I agree with the new street article in that there several risk points in sept
In fact the article does not even list them all
Assessment of the risks is key
Given all the risks, which again are more than the article lists, my analysis says srpt should be fairly valued at 15 to 18
I completely agree. 12 boys, with the boys from England, should be enough. Dystophin production seems to be near uniform once administered 4mg and up. The toxicity issues are absolutely pristine also. As Jrrt1 suggested, it seems ridiculous to force SRPT to treat the entire exon 51 population at not cost in order to get the FDA's approval. They call it a rare disease for a reason.
Also, the FDA appears to have a new mandate given to them recently where they are obliged to consider early approval for rare disease drug candidates. Etiplirsen would seem to be an ideal candidate. It makes the protein it is supposed to make, and it does so with ZERO toxicity.
The questions that remain, about dosing, etc. can be answered while those in need are receiving the drugs.
I put down what I'm doing--not what I'd like to be doing. For the first time in a long time I'd love to be buying. However, I'm still underwater (up to about 15 post-split) and have no more with which to be a buyer. It'd be great if I had all I'd invested in AVI over the years as cash and I'd certainly be a buyer here and own a lot more shares.
Besides the 4 boys on the 50 mg/kg, there will be dystrophin and the start of clinical efficacy on the 2 delayed placebo patients at 50 mg/kg. Plus the the 30 mg/kg cohort dystrophin and possibly some better clinical outcomes there.
With dystrophin as the primary endpoint, there will be 12 boys. From a safety standpoint there will be 8 boys at 52 weeks and 4 boys at 30 weeks. Plus there will be dystrophin and safety from the UK boys.
For the severe rare debilitating disease, a drug with a perfect safety record stands a pretty good chance IMHO. Almost every drug passed by the FDA has blemishes on their safety records.
the market isn't large so i don't know how much of a ramp up is really necessary. also, i think the fda is looking at this kind of how they look at companies like BMRN. the standards and barriers for approval are lowered considerably. i don't know if you can look at this drug as having to go through the normal, long, drawn out process with the fda.
MDA should take some of their cash horde ($67 million at end of 2010) and make a direct investment in SRPT and ask for a couple of board seats in return.
If SRPT is successful in this, in a few years the stock would pay them tremendously and this would fund much more development and treatments.