BMRN - NASDAQ
BioMarin Pharmaceutical Inc.
BMRN·NASDAQ
$54.66
+2.5%HealthcareBiotechnology
BioMarin Pharmaceutical Inc. develops and commercializes therapies for people with serious and life-threatening rare diseases and medical conditions. Its commercial products include Vimizim, an enzyme replacement therapy for the treatment of mucopolysaccharidosis (MPS) IV type A, a lysosomal storage disorder; Naglazyme, a recombinant form of N-acetylgalactosamine 4-sulfatase for patients with MPS VI; and Kuvan, a proprietary synthetic oral form of 6R-BH4 that is used to treat patients with phenylketonuria (PKU), an inherited metabolic disease. The company's commercial products also comprise Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, which is delivered through subcutaneous injection to reduce blood Phe concentrations; Brineura, a recombinant human tripeptidyl peptidase 1 for the treatment of patients with ceroid lipofuscinosis type 2, a form of Batten disease; Voxzogo, a once daily injection analog of c-type natriuretic peptide for the treatment of achondroplasia; and Aldurazyme, a purified protein designed to be identical to a naturally occurring form of the human enzyme alpha-L-iduronidase. In addition, it develops valoctocogene roxaparvovec, an adeno associated virus vector, which is in Phase III clinical trial for the treatment of patients with severe hemophilia A; BMN 307, an AAV5 mediated gene therapy, which is in Phase 1/2 clinical trial to normalize blood Phe concentration levels in patients with PKU; and BMN 255 that is in Phase 1/2 clinical trial for treating primary hyperoxaluria. The company serves specialty pharmacies, hospitals, and non-U.S. government agencies, as well as distributors and pharmaceutical wholesalers in the United States, Europe, Latin America, and internationally. BioMarin Pharmaceutical Inc. has license and collaboration agreements with Sarepta Therapeutics, Ares Trading S.A., Catalyst Pharmaceutical Partners, Inc., and Asubio Pharma Co., Ltd. The company was incorporated in 1996 and is headquartered in San Rafael, California.
At a Glance
Live SnapshotMarket Cap$10.56B
EPS1.8200
P/E Ratio30.03
Earnings Date08/03/2026
Earnings Call Transcript
BMRN • 2025 • Q3
Operator
Hello, and welcome to the BioMarin Pharmaceutical Third Quarter 2025 Conference Call. [Operator Instructions]. I would now like to turn the conference over to Traci McCarty. Please go ahead.
Traci McCarty
Thank you, operator. To remind you, this nonconfidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market and developments by competitors and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, such as 10-Q, 10-K and 8-K reports. In addition, we will use non-GAAP financial metrics as defined in Regulation G during the call today. These non-GAAP measures should not be considered in isolation from, as substitutes for or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are available in the Investor Relations section of our website. Please note that our commentary on today's call will focus on non-GAAP financial measures unless otherwise indicated. Beginning on Slide 3 and introducing BioMarin's management team, joining today's call, Alexander Hardy, President and Chief Executive Officer; Brian Mueller, Chief Financial Officer; Cristin Hubbard, Chief Commercial Officer; and Greg Friberg, Chief R&D Officer. I will now turn the call over to BioMarin's President and CEO, Alexander Hardy.
Alexander Hardy
Thank you, Traci. Good afternoon, everyone, and thank you for joining us on today's call. Starting on Slide 5, I am very pleased with the strong results across the business, leading us to raise full year total revenues guidance at the midpoint and reaffirm our VOX
Brian Mueller
Thank you, Alexander. Please refer to today's press release for detailed third quarter 2025 results, including reconciliations of GAAP to non-GAAP financial measures. All 2025 results will be available in our upcoming Form 10-Q, which we expect to file in the coming days. Starting on Slide 7. Strong global demand across our portfolio of innovative medicines drove a year-to-date total revenue increase of 11% compared to the same period in 2024. Revenues from VOX
Cristin Hubbard
Thank you, Brian. I want to begin by acknowledging the exceptional work from our teams across the world, whose dedication has delivered strong year-to-date results for BioMarin's commercial portfolio. Now moving to Slide 11. We are pleased that PALYN
Gregory Friberg
Thank you, Cristin. Now moving to Slide 16 and to provide a little more color, hypochondroplasia is a serious condition with a potentially broad impact on the health and daily life of those affected. Children and adults with hypochondroplasia can face significantly higher rates of comorbidities and procedures when compared to the general population, covering areas like respiratory, orthopedic, ear nose and throat and mental health. As a result, they often undergo more surgeries, adding to the overall burden of the condition. Beyond the medical challenges, the condition can affect quality of life in very real ways, making everyday tasks harder and creating social and emotional strain. These insights reinforce why it's important to advance treatment options for hypochondroplasia, a condition for which no approved therapies are broadly available today. Now moving to Slide 17. At ASBMR in September, we presented important spinal morphology data for children under 5 years old with achondroplasia. These children were treated on our Phase II CANOPY study with either vosoritide or placebo. Spinal morphology is one of the factors that contributes to spinal stenosis, a leading cause of morbidity in achondroplasia. Spinal stenosis, particularly in the lumbar region, is a serious and all too common medical complication in achondroplasia, resulting in pain, muscle weakness and reduced mobility in the most severe cases. Since measures of spinal canal reach near final size by age 5, early intervention is essential to prevent complications. Radiographs of the spine in our CANOPY study demonstrated that children who received VOX
Operator
[Operator Instructions]. Your first question comes from Phil Nadeau with TD Cowen.
