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 Ratio32.67
Earnings Date08/03/2026
Earnings Call Transcript
BMRN • 2024 • Q2
Operator
Good afternoon, and welcome to the BioMarin Pharmaceuticals Second Quarter 2024 Conference Call. Please note that this call is being recorded. All lines have been placed on mute to prevent any background noise. After the speakers’ remarks, there will be a question-and-answer session. [Operator Instructions] I will now turn the call over to Traci McCarty, Head of Investor Relations. You may begin your conference.
Traci McCarty
Thank you, Operator, to remind you, this non-confidential 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 search 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 measures 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. On the call from BioMarin management today are Alexander Hardy, President and Chief Executive Officer; Hank Fuchs, President of Worldwide R&D; Brian Mueller, Executive Vice President, Chief Financial Officer; and Cristin Hubbard, Executive Vice President, Chief Commercial Officer. I will now turn the call over to BioMarin's President and CEO, Alexander Hardy.
Alexander Hardy
Thank you, Traci, and good afternoon, everyone. Thank you all for joining us today. We're pleased to today share our strong quarterly results and the decision on ROCTAVIAN. This quarter, we've also made significant progress towards finalizing BioMarin's evolved corporate vision and strategy. This work gives us confidence we can amplify our progress and mission to deliver more impact on patients' lives and greater value for shareholders. We look forward to sharing more about our vision and strategy, the sustainable growth, accelerated profitability next month at Investor Day. Moving briefly to specific progress made in the quarter on our four strategic priorities outlined in January. Beginning with our opportunity to accelerate and maximize VOX
Cristin Hubbard
Thank you, Alexander. I'm pleased to join you on my first quarterly results call since joining BioMarin in May. Now I've been impressed by the breadth of commercial expertise and patient focus at BioMarin, and I'm excited about what lies ahead as we drive continued patient impact through the expansion of our innovative medicines. Turning to quarterly highlights. Strength across our brands drove 20% revenue growth year-over-year, including notable contributions from VOX
Hank Fuchs
Thank you, Cristin, and good afternoon, everyone. The R&D team has also been working closely with our colleagues on corporate strategy and operational efficiencies, aligning a more focused approach to R&D has resulted in great efficiencies across our VOX
Brian Mueller
Thank you, Hank. Please refer to today's press release summarizing our financial results for full details on the second quarter of 2024, including reconciliations of GAAP to non-GAAP financial measures. All second quarter 2024 results will be available in our upcoming Form 10-Q, which we expect to file later today. In the second quarter of 2024, BioMarin generated record quarterly total revenue of $712 million, representing 20% year-over-year growth and 25% on a constant currency basis, driven by continued strong demand for VOX
Operator
[Operator Instructions] Our first question comes from Phil Nadeau with TD Cowen. Please go ahead.
Phil Nadeau
On the updated ROCTAVIAN strategy. Can you talk a bit more about the changes aside from eliminating a bunch of territories, are you changing the strategy or investment in the regions in where you're going to stay U.S., Germany and Italy?
Alexander Hardy
Thanks very much for the question. So, our -- we're very pleased with the clarity that we're able to provide for ROCTAVIAN. If you remember, we said we would, by Investor Day, communicate the timing criteria for making a decision. We're pleased to be able to share this at the second quarter. So, the approach that we're taking allows us to focus geographically on those three geographies: United States, Germany and Italy also to focus our efforts within those geographies on what we have seen and we've learned to be the most important thing for the success of ROCTAVIAN, which is the patient pull-through activities mainly at the site level. So that's where we're going to focus geographically, but also in terms of types of activity within those geographies. We're also focusing our activities from an R&D and development perspective. We are stopping our life cycle development activities. Just to focus on the current main indication. We'll continue to generate long-term data, and we're also reducing our manufacturing expenses associated with ROCTAVIAN. All of this allows us to operate in an envelope of $60 million in 2025 and underpins our confidence that we're going to be able to achieve profitability for ROCTAVIAN in that time with this focused strategy.
