BioMarin Pharmaceutical Inc at UBS Global Healthcare Conference Summary

The following is excerpted from the question-and-answer section of the transcript.

Question: Eliana Merle - UBS - Analyst : <_ALACRA_META_ABSTRACT>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. Brian Mueller - Biomarin Pharmaceutical Inc - Chief Financial Officer, Executive Vice President - Finance Yes. Thanks, Ellie. Appreciate you having us. Thanks to those in the room for joining. Yes, it was a very exciting and dynamic first three quarters to 2024. Commencing with Alexander Hardy joining us, he took over his role on December 1 of last year as JJ Bienaime retired. And that was, frankly, right around the time where we entered into our settlement agreement with Elliot, and I bring that up because that involved the establishment of a strategic and operating review committee of the Board. This is a committee underneath the Board of Directors didn't have decision-making authority, but made recommendations to the full board. And that committee oversaw the comprehensive strategic review that we completed through September of last year. And we looked at every bit of the company strategically and where we could both extract and grow the most long-term value for shareholders. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 12, 2024 / 7:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at UBS Global Healthcare Conference First, looking at from a capability standpoint and from an asset standpoint, all of the great things that BioMarin has today that we can look towards leveraging more in the future and get additional growth potential. And then there's those what I perceived to be value accelerators like the growth potential for Voxzogo into these other indications where from a total addressable patient population the approved indication, achondroplasia and our planned second indication, hypochondroplasia are just 10% of the total potential addressable patient population. So there's 90% and we'll get more into the other indications course of the conversation, I'm sure. But my point is that was a major growth opportunity that we wanted to make sure we were accelerating as much as possible. Another area is cost structure and operating structure again, examined it deeply, looked at opportunities. We found some very beneficial mechanisms both balance and improve the cost efficiency of the company but also get better operating effectiveness. The primary example there is our establishment of new business units which are centered around certain of our products. You can think of these and other companies use the term therapeutic areas. But for us, that's the enzyme therapies which includes the five mostly enzyme replacement therapies, skeletal conditions which at the moment is Voxzogo but could grow into BMM333, our long-acting CNP and other skeletal conditions, indications and products and then a small focused business unit on Roctavian the gene therapy for severe hemophilia A. So that level of revenue and revenue analysis and growth potential work that we did as well as the cost structure examination really allowed us to confidently give significantly improved profitability guidance for the future. So over time, we'll be targeting non-GAAP operating margins in the low to mid-40% starting with 40% in 2026. That would be a doubling of last year's operating margin for 2023, our operating margin was a little over 19%. So targeting 40% in 2026. We'll be targeting $4 billion of total revenue in 2027 and a mid-teen revenue growth CAGR from there for the next 10 years through 2034. This is BioMarin. This is the first time BioMarin has given long-term guidance. We've historically been a high-growth company. But after doing all that strategic review, which included, by the way, the other core element was the innovation pillar of our strategy, really prioritizing our R&D, perhaps we'll get more into the pipeline. But we historically had a core for research and development strategy, which is known causation of disease, a targeted therapy, accessible endpoints and disease transformational, not just marginal improvement. We added a fifth core element. So now we talk about the core 5, which is commercial competitiveness. We want to make sure that assets can both access the markets, compete in markets if it's a competitive market by being first-in-class or best-in-class and have revenue potential that provides a return on the investment that it takes to get those drugs to market. So that was the new element. That led to the termination of five programs over the course of 2024. But the prioritization of three programs, which did meet that new high bar. So all in, it was a (inaudible) Amgen over 18 years. And our new Chief Business (technical difficulty) opportunity to accelerate our value through being more deliberate with business development. It's a great time for someone with James' track record to join the company. It really was end-to-end and top-to-bottom strategic review with a lot of great outcomes. We're now implementing the strategy and it's going well. We got -- still have a bit to go, and we have a lot of ambition, but it was -- it's a great process, and we're excited about the future.

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? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 12, 2024 / 7:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at UBS Global Healthcare Conference

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?

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.