Philip Nadeau
My question is on the 2027 guidance. Can you talk a little bit more about the scenarios you see? And maybe more specifically, why are you rescinding the guidance now? What has changed over the last year since it was initially issued?
Brian Mueller
Phil, this is Brian. Thanks for the question. I'll take that one. Since we shared the original $4 billion 2027 outlook last year, we've had a year to assess the various factors that have arisen since then, including the impact of potential VOX
Philip Nadeau
And maybe just a follow-up. I think when you issued the guidance a year ago, you were -- you suggested that competition was factored into the guidance. Do you now have a different appreciation or a different concern about how much share that competition could take, specifically against VOX
Brian Mueller
Yes. Thanks, Phil. We did the initial work last year following Investor Day when the competitor data was released and at the time, modeled some competitive impact. There was some ability to absorb that into our original forecast at the time, and we did have some other upsides last year. We have taken a different view. We've observed trends in the marketplace, both what we are experiencing in these markets as well as, again, different potential competitor scenarios. And this is not a single point in time estimate. It is not our forecast. We merely wanted to reflect what the range of outcomes could be from our view.
Operator
The next question comes from Salveen Richter with Goldman Sachs.
Salveen Richter
Could you speak to why VOX
Brian Mueller
Salveen, this is Brian. I'll take the first part of the question on Q3, VOX
Alexander Hardy
Salveen, thanks very much for your question. It's Alexander. I'll answer the part of your question around business development. First off, I would say we've got strong underlying business performance in both Enzyme Therapies and in Skeletal Conditions. I mean, 11% year-to-date growth across both business units. But we are also producing significant cash flow. Our balance sheet is very strong, and we have conviction that assets are worth more in our hands than they are right now. So business development is a very important part of our strategy right now. We have a number of deals that we're in pursuit of. We've always been a company focused on early-stage collaborations. But what is different or how our strategy has evolved is we're also looking at Phase III pre-commercial and commercial assets because, again, we think we can add value to all stakeholders with those in our hands. So we have a number of deals in the works and in sight. I mean, obviously, business development is never completely within your control, but it remains a very high priority for us, and we're looking forward to sharing more information when we have that.
Operator
The next question comes from Cory Kasimov with Evercore.
Cory Kasimov
I guess I have a follow-up on 2 questions that were asked. First of all, Brian, I appreciate the commentary you made on the previously issued '27 guidance. I think the way you framed it is helpful. However, I'm curious if you have any qualitative commentary you could also share on your prior long-term mid-2030s guidance with regard both to the opportunity for VOX
Brian Mueller
Thanks, Cory. This is Brian. Appreciate the question. And it's a similar analysis, as I shared with the 27 range of estimates there. We are absolutely planning on continued sustainable growth across the business. I'll share that we are still targeting high single-digit sustainable growth rate for the enzyme therapies over time. VOX
Alexander Hardy
Cory, this is Alexander. I'll answer the second part of your question with regard to capital allocation. It's obviously something we discuss with our Board frequently. It's very, very important, obviously, that we're really effective stewards of the significant capital that we've generated through the success of our business. We actually think that business development is the greatest opportunity for us to drive significant incremental growth rate on the top line, and that's the most highly correlated with stock appreciation. We have this strong financial profile. We have this capability in rare diseases, whether it's in research, development, manufacturing, commercialization. And we're convicted that assets are worth more in our hands than where they are right now. In an environment that we have right now in the U.S. with biotech stocks, there are many rare disease companies that are undercapitalized and underresourced and those assets are worth more in our hands. So we are -- as you can tell, we're very convicted that business development is the best use of our capital. That remains our priority, but it's something that as a Board, we constantly look at.