Phil Nadeau
And when will we hear the next update on the strategy? It sounds like this is a framework an initial strategy, but you're going to continue to identify the investment and success of ROCTAVIAN. When do you think you'd be in a position to give a further confirmation of the strategy or revision of the strategy if necessary?
Alexander Hardy
No, this is a decision of going forward for ROCTAVIAN. We will continue to update you on our progress. As we've said before, the success metric we're all focused on is patients infused and treated. And so, we'll update you on our progress on those metrics, but this is our strategy. We've reached a decision. We have confidence that ROCTAVIAN can be a contributor to BioMarin's profitability going forward.
Operator
Our next question come Salveen Richter with Goldman Sachs. Please go ahead.
Salveen Richter
I just want to dig in further into ROCTAVIAN. With regard to the breakeven situation by year-end '25, could you just speak to the metrics that are giving you confidence at this time point? Is it the demand aspect? Is it 340B dynamics improving in the U.S.? And just maybe help us understand where all these moving parts are coming from to give you that conclusion.
Cristin Hubbard
Yes. Thank you so much for the question. This is Cristin Hubbard. I think that really the progress that we're seeing, and I'll be specific in the three geographies is that in the United States, we really are encouraged with the three patients that were treated in the quarter. And really, what this is around is ensuring that the sites are operationally ready to infuse product, and we've seen a fourfold increase in this. Our commercial team is very focused here, and we've seen a fourfold increase in this just since January. But then also, as Alexander alluded to, the patient pull-through. And it's really about building comfort around working through the single case agreements that we know will be true to ensure that patients are being treated. So with that type of experience, we recognize that, that will create more progress moving forward and a comfort with doing so, and we're seeing a lot of that in the U.S. today. In Italy, in particular, we are really pleased with the rapid progress that we've made in ROCTAVIAN since only receiving pricing and reimbursement since January. So we've treated a couple of patients there, and we're seeing large amounts of strong advocacy from KOLs as well as experience in the sites. And then lastly, with Germany, as I said a little bit earlier, we have reached a negotiated agreement with the sub-insurer, and we are happy to report that we now have approximately 25% of patient lives covered in Germany, and we will continue to push forward with progress with the subinsurers there. We know there's demand. We do have KOL advocacy in Germany, and we want to continue on that front. So, these are the types of metrics that we're looking at, and I'll pass it to Brian for more.
Brian Mueller
This is Brian. Just to add to Cristin's comments, in addition to those signs of progress and our continued execution across those three markets with this focused strategy that plus this $60 million cost envelope is what gives us confidence that we can manage the asset to be profitable next year.
Operator
Our next question comes from Ellie Merle with UBS. Please go ahead.
Ellie Merle
Just in terms of 333, how are you thinking about the development of 333 relative to VOX
Hank Fuchs
Ellie, this is Hank. As far as the development plans for 333, our initial thinking is, first of all, to get it into the clinic, which will start shortly. And thereafter to proceed with the most expedited path to approval, leveraging potential for superior efficacy, superior convenience or both. As regards to the program of idiopathic short stature, the opportunity there is in patients with the greatest unmet need, potentially more specifically patients who have severe stature deficiency or patients who have been unsatisfied with available current therapy. So, I hope that addresses your question.
Operator
Our next question...
Alexander Hardy
Ellie, we'll share more information on the development plans for VOX
Operator
Our next question comes from Cory Kasimov with Evercore. Please go ahead.
Cory Kasimov
Great. I have a two-part question on VOX
Alexander Hardy
Thanks very much for your questions. I'll handle the first one and then hand over to Cristin for the second one. So, the first one with regard to the potential impact of IRA. We actually think there'll be minimal impact of IRA on VOX
Hank Fuchs
Right. And your second question, Cory, just remind me real fast?
Cory Kasimov
Yes. And the difference or the similarities or differences between achondroplasia and hypochon in terms of level of market awareness of these patients and desire to seek treatment?
