. . . maybe his optimism is also driven by the obvious added pricing power Mysimba would have in Europe . . . much like arna had here -- until they fumbled it away with an underfunded, uninspired launch . . .
. . . it's yet to be determined which of these technologies might ultimately dominate, but clearly big pharma is scrambling to form partnerships as quickly as possible -- pfizer with cellectis, celgene with bluebird, biogen & shire with sangamo, novartis with the university of pennsylvania, to name just a few . . . 2015 looks to be a watershed year . . .
. . . “Duke researchers have demonstrated a genetic therapeutic technique that has the potential to treat more than half of the patients suffering from Duchenne Muscular Dystrophy (DMD). One of the challenges of treating DMD is that the disease can be caused by mutations to a number of different DNA sequences, few of which occur with any substantial frequency. The new technique, however, gets around this sticking point by targeting a large region of the gene that contains many different mutations. The study was published on Feb. 18 online in Nature Communications. “There are no effective therapies currently available for people with DMD,” said Charles Gersbach, assistant professor of biomedical engineering at Duke University. “They have nothing to stop this right now -- and we’re trying to work on that.” . . . “While some clinical trials are under way for drugs that restore dystrophin production from mutant genes, they only target one exon and thus can only be used for up to 13 percent of DMD cases. The new approach, however, can theoretically target any region of the gene of any size. In this study, the researchers targeted a span of 11 exons that account for 60 percent of DMD cases.” . . . “Over the past decade, researchers discovered the CRISPR system that bacteria use to slice the DNA of invading viruses. Soon after its discovery, researchers hijacked the system and began using it to fix mistakes in the human genome.”. . . . for the full story, google Duke Today, Bacterial Defense Mechanism Targets Duchenne Muscular Dystrophy . . .
. . . "just traded the (OREX) approval and got out the day before" . . . and yet you've held this pig through thick and -- mostly --thin? . . . too funny . . .
. . . "as for no factory that means they pay a fee to manufacture Contrave that is not exactly what u should consider a plus" . . . really? . . . I seem to recall that -- according to the partnership agreement -- OREX provides Contrave to Takeda -- and I doubt that they would do that at a loss . . .
. . . before the Mysimba decision . . .
. . . "While the U.S. launch is still in its early stages, we are encouraged by the progress so far and by the positive reports we are hearing from Takeda's field based teams about how the product is being received by health care professionals and their patients." . . . Narachi continued: "In 2015 we are focused on the continued success of Contrave in the United States, securing approval for Mysimba in Europe, and taking concrete steps toward commercialization of Contrave/Mysimba in ex-US territories." . . . considering the company’s low overhead costs, profitability should be a strong suit for OREX going forward . . . we'll see . . .
. . . am very, very disappointed, Mr. WS Exec . . . after pumping this pig forever, you could finally have throw us a bone with some value on it . . . but, so be it . . .
. . . come on Mr. WSE -- where's that list? . . . time is money and I haven't got all day . . . I think h.r might like to peruse it as well . . .
. . . self-deception is rarely a successful investment strategy . . . if Drisapersen didn't still merit BTD, the FDA would have rescinded it -- and, more importantly, BMRN would not have bought the company . . . this is not rocket science . . . just common sense . . .
. . . so, Mr. WS Exec, with the mathematical/scientific background that has enabled you to do your own research aka due diligence, what other bio/pharmas have you deigned to purchase that are currently winners -- i.e., not losers, like this one? . . . as a professional, you must have a portfolio that is truly a paradigm of sector diversification . . .
. . . what harm, et? . . . arna currently has a BILLION dollar market cap that -- based on its fundamentals and operations going forward -- hardly seems justified . . . that said, Feuerstein is obviously not a fan of arna at this time; but if the company were to deliver in a big way, I think you'd see him change his outlook . . .
. . . unlike you, sp, the FDA had all of the data -- and they made the call . . . go to fda.gov and give yourself an education . . . it's free . . .
. . . that's obviously not how the FDA sees it, sp . . . many companies have applied for "breakthrough" status but few have been granted (see for yourself at fda.gov) . . . and though that special treatment can be rescinded, it's only happened a few times . . . we'll see . . .
. . . "Arena Pharma (ARNA . . .) and Vivus (VVUS . . . ) are falling.” Adam Feuerstein, 2/20/15 . . .
. . . "Drisapersen has Orphan, Fast Track and Breakthrough Therapy designation and is currently under rolling review as part of a New Drug Application (NDA) with the Food and Drug Administration. Drisapersen, a potential first-to-market and best-in-class product for treating a large population of patients with a rare, fatal genetic disease represents up to 10,000 DMD patients. . . . BioMarin expects to complete the NDA submission for drisapersen in April 2015". . . the "BT" designation pretty much says it all at this point . . .