Biomarin at Morgan Stanley Conference: Strategic Growth and Innovation

On Tuesday, 09 September 2025, BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) presented at the Morgan Stanley 23rd Annual Global Healthcare Conference. The company showcased its robust Q1 performance, marked by 16% revenue growth, alongside strategic plans to navigate competitive pressures and regulatory changes. CEO Alexander Hardy and CFO Brian Mueller emphasized BioMarin’s focus on long-term growth through innovation and strategic acquisitions.

Key Takeaways

• BioMarin reported a 16% increase in revenue for Q1, with plans to update its $4 billion FY27 revenue guidance later this year.
• The company is advancing its pipeline, notably with BMN 333, and expanding its geographic reach for Voxzogo.
• BioMarin is investing in AI to enhance its value chain and address regulatory challenges.
• CFO Brian Mueller highlighted a strategy prioritizing reinvestment in innovation over immediate shareholder returns.

Financial Results

• Q1 revenue growth of 16%, with plans to update FY27 revenue guidance later this year.
• Voxzogo’s 2025 revenue guidance is set at $930 million, with 75% of revenue from outside the U.S.
• The acquisition of InnoSign for $270 million aims to target ENPP1 deficiency, with potential peak sales of $500 to $600 million.

Operational Updates

• Positive Phase 1 results for BMN 333, with plans to move into Phase 2 and 3 trials.
• Progress with Palynziq in adolescence, with promising results presented in Japan.
• Voxzogo is now launched in 51 countries, with a goal to expand to 60 by 2027.
• AI is being leveraged across R&D, regulatory, manufacturing, and commercial operations.

Future Outlook

• BMN 333 is targeted for a 2030 launch, with ambitions to accelerate the timeline.
• The company is working on five additional indications for Voxzogo, with hypochondroplasia as the next focus.
• Monitoring the impact of tariffs and MFN regulations, particularly concerning Medicaid.

Q&A Highlights

• Minimal competition in genetically defined conditions in China, unlike other therapy areas.
• Strategic focus on sustained growth through internal and external innovation.
• Palynziq demonstrated strong growth, with a 21% increase in the first half.

BioMarin’s full transcript from the Morgan Stanley Global Healthcare Conference is available for those seeking more detailed insights.

Full transcript - Morgan Stanley 23rd Annual Global Healthcare Conference:

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Good morning, everyone, and welcome to Morgan Stanley Global Healthcare Conference. I’m Sean Lahman, Head of the Smithcap Biotech Equity Research Team here at the firm. Before we commence, for important disclosures, please see Morgan Stanley Research Disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative. For this session, we have the pleasure of hosting BioMarin Pharmaceutical Inc., our President and CEO, Alexander Hardy, and CFO, Brian Mueller. Welcome to both of you. Alex, would you like to make some opening comments, or do you want to go straight to Q&A?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: No, I’d love to make some...

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Yes, please.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Brief opening comments. First off, thanks very much, everybody, for joining us at this very busy conference. Sean, thanks for hosting.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Welcome.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah, you know, I’ve been at BioMarin since December 2023. It’s been a very busy period. I made a lot of changes to the company, to the strategy, the structure, the pipeline, and portfolio. I’m very proud of the progress we’re seeing. The execution of it, I think, has been really, really good quarter after quarter. I’m particularly excited about this last quarter. Why do I say that? I mean, with prior quarters, we’ve seen strong financial commercial performance. You saw that this last quarter with 16% revenue growth and leveraged many times that to the bottom line. What was exciting for me after a period of such a lot of change is to see the organization across the board performing and executing. We saw really good important news with regard to our pipeline with BMN 333, which is our long-acting CNP, a very, very important product in our pipeline.

We communicated the results that we’d seen, you know, positive Phase 1 results. Importantly, why is Phase 1 results important? Because we were looking to see, you know, what levels of free CNP we could achieve with our goal of actually raising the bar from an efficacy standpoint. We saw those results sooner, and those results are stronger than we even hoped for. We announced we were moving into a Phase 2, Phase 3 design program. In addition, we saw Palynziq in adolescence and making progress there with a new indication. We just released the results last week at a meeting actually in Japan. It’s good to see the pipeline. The last thing, sort of the trifecta, is the business development. We basically went from start to finish on a deal in one quarter.

