1) This drug is based on something called exon skipping in DNA. Only about 13% of boys with muscular dystrophy might benefit from this approach. Very few patients have been enrolled in trials and when larger trials are done in the future the chances of side effects will increase.
2) Summit, a company in UK is developing a better drug that makes a protein called utrophin that functions similar to dystrophin which is the protein missing in DMD. Up-regulating utrophin would regulate all DMD patients regardless of the genetic mutation. This drug is already in trials.
3) SRPT is burning cash and has very little reserves to go on. Don't forget what happened to Prosensa which went from $34 to $5 very quickly.
Uh, 200 million in the bank, isn't "very little reserve" you DOPE
And...................THANK YOU in advance for the covering your boss has been doing, and will continue to do. You shorts with your #$%$ up thesis, are the best.
You don't know enough, kid. In their phase I trial, there is no data supporting an increase of utrophin expression in the trial subjects. If C1100 can increase utrophin in DMD patients, it should be able to increase utrophin in healthy subjects too. So they already showed lack of efficacy in human in a phase I clinical trial.
Even Summit itself do not know why (if at all) their compound C1100 is changing Utrophin expression in human. It works in a mysterious way and without understanding the mechanism of function, C1100's safety concern is a big liability, and they reported "Inter-individual variation was observed".
007: A little truth can be quite misleading. I'm not saying this is intentional though...More specifically, other drugs using Serepta's exon skipping algorithms, which *will* be able to treat DMD patients outside of the 17% you ingenuously mention, are in the pipeline already. In addition, the effectiveness of increasing utrophin levels to treat MD, is theoretical, and, at this point, unproven.