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left2rightdoor 87 posts  |  Last Activity: Feb 27, 2015 12:14 PM Member since: Jul 16, 2012
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  • Mendell also talked about eteplirsen at congressional briefing, McDonald knows natural history of progressive disease abnd Muntoni know complexity of dystrophin expression - more new info for sarepta coming this month w Feb 3 Webinar and Feb20 FDA workshop
    March 3, 2015 from 1pm to 2pm ET
    This webinar will review the basic biology of dystrophin (the gene, structure-function of the protein, its role in the dystrophin-glycoprotein complex, characteristics of different forms of dystrophin (both naturally occurring and drug-induced truncated forms), discuss how dystrophin is measured in both preclinical studies and clinical trials, and discuss the need for and role that dystrophin analysis play in clinical trials of novel agents designed to exert their therapeutic effects through an increase in dystrophin levels. We will also discuss what is required in order to have dystrophin considered as a surrogate

  • left2rightdoor by left2rightdoor Feb 26, 2015 11:06 AM Flag

    with guidance around $2.90-$3, free cash over $200 (and a movie) cheap ATP

  • This latter interest culminated in a Department of Health funded grant (2005) to establish a consortium for a phase I/II therapeutic trial of antisense oligonucleotides in Duchenne muscular dystrophy (the MDEX Consortium). This study was completed in 2008.

    In view of the exciting data generated by this project, in collaboration with AVI Biopharma (/) we have obtained an MRC translational research grant to assess the safety and efficacy of morpholino antisense oligonucleotides repeated intravenous administration into young boys with Duchenne. This study will be completed in 2Q2010 and further MDEX collaborative clinical and preclinical studies in this area are being planned.

  • The NIAID is seeking information regarding efforts toward the development of therapeutic products that demonstrate activity in appropriate in vitro assays or in vivo models for the treatment of diseases on the NIAID Category A, B and C Priority Pathogens list (see link) More specifically, this RFI solicits information on activities being undertaken toward the development of therapeutic candidates/products, which have demonstrated feasibility of manufacturing, in vitro and in vivo evidence of efficacy, and sufficient characterization to allow the development of a draft target product profile. Candidate products of interest are those with broad spectrum therapeutic activity against viruses or bacterial pathogens and promising anti-toxins as therapeutic products.

    What company do we know that has a broad spectrum antiviral?

  • Reply to

    BCRX, BARDA discussions, WSJ: BCX4430

    by maphere Feb 11, 2015 9:40 AM
    left2rightdoor left2rightdoor Feb 20, 2015 1:27 AM Flag

    Biocryst was mentioned in a White House Fact Sheet on Feb 11

    For Immediate Release February 11, 2015
    FACT SHEET: Progress in Our Ebola Response at Home and Abroad

  • left2rightdoor left2rightdoor Feb 19, 2015 6:24 PM Flag

    Would like to get to 24 based on Rapivab sales, ongoing 4430 funding and IND submission and Opus 2+ trial pending FDA approval of HAE before the buyout at 60

  • Reply to

    How are they accounting for Rapivab

    by left2rightdoor Feb 18, 2015 11:48 AM
    left2rightdoor left2rightdoor Feb 19, 2015 1:38 PM Flag

    Relistening to the webcast at about 15:00, they sold to hospitals in 4 days $33,000, using an accounting method they call Sell-Through-Rep. So that was about $8000 a day, before CDC got on the big use antivirals kick. Because they used Sell-through rather than Sales-To-Specialty Distributors (when shipped/sold to dist) they had a deferred Rev amt of $5.6 million. Had they counted those this Qtr, they would have had a net loss of $11.7-$5.6, or about $6 million, or maybe .08-.09 per share. By conservative accounting, they will get
    all the bump from sales in the next Qtr. which could push them close to breakeven, or even, god forbid, profitability. With quarterly expenses of $18 million for R&D and $2 of G&A and interest of another $2 they have to get to revenue of about $20-25 million to breakeven or turn corner. Rapivab sales of more than $20 million along with NIAID funding for 4430. Some of those revenues are shown in the Net operating cash use of $65-80 million v. operating expenses of $75-95 million Sales revenue and govt funds must be the difference.

  • left2rightdoor by left2rightdoor Feb 18, 2015 11:48 AM Flag

    $33,000 in a few days, $5.6 million in deferred receivables? For product sold to distributors. What did they sell on a deferred basis 10,000 at $500? These analysts can't ask basic questions.