Coming in, you know, that was part of our strategy is to elevate the focus on BD at BioMarin. We needed to have a clear strategy, and we needed to have that capability in place. We’ve now got it, and you know, to have identified a deal and done it in one quarter and executed it and now integrated it. It’s a really, really nice deal. This just makes me very proud. I think we’re really delivering on what we said we were going to do.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Sure. Thanks for those comments. Maybe just to talk about the macro for a little bit. We’re doing this with all our comments, and I’d love to have your view. With China’s rising biotech innovation, how are you thinking about the competitive dynamic, and does it influence your R&D and business development strategy?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Having spent time in China in a prior role, China is a formidable force. We should be very, you know, as a U.S. R&D enterprise, we should all be very much focused on China. I think it’s a very, very important topic. When we look at specifically the space that we’re in of genetically defined conditions, we don’t see the level of activity or the level of, I think, the progress there that we see in other therapy areas. I mean, clearly, oncology, immunology, cardio-metabolism, I’m sure you’re hearing this from other people. You can see it with the deal flow. In genetically defined conditions or orphan diseases, there’s much less activity in China. It’s something we’re watching. At the moment, you know, the leadership role is in the United States, and you know, it’s our intention at BioMarin Pharmaceutical Inc. to continue to do that.

We’re clearly watching what’s going on. We have, particularly from a BD side, you know, very, very vigilant watching certain Chinese companies right now.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Sure, sure. Yeah, to have you too. It’s rare disease in the euros, more or less untouched at the visible levels, at least anyway. Third question on the macro. What’s been most impactful? I’ll try a second question on the macro. How are you currently leveraging AI or thinking about AI as a disruptive technology?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah. This is a big topic in the leadership team and with the board. I see that AI has potentially transformative potential. At BioMarin, we’ve been making a lot of changes fundamentally across the different parts of our business. AI is now something that we’re really now focused on. Our strategy is we’re kind of, you know, we’re very realistic. We know where we’re starting from. A lot of the big companies have been investing in this very significantly for a number of years. We really want to learn from what’s working and what isn’t. Our initial phase of our strategy is very much a fast follower strategy until probably now until 2027, and then we’ll move into an area where we’ll have an ambition, particularly, I’d say, in these genetically defined conditions to be in a leadership position when it comes to leveraging AI.

That fast follower doesn’t mean that we’re not doing it. We’re doing it across the value chain. From an R&D perspective, just to give you an example, we’re developing right now in hypochondroplasia. It should be the next indication, sure, we hope, for our biggest drug, Voxzogo. There we’ve leveraged AI to explain what were previously variants of unknown significance and actually tied them to hypochondroplasia, increasing the prevalent population of the disease. That helped us actually recruit the study even faster. That’s really, really cool. AI is just really well suited in genetically defined conditions. We’re dealing with huge data sets to be able to mine that. That’s from an R&D standpoint. On the regulatory side, we’re using AI to draft our first-pass regulatory documentation. We’re using it in manufacturing in terms of supply chain optimization, predictive maintenance in our plants.

On the commercial side, another really exciting use of AI, and a large part of our business model in genetically defined conditions, is finding patients. We’re using AI to predict where patients are likely to be in remote geographic areas. We can do hot spot analysis, and AI can say, you know what, representatives should go to this city and this town. We’ve seen these other cases. It’s highly likely there’s going to be another case there. We can focus our efforts in those areas. Hopefully, that gives you an idea of how a sort of a mid-tier company is starting to really leverage AI.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Fantastic. Thank you. Last question on the macro piece. What’s been the most impactful thing to BioMarin on the regulatory side? Has it been MFN? Has it been tariffs? Has it been changes at the FDA?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah. If it’s okay, Sean, I’ll just go through each of them, yes, one by one. I would say from the FDA side, there’s been a lot of very positive commentary, and the head of the FDA, Califf, has been doing this listening for us. He’s been very, you know, particularly pointed out rare diseases as an area where the FDA can lean in and continue to innovate in terms of their regulatory approach. Those are very exciting words. We’re excited to see what that translates to. Our interactions in the meantime on a number of different areas have been positive. We’ve not seen any issues with staffing levels at the FDA up to this moment. It’s something we’re watching. We got regulatory alignment with the FDA on the pivotal program for BMN 333, the long-acting CNP therapy that I talked about, the phase two, phase three.

