BioMarin at TD Cowen Conference: Strategic Growth and Innovation

On Tuesday, March 4, 2025, BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) presented at the TD Cowen 45th Annual Healthcare Conference. The company outlined its strategic direction, highlighting both achievements and challenges. With an 18% growth in 2024 and a focus on expanding its patient base, BioMarin aims to overcome competitive pressures with innovation and strategic partnerships.

Key Takeaways

• BioMarin reported an 18% revenue growth in 2024 with a 28.6% operating margin.
• The company aims for $4 billion in revenue by 2027 and a 40% operating margin by 2026.
• Voxogo’s potential extends beyond achondroplasia, targeting a population of 420,000.
• BioMarin plans to quadruple its patient reach over the next decade.
• Strategic focus includes innovation, growth, and value commitment.

Financial Results

• 2024 saw an 18% increase in revenue and a 28.6% operating margin.
• 2025 revenue guidance is set between $3.1 billion and $3.2 billion, indicating 9% to 12% growth.
• The company targets $4 billion in revenue by 2027, with a cash flow goal of $1 billion.
• Voxogo is expected to generate $900 million to $950 million in revenue in 2025, with a growth rate of 22% to 29%.

Operational Updates

• BioMarin has restructured into three business units: Enzyme Therapy, Skeletal Conditions, and Roctavian.
• The company is increasing its presence in 60 countries by 2027, up from 47 last year.
• Investments focus on expanding Voxogo’s U.S. market and enhancing international reach.

Future Outlook

• BioMarin plans to expand its treatment reach by four times over the next ten years.
• The pipeline includes BMN-351, BMN-333, and BMN-349, targeting rare conditions.
• A mid-teen compounded annual growth rate is anticipated through 2034.
• Voxogo’s expansion includes five new indications, starting with hypochondroplasia by early 2027.

Q&A Highlights

• BioMarin is confident in its competitive edge due to its geographic reach and early diagnosis capabilities.
• The company sees strong clinical progress in hypochondroplasia, with approval projected for 2027.

For a detailed understanding of BioMarin’s strategic plans and financial performance, refer to the full transcript below.

Full transcript - TD Cowen 45th Annual Healthcare Conference:

Phil Nadeau, Biotech Analyst, TD Cowan: Good morning and welcome once again to TD Cowan’s forty fifth Annual Healthcare Conference. I’m Phil Nadeau, a biotech analyst here at Cowan, and it’s my pleasure to moderate a fireside chat with Byron Pharmaceutical. Up here on the stage, we have Alexander Hardy, President and CEO, and Brian Mueller, the Executive Vice President and CFO, and Tracy McCarty from Investor Relations is also in the room. Alexander, I’ll hand it to you.

Can you give a brief, state of the company overview, biggest strengths, biggest challenges and what does BioMarin need to achieve to drive shareholder value over the next twelve to twenty four months?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Great. Thank you very much, Phil, and thanks for everybody joining us. So where is BioMarin right now? I’ve been in the role for just over twelve months now. Last year was a strategic review where we changed many aspects of the company in terms of the strategic direction, in terms of the structure.

We made important pipeline and portfolio decisions. But even at the same time, we were executing. And I think our 24 results, I’m very proud of the 18% growth, the 28.6% operating margin. We’re already starting to make progress on the long term targets we set out at our Investor Day last year. We’re already executing.

And I think that’s the key message that I want to send at this conference, is we’re making progress towards those goals that we set out. In terms of your question around greater strengths and what are the challenges of the company, I think the strengths of the company that aren’t fully appreciated is the full potential of Voxogo. There’s a lot of focus on achondroplasia. That is just the first indication. We have clinical programs away and underway in five subsequent indications with much greater potential in achondroplasia.

We’ll have a five year lead on any potential competitor in achondroplasia. We’re going to then have years of lead in all these subsequent indications. If you think of the total addressable patient population, these six indications in total is four hundred and twenty thousand patients. Achondroplasia is just twenty four thousand patients. So there’s a lot of growth ahead.

