The shares of developmental biotechs are extremely volatile with Geron Co.’s (NASDAQ:GERN) shares tumbling over 50% this year in just one day. This was after the Food and Drug Administration put their cancer treatment, imetelstat, on hold. Many biotech firms have witnessed Sarepta Therapeutics (NASDAQ:SRPT) gaining the attention of short-sellers.
The developmental biopharma gave shareholders a run for their money with its rapid changes in market share the current year. This has had a major impact on Sarepta’s (NASDAQ:SRPT) regulators and its DMD therapy named as eteplirsen. Originally, the FDA didn’t approve the application for the approval of the drug but later on altered its own decision which followed an outcry from the advocates of DMD.
Sarepta’s (NASDAQ:SRPT) eterplirsen has the potential with so many clinical candidates like the Ebola disease with its drug AVI-753. Its value validates the whole DMD platform. The stock is doing well in the market with many investors considering whether Sarepta’s (NASDAQ:SRPT) stock is good enough to buy with regulatory reviews coming up with the share trading way above their 52-week high.
The regulatory approval for Eteplirsen is to be submitted by the end of the year as Sarepta (NASDAQ:SRPT) reported to its investors through a presentation. It all depends on the type of review the FDA provides later on which may come in about six to eight months thus confirming it around the Q3 in 2015.
Investors think that with the changes at the FDA and the European Union with regard to DMD drugs makes it easier for eteplirsen to gain approval. With public pressure, the FDA agreed to carry forward with a regulatory filing. However, the drug has only been tested on 12 patients though. The results of these trials had more questions than solutions.
Rumors have it that the FDA will have Sarepta (NASDAQ:SRPT) complete somewhat of a larger trial on later-stage candidates after the data showing a weakening of the effect of eterplirsen. The question the market has been asking the company is whether dystrophin levels have any sort of clinical benefit to DMD patients or not. However, the company has completely dodged the question after requesting that the FDA use dystrophin production to measure outcomes.
There may not be a need to increase dystrophin in patients if the outcomes of the trials don’t have improved locomotor or respiratory functions. The point is that there may not be enough data to measure the relationship between dystrophin levels and the outcomes in the clinic. Sarepta’s (NASDAQ:SRPT) stocks have been volatile for years now with the short seller and high institutional ownership.
The company hasn’t been too responsive in the clinic to carry forward with the gain of approval by the FDA for its eterplirsen. It may be a while before the public gets what it wants as more tests and trials are carried out. Experimental treatments are applied worldwide and the public is always the one to wait for science to run its course.