That was really, really important to get that clarity. It gave us good alignment and good feedback on the Voxzogo Canopy clinical trial programs, which was the four subsequent indications, which is hypochondroplasia, and then on the Palynziq adolescence. So far, we’re seeing good traction from the FDA. On the tariff side, we’re all waiting for the 232, the result of the investigation, the 232 investigation. BioMarin Pharmaceutical Inc.’s manufacturing footprint, for those that don’t know, is we have a U.S. footprint and a European footprint. We are in Ireland and the U.S. We’re a net exporter from the U.S., so we export a bit more than we import. We are quite well positioned in that two-thirds of our revenue is ex-U.S., one-third U.S. If the 15% tariff the EU holds, I think that will be a scenario which will be the lower end of potential impacts for us.

If that was to be implemented tomorrow, we would be pretty confident, right, Brian, with regard to our 2025 guidance. It’s something we’re watching. We’ll have to see what the 232 looks like. We have a whole range of mitigations that we could employ depending on how the tax bill. Then MFN. MFN is, of course, very, very much in movement right now. I think we’re entering into a really critical time to see how the administration approaches this. I think from BioMarin Pharmaceutical Inc.’s perspective, again, that ex-U.S., U.S. revenue split plays in our favor compared to most companies in the sector. Two-thirds of our revenue is outside the U.S., one-third in the United States. Rare diseases and our own experiences, we have much tighter pricing corridors than most companies. I think the thing we’re specifically watching is MFN in Medicaid.

Again, being rare diseases, we have very little exposure to Medicare. Medicare is less. It’s a low single-digit % of our population in the U.S. is covered by Medicare. Medicaid is really important for these genetically defined orphan diseases. There’s very high levels of disability. Medicaid is about a third of patients are covered by Medicaid. You know, very concerned if MFN was applied in Medicaid, what it would create in terms of disincentive to invest in developing for rare disease drugs. We’re very active in trying to make sure that MFN doesn’t happen. Specifically, MFN in Medicaid would be a setback, I think, for innovation. BioMarin has a number of hedges, but we’ll just have to navigate these two things.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Awesome. Thank you for such a candor. That was great. On to more specifics on BioMarin.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: I’m coming from an Australian. That’s when somebody says Canada, that’s good. That’s a high bar. That’s great.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Thank you for that.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Throwing you off your game here.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Oh, no. I’m right on it. Don’t be fooled by the relaxed nature. Can you talk about the recent InnoSign acquisition and why it was a good acquisition? How long have you been evaluating it? Do you foresee those, or when do you foresee those revenues being a meaningful contributor to BioMarin?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah. I mean, we’re very excited about the InnoSign acquisition. As a new CEO coming in, you know, people are watching your first deal. As part of our strategy, we really saw the role for BD in the future of BioMarin as being supplementing the internal innovation with, I think, a really compelling proposition to all of our stakeholders. I would say, you know, to investors, you know, we have this geographic footprint in 80 countries. We have this capability set that you believe that, you know, assets are worth more in our hands than where they’re sitting right now. Because in many cases, these rare disease companies, you know, undercapitalized, under-resourced. If they’re lucky, you know, they’ve got one or two assets. They’re focused very much on the U.S. The prevalence of these diseases are generally, you know, outside the U.S. and Europe.

You take achondroplasia, for an example, which is what our drug Voxzogo is indicated for. 70%, almost 70%, 68% of patients are outside the United States, Europe, Australia, New Zealand, and Canada. I mean, almost 70% of patients. You need that geographic footprint. We have that. We built that up over 20 years with our enzyme therapy business. When I came in, we started looking at InnoSign. We liked the first indication that they’re working on, the ENPP1 asset. I think there’s a little promise with other indications, but they had proof of concept in the ENPP1, but didn’t have the capital to complete the Phase 3. We didn’t like the value at the time. We waited our time, and a process was then started. We executed really well in that process. You know, we paid $270 million for a first-in-disease asset.

There were no competitors in development, with peak sales potential of, I don’t know, $500 million, $600 million. So $270 million in asset, with the first readout from their pivotal studies in 2026. We could potentially have the first approval in 2027. You can see why, you know, I was particularly excited about this deal. It’s been well received as well. We aim to do more.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Okay. Wonderful. In your Q2, you noted that the company plans to provide updates to the $4 billion FY27 revenue guidance later this year. Also, the 2025 guidance currently does not incorporate IP R&D expense associated with the InnoSign acquisition. Can you provide any more color on those threads?