I think that is one of the big opportunities in skeletal conditions. And then in terms of the other aspect, I think, is underappreciated is the enzyme therapies. We this is the history of the company was in enzyme therapies. We have six of them. They are, I think, underappreciated in terms of their growth potential.

Over the last three years, if you look at 24 prior three years, growing at an annual rate of around 9%. Okay. Yeah. 9% CAGR. And it’s part of our long term financial guidance.

We said high single digit growth. And I think that’s not fully reflected certainly not fully reflected in the valuation or the models that people have. I think in terms of the challenges, I mean, clearly, we hear it from investors that they’re thinking about competition. And we have this five year head start over any potential competitor that may come to market. We have intellectual property, of course, no doubt we may touch on that in our conversation.

But we’re readying to face competition. We will have such a lead. We will have the the indication in infants in achondroplasia. And this is where the guidelines say that that, patients should be diagnosed and treated as soon as possible after birth. And we’re now treating patients within days of birth.

That will be years before competitors have that indication. And should they even get that indication, I think competitors that are different mechanism of action, that burden of safety, this is a no risk population when you’re dealing with infants. Its efficacy, safety and convenience is down the list in terms of priorities from a caregiver and an HEP standpoint. So I think that’s probably the thing that we need to do more of is convincing people that we’re ready from a competitive standpoint. I’m very confident whether it’s the IP or whether it’s our ability to compete in the market given the advantages we have as being first mover.

Phil Nadeau, Biotech Analyst, TD Cowan: Can you discuss your long term vision for BioMarin? It was partially laid out through the guidance that was given at last year’s Analyst Meeting in terms of the long term financial targets. Maybe outline those. But then also, what is your long term strategic goals for the company? Yeah.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: So I mean, I came to BioMarin. I saw all the potential to create an iconic build an iconic biotech company. And as I’ve been here now a year, I see all of that only with greater clarity. So we have a strategy. It’s now very clear and laid out three strategic pillars: innovation, growth and value commitment.

We are always been around innovation. We’re going to continue to disproportionately invest in innovation. And our goal is to, over the next ten years, to expand the number of patients who benefit from our treatments by a factor of four times. So that’s the main thing that I’m driving is for BioMarin to be a primary growth innovation biotech. As we do that, we will aim to deliver significant value to investors.

So billion in revenue by ’27, ’40 percent operating margin by 2026 and forty percent plus thereafter. And cash flow generation of billion in cash flow in 2027. So these are the elements. That’s the strategy. Our goal is to significantly help patients and also create significant value for investors.

Phil Nadeau, Biotech Analyst, TD Cowan: What role will business development play in the long term strategy?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Yes. So when we articulated the strategy last year, we said that there was going to be an increasing role of business development going forward for BioMarin. It was not included in the guidance numbers. So I think that’s important to highlight. It’s incremental.

It will be incremental growth on top of that. Now why is business development an important part of our strategy going forward? It’s because we have this capability, which is pretty unique. It’s the ability to research, develop, manufacture and commercialize drugs, rare disease, genetically defined condition drugs. And that’s a really compelling proposition.

Very few companies have, for example, that global footprint, which you need when you’re dealing with genetically defined conditions. I think we’ve started to really shed light on this for people. But if you look at achondroplasia, only ten percent of the patients with achondroplasia are in The United States, Sixty Eight Percent of them are outside The United States, Canada, Australia, New Zealand. And that also plays out to an even greater degree in the enzyme therapy, the MPS space in those diseases. So we have a unique capability.

We are now because of our profitability, we are now cash flow positive. And we see a lot of interest in companies in terms of partnerships at the product level to take advantage of our capabilities to get the drugs approved and commercialized outside The United States. So we didn’t rush into doing business development last year. I wanted to make sure we got the strategy really clear, what we were really defined, what is our sweet spot, what are we really good at, where do we have an opportunity to win. And I also wanted to build our business development capability.