Operator
The next question comes from Joe Schwartz with Leerink Partners.
Joseph Schwartz
For the upcoming BMN 333 PK data, what specific exposure levels would give you confidence that you can achieve clinical superiority over VOX
Gregory Friberg
Thanks, Joe. This is Greg. I'll take a stab at both of those. With regard to the exposure levels, the stated level that we were looking for from our Phase I PK study was while we were looking at the free CNP levels. So in the case of VOX
Operator
The next question comes from Jessica Fye of JPMorgan.
Jessica Fye
I had a couple on the guidance and a couple on the pipeline. On the guidance, I don't have FactSet. What is the 2027 FactSet consensus, excluding ROCTAVIAN, just we know what that lower bound is? And then the second one on the guidance, maybe just asking about the other 2 elements of the longer-term targets that I don't think Cory mentioned. Should we still expect 40% non-GAAP operating margin starting in '26 that could expand to the low to mid-40s and the greater than $1.25 billion of CFO starting in 2027? Or were those sort of top line dependent and more in question now? And then the 2 on the pipeline, for 351, my understanding is we'll get 6-month biopsy data for the 6-milligram cohort. What should we expect for the 9-milligram cohort? And second one on the pipeline is for hypochondroplasia, can you remind us of the powering for that trial? And is that fig sufficient in your mind? Or is there some minimum delta on efficacy you want to see to meet your target product profile?
Brian Mueller
Thanks, Jess. This is Brian. I'll speak to those first couple. So first, just to clarify that FactSet math for you, we are showing FactSet total revenue consensus for 2027 of $3.725 billion, and that includes $75 million of ROCTAVIAN. So that without ROCTAVIAN consensus would be $3.65 billion in '27. And then with respect to the 40% operating margin target next year, that does remain our target. Just a reminder that we've grown profitability and operating margin significantly over the last 2 years due to our strong underlying execution and the focused cost transformation, we do expect that to continue heading into next year and hold that 40% target. I will say that our operating margin objective is rooted in driving efficiency in the business through cost and process transformation, but without compromising value-creating activities. So in the event, in the lower end scenario over time, if we do face a trade-off, and this is less next year or more beyond, if we face a trade-off between preserving those value-creating activities and hitting the 40% margin, we will prioritize value creation to maximize long-term shareholder value. But right now, cost transformation and the target for next year is on track. We'll be prepared to update that again when we issue '26 guidance early next year.
Gregory Friberg
Thanks, Jessica, and this is Greg. Let me take your 2 pipeline questions. With regard to 351, just as a reminder, what you can anticipate is a top line result that will be a combination of all the safety data that we have. That will be the 6-milligram and the 9-milligram per kilogram cohorts as well as the early data that we have, we'll be looking at the 12 milligram per kilogram, which is currently enrolling. What we will be also looking at is biopsy results and dystrophin levels from muscle biopsies in both the 6 and the 9-milligram per kilogram cohort. Our goal, again, is to have a level that predicts it steady state that we would be hitting this 10% level, which is a quite ambitious target. That's not correcting for fat and muscle content. That is a level that has yet to be seen in programs targeting exon 51. With regard to hypochondroplasia, we powered the study to measure for an AGV delta similar to what's been seen with VOX
Brian Mueller
Sorry, Jess, this is Brian. I'll come back again because I realized you had another part to your question about that '27 cash flow and that greater than $1.25 billion. So I'll use your words there. That was top line dependent. And therefore, in the lower case scenarios would be somewhat proportional to the overall revenue scenario. But I will say, again, we're generating significant operating cash flow today, almost $370 million this quarter, over $700 million year-to-date. We've got a number of working capital optimization initiatives that we're introducing across the enterprise over the next 2 years. As Alexander touched on with respect to our capital allocation strategy and business development, these cash balances and the sustained cash flow that we're building has the opportunity to be -- opportunity to be deployed as growth capital going forward. So it's a top priority for the company. But in short, that $1.25 billion was tied to the $4 billion.
Operator
The next question comes from Paul Matteis with Stifel.