Hank Fuchs
Yes, hypochondroplasia doesn't occur as early in life for diagnostic purposes. So, it's underdiagnosed, and we're undertaking a lot of effort to increase its diagnosis. Having said that, we're really quite pleased with the rate of progress of the feeder study, and I'll give you more of an update about that. But as with a lot of genetic conditions, once a diagnosis -- once the therapy is in place, then there's more diagnostic effort undertaken once there's more diagnostic efforts undertaken, there's more ambition to try during the development program, we're hoping to try to begin the process of increasing awareness and ultimately uptake in the hypochondroplasia population.
Operator
Next question comes from Jessica Fye with JPMorgan. Please go ahead.
Jessica Fye
How are you thinking about the evolution of the competitive landscape in achondroplasia when we think about TransCon CNP and infigratinib potentially on the horizon? And maybe as a follow-up, would you ever consider evaluating VOX
Alexander Hardy
Thanks very much for your question, Jessica. So overall, we feel very confident about our competitive profile in achondroplasia and in the subsequent indication. As you know, CNP pathway is amenable to all the five different indications that we're now pursuing after achondroplasia. And even with achondroplasia, we think with the movement in achondroplasia to treat the youngest infants zero to five population, which is very, very part of the growth, but also very important in terms of optimal patient outcomes. The early start treatment, the greatest possible impact on the child's health outcomes as well as growth. So, we think that's a really compelling position in achondroplasia. The lead we have the safety profile of the durable efficacy, we feel very, very good about achondroplasia. And then obviously, in the subsequent find indications where we think we're going to be very competitive. With regard to your second question, I will hand it over to Hank to answer the possibility of the combination approach.
Hank Fuchs
VOX
Operator
Our next question comes from Paul Matteis with Stifel. Please go ahead.
Paul Matteis
Great. As you think about the next leg of growth for VOX
Cristin Hubbard
Yes. Thanks, such for the question, Paul. I will say, I don't know the exact numbers the actual treaters per say that you're referring to, but I will say where our efforts are focused. So in the United States, for instance, where we believe there's going to be a large growth opportunity for us. This is really an area where we do see more decentralized care. And so what I mean by that is you certainly have it treated by some geneticists in some offices and in fact, some pediatric endocrinologists as well. But what we're finding is a lot of these patients are also sitting with general pediatricians. And so what we're really focused on is ensuring that we have awareness around VOX
Operator
Our next question comes from Joseph Schwartz with Leerink Partners. Please go ahead.
Joseph Schwartz
I'll ask about a couple of your priority pipeline programs. First, on BMN 333, I was hoping you could give us some insight into the long-acting technology that's used there. And also help us envision the expected time to market for BMN 333. And then, what are you hoping to see for BMN 351 at the target dose or doses since it's moving behind several other next-gen next gen Exon skippers, it would be helpful to hear what you hope to see efficacy wise at different dose levels?
Hank Fuchs
As far as the specifics about 333 in terms of design, we really tried to leverage a lot of safety and information that's already available on linking chemistry. I'll get into that in a little bit more detail at Investor Day. Obviously, one of the other critical aspects of the program is to ensure sustained exposure without a lot of thoroughly high exposure or dumping is sometimes is referred to in drive delivery circles. So, I'll get into a little bit more details, but really a big orientation around safety and consistency of exposure. On 351, acknowledging that there are a lot of competitors, it doesn't seem like anybody is really able to get to high levels of dystrophin either by conjugating polymorphelino with CAGS or with transferrin receptor antibodies. And so what our strategy has been all along, is to try to develop a more potent molecule by virtue of targeting a different regulatory site in pre-mRNA processing. The advantage of that approach has been twofold: One in preclinical miles. We can get dystrophin expression up to 40% in not just skeletal muscle, but also in high levels and diaphragm, high levels in heart. And the second is that we can do that at levels where we have confidence about the safety profile, given our experience with [indiscernible]. I'll give more detailed updates on status of the program and expectations to when you're going to see data at Investor Day, but we're very encouraged by the progress that we're making in 351.