We have a history of doing early research collaborations, but clinical stage collaborations, we want to make sure we’ve got the capability to identify deals, do good deals. And then most importantly, where many companies go wrong is in terms of integration and actually realizing the value from a deal. And so last year, we at the end of last year, we brought in James Sabre, who is a recognized leader in the industry and business developments. We now have all the elements, the clear strategy, we have the capability and we have the capacity and we aim to do business development, which is incremental to the business plan we have.

Phil Nadeau, Biotech Analyst, TD Cowan: What type of assets would be of interest to you? Would you buy and market a product? Would it be early stage? What do you think fits best with your current portfolio? And similarly on risk profile, do you look more at the lower risk end of the spectrum, high risk?

How do you think about building a portfolio externally?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Yeah. So the lens we’re looking through is is defined as such. Okay. So it it has to be genetically defined conditions. That that’s that’s what we do.

That’s what we’re really good at. Has been consistent with the the the core five. So this has served us very well as a core four. We’ve added a fifth element. So this is genetic, targeted, accessible, transformational and then commercially competitive.

These are the five criteria. And then on top of it, ideally these assets fit within our business unit structure. I hadn’t touched on that in the comments so far. But with our strategy, the three pillars of our strategy, we’ve actually reorganized the company into three business units: Enzyme Therapy business unit, Skeletal Conditions business unit and a Rotavian business unit. That allows us to have that focus, that accountability to be able to drive what have actually three very, very distinct businesses.

So ideally, it will be asset that fits into those. We’re prioritizing product partnerships or product acquisitions, not M and A, and of course, very much with a lens of value creation. This is the advantage that again, business development is not included in that long term financial plan and the long term guidance. So we’re doing business development from a position of strength, not from a position of weakness. So therefore, we can be really picky and really focus on things which really initially just drop right into that capability and that framework.

So you can imagine us doing things which actually involve minimal incremental investment can take advantage of the infrastructure we already have. So clinical stage, ideally, we’ll continue to do research partnerships. We’ll just do those at a steady clip. We did one of those last year. We’ll continue to do those.

But what is new is more clinical stage launching in the next several years.

Phil Nadeau, Biotech Analyst, TD Cowan: Perfect. Last week or the week before, you provided 2025 revenue guidance $3,100,000,000 to $3,200,000,000 that’s 9% to 12% growth. Can you talk about the pushes and pulls in that guidance? What can drive upside? What are the risks stating?

Brian Mueller, Executive Vice President and CFO, BioMarin Pharmaceutical: Yes. Thanks, Phil. Appreciate the question. So, yes, we’ve been pleased with the revenue growth over the last couple of years and the strong performance in 2024. We remain committed to that $4,000,000,000 by 2027.

And we believe that this 2025 guidance is an enabler to that. To your point about pushes and pulls, one element that we’re observing when you look at 2024 into 2025 is the really strong performance in a couple of the enzyme therapies, ElduraCyme, which is marketed by Sanofi Genzyme grew 40% last year. And our revenue to Sanofi is based on product supply and is less directly tied to end user demand. Likewise, Naglazyme, one of our enzyme therapies grew 14% last year, which was really strong. I’ll share that the underlying dynamics across the entire portfolio for enzyme therapies are that we’re growing patients annually, including last year for Nagozymes as an example in kind of the mid single digit range.

That’s the baseline type of growth that we’ve seen. So for Nagozhyme to grow 14% last year, that means that there were some ordering dynamics. In our global business, we sell to a number of large single national payers that tend to order, perhaps just a couple of times a year. Sometimes you see this at the quarters when we’ll get a couple on a particular quarter, revenue will be high. If we don’t get a couple of the larger orders, it can be low.

So it can skew the quarters. It’s less that dynamic and more just the global order dynamics. So my point is once you normalize some of those trends in ’twenty four looking into ’twenty five, you get to this high single digit target rate for the enzyme therapies. So we’re going to continue to be subject to some level of those global distributor dynamics. We do expect a good portion of our growth in 2025 to come in the second half of the year.