Julian Pino
This is Julian on for Paul. You talked about how your views have changed on the market as well as in thinking about some of these best case scenarios and some of these more bearish scenarios. Can you talk about the contributions of potential commercial competition versus the risk to some of these competitors entering the market? And how much do you think could be attributed to your overall view on being able to have a positive outcome in litigation? Just curious on what you think about that. And then further, on the DMD program, can you just talk a little bit more about the 10% bar that you're sort of setting for yourselves there? Obviously, I think a lot of investors believe that the bar for regulatory approval is meaningfully lower when thinking about exon skippers that are currently approved. So just thinking about what sort of informs that view and if there's any sort of outside case that you could push a program forward that doesn't meet that bar.
Brian Mueller
Thanks for the question. This is Brian. I'll start with just outlining those bookends of the lower case estimates and the higher case estimates again. And then I'll hand it over to Cristin to make a couple of remarks on the specific market trends we're observing. So for the sake of making these assumptions and developing these -- the lower end of these estimate scenarios, again, we took the assumption that 2 competitors come to market and that by the end of '27, they've been successful with their launch and take significant share. And then in the higher case estimates, and there's a range in between outcomes, of course, that includes either less competition or success with our intellectual property defense. And again, neither of those are our official forecast. We are illustrating what the revenue impact of those potential outcomes could be over time. And again, at the lower end, comfortable with consensus today. In the higher end, we can still get back to $4 billion. And again, this is excluding ROCTAVIAN. Cristin, do you want to comment on that?
Cristin Hubbard
Yes Brian. And so yes, so looking at the overall trends, I just want to note what Brian, you said earlier and we said in the prepared remarks, and that is that we have continued to add patients on treatment with VOX
Gregory Friberg
Thanks, Julian. This is Greg. Just if I could tackle the DMD question, if that's okay. Yes, we have set a pretty ambitious bar with the 10% level. Just to back up a little bit, of course, Duchenne's muscular dystrophy, the name of the game is opening a therapeutic window in these patients and delivering meaningful results. We've made some choices with the way we've engineered this molecule. We've chosen to use so-called phosphorothioate chemistry instead of what most exon skippers use, the morpholino approaches. That opens this opportunity, again, to open a large therapeutic window for what we think will be a potentially dramatic effect. There are some challenges associated with that as well, though. Weekly administration is required. And the reality is that steady state because of the very long tissue half-life will be out at a year or more. And so what we've done is we've set an ambitious target. We know that we're looking at biopsies at the 6-month time frame. Now as a quick reminder, if we see something between about 3% and 5% at 6 months, that will translate in our model to 10% at steady state. We chose that number because of the human genetic data that suggests dramatically improved functional outcomes in patients that reach those sorts of levels, similar more to a Becker's muscular dystrophy. And we think in the face of some of the characteristics of this molecule, we think that, that sort of doubling of dystrophin as compared to some of the data that's been produced with other exon 51 skippers would be an undeniable advance in the field. And so while we also will be looking, of course, at functional data, we'll look at the totality of the data, that 10% bar is our true north right now to deliver something meaningful for patients.
Operator
The next question comes from Chris Raymond of Raymond James.
Christopher Raymond
Just 2 actually for me. And Brian, I heard what you said about '27 not being guidance, just a sort of range of outcomes, but -- so I won't say it's this way. But is it safe to say you're more concerned about TransCon CNP [indiscernible]? And I guess it is -- when you talk about that FactSet number being sort of the low end, is it your assumption that Ascendis gets first cycle approval with just 1 year's worth of data when their PDUFA date comes next month? Or does that even -- does that factor into your thinking? I know it's a little bit removed from 2027, but just kind of are you getting that granular in your thinking? And then maybe an M&A question, Alexander. You guys talk about wanting assets that are under resourced and could be better served by the BioMarin infrastructure or the Inozyme asset, 401, can you maybe talk about how you have leveraged and improved upon Inozyme's efforts in terms of patient identification, outreach, other things that you've done to make that asset better?
Brian Mueller
Thanks, Chris. This is Brian. I'll start. I appreciate the question. With respect to the 2 potential competitors, first, I'll say I don't think we're going to comment on them versus each other, one versus another. But I will say, this is, I think, the heart of your question, what we've assumed in those lower case estimates that I referred to in that lower end of the range, we have assumed middle-of-the-road assumptions with respect to that -- those companies communicated time lines for their approval, one of which has a PDUFA next month, as you noted. And then following those action dates and communicated approval and launch time lines by those other companies, we then model what a successful launch for those competitors could look like. And that's where we get to this point of the lower end estimate where VOX
Alexander Hardy
Chris, I'm going to -- I'll take the first part of the question and hand it over to Greg because obviously, 401 is very much in the development stage right now. But overall, what BioMarin is now is we're executing rare disease at scale. I mean we're in 80 countries with an incredible muscle. And we're confident that that's going to magnify the potential impact and ability to reach patients with these genetic conditions all around the world. Our capabilities, the ability to find patients to start them on therapy and then keep them on therapy, these are capabilities we've built over 20 years. So very confident that should this product be approved, we'll be able to leverage that and achieve significant things for 401. But right now, our focus is on the execution of the clinical program, and I'll hand it over to Greg.
