So there is this new Will Smith movie coming, "Concussion'" which portrays the doctor who fought against the billionaires of the NFL to confirm CTE issues from repetitive head trauma. The trailer shows the skepticism and worse that he encountered.
Fast forward to today, where the NFL acknowledges the problem and has implemented new protocols for injured athletes to follow. The billionaires accede to the experts, and they appear to be supportive,
Perhaps there's a parallel to Sarepta. From the early dog videos to the continuing results from our dozen P2 boys, it's obvious that eteplirsen bleedin' works, mate, and that PMOs hold tremendous promise for the future of medicine. But we have been blunted, taunted, doubted, bully ragged, denied....well, you get the picture. But....maybe that's all about to change? The billionair bio -- the best in the orphan drug business -- watched and learned how data really DO matter. The heads of charities gained headaches that neither Advil nor Tylenol can help. And the stage is set for an Adcom or outright approval that will put eteplirsen into the hands of boys that have been hoping against hope for a therapy that flat-out WORKS. And if any of the panelists feel any pang of guilt for going thumbs down after a 16-yr.old and other pleaded for assistance, they can take heart that they actually did these boys a favor. In short order, they will be on etep without worrying about becoming a Farkas statistic.
There's so much work left to be done, but I really think the walls of resistance are crumbling. And while we suffer some of the indignities posted here, we also embrace the perspectives and outright brilliance of quite a few people who are determined to see this drug earn the acceptance it deserves. If they profit as investors along the way, great. But I think the consensus of this team holds that "a solution for these boys" is the most important outcome. I wish a happy and safe Thanksgiving to all of you.
Agreed, Gary. Nice conclusion: an approved drug "is his legacy." We could do worse than have CG as our continuing advocate.
In the court of public opinion, the agency would risk being perceived as doing a back-room deal if they give us a green light without an Adcom. And if those same agency brainiacs from yesterday parse our data for our panelists, I think there is little chance the group would go rogue or renegade. I think the FDA knows what it has in our NDA, and the first part of their plan -- (dispatching drisa) -- has been checked off. The second part of their plan is to make themselves look really really smart for "finding" the first drug for DMD. The Adcom gives them the venue for that coronation -- and if they move up the date, all the better.
Is drisa relegated to the scrap heap?
Yes. Sarepta is now the only game in town.
Does the FDA want and need a DMD drug?
Yes. This is a closely watched battleground for FDASIA mandates, and the agency would be insane to reject a drug with efficacy and unblemished safety. The bd will be challenging, but the agency understandably needs to kick the tires before buying our vehicle.
What's the best investment strategy here?
People still have a hard time seeing Sarepta crossing the finish line. That doubt helps suppress our pps, which is really a gift in disguise. If you believe in the science and the company, you know things are really solid at this point. Everything else is just details.
(Thanks for yesterday, Stocktrooth. This board has its share of certifiables, but it also has some pretty good folk, of which you are one.)
I just read AF's story. He stuck a fork in the drisa turkey and deemed it cooked. That's about as good as it gets, given how he cites analysts still predicting a better than 50 percent chance of drisa approval. A few logs have been dislodged from the dam, and this thing will eventually give way. When Cramer goes long on us -- and he will -- that's the signal to mortgage the farm.
That's my guess too. The FDA brainiacs who picked the drisa turkey apart today know a good data set when they see it -- and they already know what etep's NDA is all about. Pushing our Adcom up makes perfect sense to them....
Where were these FDA brainiacs in 2013 and 2014? Strongly suggestive that higher-ups pulled our plug for reasons unrelated to our data??
He blogs about BMRN making "long term commitment to DMD" -- and then adds an illuminating parenthetical: "but where is the confirmatory trial?"
BMRN ain't gettin' no free passes from Adam today......
SERIOUSLY?? Sunshine laws were enacted for a simple reason: if you're not being transparent in your meetings, the perception is that skullduggery is being waged. Unreal.