All right. Good morning, everyone. Thank you so much for joining us. My name is Whitney Egem. I'm one of the biotech analysts here at Canaccord, and it's my pleasure to introduce Biomarin Pharmaceutical this morning. And on behalf of Biomarin, we have Brian Mueller, CFO. Thank you for being here. So just to start off at high level, for anybody who's not familiar, for anybody who that might be, I don't know, Biomarin is a household name, but for anybody who is not familiar, can you briefly introduce the story? Like, who is Biomarin currently, and where are you trying to go over the next five, ten years?

Yes, thanks, Whitney, for having us. Really glad to be here. Thanks, everyone joining in person and those online. Yeah, so I do think there's some folks that may not be familiar with Biomarin, so I thought I'd give a little bit of maybe the past, present, and future, and then we can get into more details in the business. So Biomarin Pharmaceutical is one of the largest rare disease focused companies. The company's been operating for a bit over 25 years. And it's interesting, back when we started our journey, there were only a handful of rare disease companies. There was Biomarin, there was Genzyme, Shire, TKT. However, those other companies are now part of larger pharmaceutical companies because they were acquired over the years. And while now there's hundreds of other rare disease companies, BioMarin spent the last couple of decades plus growing and scaling while retaining that rare disease focus. We've got six first-in-disease medicines, nine approved therapies overall. We've got higher-than-average success rates in the biopharmaceutical sector, and that's largely because of our focus, not just rare disease, but genetically defined conditions. So we ensure when we embark on research programs that we understand the underlying genetic causation of the condition. And then we design a precision medicine aimed directly at that genetic cause. That comes with a number of competitive advantages in biopharma. First of all, rare disease, patient populations are often smaller, which means smaller studies, which can be run in shorter amounts of time. There are certain regulatory incentives like orphan drug protection and other accelerators in the regulatory pathways, and a lot of community support. So rare disease patient advocacy is very strong. So we partner with regulators, the patient communities, and that's been a significant part of our success. But now we've grown and scaled the business into close to an expected $4 billion of revenue this year. We've built global, world-class biopharmaceutical capabilities. We have in-house manufacturing, end-to-end research and development and commercial capabilities. We operate in over 80 countries worldwide. So again, rare disease at scale and one of the leaders. Shifting into the present, we just reported our Q2 financial results last week. We had nearly $1 billion of revenue for the quarter, which was 20% growth year over year. We raised our guidance to $3.875 billion to $3.925 billion for total revenue. We also raised our non-GAAP earnings per share guidance from $4.90 to $5.10. Another significant announcement last week was in closing the acquisition of Amicus Therapeutics during the quarter, we shared our plans for the Amicus business. Amicus is a rare disease-focused company based here on the east coast. They had two commercial assets, Gallifold for Fabray disease and Pombability and Upholda for Pompe disease. These are both high growth assets and very complementary to BioMarin's business. We announced the acquisition back in December and closed it during Q2. Part of the strategic rationale for this transaction was taking these two high-growth medicines in a small company into the Biomarin infrastructure and global platform where they should actually be more valuable. And what we shared last week was our aspirations for the Amicus business. We shared our view on peak revenues for Gallifold, which is $1.4 billion, and for Pombility and OpFolda, $1.2 billion, both in the mid to late 2030s, respectively. We shared our view on synergies. We believe that we can synergize approximately 50% of the legacy Amicus operating cost base. Again, two companies together, Bomberin at scale. We have the platform. So when it comes to infrastructure, Most of the synergies are coming from general and administrative expense and other business support. That's where the bulk of this 50% synergies comes from. Very importantly, we preserved and left intact, and if anything, we're investing in the amicus sales and marketing, all of those customer-facing capabilities. Again, these products are in their high-growth phase, so it was important that we preserve that growth and invest in it. So we weren't aggressive with any synergies within sales and marketing. So with that revenue growth profile of the Amicus business and with some of those cost synergies, what that results in is a substantially accretive business over time and significantly increased cash flows. We shared that we accelerated our deleveraging target. Biomarin took on leveraged debt for the first time to do this acquisition. And at the time of the acquisition, we shared a leverage ratio target of less than two and a half times within two years of closing the transaction, which was, again, just back in April. After doing our work on both that revenue growth and the synergies, we accelerated that deleveraging target by roughly a year. And we think we can be at that less than two and a half times by the middle of next year. Then the last thing on the accretion is, again, the combination of the revenue with those cost synergies. Over time, over the next few years, say by 2030, we think the amicus business can operate at a 60% operating margin, also contributing to what is already a healthy biomer in operating margin. So that's some of the present. And in the future, our strategy is to realize the growth potential of the business. lots of commercial execution to do both on our, you know, base legacy business and in successfully growing and completing the Amicus integration. I should have mentioned in the sort of past to present, we also transformed the company over the last couple of years. Significant changes in our focus, in our operating model. We substantially improved our profitability and cash flow. It was great timing that we did that because now when we layer on the Amicus business, It's on top of this re-engineered, transformed, bio-marin operating model. And so we're expecting to generate significant growth in revenue, earnings per share, but most importantly, cash flow, because cash flow is the vehicle to be able to reinvest in the business. Our top capital allocation priority is to continue to invest in future growth and thereby shareholder value. We talk about sometimes this virtuous cycle where we're growing and reinvesting in the right assets, both our internal innovation and inorganic business development, external innovation, continuing to grow and reinvesting. So, again, this virtuous cycle with a flywheel effect, that's the strategy.

