There are three possible outcomes.
The best for us and for the families is AA in the next 30-60 days. Such an outcome would send the stock over 100. For SRPT, this would be just the start. They would move on the develop exon skippers for most of the other DMD kids, and then use exon skipping technology in a variety of diseases and even infections to create a broad pipeline of new products. Eteplirsen is just the appetizer. In this scenario the FDA would want the company to essentially conduct a P4 trial with drug on market to prove that it works, if it fails to live up to its billing, the FDA could pull the plug on it (see Xigris).
The worst outcome would be the FDA requiring a full P3 trial with at least 18 months if not 2 years of follow up. That would take probably 6-9 months to set up and create an SPA with the FDA, and another 2-3 years to run. We would get data in 2016 or 2017. Such an event would probably knock the stock price back into the teens but would be a buying opportunity for those with a 3 year investment horizon.
The third, and I think most likely outcome, is that the FDA will ask for an additional limited trial looking at dystrophin production at 6 months and 6MWT stability at 1 year. This could be done by end of 2015. Such an outcome would not knock the stock price back much, might even hold steady.
I bought into this company last summer but then bailed with a small loss in October when the biotech sector was getting crushed and my really big holding was dropping enough to make me nervous about a margin call. Pure luck but I missed the meltdown to 12 dollars as a result. Since then have been buying back in average price about 18 dollars at this point. This is what I see as a physician, and I have read the Annals of Neurology article in its entirety.
Eteplirsen has a convincing mechanism of action. The production of a truncated dystrophin protein converts the DMD patient into the equivalent of a Becker's MD patient, Becker's patients produce a truncated dystrophin also. Becker's patients have lifelong weakness but do not lose mobility or end up on ventilators in their teens. In the muscle biopsies we see consistent production of significant amounts of truncated dystrophin in all 12 boys. We also see over 2 years of muscle strength stability, which is completely the opposite of the natural history of DMD. Given this, it is highly likely that eteplirsen is an effective therapy for DMD. If my child had it, I would demand it right away. There is no toxicity out to 2 years.
The FDA is uncertain what to do. To approve a very expensive drug for use on children based on 12 patients would be highly unusual to say the least. They do have the legal authority through AA, but this is a real stretch and there is no historical precedent for this. The FDA by its nature is extremely cautious. No one wants a repeat of Vioxx or thalidomide.
On the flip side, the desperate and immediate need of these children is obvious and overwhelming. For them, a delay of 6 months not to say 3 years is huge. Some of these kids will die while the FDA makes up its mind. The parental and political pressure will be huge. How the FDA's conservative nature and this parental pressure resolve itself is anyone's guess. Continued...
Immu is my largest position, owned it since 2006, went 100% long in spring 09 when market melted down and IMMU dropped below a buck. The company has plenty of cash and three major plays. They have a Mab for SLE that is partnered with UCB, the P3 1600 patient trial is supposed to fully enroll in next couple of weeks and topline data in early 2015 . They also have a hot Mab for pancreatic CA that just started a P3 trial (no partner for now on that), and they have two different ADC Mabs to solid cancers in P2 trials which I suspect will get major pharma partnerships this year. If you are interested in the company the best board is on Silicon Investor, the YMB is mostly useless. I like TTPH, I think that if eravacycline works it should do 500 million in annual sales and we should get a market cap of 3 billion plus.
I'm a critical care doctor and deal with multi drug resistant infections all the time. Looked at their p2 and lab data and the lead antibiotic enavercycline looks great. It has activity against all the major drug resistant bacteria except pseudomonas. In the P2 it was equally effective to meropenem which is one of the most potent IV antibiotics. Tetracyclines are very safe antibiotics and don't have major toxicities such as kidney failure that limit their use. The fact that enavercycline can likely also be given orally makes this a potential blockbuster drug. If the P3 comes in showing effciacy, this could be a billion dollars annual worldwide sales. The problem for the last 4 decades was that tetracyclines were so complex they could not be synthesized and modified from scratch, they had to be generated from tetracycline producing bacteria. TTPH has patented a chemical process that allows them to build tetracyclines from scratch and modify them to overcome current bacterial resistance, they have made over 1000 different variations in the lab and took the best compound to be their lead product. They have 2 or 3 more on the way in the pipeline. This stock is way under the radar if you look at the anemic volume, but someone is buying.