I love the idea but it would never happen. No way the FDA would ever allow a human element into a clinical review, known for being unfeeling and filled with Adcom members who put their own careers ahead of patients needs.
At least SRPT is better than TTPH that I've been watching (too risky to buy). After hours it lost 80% of its value as it didn't meet the primary endpoint of the Clinical Trial.
How did BMRN avoid a collapse when Drisapersen didn't meet its primary endpoint?
Worse than that, I just hocked pearsby's wife to get money to buy SRPT shares.
Now, how much can I buy for 25-cents?
I've got some news for you, you were wrong.
Now before you get offended, hear me out, because you'll realize I'm actually giving you a compliment - a well deserved one at that. From the posts I've read of yours, I've become a big fan of yours. You are usually right in what you say, so on the rare occasion you are wrong, that is a new thing, therefore "news".
I typically bypass the turkeys and morons, putting them on ignore, because of their hate filled junk that makes them look like short selling bashers.
I do seek out your posts and a few others because they are typically informative, useful, and insightful, and for that, I say thank you.
Now what news were you expecting today? And do you think that any meaningful news would/could/should be released after market close tonight?
Says the guy who not only wasted time reading my dribble, but wasted even more time replying it it.
Another stock to watch is PTCT which is about to release their late stage clinical trials results on efficacy and safety. The stock is repressed substantially and looks like a screaming buy, but I'm just not sure if they will have good results in the latest round of clinical trials. News reports set it up as one of the biggest binary events of the year. Stock could quadruple, or get cut in half, so not for the squeamish.
Then again, I thought SRPT would have been over $60 by now given the acceptance of the NDA.
Anyway, anybody have any thoughts on PTCT?
Fails miserably? Based on what. It is already approved in Europe for use, so it must have some proven efficacy & safety. It isn't a competitor to SRPT in any way, so not sure what the arbitrary objection is.
I'd buy the option but the premiums are so high. I guess people are thinking it is going to get approved. The guy at Citi put a target at $124/share if approved (at $38/share now).
So far so good. No adverse events reported and efficacy is good. Waiting on official update though.
I expect news by the end of October on the late stage trials that include some outstanding results from the patients taking the drug in Europe. Stand by for some very positive news coming before Halloween.
You will be richly rewarded. Apparently the confirmatory trials in Europe are going very well.
Really? So you have seen the research he has done and his e-mails back and forth with the European doctors? I didn't know you had that kind of access - which of course would be required for you to provide that kind of summary judgement.
News is that while there are few patients on Translarna the results are very good. The problem is getting the countries in the EU to cover the cost, which is why it is slow on the uptake so far. Still, the favorable results will help get more countries to cover the cost while helping them gain approval in the US.
Not from him but from others that are directly involved in treating patients in Europe that reported that adoption is slow due to many countries not covering the cost yet, and that patients that are under treatment are responding very favorably. The only problem this doc had was that the patient count is few.
As of July 28th, there are 106 DMD patients on commercial therapy, including patients from both direct commercial sales and reimbursed early access programs. New countries recently added include Denmark, Norway, and Brazil. Translarna received marketing authorization from the European Medicines Agency in August 2014 for the treatment of nonsense mutation Duchene muscular dystrophy in ambulatory patients aged 5 and over, representing the first-ever treatment approved for the underlying cause of the disease.
Top-line data from Phase 3 ACT DMD expected in the fourth quarter of 2015. In December 2014, PTC began submitting a rolling new drug application (NDA) to the FDA for the approval of Translarna in nonsense mutation DMD. Top-line data from the company's ongoing Phase 3 ACT DMD trial is expected in the fourth quarter of this year. PTC anticipates a potential US approval and commercial launch of Translarna for nonsense mutation DMD in the first half of 2016.
Good news came in and it was that they filed in Canada to get Translarna approved for use there. Another country, another revenue stream.
Keep plugging away ...