Brian Mueller, CFO, BioMarin Pharmaceutical Inc.: Yeah, Sean, thanks. I’ll take that one. We are working through a comprehensive review of all the assumptions at the product level within our portfolio in terms of our current 2027 expectations. There’s been a lot that’s changed both internally within BioMarin and externally. We just want to take full account of all those assumptions and work them back through our models. More importantly, almost than the revenue expectation of 2027 itself, is we want to provide more clarity and transparency around some of the assumptions, again, at the franchise or product level so that it can be better understood by investors. We appreciate that last year when we talked about, you know, that one big number, $4 billion in 2027, it was an outcome of our full strategic review.

We tried to give some clarity with the growth rates at the business unit level, but we don’t think it was enough to fully understand how, given all of these circumstances that are out there, we think we can continue to grow revenue at BioMarin. We are doing that work, and we’ll work with them.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Thank you. Maybe to go product-specific on Voxzogo, so it’s been launched in 51 countries, I think, more recently, up from 49 in Q1. Where do you stand on near-term country additions and reimbursement wins, and how should we measure our progress against your longer-term ambitions?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah. The growth opportunities that we have in front of us for Voxzogo and achondroplasia, I mean, clearly, we’ve got these five subsequent indications that we’re working on too, with hypochondroplasia being hopefully the next one. With the achondroplasia, there’s truly really two key growth opportunities that are ahead of us. One is fully penetrating the U.S. market. We’ve been really devoting additional resources there. It was a slower launch than the rest of the world, but it’s the largest single market that we have of about 10% of prevalence. That is very important. We’ve been pleased with the progress we’ve been making there. The other part is fully occupying a full global footprint in these underpenetrated markets. We’re still, you know, you mentioned with, you know, 51 markets now, that the most recent launches have not been in small markets.

We’re still launching in reasonable-sized markets and making really good progress. There was one, I think we got, I’m not going to mention names of markets because clearly we want to, from a competitive standpoint, be confidential around that. In a market, I think we got approval last year, we’ve already achieved penetration of about a third of the patients. We should be aiming for about 60% by the end of this year. This is not a small market. It’s really exciting that we’re still on these. It’s sort of interesting seeing these sort of rare disease dynamics that, you know, it’s a pretty short ramp to peak, but, you know, they start when the country comes online. We’re still filling out that footprint. Again, we’re in 80 countries. We’re now 51. We’re aiming for 60 by 2027. That’s another large part of the growth.

From a competitive standpoint, that’s a real competitive advantage. That geographic footprint is a competitor is going to have to be successful in many, many countries outside the United States and Europe.

Brian Mueller, CFO, BioMarin Pharmaceutical Inc.: Sure. Could you talk, as I say, just to add, it shows up in our current revenue profile today. Our Voxzogo revenue guidance for 2025 is $930 million. Roughly 75% of that revenue is from outside of the U.S. Back to the competitive standpoint, we’re talking about a competitive space and a smaller fraction of our total Voxzogo revenue and, of course, portfolio revenue.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Sure. I guess you stand back and look at where your share price is, and everyone seems to think it’s worth double the way it’s trading, at least on the sell side. The overhang seems to be Voxzogo and what happens with Transcontinental PC, just to get your agreement on that if that’s what you think the major overhang is. Maybe walk us through your competitive positioning of Voxzogo and your early indication on an age-related basis, your geographic spread, and maybe why you might not necessarily think there is a material risk to Voxzogo.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah, I mean, that is the big thing that we hear. The standing in the, you know, again, we’re executing, delivering significant revenue growth and profitability. We’re doing the things we set out to do and said we were going to do and hitting those marks. Still, the stock price doesn’t reflect that. The big overhang is this Voxzogo thing. I would say that it is, it’s one-third of our revenue. If you look at the implied valuation, basically, people are giving us no credit at all for Voxzogo. The enzyme therapy business is growing what we said it would grow, a high single digit, very, very consistently. We’re just seeing the beginning of the benefits. We moved structurally to a business unit structure to allow focus on these different segments. It was underappreciated internally and externally.

When it comes to Voxzogo, a third of our business that dominates so much of the investor perception, I think there’s real reasons to be more confident than the external world is giving us credit for. The first is this geographic footprint where we’ve already talked about the presence in all these countries where that’s where the patients are. That’s where our revenue base is. 75% is outside the U.S. We built that over 20 years. We’re very, very good at commercializing outside the U.S. The other aspect is that achondroplasia and the guidelines say diagnose and treat children as soon as possible after birth. The majority of children in the U.S. are actually diagnosed prenatally. Often, the decision to treat is made prenatally. We will have, for a while, been the only one that’s indicated in infants.

