Why This Stock Is Poised for Long-Term Success

By Terry Chrisomalis - March 25, 2013 | Tickers: BMY, PFE, SRPT | 0 Comments

Terry is a member of The Motley Fool Blog Network -- entries represent the personal opinion of the blogger and are not formally edited.

There is currently a big medical need for a drug that treats Duchenne Muscular Dystrophy. This disease basically halts motor function and movement as the years go bye. There are hardly any options for these patients, and a breakthrough drug from a pharmaceutical company can prove to be monumental for them.

One such company has achieved tremendous results in Duschenne Muscular Dystrophy, and this company is known as Sarepta Therapeutics(NASDAQ: SRPT).

Results that matter

On Oct. 3, 2012, Sarepta Therapeutics announced results from its phase two trial in Duschenne Muscular Dystrophy. Patients in the study, who took eteplirsen, were on average able to walk 89 meters more than those who just took the placebo drug instead after 48 weeks.

What's even more incredible is that when patients had muscle biopsies performed on them at the beginning of the study, researchers noticed that they had no dystrophin proteins being created. But by the end of the study, the levels of dystrophin protein had increased to 52%. Obtaining 52% for patients is great, as that is considered to be normal movement levels for functional humans.

One hit wonder

When many people think about Sarepta Therapeutics, they only think about their Muscular dystrophy drug. But in truth, this RNA based pharmaceutical company is not just a one hit wonder. They have received a contract from the government, worth $291 million, to fund trials for phase one testing for Ebola and Marburg virus. This opportunity is great, because currently there is no effective therapeutic for either Ebola or Marburg virus, so advancement in these two drugs can also prove to be profitable.

So, Sarepta Therapeutics has much more to offer than a one trick pony drug, and it's basing all of its drugs on RNA technology. A key difference about Sarepta's RNA technology, compared to other RNA companies, is that Sarepta can activate both a messenger RNA and a precursor messenger RNA. In other words it can turn gene expressions on or off, while other competing RNA technologies can only turn off interference.

Original post:
Why This Stock Is Poised for Long-Term Success