NDA is expected to be filed in Q1 or Q2 of 2014. Time is money and I do not see a reason to hold a over $1 Bil. market cap for a drug that won't even be seen by the FDA until 2014 and approval much later, assuming it gets approval. The FDA may also require a phase III just to ensure safety across a broader range of various of people. I know it targets a specific SNP but tolerance could vary across the rest of the DNA profile. The best way to test that is to have a larger body. I'm sure I'll be told about the FDA saying size does not matter, but in the case of safety it would be better and what the FDA has said is just preliminary. It could and should change in the interest of safety. Which would push any possible approval even further out.
"targets a specific SNP", I heard of pseudoscience, today I see a prime example of pseudo-scientist. Do you understand that you copying-pasted? You don't understand Etep's MOA. What is the SNP here?
do you understand that if it is copy-pasted then it was written by someone else, somewhere else??
Are you going to tell me that the mutations are not SNPs?
What has happened here is simply a case of typing of speed and brevity and not making myself clear to those who wish to nit pick every word.
All I was saying is that this drug targets a specific subset of DMD patients, specifically those with specific mutations (SNPs). In short, these patients all have the same problem, but that is only one region of the patient's whole genome. While this one region is the same, the rest is not. Thus safety cannot be automatically guaranteed unless there is genetic diversity in the patients.
Happy now? Or do you still want coverage on how exon skipping works?
what always amuses me about pre-approval drug stocks are the stock holders doing the job of the FDA.
If everything was a slam dunk there would be no need for trials. Yet there are trials. None of you are on the inside of the FDA (I hope not) so no one can be sure of what they will approve or not.
This has minimum of 18 months to even make it to approval. The months away mentioned in the replies is for the NDA filing. just the filing. Approval and then manufacturing process approval will take much longer.
And, as mentioned, this is assuming the FDA does not ask for a phase III trial.
Other investment ideas? Well, how about shorting MSFT. it seems like easy money to me. I think the Mulally rumor is just #$%$, and even if it is true then it would be another sell the news situation like the Ballmer news. MSFT is only a little under 7% off its pre-earnings disaster 52 week high. This short will make money in the next few weeks instead of waiting for 18 months for a possible approval that no one can guarantee here since they aren't privy to the FDA's viewpoints on the data.
Sentiment: Strong Sell
Give me one long ideal. Just one.
No doing the job of the FDA anymore than you are.
Hoping the drug works for a variety of reasons including and so far in the patients it has been tested in, something is happening. Boys who normally would be progressively getting much worse aren't. What explains that? Placebo effect? Chance? Did they pick the 6 kids who have some yet to be discovered version of DMD which has a different prognosis? You tell me what explains that these kids aren't declining. Because I cannot think of another reason other than eteplirsen works.
It seems to me that in your ingeniousness, you've lost a simple truth... it's 2013 now... and 2014 is just 3 months away. You make it seem as though we're decades out. So, why the hell NOT hold? The drug works, the tests continue to prove that. The FDA seems to be on board. As of right now... there is NO short thesis.