Operator
Our next question comes from Akash Tewari with Jefferies. Please go ahead.
Unidentified Analyst
This is PV on for Akash. So, on BioMarin 351 for DMD, how should we think about the translation from animal to human models when it comes to dystrophin expression. In mice models, you showed normal dystrophin levels as high as 98%. I guess, how well does that data translate to humans perfect normal dystrophin? And also for borrower success, what would you need in order to move forward.
Hank Fuchs
Yes. Well, I think one of the challenges of translation is that there hasn't been a lot in humans that's really moved the needle in terms of dystrophin inspection. So, a little bit of that question is TBD. What we talked about at last year's R&D Day was based on our animal model where if we can achieve the tissue concentrations of 351 that we achieve [indiscernible], we should be in a very much higher level of dystrophin expression and partly we based it on a relatively unique animal model, which carries the human equivalent of the gene that is to be skipped. And so, we have a very fulsome assessment in vivo skipping potential. And we've got a lot of safety data, both from rodents on human primates and humans on our class of compounds. And so, we're optimistic based on that, that we'll be able to see much more meaningful levels of dystrophin expression. And of course, it's really the near full-length dystrophin that motorizes the muscle, if you will, that will enable much greater function of the muscle. And so far, that hasn't been achieved. And that's what we're looking to achieve with 351.
Operator
Our next question comes from Chris Raymond with Piper Sandler. Please go ahead.
Chris Raymond
Just maybe two questions. VOX
Hank Fuchs
Sure thing, Chris. The challenge oftentimes in genetic diseases is the diagnostic odyssey and the delay. And we hope that during our development program, we can overcome some of those barriers and start to teach our colleagues that early diagnosis is really the path to improve overall outcomes in genetic conditions. As to regulatory requirements for the under three population, that's a TBD. The one thing I'd say about that is, we've got a lot of wind at our back with health authorities around the world who recognize the safety and the efficacy of VOX
Operator
Our next question comes from Gena Wang with Barclays. Please go ahead.
Gena Wang
I will have a few questions regarding ROCTAVIAN. Given your comments on 2025, is it fair to assume that you actually your assumption is about 30, 40 patients that will be treated in 2025 in order to make breakeven? And then I have one question regarding the Germany. That 75% of the insurance, why -- what's the reason they didn't cover now? And when do you expect to cover will be in place. In the U.S., the three patients that treated were these from three different sites? How many is now past contracting phase and in the process of payer discussion?
Brian Mueller
This is Brian. I'll start with your first question. We're not giving specific ROCTAVIAN patient or rent revenue guidance today. It was important to talk about the progress we're seeing in the launch that Cristin covered earlier. And then this cost envelope for next year and the goal of getting to profitability. So, framing it up in that way as demonstration and articulation of the strategy, so you can view that as, again, minimum level of revenues, if you will, but not getting into further specifics at this time.
Cristin Hubbard
Yes. And I'll take the second two questions. So, with regard to the sub-insurers, I mean, it's impossible to give you an exact time of it, but I can say that we're making progress in these discussions that we're having. And really, we're just pushing through insurer by insurer and making sure that we are addressing the needs, which, of course, are going to be about risk carrying. On the U.S. side, you asked about the HTCs that have been treated. We've had those three patients treated. They were all in separate HTCs and we're actually geographically dispersed across the U.S.
Operator
Our next question comes from Kostas Biliouris with BMO Capital Markets. Please go ahead.
Kostas Biliouris
Congrats on the strong quarter. One question from us on ROCTAVIAN acknowledging that HEMGENIX in hemophilia B was approved six months before ROCTAVIAN in the U.S. and that the hemophilia depopulation is 4x to 5x smaller than the hemophilia A. How are you benchmarking ROCTAVIAN revenues versus HEMGENIX, which generated about $15 million to $30 million in the first half of 2024 based on our estimations.
