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Expert Interview

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

A Second Opinion: Preparing for upcoming 3 year data on BioMarin's gene therapy ValRox in treating patients with hemophilia A

Ticker(s): BMRN, ONCE, PFE

Who's the expert?

Institution: Orthopaedic Hemophilia Treatment Center

  • Medical Director of the Orthopaedic Hemophilia Treatment Center and Hemophilia Foundation of Southern California.
  • Currently manages 120 hemophilia A patients and 40 hemophilia B patients.
  • Coordinates the federally funded Region IX Hemophilia activities for the Orthopaedic Hemophilia Treatment Center.

Interview Questions

Please describe your clinical practice; roughly how many patients with hemophilia do you currently manage?

Added By: joe_mccann

What are your expectations for the 3 years outcomes data to be presented at the upcoming ISTH meeting?

Added By: joe_mccann

The median factor VIII activity level was 89% after 1 year, but drifted down to 46% after 2 years. 

  • What is the minimum medium level you need to see here to be enthusiastic about the program?
  • Do you need to see a slowing rate of decline? By how much?
  • Is there any chance it could improve again by year 3, or is there only a chance to get worse?
  • If it stays about the minimum threshold to induce clotting is that enough?

Added By: joe_mccann

If patients median factor VIII activity level  continues to decline, how many years above the minimum threshold would be appealing to you? Your patients?

Added By: joe_mccann

What other key endpoints, beyond median factor VIII activity level are you most focused on?

Added By: joe_mccann

Do you view annual bleed rate or median Factor VIII activity levels to be a more important endpoint at the 3 year mark?

Added By: joe_mccann

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