I don't think they'll be negative. In fact, I believe they will overall be positive. But I do think there will be some criticisms that the market will initially view as negative, namely the small P2 trial and questions around dystrophin. My best guess is that there will be an initial sell-off on the Adcom docs, followed by some pretty heavy buying and ending green on the day.
Honest question: Do you have friends? You know, like, people who legitimately like you? I find myself thinking you probably don't or that the people you spend time with don't have the heart to tell you that you're insufferable. The good news in your case is that you're probably too sociopathic to realize this. Regardless, can't wait for that bankruptcy to hit! Hoping it comes sooner than later.
They increased, but I doubt it's too concerning if that happens, so long as eteplirsen is approved around the same time. No way any rational doctor would prescribe drisa over etep with such a label, and I don't think insurance companies would be able to force patients into taking drisa given the implications, regardless of how much lower it will cost.
The extension trial hasn't been going on for almost 3 years because it was never started as a trial. In other words, no data was collected. Just anecdotal evidence from a few parents. Patients were receiving drisa through compassionate use program. In this case, the company is under no obligation to collect data, and so they didn't.
If BMRN was hoping that etep had safety issues, then wouldn't start the study now. They'd wait until those etep safety issues popped up first. So I don't think your reasoning holds water. By starting now, they can at least capture some of the EU market -- 220 boys to be precise. If etep were approved in the EMA before a new drisa study started, drisa wouldn't get any boys to enroll.
Yes, they would bother with it. For the reasons I listed: PRV and royalties. They tried getting drisa approved without starting a study and failed. (It's much easier to pay for a study with an approved drug than without.) As for your speculation, I think BMRN is wise enough to realize at this point that safety issues with eteplirsen likely aren't going to occur.
The fact that BMRN will be choosing which boys enroll (and let's be honest, boys who had adverse side effects aren't going to enroll anyway) is indicative that they are just going for approval at this point for the reasons I mentioned, and aren't concerned so much with market share because they realize they are likely to lose that war now.
I should also say that I think there's a reasonable chance that the FDA flat out says they think doing a new study for drisapersen is a bad idea. Which would be appropriate.
I should add that I think there's a reasonable outside shot that the FDA flat out says they think doing a new study for drisapersen is a bad idea. In which case, eff you BMRN. You deserve it.
Why would BMRN start an extension study now? Because they are desperate and know that they will likely receive a CRL asking for completion of a new study. They had hoped they could do so without initiating the study, but have realized that ain't happening. I still believe they receive a CRL, and that eteplirsen is approved. It is possible, however, that they are doing this study now because they believe the FDA will see their good faith effort and approve drisa now, thanks to heavy lobbying. But I don't think that's very likely. The FDA was pretty clear in their view of drisapersen at the Adcom.
So why do a study for a drug that is complete #$%$? Simple. The study is rigged. BMRN can, and will, choose which boys from previous trials will take part in the new study. Who will they choose? Obviously boys who showed the least side effects, and yes, I think they will be able find enough that had minimal side effects. After all, the primary endpoint is safety. (Efficacy is secondary. What a joke.) This is smart because it increases the odds that drisa eventually gets approved in the U.S. and/or EMA. Even though eteplirsen will dominate the market and drisa won't make a dent outside of boys in the extension study, if drisa is approved, it gives them a PRV voucher, which should be worth $200 million at a minimum. On top of that, if approved, they will have a much stronger claim to push for royalties from SRPT.
Love them or hate them, BMRN is savvy. They are rigging this study to hit the primary endpoints, and they probably will. The good news is that it's still a gamble, and the FDA and EMA will hopefully see right through their shady plan, particularly once it's clear that eteplirsen clearly works with minimal side effects.
Two independent agencies. They don't typically confer on whether or not to approve a drug. And I doubt the EMA would sway the FDA in any way. (Also, conferring wouldn't even be necessary. It's pretty clear how the EMA feels about drisa.)
I never biotechs that are heavily promoted. (Or really, any at all. KBIO is a great reason not to.) If it had long-dated puts, I would absolutely buy those.
AVXL is a complete scam. Oneway seems like a decent fellow, but I have no idea why he pumps that junk. The best bets are biotechs with low market caps and drugs that have performed well in large, well controlled Phase 2 studies on their way to Phase 3. And also treat conditions with a multi-billion dollar market. The two most promising in that category, in my opinion, are AKBA and TRVN.
No, I'm not sure. You could be correct.
Also, it was May 50 calls. Not 55. But probably just the hacker making it seem that way.
I'm actually of the belief that one of these scenarios is most likely:
1. The FDA simply pushed it back due to the holidays.
2. The FDA is going to issue a CRL for drisa, but not until after it approves etep. (Tentatively pushing BMRN's PDUFA date to January can just as easily be tentatively be moved back again.)
3. The FDA is going to approve both drugs at nearly the same time, drisa with a huge black box warning, and etep with a safe label.
I'm actually leaning toward #2. Even if drisa does get approved, I see no way of that happening without both a black box warning and etep being approved at the same time. Just won't happen.