. . . anything is possible, d7, including a buyout of this pig . . . but at what price? . . . not much, I suspect, as the FDA will likely put etep on a pretty short leash . . . that said, I've been wrong before . . . more interesting to watch, I think, will be etep's pricing if it gets the nod . . . wait for the hue and cry, if srpt tries to gouge . . .
. . . "Then lets add the fact that 37 of the last 42 drugs that the decision was delayed on were approved." . . . and your source for that pearl? . . .
. . . I'm mistaken? . . . really? . . . you sound like every other overly emotional retail investor who is in over his head on a long-shot bet -- a position in which you have limited information, a highly active imagination, and zero control over the outcome -- if you want to live in that world -- swell . . . it's no skin off my nose . . . but as I told mw (in more detail, below), I think etep has a small shot at approval -- that's why I hold shares in srpt -- for the chance at long-shot winnings . . . is it likely to pay off -- no . . . does my ownership satisfy any moral imperative -- absolutely not . . . it’s just a bet -- one that I can afford to make and lose -- to feed my need to gamble on occasion . . . understand? . . . verstehen sie? . . . capiche? . . .
. . . you, apparently, want to live in the world of pumper mania, mw . . . no matter how negative the facts, you folks read the tea leaves and concoct the flimsiest of positive spins and -- worse -- conspiracy theories to rebut reality . . . sure, etep has a small shot at approval -- that's why I hold shares in srpt -- for the chance at long-shot winnings . . . is it likely to pay off -- no . . . does my ownership satisfy any moral imperative -- absolutely not . . . it’s just a bet -- that I can afford to make and lose -- to feed my need to gamble on occasion . . . I remember taking a flier years back on Human Genome Sciences (HGSI) at $1.75 . . . they had a lupus drug finishing phase 3 trials, and analyst predictions, as I recall, were universally grim . . . I enjoyed selling the last of that position near $30 . . . long-shots can pay off, mw . . . just not very often . . .
. . . I don't see big pharma as a buyer, because they're already invested in gene therapy outfits that are likely to bury srpt's technology . . . even BMRN's latest successful effort in hemophilia A is a pure gene therapy play . . . I think the industry thinks -- as I do -- that oliglops are flops . . . we'll see . . .
. . . I don't think etep can win on its merits at this point, mw, so its only chance for a near-term aa, imo, may be political pressure; however, I think it's more likely that the FDA throws srpt a CRL softball, like some guarantee of aa in 12 months -- if the data continues to show a trend of clear patient improvement . . . that said, I, personally, don't think the drug works . . . I don't think the FDA thinks it works . . . but I also think that srpt knows that the window of opportunity is rapidly closing on this technology, as gene therapies -- offering the promise of permanent cures -- continue to make dramatic advances with big pharma support. . .
. . . "There are numerous pitfalls attempting to force Sarepta to treat an entire class of DMD sufferers for free via CU" . . . wrong again, g3 -- as usual . . . the FDA does not FORCE any company to provide a drug for free under CU . . . get the facts straight for change directly from the FDA website . . .
. . . the point, d9, is that the FDA's number one consideration is EFFECTIVENESS -- which according to the agency is something etep clearly lacks . . .
. . . agree, dk, that it's all about the money . . . but, personally, I'm lookin' for a four-fold gain before I dump this pig of a placebo . . .
. . . yeah, little marco is an absolute expert on dmd . . . in fact, I'm betting on misguided political pressure to push this pig across the finish line . . . (holdin' my shares, holdin' my nose, and hopin' for $60 and a quick exit) . . .
. . . last time I checked, s2, ONCE is not working on dmd . . . but others, who are, expect to start dosing patients next year (and I would expect dramatic results on dystrophin production within a matter of months) . . . just sayin' . . .
. . . you miss the point, d9 . . . Z was approved because it was shown to be highly effective against MS, though some (not all) may experience serious side effects . . . etep's problem appears to be an utter lack of performance . . . if it doesn't work, why bother with it at all? . . .
. . . the big feature story last night focused on a brother and sister who were going blind due to a genetic retinal dystrophy . . . now, after a single genetically engineered treatment, these kids are recovering their sight . . . the treatment, developed by Spark Therapeutics (ONCE), was described as part of a "new paradigm in medicine" for treating/curing genetic disease . . . it uses a harmless, adeno-associated virus to encapsulate and deliver the required genetic material into target cells . . . surely, the folks at the FDA must be loath to even consider approving a performance stinker like etep, when they are, in fact, witness to every aspect of this truly revolutionary new approach to creating cures for potentially every genetic disorder out there -- including DMD . . . just sayin' . . . (and, yes, I own ONCE as well) . . .
. . . since you're obviously not the sharpest knife in the drawer, g3, I'll say it once more --slowly: "watch . . . for . . . the . . . first . . . dmd . . . patient . . . to . . . be . . . dosed . . . with . . . a . . . potential . . . genetic . . . cure . . . in . . . 2017" . . . if you still don't understand, maybe you can find someone else to read it and explain it to you . . .
FDA website: "Wherever possible, use of an investigational medical product by a patient as part of a clinical trial is preferable because clinical trials can generate data that may lead to the approval of products and, consequently, to wider availability. However, when patient enrollment in a clinical trial is not possible (e.g., a patient is not eligible for any ongoing clinical trials, or there are no ongoing clinical trials), patients may be able to receive the product, when appropriate, through expanded access." . . . individual patients need only apply; however, srpt is not required to play ball, and I think they've already said as much . . . and, of course, insurers are likely not obliged to cover any associated costs . . .
. . . "they logically have to incorporate into their thinking what a CRL would mean for future investment in DMD" . . . more likely, mh, the FDA is fully aware of new genetic technologies that are rapidly coming on stream that very likely will obsolete sprt's antisense nonsense approach . . . why do you suppose big pharma partnering in this technology has all but dried up? . . . watch for the first dmd patient to be dosed with a potential genetic cure in 2017. . . just sayin' . . . (still holdin' srpt, holdin' my nose, and hopin' for $60 and a quick exit) . . .
. . . au contraire, f0, I lost money -- though mostly on srpt . . . but I prefer -- as a longtime biotech investor -- to take the long view . . . yes, I sold some BMRN at $140 to buy more srpt as a hedge and both got killed by the FDA . . . that said, my considerable stake in BMRN was originally purchased at $17 per share, and I had been selling it off since it broke $100 . . . and, as I posted here some months back, I sold the last of it to buy more BLUE, which is where I think the future of medicine is headed . . . just confessin' . . .