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: Jess Fye - JPMorgan - Analyst
: Great. And maybe while the rest of the team is coming up here, I can just start with the first question. We're getting a bunch of investor questions
on the back of the litigation that you just announced. Can you outline the potential outcomes that could result from that litigation in Europe against
Ascendis? I think a lot of investors view this as sort of a best-case scenario as royalties to BioMarin versus actually blocking them. But is that accurate?
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JANUARY 13, 2025 / 5:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at JPMorgan Healthcare Conference
Question: Jess Fye - JPMorgan - Analyst
: Got it. Maybe a question for Greg and thank you for the presentation on the pipeline. Can you expand a little bit on the nonhuman primate data
for the BMN 333 and how that compares to other long acting CNP products? What levers do you have to accelerate development there? For example,
could you go straight from this Phase 1 to a pivotal trial? And then I just want to clarify, I think you mentioned PK data at a conference in the first
half of '26, but you get the data by the end of '25. When do we get the data?
Question: Jess Fye - JPMorgan - Analyst
: Maybe one for James, and I'll kind of keep it open ended here. What kind of business development deals do you want to do?
Question: Jess Fye - JPMorgan - Analyst
: Great. So maybe one for Cristin. Thinking about the real world, how are VOXZOGO's new treatment guidelines going to impact awareness and
uptake?
Question: Jess Fye - JPMorgan - Analyst
: Let's see. Alexander next. You started out the presentation by saying it's your 13-month anniversary at BioMarin. Of all the decisions you've made
over the past year or so, what do you think is going to have the most impact on BioMarin's growth strategy or trajectory?
Question: Jess Fye - JPMorgan - Analyst
: Maybe one for Brian. As the company becomes increasingly profitable, growing cash, how should we think about deployable cash over the coming
years?
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JANUARY 13, 2025 / 5:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at JPMorgan Healthcare Conference
Question: Jess Fye - JPMorgan - Analyst
: Maybe coming back to the pipeline on DMD, you kind of gave us the teaser talking about these two patients with measurable levels of functional
dystrophin. Anything you can quantify about those measurable levels, any more to share there?
Question: Jess Fye - JPMorgan - Analyst
: Great. With that, we are about out of time. So we'll leave it there. Thank you.
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