Accelerated approval doesn't mean the fda lets you start a phase III study and waits for more years for the outcome before the drug produces income.
Accelerated approval means: Since the surrogate marker (dystrophin in this case) ALREADY is shown to be produced by the drug, when accelerated approval happens it is right THEN approved, (as in --you can charge your fee for it). The ongoing study of more patients occurs while the drug company generates income from the drug; the larger the confirmatory study the larger the income.
So when Chris talks about a larger study in 2013, he isn't talking about a phase III where you have to give away the drug while it gets further proven, but rather a Phase where the company is marketing the drug while the study proceeds. (ie income gets produced right away.)
Maybe I'm missing something but if my reading of the above is accurate, there's a short time to a LARGE market cap based the above noted income, and capital generation to further the production, and thus,
And a short time to when the boys can start getting real help. imho
Sentiment: Strong Buy
You all assume the parents and a motivated congress just sit by and say what's a year or two? Just wait the activist to unlease a war for action . AP. a layup. And funding coming! Trust me!
Sentiment: Strong Buy
Obviously some insurances will not pay for this treatment before it is fully approved after the confirmatory study...I am sure many of us might have a small print in our policies where the insurance comps might still call this 'experimental' before fully approved after the confirmatory study. Also, as many of you know, most companies producing these expensive life saving treatments have implemented programs giving the drug free to the non-insured, but I doubt Sarepta can do this in the first years before they start making profits.
Nice to see some good dialogue instead of idiotic short or long #$%$. That said, has anyone talked to IR to confirm or disavow any of the talking points you all bring up here? I haven't talked to their IR yet. I wonder if they can even address your questions. TIA....
Exactly. The CEO is not going to take Accelerated Approval for granted when making projections. As has been mentioned by Gary and others, he is not going to make any statements that disrespects the formal FDA application process. But make no mistake about it, this company and most of the DMD community will be shocked if Accelerated Approval is not granted. It will be granted in the first half of 2013. The information moving the stock in the meantime are simply mounting morsels of evidence that add to investor confidence of its inevitablitiy. It benefits the kids because the drug becomes available to everyone. It benefits the company because they are realizing their potential.
Anyone who suggests that Accelerated Approval has been priced into this stock is clueless what the price will be when Accelerated Approval is granted. That is the day the stock will approach $160 or more. Enjoy the journey!
Sentiment: Strong Buy
I do not think this is correct. The company covers the cost of the drug for patients enrolled in the confirmatory study. The other boys, NOT included in the study, have access to the drug for the price of the treatment (their insurance company pays for the drug in many cases but not all). Sarepta cannot charge insurance for boys enrolled in the trial obviously, that would be ridiculous to run a clinical trial paid by insurance companies. However, all other boys not included in the study (due to inclusion criteria and/or not willing to participate) might have access to the drug if their insurance (or someone else) pays for it. Obviously enrolling in the trial will be an advantage since the treatment price is covered by Sarepta and your insurance is not billed. On the other hand, the boys will have to go thorugh biopsies, testing etc. while taking part in the confirmatory study. So NO, it is not in Sarepta's advantage to have a very large study as they will have to give that drug for free. That is why they are hoping to have only ~60 boys enrolled in the study...so they can make money from the rest of the DMD boys whose insurance would pay for the treatment.
Prosci, if this is correct then I see NO advantage to the company for AA and don't know why so much hope has hinged on it--it would be no different from a phase III without AA and all the dilution we've hope to avoid would NOT be avoided as it would be just as expensive to the company as if AA had not occurred.
I hope this is wrong or those predicting major dilution will turn out to have been correct and the company's near-intermediate term prospects are in fact fairly poor from likely price appreciation standpoint.
Also, an approved drug (I suppose even under AA) shouldn't be considered experimental for insurance purposes, IMO.