. . . (April 30, 2015) "We begin 2015 prepared to execute on a number of transformational milestones," said Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin. "Having completed the submission of the NDA for approval of drisapersen in the U.S., we will turn our attention to filing for approval in Europe this summer. . . . Moreover, we believe a near-term approval of drisapersen will drive us to profitability on a non-GAAP basis in 2017.” . . . No doubt BMRN is expecting to cash in on Drisapersen’s Orphan and Fast Track status, as well as its Breakthrough Therapy designation by the FDA. . . .
. . . despite what is routinely posted here, r9, the FDA has not rescinded D's orphan, FT or breakthrough status, so I do see it as the preferred candidate . . . regarding pricing, it almost appears that some drug makers might be jacking up prices as quickly as possible in anticipation of some added form of cost control in the not-so-distant future . . . certainly pharma pricing will face a brave new world when the first real cures for genetic disease are approved here in the U.S. . . .
. . . if you really are serious, fr, let me clue you in . . . I have bought and sold this equity twice before over a period of years and currently own it again at about $32 . . . I am in it and BMRN as a hedged trade . . . I'll probably make money on good dris news but I'll likely make even more money on good etep news . . . if both of these slightly-better-than-placebo drugs are rejected, however -- well, that's another story . . . that said, the fact that you may actually think you fully understand either of these drugs or, for that matter, the motivations/truthfulness of anonymous posters on this board does not -- I think -- bode well for your stock trading over the long term -- beyond pure luck, that is . . . just sayin' . . .
. . . though I suppose the numbers could have been better . . . that said, it was the Q&A that was most interesting . . . lots of questions regarding dris and etep (though not specifically named) . . . one question, regarding toxicity, led to a lengthy, detailed response from BMRN execs who characterized the drug's adverse effects as moderate, seldom seen, and only temporary in duration in the 300 patients treated over the years . . . that's certainly different from what I see routinely posted on this board . . .
. . . actually, ge, BMRN execs did point out the need for genotyping all prospective patients before treatment . . . as for what specific genetic profile they'd be hunting, I have no idea . . . had you listened to the call, you wouldn't have to ask me . . . regarding etep, its relative safety profile -- and true efficacy, for that matter -- won't be fully known, imo, until they've treated a comparable number of patients -- say 300 or more over an extended period . . . regarding BMRN's ability to generate a return, that, of course, will also hinge on the treatment's price . . . I also would expect BMRN -- with little build-out -- to effectively field its product at a far lower cost per patient than srpt . . .
. . . could not care less about zf embryos, g3 . . . or, for that matter, what you characterize as kidney-threatening toxicity . . . the BMRN execs said that the few low-grade cases of np may not even have been attributable to the drug . . . as an investor in both BMRN and srpt, I only have to decide who to trust on this issue -- BMRN, a highly respected world leader in providing orphan drugs or some anonymous message board posters with an ax to grind . . . hmmmmmmmm . . . even if I only owned srpt, I would have listened to the BMRN cc . . . did you? . . .
. . . if srpt were truly the risk-free investment the round-the-clock pumpers here purport it to be, I don't think it would be sitting at a lowly $34 per share . . . that said, I do think the FDA at some point is likely to hold its nose and conditionally approve both slightly-better-than-placebo dmd drugs; hence, I am . . . (long the hedge: BMRN + srpt) . . .
. . . and praying that those nasty shorts take it even lower (why not $1?) . . . then you could really load up . . . you could remortgage your house, pawn your wife's wedding ring, and sell the family dog to raise more cash to buy-buy-buy! . . . and who wouldn't, if you know for a fact -- to hear some of you tell it -- that an FDA approval in six months (or sooner!) is an absolute, can't-miss sure thingy . . . (long the hedge: BMRN + srpt) . . .
. . . jeez louise, g3 -- enough already. . . I'll try to contact Ms. Furlong/PPMD myself . . . I swear, you people are like children . . .
June 8, 2015: "BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for drisapersen, an investigational antisense oligonucleotide drug candidate for the treatment of the largest subset of Duchenne muscular dystrophy (DMD) amenable to single exon skipping. . . . In Europe, it is estimated there are 23,000 boys with Duchenne Muscular Dystrophy, and approximately 3,000 of those would be candidates for drisapersen. In BioMarin's commercial territories, approximately 85 percent of Duchenne patients are located outside of the United States, including Western Europe, Middle East, Eastern Europe, Latin America and Japan. Western Europe has the largest patient population among those areas, exceeding the United States by around 30 percent. . . . The company recently submitted a New Drug Application (NDA) to the United States Food and Drug Administration (FDA) for drisapersen in April 2015.” . . .
. . . hats off to blue, indeed, b9 . . . but don't confuse its efforts to genetically engineer one-time, permanent cures for orphan diseases with the chemical therapies now being developed by srpt and bmrn . . . just sayin’ . . .
. . . you're not alone, p9, in questioning etep's efficacy . . . mr. market gives srpt a market cap of just $1.2 billion -- granted, that's a princely sum for sure, but it's really peanuts for a company that supposedly owns all rights to a DMD drug that's destined to be a clinical slam dunk . . . not sayin' it won't happen, but there's still plenty of risk between here and there . . .
. . . potentially more choices ahead for these patients . . .
. . . BPs have been partnering like mad with "real" genetic engineering outfits like BLUE because they hold the promise of one-time permanent cures . . . something not offered by oliglops . . .
. . . as I posted earlier . . . "on Monday . . . I sold more shares of BMRN and, for each, I was able to get 4.7 shares of srpt for my DMD hedge" . . . in other words, hr, I'm here because I own BMRN and trade in and out of srpt (now currently "in") -- not because I think etep's likely to be approved, but because there's probably decent money to be made on run-ups to binary events going forward . . . good luck with your bets . . .