Competitors are going to have to produce that data and get regulatory approval. They will mainly be dealing with a switch market because the incident market will be predominantly in many of these developed, highly penetrated markets, in the younger age group. Switching dynamics in pediatric rare diseases, if the patients are doing well, then this is not something that is going to happen across the board. There will be some patients. I think this is misunderstood. We’re not saying that no patients will be switched, but we’re also not saying, and it’s not credible to say that all patients will be switched. Voxzogo is somewhere in between, and that’s still a very viable and growing skeletal conditions franchise for us. On top of it, we obviously are bringing BMN 333 forward, which we already touched upon.

We’re aiming there to actually surpass the efficacy of Voxzogo, which so far nobody, we’re going head-to-head against our own drug in our phase 2, phase 3 design. That will be a reason why patients will be interested and caregivers will be interested in switching patients, for additional growth velocity and, most importantly, benefits beyond height.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Right. Think about the, I guess, it might be prevention of switches, but the Canopy clinical trial program, maybe touch on that as something that you think whether investors are focused on or not focused on. Maybe that is a defense strategy, if you like, if you did lose in achondroplasia and try and net that off if that makes sense.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah, yeah. I mean, you know, just to put this into perspective, we’re developing, we’re already indicating in achondroplasia, and we’re now indicating in, or pursuing indications in five subsequent indications. The total addressable patient population that we’re aiming for across all of those six indications is 420,000 patients. Achondroplasia is 24,000. The way I think of this is, I mean, certainly in achondroplasia, we have got a lot of growth ahead of us still. Vimizim overall, with all of these indications, we have much, much more growth in these indications. We’re aiming to be in those indications and to establish that leadership position well ahead of any competition. It’s important to note that some of the other MOAs like FGFR3s will not, scientifically, there’s not a strong hypothesis for them to work in many of those indications. We should have some white space.

We’ll certainly have time, we believe, to get ourselves established. Those are large patient populations. Those studies are recruiting. As I said, hypochondroplasia has finished recruitment. We’re now waiting for the results. We’re recruiting the Phase 2 studies for the four subsequent indications.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Got you. Maybe move to a little bit more longer term, but on BMN 333, which is showing extraordinary promise with the data that you released. Can you talk us through getting to the registrational study and sort of looking at longer-term, you know, potential launch plans? Like how far are we potentially thinking?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah, yeah. I mean, why did we make such a big deal over Phase 1 clinical study? Obviously, we wanted to see the safety profile, as you would expect in a Phase 1, but we were very interested to see the PK data in the healthy volunteers. We had this hypothesis that we would be able to achieve two to three times the free CNP levels that have been hitherto shown by other long-acting CNP agents. That’s what we’ve seen already in that Phase 1. We’re still going through the dosing cohorts. We declared success after three. Two of them exceeded what our target was. That was enough for us to go with green light for Phase 2, Phase 3. You asked then about the approach now for the registrational study. It is an operationally seamless Phase 2, Phase 3 study.

This will take three of the dosages from the Phase 1 study into the Phase 2 study, together with a control arm of Voxzogo. Again, we’re aiming for superiority. We’ll then select one of those dosages and move it into the Phase 3. We’ll carry the sites through, so there will be no minimal delay. As I already mentioned, we already have FDA alignment on the design of the Phase 2, 3 study and the endpoints. We think that this study will be really compelling from a recruitment standpoint because, again, the control arm is Voxzogo, the standard of care. We think there’ll be a lot of patient and investigator interest in it. The timeline is, at the moment, we’re saying publicly 2030.

As you can imagine, after the superiority from an efficacy standpoint, that’s the next most important thing for us is speed to get this to patients as quickly as possible to raise the bar, you know, really meaningfully from an efficacy standpoint. We’ll keep you updated. At the moment, we’re saying 2030 with a lot of ambition to move that up.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Wonderful. Wonderful. Thank you. That was on Palynziq. Some strong numbers, 1A to 25. I believe there’s some more data around on adolescents. You’d potentially utilize that data to further accelerate sales.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah. So, you know, there’s a lot of interest in the overall PKU space. There’s a lot of buzz and noise right now. It’s an important part of our enzyme therapy business and our franchise. We’ve been the leaders in PKU. We’ve had Kuvan and now we’ve had Palynziq. We saw, as you mentioned briefly there, Palynziq is growing extremely well. It’s grown in the first half by 21%. We’re really seeing strong patient enrollment in the second quarter. We’re confident of its competitive profile. It’s really dealing with a very different target patient population than any of the new entrants. This is for the severe patient. It has really unsurpassed effect on Phe levels. You just saw that adolescent data, almost 50% reduction in Phe levels for those adolescent populations, and an opportunity to significantly improve diet.