If you look historically, including last year, Q1 can be a slight a slider growth quarter. Also important to note is the potential of these business units that Alexander mentioned. We spent the latter part of 2024, you know, reorganizing and implementing this, these business units. So tactically speaking, getting personnel, into their roles, has just been, you know, through the first couple months of this year. And we’re seeing great progress on the potential in terms of the global knowledge sharing some of the benefits of the extreme focus and accountability within these focus therapy areas.

But it’s going to take some time to truly realize the potential of those, whether it be the enzyme therapies or Voxo Go. So the patients that we’ll be adding in the first half of the year won’t necessarily show up in terms of significant revenue growth until the second half of the year. Those are probably the two largest things to pay attention to.

Phil Nadeau, Biotech Analyst, TD Cowan: And maybe one more general question before diving into some of the franchises. In terms of operating margins, your guidance is 32% to 33%, I believe, in 2025, going to 40% over time. It seems like based on our model that 32% to 33% assumes modest growth in OpEx in 2025. Is that reasonable? And are you able to get that leverage out of the business because of the reorganization of the business units?

Or what has permitted you to grow revenue while not really increasing expenses at least this year?

Brian Mueller, Executive Vice President and CFO, BioMarin Pharmaceutical: Yes, thanks. Great question. And again, we’re really pleased with the operating margin performance last year, 28.6% in 24% versus 19.4% in 23%. Percent. We’ve got this target of 40% non GAAP operating margin in 2026, which would be a doubling of that 2023 margin.

So when we look ahead through the chapters, if you will, of operating margin in ’twenty four, ’twenty five, ’twenty six. A couple of things I’d point to is, one, we never thought it would be linear and the 2025 plan is on track with the 2026 target. To your point, yes, if you do the math down the P and L, we are growing OpEx, but at a modest level. However, it’s really important to note that in 2025, we’re realizing most of the impact of last year’s transformation, including the restructurings and changes to the operating model. Last year, we spent a lot of time on not just operational efficiency, but organizational efficiency, prioritizing our spend in the right places, you know, an enhanced view of the portfolio through our governance process, you know, focused on what is now the core five research and development criteria that Alexander mentioned, that led to the discontinuation of five early stage programs next year, plus the operational efficiencies.

Those dollars are being reinvested now in the right high value tactics. So absolute dollar level growth may be modest, but it’s a different type of spend on the right activities. Some of the priorities this year are accelerating the Voxogo indication expansion. So these five additional indications, full systems go there, as well as the prioritized early stage assets. So BMN three thirty three, our long acting CNP, for skeletal conditions, BMN three fifty one for Duchenne’s muscular dystrophy and BMN three forty nine for alpha one antitrypsin deficiency.

Those are prioritized moving those along. Again, all within this cost envelope that’s enabling the 40% margin next year. And then likewise, I’d say we’re we’re making a number of strategic investments in sales and marketing as well. There were also restructuring elements of our SG and A line item, which then provide the capacity for this incremental investment, whether it be skeletal conditions, and maximizing Vatogo, for example, in The U. S.

Deeply increasing penetration there and then within the enzyme therapies just global tactics.

Phil Nadeau, Biotech Analyst, TD Cowan: Perfect. Shifting to Voxogo where investors are very keenly focused, guidance for this year is $900,000,000 9 50 million dollars that’s growth of 22% to 29%, very healthy growth, but a bit of a deceleration from prior years. Where are we in the Voxogo launch? Which territories are driving growth? Which ones are more mature?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Yes. So we’re very much on track for what we laid out in terms of the financial guidance of 25% CAGR for Voxogo over this period to 27%. We’re very much on track and 25 growth rate is consistent with that. I mean, clearly, as the product gets larger and larger, it’s on track to obviously hit the blockbuster status in the next several years. It’s a larger base, so that percentage is obviously going to ameliorate.

But we’re very much still with more growth ahead of us than behind us with Voxogo. The U. S. Market was I think one of the interesting elements is we shared the revenue breakdown for Voxogo last year. It’s 24% of our revenue came from The United States for Voxogo, 76%, therefore, is outside The United States.

