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Expert Interview

Slingshot members are talking to an expert! The topic is:

Making sense of BioMarin's 3 year update on ValRox in Hemophilia patients

Ticker(s): BMRN

Who's the expert?

Name: Dr Guy Young - MD

Institution: University of Southern California

  • Director of the Hemostasis and Thrombosis Center at Children’s Hospital Los Angeles, Professor of Pediatrics at USC & Chair of the Scientific and Standardization Committee on Factor VIII, Factor IX, and Rare Bleeding Disorders of the International Society on Thrombosis and Haemostasis.
  • Currently manages 320 patients with hemophilia A and B
  • Research activities encompass clinical trials in hemophilia, the use of novel anticoagulants in children, and the development of the global hemostasis assays to monitor the effects of medications used to treat bleeding in hemophilia.

Interview Questions
Q1.

What was your impression of the recently updated 3 year patient data for ValRox?  LINK

Added By: joe_mccann
Q2.

More detailed questions to come once community interest is demonstrated!

Added By: joe_mccann
Q3.

How do you view the three year chromogenic factor expression update?

  • Median 1/2/3 year expression (IU/dL): 60.3, 26.2, 19.9
  • Mean 1/2/3 year expression (IU/dL): 64.3, 36.4, 32.7

  • Added By: joe_mccann
    Q4.

    Biomarin discussed on the call an internal model that projects the reported three years of expression levels will translate in to 8 years of a durable effect. I believe BioMarin is using non-hemophilia A specific gene therapies to model this forecast - do you put in any weight in to BioMarin projecting out 8 years of durable benefit with the data generated? Would this have any impact on your willingness to prescribe?

    Added By: joe_mccann
    Q5.

    What was your general view of the update? Did the update make you incrementally more positive or negative on ValRox?

    Added By: joe_mccann
    Q6.

    Biomarin commented that it believes expression levels are plateauing, would you expect the 4 year update to decline or show the plateauing?

    Added By: joe_mccann
    Q7.

    What do you make of the median expression continuing to decline but the mean expression somewhat stabilizing? Which measure is more meaningful?

    Added By: joe_mccann
    Q8.

    Any other thoughts on the three year update for the phase II high dose?

    Added By: joe_mccann
    Q9.


    What do you make of the other three year data updates: 96% mean reduction in factor usage and 96% reduction in annualized bleeds?

    Added By: joe_mccann

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