Quite a few patients achieve an unrestricted diet, which obviously approaches normal life for these patients. We really think there are very different segments that any competitors are going to come, and we’re pretty confident that Palynziq is going to continue to grow.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Wonderful. Are there any steps guiding a supplementary BLA submission in the U.S. and EU? What sort of revenue contribution would you expect from the label inclusion to 12 to 17-year-olds?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah. I encourage people to look at that data, as I’ve already mentioned. It was published last week after a meeting in, or it was released last week at a meeting in Japan. This represents about a 10% increase in the patient population, which is meaningful. The vast majority of patients, though, in PKU are still untreated. This is another growth opportunity. We’re still, even without this, nowhere near fully penetrated with Palynziq. This will just add to that. I think it’s exciting to have the prospect, the adolescents, you know, the children are generally quite well controlled with diet and parents overseeing their kids. When it comes to adolescents, this is really an important stage to try and get these patients well managed before they’re sent off to college and into the world.

Now we have the prospect, you know, should the drug be approved, to be able to offer them the segment. There’s a lot of excitement amongst the treatment community and the patient community about this.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Thank you. I believe there’s a recent approval in the PKU space. Does it change your view on the competitive dynamic market share assumptions?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Yeah. As I mentioned, I think this will really be a different patient population. With the new competitor coming in, I think they can have a very successful launch and Palynziq can continue to grow because really our sources of patients are very, very different. They’re more likely to cannibalize Kuvan, which is not a significant product for us as it’s already after its loss of exclusivity. I think that’s where they’re going to be the majority source of their patients, and we’re going to be more focused on the severe segment.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Sure. Taking a step back and looking at the investment case, there’s, you know, I think you mentioned 30% of the revenue is coming from Voxzogo, and you’ve got a balanced portfolio across the rest of the business. For biotech companies, cracking profitability and generating cash flow is important. I think you talked about segmenting the business into operating units. There’s a lot going on in the R&D pipeline. What’s your thinking from here as investors? Do we think about enough appetite in your current R&D portfolio to absorb cash flows? Do you think about the business development opportunities? Do you think mid to longer-term, more formal capital returns? How do you put all those pieces together?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Maybe I’ll start and then hand over to Brian. You know, we’re focused on long-term sustained high levels of growth on the top line, and we think we’ve got the ability to do that. The modification of our strategy is internal and external innovation. As I’ve already covered, you know, we think that we can deploy capital really effectively to supplement our growth rates. As an established player with that geographic footprint and that capability and with the cash flow, we think we can do deals. Those deals will be worth, those assets will be worth more in our hands. That’s our primary goal with our capital allocation, to have a blend of both internal and external innovation, continue to be an innovation-driven company, but delivering sustained high levels of profitability, which I think we’ve already demonstrated we can do.

Brian Mueller, CFO, BioMarin Pharmaceutical Inc.: Sure. Thanks. Maybe I’ll jump back to the investment thesis part of the question. We think we’re uniquely positioned because we believe we can continue to grow our existing commercial franchise. We also believe we can continue to innovate and invest in research and development, but also increase profitability and operating cash flow. As we improve and accumulate cash flow, it becomes a virtuous cycle, where we can just reinvest that back in this business as well through external innovation, which gets the capital allocation. Alexander noted it well. We believe that investing in long-term growth over return to shareholders has the opportunity to create more value for shareholders over the long run. We look at all mechanisms and vehicles for capital allocation, and we discuss it with our board regularly.

Even at current price levels, we believe that investing in future revenue growth and thereby profitability and cash flow is the best growth opportunity.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Wonderful. One final question. Is there anything that I didn’t ask that I should have?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: We didn’t go further into the pipeline around 351 or 349. Maybe another time, we would love to talk about it.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Sure.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: Innovation is very much the lifeblood. Highly innovative drugs that really make a big difference for these diseases and genetically defined conditions, that’s what we’re really excited talking about.

Sean Lahman, Head of the Smithcap Biotech Equity Research Team, Morgan Stanley: Wonderful. We’re out of time, but thank you, gentlemen, for your time today. It’s been great.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical Inc.: All right. Thank you very much.

Brian Mueller, CFO, BioMarin Pharmaceutical Inc.: Thank you, Sean.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.