The U. S. This is very unlike, right, most biotech and pharma products. This means that The U. S.

Has a lot more room to grow. It also means that those ex U. S. Sales are in that geographic footprint we talked about and which would therefore be tougher for competitors to penetrate. So our focus in terms of growth for Voxogo is U.

S. And you heard from Brian. We both talked about the business units. You heard from Brian that we’re increasing our level of investments. We’ve just added an additional field force in The United States.

We’re increasing our marketing spend in The United States, increasing our clinical coordinator resources. This will help us, we think, to even accelerate the growth in The United States. So U. S. Is really critical.

And then ex U. S, we’re still expanding out into that geographic footprint that we have with the enzyme therapies. So last year, we were in 47 countries. We aim to be in 60 by 27. We still see a lot of potential in terms of penetrating the prevalent population in many of these large U.

S.-ex European large markets.

Phil Nadeau, Biotech Analyst, TD Cowan: I think investors are very keenly focused on competition, as I’m sure you’re aware. What gives you confidence that you can grow in the face of competition? You can grow the Voxogo franchise in the face of competition?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Really, really great question and a very, very important one. So we’ll just put the whole intellectual property topic aside, just park it. We didn’t include it in our long term assumptions. We think we’re going to be successful competing. The reason why we think we’re going to be successful competing should and if a competitor come is a number of factors.

One is the geographic footprint. We’ve talked about a lot that any competitor has got to have really strong capabilities outside Europe, outside The United States to be successful in achondroplasia. The next area is indication down to to birth. Competitors are not going to get that initially. Some competitors are going to, I think, probably have challenges with the safety profile required to have an infant indication.

The third point I would say is that caregivers and patients, I should say HCPs, are really motivated actually less by the height benefits of drugs in achondroplasia and more about addressing all the attendant health issues that come that can come with achondroplasia, things like spinal stenosis, forum magnum compression, the need for surgeries later on in life, cranial facial issues. And we are producing and it’s the benefit of having a five year head start and now six thousand patient years’ worth of data, we are producing both a safety database and an efficacy database on the effects on all these other things, which are actually the most important thing with regard to initiating treatment. So I think those are the key points. Anything I missed, Brian?

Brian Mueller, Executive Vice President and CFO, BioMarin Pharmaceutical: No. I think you have it. Okay.

Phil Nadeau, Biotech Analyst, TD Cowan: It seems like the biggest potential benefit of the competitors is convenience, whether it’s oral or less frequent injection. How does convenience factor into the treatment decision in achondroplasia, in the context of some of those other health issues you talked about as well as long term safety and efficacy data?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Well, in pediatric rare disease and achondroplasia is no exception here. There is a hierarchy of needs. And it is, I think it’s best described as safety and efficacy and then much further down. If those are satisfied, then it becomes much more about convenience. And when you’re dealing then, just a further lens down, if you’re dealing not just with pediatric rare disease, but you’re dealing with initiating treatment in infants within days of birth, then safety, this is a zero risk proposition, is absolutely critical.

So safety becomes even more important. And that, you know, that is something that, it’s not only the regulators that need to be satisfied, it’s going to be the HCPs and the caregivers. And what you see in and I think it will be exactly the same here is I think investors are looking at this as a sort of very binary proposition. And in reality, and if you look at all the analogs, this is just not how it works in these pediatric rare disease states. Patients are doing well and they do very well on Voxergo.

We have a ninety five percent compliance rate for Voxergo. What that tells you is that patients and caregivers deal with the daily injection because they’re also seeing those benefits. And again, those analogs show that when patients are doing well, and they’ve incorporated into their routine, there’s not a lot of interest for a wholesale switch.