Excellent. Okay, very, very helpful and a lot to dig into there. I think we'll stick with Amicus and start there. So as you mentioned, de-levering one year sooner than originally thought. You touched on it, but can you give a little bit more color around what is driving that? Is it really upside on the revenue front? Is it more synergies, or are there other levers that are kind of different as you got in there versus more close?

Yes, of course. Thanks, Whitney. Great question. Yeah, it's multiple levers. So I might start with where I kind of finished the opening remarks with that transformed Biomarin-based business. We've gotten to a level where we're generating not just significant levels of operating cash flow, free cash flow, but that EBITDA base for leveraged debt. So even without the amicus business, we've got a healthy, growing, solid base of cash earnings, if you will, and growing into next year. On the leverage target itself, I'll share that with Bomarin as a first-time debt issuer, it was very important that we go on the record at the announcement of the transaction with a sound financial policy. That was that less than two and a half times within two years. I will share that there was some room in that. If you were to model out our EBITDA in the debt we raised, it was about $3.6 billion. We also have a $600 million convertible note on the books, but that actually matures next May. So that helps deleverage a bit in itself. So it was a healthy situation to begin with, is my point. And then to your question, once we layer on the amicus business, because we are growing revenues, we think there's more of an opportunity than, again, amicus standalone. We can get more into what those levers are as well. but then with the cost synergies, the combined EBITDA profile over the next 12 months really got us comfortable that we can accelerate that deleveraging target.

Okay, really, really interesting. And then going back to the revenue guidance for Gallifold and POMOP, I'm just going to use the short version.

Sure, I might as well.

Yes, exactly. 1.4 billion for Gallifold and 1.2 for POMOP. I think that was ahead of consensus and kind of how we had all been thinking about it. So can you talk us through what's driving that as well? And maybe as you're doing that, probably part of the answer is around retaining the existing salesperson relationships and just kind of help us understand how all that works together.

Yeah, of course. Yeah, maybe picking up where you left off. So really important to retain, preserve and grow the capabilities because both of these medicines are still in their high growth phase. the capability that applies to both Gallifold and Palmop is the Biomarin global scale and capabilities. There's a couple elements of that. First is global reach. I mentioned a few moments ago that Biomarin commercializes its product in 80 markets globally. Amicus had previously commercialized Gallifold in about 40 markets and POMOP in about 15 markets. So that global expansion is a huge opportunity for us. To be clear, we're not expecting to take both products into all 80 markets. Some of those are small and unique to just a couple of assets in our portfolio, but nonetheless, the pure geographic expansion is one lever. Second is just some of those global capabilities. It's not uncommon for earlier stage commercial companies as they're growing when they do expand internationally to use a partnership distributor model. Biomarin does use distributors, but in most of our countries, we've got Biomarin capabilities on the ground. This is both marketing and traditional commercial, market access, medical, regulatory when you get out to several of these international markets those are very complex they take time but biomarin again has been doing it for for over 20 years so that should also be not just an accelerant but an improved capabilities and then a couple nuances on the on the medicines themselves for Gallifold and Fabray it's believed that Fabray is still significantly underdiagnosed And this has been one of the areas where Biomarin has built outstanding capabilities over the years, which is improving diagnosis. When I mentioned in my opening remarks that six of our medicines were first in disease, that's not just novel science in bringing a therapy to these disease areas that had no therapy available before. On the actual commercialization front, we actually had to build those markets. You know, there's often not a treatment home or, again, advocacy may be dispersed. So building these markets is what Biomert has done. Fabray, Gallifold was not the first to market, so you can say that the Fabray market was already built. But this underdiagnosis, we believe, is a big opportunity. We're going to be working on things like AI-enabled patient identification, newborn screening globally and in the U.S. That's done at the state level in the U.S. And then familial genetic line screening because Fabray is genetic and can manifest itself in different levels of severity, in different levels of symptoms. It's been seen already that if you do genetic family cascade screening, one patient can lead to many. So those type of efforts, again, this is the type of infrastructure that we've built over the years. And from Pombility and Alpholda and Pompe disease, it's a little bit of a different strategy. That's more of a switching strategy. Again, there are other products on the market for Pompe, traditional enzyme therapies. However, it's been seen that the efficacy for some of the existing therapies can wane, and there's an emerging body of real-world evidence for improved outcomes with POMBILITY and OpFolda, so getting that messaging out there through the medical community, driving these switches, plus the geographic expansion, is the strategy for POMOP.