Gregory Friberg
Thanks, Alexander. Yes, Brian, just as a quick reminder, again, the deal closed on July 1. So we're not quite 4 months into the integration at this point. It's premature to cite, I think, too many examples of the impact that, that scale of our capabilities and resources can have on the totality of the program. It is very early days. But I will reassure you again that we're leveraging all of our capabilities, whether it's interacting with regulators around the world, whether it's looking for additional indications. And that first -- that first sign, I think, that we'll be able to talk about in future calls will be our preparation that's ongoing for an adult indication in this ENPP1 deficiency area. We're very much looking forward to taking this asset that the Inozyme team, quite frankly, did a remarkable job being able to recruit these very difficult to find patients, difficult to reach patients on to a pivotal study. And we're looking forward to turning the card over for the ENERGY-3 study in the first half of next year.
Operator
The next question comes from Sean Laaman with Morgan Stanley.
Sean Laaman
I just get your latest thoughts on orphan drug exclusivity, kids under 5 and what you think about the potential switching to a competitor as the first one. And the second one, if I'm getting the narrative right, without business development, BioMarin is a capital accumulator. Just to get your thoughts on what you think your balance sheet capacity is and what you see as an efficient balance sheet structure.
Cristin Hubbard
Sean, this is Cristin. I'll take the first question. And I think it really comes down to that element of a patient switching. So assuming -- as we're looking at there being a potential for more therapies on the market for the treatment of achondroplasia, we do think that there's a distinct difference between patients that are new to therapy that are naive and the choices that they will make and importantly, those that are already on therapy and seeing great efficacy. So what we hear in both our market research and in our conversations with physicians and families alike, we hear that the majority of patients when they are looking at the opportunity to switch, if they are satisfied with their treatment, they will more likely than not remain on therapy. Now of course, some will choose not to, but that is irrespective of orphan drug exclusivity. That is just a decision that a physician, caregiver and patient are likely to make in that moment. We do think that the adherence rates that we see on VOX
Brian Mueller
And I'll answer the firepower part of your question there, Sean. This is Brian. We estimate that our total firepower is between $4 billion to $5 billion. We're just reporting $2 billion of cash investments on hand, a significant portion of which is available to invest in future growth. And then with our current and growing EBITDA profile, we do have a chance to leverage our earnings and assuming a reasonable ratio, we believe that in total, we've got $4 billion to $5 billion to deploy as growth capital.
Alexander Hardy
Sean, I just want to jump in. Did you -- was your question around orphan drug exclusivity as well?
Sean Laaman
Sure. It was.
Alexander Hardy
Okay. I apologize about that. Yes, I mean, we've submitted a petition to the FDA concerning the orphan drug exclusivity for VOX
Operator
The next question comes from Akash Tewari with Jefferies.
Unknown Analyst
This is [
Brian Mueller
Thanks for the question. And yes, I tried to capture the primary takeaway from this exercise, which is that we've modeled a significant level of scenarios across all of our portfolio, across all markets and given the various key assumptions. We did that given the significant level of investor interest on the topic. We appreciate that when we gave the original $4 billion guidance at Investor Day last year, that was before seeing the first competitor data here and have made some updates along the way but really outlining the full range of outcomes and including a lower case that has 2 competitors launching successfully, but yet our revenue still landing at current consensus for '27, we thought would be useful. I am not characterizing that as a worst-case scenario nor am I characterizing the $4 billion as a best case scenario. We just decided to share with you a range of our lower case estimates and our higher case estimates. And on the upside, that would include, I mentioned as an example, intellectual property defense success, but it could also include success across the entire portfolio or competing successfully.
Operator
That concludes the Q&A portion of the call. I will now turn it back to BioMarin's President and CEO, Alexander Hardy.
Alexander Hardy
Thank you, operator. We are pleased with the third quarter results across the business, leading us to raise full year total revenues guidance at the midpoint and reaffirm our VOX
Transcript from October 27, 2025
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