Phil Nadeau, Biotech Analyst, TD Cowan: In your opening remarks, you talked about the other indications, the size of the markets as well as the development program. Can you maybe dive into that in a bit more detail? Where is VLXO go in its development plan in like hyperchondroplasia and the other potential indications? And what are the size of those patient populations?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Yes, absolutely. I mean, that’s the other element I think that investors need to understand. I’m going to start it off talking about the full potential of VoxSOGO and how achondroplasia is just beginning as the five subsequent indications. As competitors come into if they come into achondroplasia, we’ll be launching in hypochondroplasia and will be well underway with those other indications with many times the growth potential of achondroplasia. So the hypochondroplasia is the next indication that we’re seeking for VoxSOGO.

The clinical development has gone extremely well. It’s running ahead of our timelines. We’ll, we project to finish recruitment of the pivotal study the first half of this year. We’ll then have data at the beginning of twenty twenty six, and we’re projecting right now for approval beginning of twenty twenty seven. So hypochondriplasia is about fourteen thousand patients potential.

Contrast that with twenty four thousand for achondroplasia. We’ve actually we’re going after the most severe phenotypes. That’s why the genetic prevalence is about the same, but we’re focused on the more severe phenotypes. And as we’ve got the proof of concept data from Dauber, we saw AGV or growth levels actually numerically higher than achondroplasia. We think this is highly derisked from a clinical trial and a regulatory standpoint.

Brian Mueller, Executive Vice President and CFO, BioMarin Pharmaceutical: And just bringing that back to the competition, Bill, if you think about the lead we have in achondroplasia, the lead we have in these other indications and the fact that these other indications with scientific proof of concept are actually significantly larger than achondroplasia, we believe that provides an avenue for growth even in light of competition.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Yes. So you asked about the other indications. So the next one I would focus on is idiopathic short stature. That is a very large patient population, about 170,000 patient population globally. Growth hormone is already approved in The United States, so these patients are treated.

There’s an unmet need of sustained growth. You see a waning effect with growth hormone. We’re right now in our Phase II recruiting our Phase II study in idiopathic short stature. We’re defining what the right dose is. It’s a different disease than achondroplasia and hypochondroplasia.

We also again have a proof of concept from Doctor. Dabra in the National Children’s Hospital. So I think we think that BOCSOGO has a role to play. And then we have Newnans, Turner’s and Shox. This we’re doing in a basket style study or three indications.

We’re in Phase two for those indications as well. Estimated launch timing is 02/1931.

Phil Nadeau, Biotech Analyst, TD Cowan: Perfect. Maybe in the last minute just turning to the earlier stage pipeline, BMN-three fifty one has some proof of concept data coming up in the back half of this year. Can you discuss how that molecule is different than the other skipping agents? And what BioMarin would consider proof of concept in the upcoming data releases?

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Yes. I mean, this is this was fun in the fourth quarter to start. I mean, as a biotech company, we are about innovation. Investors starting to ask us a lot about these readouts because we have these three readouts this year, proof of concept for March, ’3 ’30 ’3 and then the Phase III data for Palynziq in adolescence. So three important data readouts and proof of concepts and hopefully growth catalyst for the outgoing years.

March is really unique in terms of its chemistry, its skip site. We have a it’s an ASO, oligonucleotide. We have a very high bar TPP that we’re aiming for this year, which we’ll see the data for, which is a 10% effect on full nearly full length dystrophin, which we achieve is going to be significantly better than anybody else has demonstrated. So this has been this is something that’s come out of our research group. We’re really excited about the chemistry.

We’ve already dosed both cohorts for the proof of concept. And we’re waiting now for the twenty six week data. We’re going to share the model prospectively so people can see what our modeling is because basically at twenty six weeks, you still have not seen the full effect. Probably the full effect is still going to be sort of fifty two weeks. So it’s a sort of modeled S curve shape.

And again, our goal is double digit effect on dystrophin. And we think there will be a best in class.

Phil Nadeau, Biotech Analyst, TD Cowan: Perfect. With that, I think we’re out of time. Thank you for an interesting question.

Alexander Hardy, President and CEO, BioMarin Pharmaceutical: Thanks very much, Bill. Thanks, everybody. Thank you, everybody.

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