Sarepta Therapeutics Inc (NASDAQ:SRPT) On Right Track
Sarepta Therapeutics Inc (NASDAQ:SRPT) focuses on development of treatment for rare and infectious diseases. Its prime candidate is eteplirsen, being developed to treat Duchenne muscular dystrophy (DMD). This disease affects one in every 3,500 boys and is prevalent worldwide.
DMD is a rare disease with a small but well defined patient population. It is estimated that there are approximately 35,000 patients in the U.S. and Europe alone. Unlike other diseases affecting children, this can become fatal and hence need to be treated at the earliest. There are, however limited therapies available.
Sarepta Therapeutics Inc (NASDAQ:SRPT)’s eteplirsen is undergoing extended phase-2b trials and they have shown encouraging results. This drug has been able to help patients in the 120 week period, a trial involving 12 patients in the age group 7-13 ages showed considerable stabilization in walking ability. The 6-minute walk test conducted at 120 week mark showed that patients undergoing the treatment experienced at decline of only 13.9 meters in walking distance. They showed a treatment benefit of 64.9 meters over the placebo group. Other companies trying to develop treatment for the same disease have had very bad trial III results and the U.S. FDA may want a bigger or more sustained benefit before giving it approval. Sarepta had approached the FDA in November for marketing approval for the first half of 2014, but the FDA felt it to be premature. The company will now be looking to finish the design of a late stage trial during the first quarter of 2014.
Other Pipeline Products:
Unlike other bio-pharmaceutical companies, Sarepta Therapeutics Inc (NASDAQ:SRPT) is not a one product wonder. It has several other products in the pipeline and they are at various stages of development. The company does not have a saleable approved product in its stable though. Sarepta is developing treatment for both rare and infectious diseases. Other products being developed include treatment for Ebola, influenza and Marburg.
Analysts are also confident that the company can deliver results and have given it a ‘buy’ rating.