Okay. So, excuse me. While the commercial team is executing on all of that, that's going well and harvesting those synergies. Can you talk to us about BD and where you're going next? I think, again, when the deal was initially announced and the delevering timeline was a little bit longer, the goal, you had kind of said, well, we're still interested in BD, but it'll be smaller kind of pipeline. Does the delevering timeline change impact that all or kind of what's the mandate for the BD team now?

Yeah, thanks. Thanks, Whitney. Great question. Yeah, I'd say a couple of keynotes on the business development strategy and as it relates to the deleveraging strategies, as you noted. So first of all, the Amicus transaction was unique for us. Again, very large, close to $5 billion of total purchase price, commercial stage assets, a company already at somewhat of a reasonable scale, as they had several hundred million in revenue, and again, were a global company themselves. But because of that high strategic fit and the ability to transact, that was the right transaction to do last year. In the end, again, because of the growth and the synergies and the improved profitability and cash flow profile for the company, it actually serves as an accelerant to our long-term BD strategy. The current focus is building out our pipeline at the moment on the BD. That's what the BD team is focused on. Even without considering the leverage profile, we believe we can and will continue to execute on early-stage clinical deals that can fill up that early-stage pipeline. And we'll always do early stage collaborations. We announced just a couple of weeks ago a collaboration with the N. Lorem Foundation for Renew Disease. Again, not very material, and that's how Biomarin over time has had a lot of success and built our profile. It's this early stage in licensing where then you wrap around the Biomarin development capabilities and we make it a Biomarin asset over time. We will always do those. And then as we delever and have the opportunity to look at larger deals again. The priority will still be building out the pipeline, you know, so then it could be, you know, mid to late clinical stage assets, but still a bit early to comment on any specifics in terms of size or timing.

Okay, okay, sounds good. So maybe to try on a different specific. In terms of like therapeutic vertical modality, are you looking broadly? Presumably rare disease is the lens, but are there any other filters you place as you look outside?

Yeah, thanks. Great question. So yes, rare disease focus. And then further within there, genetically defined conditions. Again, we think this is very important. I mentioned it as being part of the foundations of the company in our past success. So the first priority within those parameters will be areas where we can leverage our in-house capabilities. I already mentioned in-house manufacturing. We also have a robust global external manufacturing network. We can manufacture and develop in multiple modalities, large complex biologics, oligonucleotides, even small molecules. And then also just research and development, regulatory clinical operations, commercial capabilities, so making sure there's a strategic fit there so that we can get the leverage from our own infrastructure. Further focus next would be within our therapeutic areas. So we've got two business units, metabolic conditions, which includes kind of the legacy biomer and enzyme therapies, and then gallifold and pombility and outfolda. And the second business unit is skeletal conditions, which today includes Voxogo for achondroplasia, on file for hypochondroplasia, BMN-333, the next generation CNP, also in the skeletal conditions business unit. So you can think of assets that fit not just within rare and genetically defined conditions, but within those business units, lots of leverage. And we are open to expanding into other therapeutic areas as a third priority, where we can still get some of that leverage from the base infrastructure that I mentioned.

Okay. I could keep asking you questions but I will switch over to Voxogo. And again, at the quarter, you raised guidance for Voxogo to at least $1 billion. So what are the drivers of that raise? And I guess what gave you confidence at this point to do that relatively early into a competitor launch?

