I think it will demonstrate good efficacy (though not necessarily great - but definitely good enough for approval, and for use) and very good safety profile. There are no other treatments, which is why this one is so important.
With respect to this latest news, probably 3-6 months. It can be accelerated since there are no current treatments in this case and that could expedite approval.
Sure the patent win for BMRN means many young boys will die, but who cares. As long as business wins out, that is a sacrifice we are willing to make!
It also doesn't matter that the whole thing will be overturned on appeal and the net effect will just delay dying boys from getting life saving medication. The point is everybody at BMRN knows Drisapersen doesn't work and causes liver toxicity, but who cares, as long as shareholders make MONEY!!!
please reply to this message if you believe little boys should die so you can achieve profits you couldn't get otherwise since the BMRN drug can't compete.
Given the favorable rulings in many countries around the world including the EU, the approval probability by the US FDA is extremely high. This is based on the efficacy and safety profile of Translarna, and not even considering that the FDA appears to be approving every drug submitted (see TheFool article that explains that the FDA seems to be approving all NDAs submitted, and they are filtering out all problems before NDA filing).
"must certainly be going badly"?
Not at all. The trials are apparently going well in terms of efficacy and safety, the only issue I know of that is less than optimal is the speed of adoption/approval in covering the cost. There have been no issues reported that I have seen about any adverse events or problems of any kind.
Why would you make that up?
He said that he expects the phase 3 to be successful. With regard to the current decline he said it is a market-wide downturn across the biotech space and nothing specific to PTCT at all.
There is no hope for any biotech stock anytime in the near future. This is a bad bear market so I'm going to keep what I have, but no way in hell I'm putting any more money into this crooked market.
Yeah, that is the same story I heard back in March at $70/share, that it was going to the moon. Forgive me if I'm skeptical, and unwilling to put any more money into such a crooked market. I'll keep what I have, but it has a long way to go to get back to where I bought in, and the current chart shows no letup in the nosedive it is in. So no thanks, I'm not going to put any more money into the stock market. Anywhere.
Are the fund managers intentionally driving the price of SRPT down, so that when October 1 news comes, and the stock is up 25% in a day, it really only comes back to the previous level of $40 that we hit before the meltdown?
Clicking on your ID it appears you have secondary-on-the-brain. You seem obsessed with a secondary offering. I wonder why?
Where is simp's beloved hero Hillary to help that boy get medicine? You'd think that would be a headline grabbing situation that would help get a politician elected.
Nope. It is because Hillary Clinton ran her mouth about how she would curtail drug pricing. While she specifically targeted high priced drugs, the whole biotech sector took a 10%+ hit (some 5% some up to 25%) due to this politician's reckless statements. Yet again proving that all politicians put their own craving for power ahead of everything else regardless of who they hurt and how bad.
The Fool article supports the others, but requires intelligent thought to back it out. In order to complete the NDA for the FDA by 12/31/2015 they need time to prepare the data, run it by the FDA before officially filing it with them, update the package based on the FDA feedback, then formally submit the package for the FDA to officially file.
3-months is a very standard timeline to complete the trials, prepare the NDA package, meet with the FDA (once all data is collected and prepared), get feedback, incorporate the feedback, and then submit full package by 12/31/2015.
The Fool reference requires interpretation. In fact just about EVERYTHING requires interpretation, short of being delivered on a silver platter on the company website as an official news release.
No, nothing I have is proprietary, non-public or even all that special. I just connect the dots based on public information.
Actually no. The downside risk if they missed the P3 primary endpoint would be around $24/share (per analyst comments). The upside potential is $100+. So the risk reward potential isn't there, especially considering the EMA already approved Translarna, so the precedent is favorable to Translarna.
Actually, England didn't deny approval, they deferred it to the National Institute for Health and Care Excellence (NICE). NICE will conduct a drug review, which should be reported back in early 2016. So England didn't agree to cover Translarna for funding, they deferred the issue to NICE which apparently has favorable data according to the Dr.'s conducting the trial.
4th quarter 2015 is updated P3 results for Translarna. They don't say when in the 4th quarter, just that it is coming in the 4th quarter, so that could be anytime up to 12/31/15.