Sarepta Announces Positive Results…Shares Rise
Sarepta Therapeutics, Inc. (SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases, today announced muscle biopsy results from its 4053-101 study, a Phase 1/2 first-in-human study conducted in Europe to assess the safety, tolerability, pharmacokinetics, and efficacy of golodirsen in 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53.
Sarepta further reported that the study achieved statistical significance on all primary and secondary biological endpoints. the Company also stated that the results further validate the Company’s exon-skipping platform for the treatment of DMD.
Dystrophin is a protein found in muscle cells that, while present in extremely small amounts (about 0.002 percent of total muscle protein), is crucial in strengthening and protecting muscle fibers. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas of the body. The condition is universally fatal, and death usually occurs before the age of 30 generally due to respiratory or cardiac failure.
Shares of SRPTclosed today’s trading at $46.63 up 13.75%!
Biotech firm Atossa Genetics (ATOS) a clinical-stage pharmaceutical company developing novel therapeutics and delivery methods to treat breast cancer and other breast conditions closed today’s trading up 2.24%! Learn more by visiting the Dedicated Company Page at Vista Partners for ATOS.