. for TXA127.