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: Eliana Merle - UBS - Analyst
: Maybe just a high-level question before we go into all things. ERT and Voxzogo, you guys underwent a large strategic review and presented some
of these findings at the Investor Day. Maybe high level, recap sort of where you see the trajectory for the business and your confidence in this
trajectory in light of some of the debates around competition.
Question: Eliana Merle - UBS - Analyst
: Great. And I know everyone here is probably eager to talk about Voxzogo, but something I do want to talk about first is the ERT business. And in
particular, something I think that's not talked about enough is the mid- to high single-digit growth that you expect, which does imply somewhat
of an acceleration, but it's also notable considering the ERT revenues are probably some of the most durable that we see in biotech. But let's talk
about the specifics here. What gives you the confidence that this is a business that can grow in the mid- to high single digits for years to come?
Question: Eliana Merle - UBS - Analyst
: Maybe just if you can give us any more color on the product mix as well as the geographic mix of where you expect this growth to come from?
Question: Eliana Merle - UBS - Analyst
: All right. Great. Exciting things to come. Pivoting to Voxzogo, now competition here has been a major topic of conversation. How do you see the
trajectory for Baxogo putting together both the opportunity in achondroplasia, where you're currently growing?
But then also with expansion indications and seeing the recent Ascendis data, how should we think about sort of the mid- and long-term trajectory
of Voxzogo?
Question: Eliana Merle - UBS - Analyst
: Interesting. And when it comes to BMN 333, I guess you are maybe beginning to dose very soon or already have perhaps.
Question: Eliana Merle - UBS - Analyst
: Okay. How should we think about when we could see initial data? And maybe just how confident you are in this program and its ability to materialize?
Question: Eliana Merle - UBS - Analyst
: Okay. I won't press you more on that for now. Maybe just turning back to the near-term Voxzogo growth and thinking about the sort of overall
TAM size in achondroplasia, maybe just what have you been seeing recently? You've been doing some geographic expansion, but then also you've
mentioned growth in the US, particularly under ages five or up to age five. Maybe just what's the mix that you've been seeing in terms of growth?
And then from here, particularly without the supply constraints in the near term, kind of the contribution of growth from those geographies?
Question: Eliana Merle - UBS - Analyst
: And in terms of hypochon, I mean this is something that theoretically could be launching right at the same time that you would be facing increasing
competition in achondroplasia. So just putting those two dynamics together, how are you thinking about the hypochondroplasia market? And
specifically, what we could see in terms of how many patients are currently diagnosed, is this something we could see a bolus upfront for.
Question: Eliana Merle - UBS - Analyst
: All right. Great. Pivoting for a moment to the pipeline. So for 351 you've said that you will have the 26-week dystrophin data internally in the second
quarter next year. How should we think about when we will see this data and specifically what you're looking for in terms of potentially moving
the program forward?
Question: Eliana Merle - UBS - Analyst
: Awesome. Yes, we're looking forward to it. I know you have mentioned at Investor Day looking at business development, how should we think
about the size and potential timing?
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NOVEMBER 12, 2024 / 7:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at UBS Global Healthcare Conference
Question: Eliana Merle - UBS - Analyst
: Absolutely. It looks like we are at time. But thank you all so much for joining us.
Question: Eliana Merle - UBS - Analyst
: Thank you, guys.
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