I guess analysts haven't read past the headlines, and CNBC's error didn't help things any. Why does it work like that? Why doesn't CNBC get my stocks mixed up with one that has great news causing an overnight doubling? Why is it they always screw up the bad news? Almost like they are manipulating the stock or something.
Anyway, with the modified intended to treat (ITT) group in the new/updated NDA we should see a very strong uptick in the stock along with a very favorable FDA Adcom. Good notes/briefing, good positive advisory committee, and a strongly favorable vote.
They will file the NDA before year end, then about 4-5 months later will be the Adcom and final approval decision about a month later (within 6 months of filing the NDA).
Yeah, I found it. Some 2-bit moron at CNBC who doesn't have a clue, and shareholders pay the price.
Shame on CNBC for their inaccurate reporting (per gogate02). I didn't see it but something is driving this stock lower without reason. BMRN failed the drug Adcom, not PTCT.
That's the whole point and the problem, you have to look beyond the headline (and the FDA will). The headlines read that the drug didn't work for all patient groups. That is true, it only worked on 90% where the patient had not completely lost ambulatory ability. For those 90% the drug worked extremely well and was safe.
Maybe people won't get it until Ataluren is officially approved by the FDA. Then they will understand.
Should be, but isn't, because people haven't figured out yet that Ataluren treats a different subset of DMD than other drugs. There is no treatment whatsoever for the PTCT patient subset. Plus. There are no safety issues with Ataluren.
Looking at pre market trading it is clear that some people don't understand that BMRNs failure yesterday has nothing to do with PTCT or its prospects.
1) Ataluren treats a different set of patients, those with a nonsense gene.while SRPT and BMRN are in direct competition to treat the same patient group, that is separate from what PTCT is focusing on.
2) The safety profile of Ataluren is outstanding.
3) The modified Intend To Treat (mITT) group in the Phase 3 trials of Ataluren shows outstanding efficacy. No subgroup in the BMRN study showed any discernible benefit over placebo.
BMRN failed because it would kill patients (FDA's Farkas said yesterday). PTCT has no such problem.
BMRN also failed because it didn't show efficacy. PTCT dis show it, although only for patients who weren't in advance stages of DMD and had already lost the ability to walk. Who knows, it may just take longer for those smoldering embers to catch fire, because it clearly works for a large portion of the patient population.
I think that Ataluren will be approved because there is NO treatment for the DMD kids with a nonsense mutation. Drisapersen and Eteplirsen won't work with this set of patients, Ataluren is their only chance.
PTCTs modified intended to treat (mITT) shows outstanding efficacy and perfect safety, which makes it a strong and compelling drug for approval. BMRN's drug had severe safety problems and no efficacy in any group, or subgroup, which was their problem yesterday.
Right now I peg Ataluren's chance of approval around 90%.
Will there be a stampede for the door?
Yes, delisting IS happening. Company can help the situation but they have to take action to prevent the delisting from happening. Otherwise, they will get delisted.
That is certainly one way to view that. Another way is that they are in the process of being delisted unless they take action and change things and get updated paperwork filed with the SEC. By a new CEO who is a kid who doesn't know anything about business and will learn at the expense of shareholders.
Posted from BMRN board: As AF points out, the vote is not even a traditional yes or no on efficacy and safety (two separate votes) - instead they are asking them to vote on whether the 3 separate trials either strengthen, weaken or have no effect on approval and then a separate vote on whether safety strengthens, weakens or has no effect on approval - this is a very unique format and completely dismisses the pooled analysis that BMRN portrayed in today's press release. Given the FDA's issues and conclusions on the separate trials and the even worse assessment of safety, it is hard to imagine many "strengthen" votes?
Is this right?
Not true at all. 100% of the boys treated with SRPTs Eteplirsen are still walking. That drug is bothe safe and effective.
Zero side effects, zero adverse events.
Thank you, I did not know that. I will be attending the Adcom in person, so it will be interesting to see how that goes.
I agree that it is a positive for PTCT. I think there was a mis-perception that because BMRN was knocked for not showing any efficacy, that PTCT might be in the same boat - but it isn't - because they have very specific groups that showed high efficacy (those that had not yet lost ambulatory function). Only where the disease was very advanced did they not show efficacy, so if you narrow the intended patient population (which is what PTCT is doing in the revised NDA submission to the FDA) then they are all set as they can show very high efficacy.
Once institutional investors realize this it will power up to $40+.
Now it appears there will be a vote of no on Drisapersen based on the harsh FDA comments released today. I used to think the FDA would approve both Drisapersen and Eteplirsen but no longer.
Watch today as BMRNs Adcom briefing documents were released with harsh criticism from the FDA on efficacy. No specific patient group had consistent benefit.
Now look at PTCT and you will see a very strong efficacy case for patients who had not lost ambulatory function yet. That is the main difference - PTCT has a very strong group of patients that showed significant improvement over placebo. While not as good as SRPT, it is far better than BMRN. Since SRPT treats a different patient group, the two aren't in competition.
Then again Sarepta could buy PTCT and have a commanding lead in the DMD space while leveraging Translarna into other diseases like CF.
I agree that long term DEPO will be better off not being acquired, but it will take more than a year to recover the share price to what Horizon would have given shareholders instantly. That opportunity cost can never be undone because of compounding.
A year from now it will likely be a wash, but I don't like money to sit dead for a year. I want instant gratification and I want it NOW.