Yeah, thanks. Great question as well. Strength, strong first half performance is the start and confidence in the second half of the year. Vox Ogo revenues grew 14% in the first quarter, and we added 20% new patients globally for Vox Ogo. And then, again, confident in the outlook for the second half. So that really gave us the confidence to increase the revenue guidance, which is now a floor of $1 billion, Biomarin's first potential blockbuster therapy. At the beginning of the year, we highlighted a handful of variables in the 2026 guide. One was a couple of international price renegotiations that were in process at the time. This is a normal part of the process. Voxogo has been on the market for five years in most countries. And outside of the U.S., there's often a process to either renegotiate or change over from a name patient sale model to a you know, national formulary model that involves a negotiation. One of those was resolved with a good outcome. The other one had some initial setbacks, but remains in process, but nonetheless was a net positive. So that was one of the variables that was partially solved for. And in the guide does include the different potential outcomes for this year. And then the other dynamic was patient switching. As you mentioned, a competitor came to market earlier this year, so that remains another factor. And then just the variability of estimates at the beginning of the year and execution, and that's where I come back to how I started with that strong first half and confidence in the second half.

Okay, got it. That sounds good. And then I guess you mentioned approved Voxogos, sticking with that, approved an achondroplasia, hypochondroplasia, is up next. How should investors be thinking about the revenue trajectory post-approval there? Is it going to be a bolus? Is it going to be a sharp inflection, more of a steady chug?

Yeah, thanks. Very excited. This is Bob Marin's first opportunity for a kind of a true label expansion into a new indication. So Voxogo was approved in 2021 for the treatment of achondroplasia which is the most common form of dwarfism. Hypochondroplasia is a genetically similar condition as achondroplasia. It's the same pathway where the active ingredient in Voxogo, the CNP analog which acts downstream from the FGFR gene, we believe has and data shows has the potential to be efficacious in hypochondroplasia as well. We were pleasantly surprised that the phase three study that we announced earlier this year, the data exceeded our own internal expectations. There's been an investigator-sponsored study going with Vox Ogo in hypochondroplasia. Dr. Dauber, his study, he released three-year data, which again showed sustained efficacy for Vox Ogo. So with that phase three data in hand, we announced last week that we filed the supplemental NDA with the FDA in record time. Again, AI enabled. We benchmarked it and were top quartile with the best companies out there in terms of phase three data timing to filing. Now the FDA still needs to review and accept the file. We'll share an update on our three call on that, but we did want to share that the filing at least was submitted. So if accepted, that would mean a potential approval and launch next year. Achondroplasia, we estimate at 24,000 patients worldwide. Hypochondroplasia is a bit smaller. We estimated at 14,000, but still very substantial. And as you touched on, the market preparation are the key efforts right now. Now, of course, we can't, you know, officially market until approval, but we can prepare the market on the medical side, building condition awareness, building, you know, the case for Vox Ogo as a mechanism. We should have as a tailwind the trust and history with Vox Ogo and achondroplasia, which was, you know, very well received. There's thousands of patients that have had years of positive experience on Voxogo. So that's a good starting point for hypochondroplasia. Diagnosis is going to be one of the challenges. Hypochondroplasia is more difficult to diagnose than achondroplasia. And that's why, again, these medical efforts will be important. So that's the work that we're doing. I can't predict at this time a bolus or what the ramp might look like. we'll talk more about the market as we get closer. But I will say that different from other therapies where if we've had a rapid development pathway get approved and then we're building the market as we launch, the advantage here is at least with Voxogo and achondroplasia, a lot of that market preparation has been done. So now it's hopefully a matter of just adding hypochondroplasia into that Voxogo ecosphere.

Okay, perfect. We are out of time. I'm going to squeeze in one last question. I didn't get to ask about 133, which is the long-acting CNP or PoundZ, but I'm going to skip ahead to the FSGS program from Amicus. And can you just talk about the level of internal focus and enthusiasm there? And is there any potential to accelerate that by chance?

Yeah, thanks. We're very excited about what was DMX200 at Amicus is now BMN820. This is a small molecule for FGFS, kidney disorder. uh i'll share that when we originally did the amicus transaction uh when we did our valuation because that was a development asset and because there was so much value and we were focused on gala fold and pombility and outfolda we did not assign a significant amount of value at the time of the deal to bmn820 however since uh digging in after completing the acquisition uh we're very excited about it. While there is another therapy on the market for this condition, it's a relatively narrow label. So BMN-820 is a novel approach and a potentially larger patient population. Bomarin has U.S. rights only. Dimerix is the developer. But we estimate that at about 30,000 patients in the U.S. So potentially broader label, slightly different mechanism of action. The other positive development is that the FDA has agreed to proteinuria as the primary endpoint, which is a positive. So we just need to let the study run now. It is a two-year study. It was fully enrolled recently. Just a reminder, Dimerix is the developer. So Biomarenoramicus actually doesn't do a lot of the operational R&D, but we'll be watching it closely.

Excellent. Thank you so much. I appreciate everything.

Thanks, Whitney.