The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Aspen Mori - BofA Merrill Lynch, Research Division - Analyst
: Yes. So a couple on the WFH update. I guess, first off, maybe you could keep elaborating. It's interesting to hear some of the feedback you got
specifically on the 4-year data from WFH, obviously, is a virtual. So couldn't get a whole lot of doctor feedback on that. But -- and then in addition
to that, there was maybe a thought that the low-dose data at 3 years declined by maybe a rate that's a little faster than some people had expected.
Maybe you could talk about that and what your read-through there is for the overall valrox profile and durability?
Question: Aspen Mori - BofA Merrill Lynch, Research Division - Analyst
: Okay. And then you mentioned earlier that numbers don't matter so much. How the patients are doing, whether or not they're bleeding or -- I
mean, it's not like they're obviously not, but I guess going forward, do you think that when we're considering the competitive landscape in the
gene therapy hemophilia space, rather than focusing on the fact rate levels, do you think that do -- does it make sense to go to compare bleed
rates? Or is there maybe another metric that you think might make sense to say to stack up between 2 indifferent therapies?
Question: Aspen Mori - BofA Merrill Lynch, Research Division - Analyst
: New standard. And Hank, from a life cycle management perspective, obviously, you have the launch ahead of you, but what would you say for
ROCTAVIAN indication base? Is it expanded in hemophilia through different age cohorts? Is it more on the AAV5 delivery system? Just a word on
that.
Question: Aspen Mori - BofA Merrill Lynch, Research Division - Analyst
: This is Aspen again. Maybe to follow-up on that and to zone in on the gene therapy 307. How are you guys thinking about the competitive landscape
there in terms of the other competitors developing gene therapy? And how is this discussion different than maybe the ones you had over the past
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JUNE 24, 2020 / 3:30PM, BMRN - Biomarin Pharmaceutical Inc at Bank of America Merrill Lynch Napa
Healthcare Conference (Virtual)
few years about valrox versus its competitors? Is there anything that you would highlight there to, I don't know, maybe I guess, avoid the new
pickiness that kind of happened over the last couple of years with the factor VIII goals?
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