"Those muscles were supposed to be impervious to Eteplirsen." Really? Where did you read that diaphragm and heart muscle tissue was thought to be impervious to Etep.? Obviously, it would be unethical and medically risky (unless in primates) to sample these critical muscle fibers.
Not to throw cold water on your insight, but an off target positive effect isn't the same as an "adverse" event.
It's been my contention all along that the pulmonary benefits, evidenced later in Study 202 (which were not a clinical endpoint), would weigh heavily in favor of approval during AdCom. It remains to be seen if Etep will have a broad spectrum pulmonary benefit to DMD boys with other exon deletions, other than exon 51.
I'm totally with you on this! By rejecting FDA's guidance in and recommendation (in '14) about conducting a confirmatory trial, they snubbed their nose at FDA, saying essentially: "We're going to do it our way, and explain ourselves later, at AdCom." Bad idea, no?
No. FDA doesn't accept NDA's based on the potential approval of a drug. Accepting BMRN's NDA doesn't mean that FDA agrees with their data set, it just means that BMRN had all their t's crossed and i's dotted. BMRN knows how to do that.
IMO BMRN's gamble all along has been that 1) they would have first mover advantage b/c SRPT wouldn't get the NDA job done w/ the small N, (+) C.G.'s hx of contentious relationship with FDA. And/or 2) They would get royalties from $SRPT if Etep got approved. Seemed like a slam dunk to them! Looks like it's going to be a FAIL on both counts.
H2:15 Potentially initiate 2 confirmatory studies to support potential accelerated approval.
Apparently BMRN is going to seek AA first, and then agree to initiate confirmatory studies if AA is granted. Seems a bit on the overconfident side the equation. The FDA had already asked BMRN to conduct a confirmatory study in '14, which they declined to do.
I disagree: his attacks have only been to examine the short thesis. He's not cheerleading for either buy or sell side investors, but his leanings have been more positive than not for SRPT. All this talk of conspiracy w/ shorts is just speculative nonsense.
Not so sure about that; "Shorts are not fools." The hand writing has been on the wall for quite some time. They just chose to ignore it and stay entrenched in their Small-N mantra. Seems foolish to me!
Or, as an alternative theory, the data will be "noisy" enough so that the experts will agree that it's good, viewed through their lens, but Wall St. won't be impressed. It will be a push/pull and the stock won't have budged much at the end of trading, even though it will be +/- 15% throughout the day.
A PR tonite would only happen if there was positive "D" data, which Kaye has stated he thinks the FDA will be "comfortable" with; both re-reads and 4th Bx. We know the 6-mwt will show decline from previous test, but it will probably be better than natural Hx. Noisy data won't be PR'd, as you suggested. I think the 7am ET CC will give the market plenty of time to digest the news, either way. We'll know soon enough.
Hard to imagine that FDA would have any lingering doubts about approval if the upcoming data release (compared to natural hx) is overall positive. Clearly, the earlier tx begins the better outcome, but it will still put DMD boys in a Beckers DMD state, but significantly better than the alternative; their health overall vastly improved.
I think Roth is hedging the $50 pt b/c they are unsure about BMRN's ability to get Drisa past FDA AdCom
Not so sure about the "permanent" part. I think it's going to take more recovery time than usual. We're in deep correction territory here; more fear than greed, which always creates stronger sentiment, and makes things appear worse.
AQX-1125 looks extremely promising for it's anti-inflamatory properties in many different indications. The clinical benefits appear robust at the 12 week mark. The great unknown is what potential adverse effects might surface with long-term dosing. If no serious AE's develop over time, this drug will be a blockbuster, imho. The Baker Bro's apparently believe in the potential here. They aren't always right in their conviction; witness XOMA, but their winners outnumber losers over time.
Just to clarify about 6-mwt. It's not that this data isn't important, it is. A decline would be expected (short fodder in the past), but if the decline is significantly better than natural history, then it's a win. Positive "D" data will also be a win, and pulmonary data (even thought it wasn't a clinical end-point in Study 202, will also be a win.
In totality, this data is what Ed Kaye referred to as the reasons why: "....it's not unreasonable to expect AA."
Let's review. Here's where we're at w/ Fast Track and Rolling NDA
1) More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
2) More frequent written correspondence from FDA about such things as the design of the proposed clinical trials
3) Accelerated Approval: meant for drugs that demonstrate an effect on a surrogate, or intermediate endpoint reasonably likely to predict clinical benefit.
4) Rolling Review: a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed.
At WMS, forget 6-mwt data; expect continued slippage. All that matters is this: If the 4th Bx data and re-reads of previous Bx data are strong (see #3 above), then AA is a done deal, as Ed Kaye implied at the recent Morgan Stanley presentation. Kaye: "Based on the data, and based on FDASIA, it's not unreasonable to expect accelerated approval."
Cobbled these together from what I heard and a couple other sources, who were also listening.
With after hours trades, SRPT up 3 bucks today. Not bad. Hope data from WMS doesn't disappoint, but Kaye did say AA was definitely possible with what data FDA has.
Kaye also said that they are committed to addressing treatment for the other 8 exons (6 of which are smaller subset of DMD boys), and that they are in discussions with FDA to further this objective. He sounded solid, fielded all questions extremely well, and seemed relaxed and confident. Who needs a CEO when Kaye has the reins?