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Thread: Spark SPK-9001 Gene Therapy Trial "Effectively Cures Hemophilia B"

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    Spark SPK-9001 Gene Therapy Trial "Effectively Cures Hemophilia B"

    I have the print edition of Winter 2017 Hemaware Magazine in front of me... its not up yet on the website; will bump when its updated

    https://hemaware.org/current-issue

    Front cover says:

    GENE THERAPY BEGINS TO FULFILL ITS PROMISE


    Spark and BioMarin both released data saying that they could effectively cure patients with hemophilia. That's a watershed.

    here's some snippets from the web:

    http://www.satprnews.com/2017/02/22/...ss-highlights/

    highlight of the year was the release of encouraging investigational SPK-9001 Phase 1/2 trial data that give us hope that we may be able to eliminate the need for regular infusions to control and prevent bleeding episodes for people living with hemophilia B
    http://www.economiccalendar.com/2017...apeutics-once/

    Spark is also developing a hemophilia B treatment, in a collaborative effort with Pfizer Inc (NYSE:PFE), called SPK-9001. The product was developed for patients so that they don’t need to undergo regular infusions to manage and prevent bleeding episodes, and has been granted breakthrough therapy designation by the FDA.
    The two companies just finished enrolling ten patients in a Phase 1/2 trial evaluating SPK-9001, and Spark already announced that nine of the participants have observed a beneficial effect from the treatment. Eight of those nine patients experienced a 100 percent decrease in the use of factor concentrates an in the number of bleeding episodes.
    http://www.streetinsider.com/Analyst+Comments/Spark+Therapeutics+(ONCE)+PT+Raised+to+$76+at+Stif el/12583754.html

    Stifel analyst Stephen Wiley reiterates Buy rating and raises price target on Spark Therapeutics (NASDAQ: ONCE) to $76 (from $73), as the company reported a quiet 2016 fourth quarter ahead multiple data catalyst for the first half of 2017.
    Click image for larger version. 

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    My son has Hemophilia B; aside from bleeding episodes and IV therapy every 2-3 days, his "cost of care" is between $500k and $1m annually.

    The annual cost of care for the 50k Hemophilia B patients in the US alone is about $50 Billion; about the size of the US trade deficit with Mexico. There are 4 times as many Hemophilia A patients.


    more on BioMarin's BMN-270 trial "cure" for Hemophilia A

    Over the weekend BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its positive interim results of an open-label Phase 1/2 study of BMN 270, an investigational gene therapy treatment for severe hemophilia A, which will be presented as part of a company overview at the 35th Annual J.P. Morgan Healthcare Conference in San Francisco, Calif. These data are an update from previously reported results presented in July 2016.

    A total of nine patients with severe hemophilia A received a single dose of BMN 270, seven of whom have been treated at the highest dose of 6 x 1013 vg/kg. As of the Dec. 9, 2016 data cutoff, post-treatment follow-up ranges from 34 to 50 weeks. Median Factor VIII levels for the high dose cohort have been consistently within the normal range from 20 weeks through 44 weeks of treatment. (See Table 1) For those seven patients, as of each patient's most recent reading, six of seven patients continue to have Factor VIII levels above 50%, as a percentage calculated based on the numbers of International Units per deciliter (IU/dL) of plasma, and the seventh continues to be above 15%. (See Table 2)


    For the six patients at the high dose and previously on a Factor VIII prophylactic regimen, the mean annualized bleeding rate dropped 91% from 16.3 before the BMN 270 infusion to 1.5 two weeks after being dosed (median annualized bleeding rate dropped from 16.5 to 0). For those same six patients, the mean annualized Factor VIII infusions fell 98% from 136.7 to 2.9 (median annualized Factor VIII infusions fell from 138.5 to 0). (See Table 3)


    Since the last update, six of the seven patients at the high dose as of the most recent reading are within the normal alanine aminotransferase (ALT) range, and one patient is less than 5% above the upper limit of normal, which is 43 U/L for the central laboratory in this study. (See Table 4) Patients successfully tapered off of steroids with no lasting significant impact on Factor VIII expression or ALT levels. The requirement for prophylactic corticosteroids has been removed for all newly enrolled patients in this study.


    Study medication was generally well tolerated. No serious adverse events were observed, and most common adverse events were mild in severity.

    The next steps for the program are to begin a potentially registration enabling Phase 2b study in 3Q 2017. In addition, the company is expected to commission its commercial gene therapy manufacturing facility by mid-2017.


    "These data continue to show promising evidence that restoration of clotting function can be achieved by gene therapy,"
    http://www.streetinsider.com/Confere.../12405628.html
    Last edited by presence; 02-25-2017 at 12:08 PM.

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  3. #2
    Wow. How fantastic would that be!



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