  • On February 12, the National Institute of Allergy and Infectious Diseases (NIAID) exercised an additional option under its BCX4430 development contract, which provides $2.7 million to BioCryst for I.V. BCX4430 investigational new drug ("IND") enablement and submission. $25.0 million of option funding has been awarded to date under the NIAID contract, which totals $29.1 million, if all options are exercised.

  • left2rightdoor by left2rightdoor Feb 17, 2015 11:40 AM Flag

    SRPT gets downgraded, big institutions move in with 13Gs, THEN BofA decides eteplirsen likely to get approved. Coincidence, think not. Just manipulation by big institutions.

  • left2rightdoor by left2rightdoor Feb 14, 2015 1:21 PM Flag

    The US Department of Agriculture says the avian influenza found in a flock of chickens in Parma (Idaho) last month [January 2015], came to Idaho from Southeast Asia.

    Idaho's State Veterinarian Bill Barton says the sickness was likely carried here by wild waterfowl like ducks and geese. Avian influenza is fairly common in wild birds. While Barton says they have a high tolerance for it's often lethal in domestic poultry. So far, no bird flu strain that has been found in Idaho is harmful to people.

    "With the migratory routes of wild birds in Asia, and then the migratory routes from Asia over to the US, there's a lot of co-mingling of birds," Barton says. "Among poultry and wild birds, avian influenza is easy to pass from one bird to another."

    Barton says bird flu is most commonly transmitted through feces. A migrating bird could easily get the virus by stepping in its Asian neighbor's waste and later pass it to an Idaho chicken through its own.

    He says the strain of bird flu found in Idaho likely came down what's known as the 'Pacific Flyway.' That's a migratory path from the Arctic, down through the western US and beyond.

    Jeff Knetter, Idaho Fish and Game's top bird expert, says that means there are a lot of suspects for who could have brought bird flu to Idaho.

    "There are a variety of species that sort of co-mingle along the Bering Strait, northern Alaska, or even northern Russia," he says. "Those could be snow geese, they could be northern pintail, mallards, green winged teal species, American wigeon -- all species that you can see in the Treasure Valley [western Idaho and eastern Oregon]."

  • Columbia Wanger Asset Mgt 3.272 mill shares, 7.9%
    Blackrock 3.2 mill shares, 7.8%

  • For pandemic influenza - seems like a market for Vaxfectin

    olicitation Number:
    15-100-SOL-00003
    Notice Type:
    Solicitation
    Synopsis:
    Added: Dec 18, 2014 3:43 pm

    Medical Countermeasures for Pandemic Influenza Preparedness and Response

    Human influenza pandemics continue to be a public health concern. Four human influenza pandemics have occurred in the past century. At this time it is uncertain which influenza virus variant will cause the next pandemic.

  • rumors that the king of all tech companies, Apple, is working on a Web-based pay TV service of its own. This would appear to be an idea whose time has arrived.

  • left2rightdoor by left2rightdoor Feb 9, 2015 10:03 AM Flag

    In their report, “Human Mobility Networks, Travel Restrictions, and the Global Spread of 2009 H1N1 Pandemic,” published in the journal PLOS One, the scientists offered a dire conclusion: “It is unlikely that given the ever-increasing mobility of people, travel restrictions could be used effectively in a future pandemic event.”

  • This RFI specifically seeks information for qualified pharmaceutical manufacturers with drug manufacturing expertise and experience capabilities with the ability to provide antiviral medication to support the United States of America and the continuity of government. The antivirals must be approved by the Food and Drug Administration and demonstrated by the Centers for Disease Control to be effective in treating Influenza A, H1N1 Influenza and H5N1 Avian Influenza.

  • Rapivab for 1 day delivery

  • Why can't yahoo do the same- anybody who posts over 4 messages a day should get sequestered.

  • Yahoo at 10.9% marked Yahoo's highest U.S. search share for more than five years, according to the StatCounter report. Default for Firefox, going for Apple, Core will prove out incremental $15/share suggested by Goldman prior to Spinco announce.

  • Too complex, thorny to take over Yahoo. That's what hedge funds are for, to execute difficult binary event transactions that produce, release incremental value. Here the incremental value is above $10-15 billion.
    There are financial engineers and law firms that can do that. Goldman alluded to that in their pre-Spinco "neutral" rating, saying Spinco was worth $15 incremental per share, and remaining business about the same.

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