Brian Mueller
Yes. Thanks for the question, Kostas. I'll jump in on that one, Brian, since I've been tracking the HEMGENIX launch along the way. There's different circumstances there between the two launches. Of course, there's differences as well, but we're not getting into launch comparisons or benchmarking HEMGENIX, more of a qualitative estimate. Again, we are going through similar contracting in access challenges. So, there is a comparison to be made, but we don't do that quantitative level.
Operator
Our next question comes from Mohit Bansal with Wells Fargo. Please go ahead.
Mohit Bansal
Just want to reconcile some of the VOX
Brian Mueller
Yes. Thanks for the question, Mohit. And there are going to be different dynamics from quarter-to-quarter in terms of the patient growth rates and revenue growth rates. I tried to address that a bit in the prepared remarks, but I might even point you to the last quarter where there was a significant amount of functions adds, but you didn't see that necessarily a revenue growth. And this time around in the second quarter here because of that order timing that you noted, thanks to increased supply becoming available earlier, that dynamic flipped the other way around. And this time, revenues were ahead of patients a bit and recognized also that in the second half, we could see patient growth rates exceed revenue growth rates in either Q3 or Q4. But I mean, big picture, we're in the third year of this launch. We're still less than 20% penetrated with a long way to go globally. We're on track for VOX
Operator
Your next question comes from Olivia Brayer with Cantor Fitzgerald. Please go ahead.
Olivia Brayer
As you consider profitability next year and just broader uptake of ROCTAVIAN, how are you thinking about sales contribution and breakdown across the three geographies that you're focused on? I'm just trying to get a sense for whether you expect the U.S. to really make up the majority of ROCTAVIAN sales going forward.
Brian Mueller
It's a good question. Noteworthy there just in terms of the U.S. versus ex-U.S. dynamic. The price of ROCTAVIAN revenue per patient on average in the U.S. is going to be higher. So, as they continue to make progress in the U.S. market, those patients are going to contribute more revenue. Again, we're focused on these three markets, both with the restructured cost envelope as well as the tactics in this dedicated and focused business unit that will be working exclusively on ROCTAVIAN. It's going to allow the rest of the business to focus on the remainder of the portfolio. So, we're viewing this as the three markets together, not getting into further details of each of those individual markets at this time. But important to know that those three markets are the focus area and that will be the contributors to revenue in 2025.
Operator
This will be our last question. Our last question comes from Luca Issi with RBC Capital.
Luca Issi
Congrats on the progress. Maybe two quick ones [indiscernible]. You obviously prioritize in Italy, Germany and the U.S., but what's the plan for the other geographies where you still have rights? Is there any plan there to find a partner or try to monetize that in any capacity? Any color there, much appreciated. And maybe sticking to [indiscernible]. Can you maybe just talk about net pricing in Italy versus the U.S. I believe last quarter, you reported $800,000 for the only patients treated in Italy versus today $7.4 million from three patients in the U.S. and two in Italy, that would imply net pricing of $1.9 million in the U.S. and $800,000 in Italy. Is that right? And you still how should we think about pricing in Germany?
Brian Mueller
It's Brian. I'll take that one. So first of all, in terms of the focus, it's an important question because the key element behind the driver of the $60 million cost structure for next year is focusing entirely on supporting patients in those three markets as well as the long-term overall clinical and regulatory commitments that come with ROCTAVIAN that means that we are not investing in additional markets at this time. As we prove out and continue to gain traction and get confidence in ROCTAVIAN's overall progress over time, we will retain the right to make select investments that are value accretive to the assets over time. But important to note that it is just those three markets commercially at this time. And on pricing, I'll share, again, it's going to be dynamic over time with variations in the global pricing. I will share that the net revenue that we recognized for those five patients across the two regions where we had sales in Q2 was consistent with our expectations and prior communications. The largest gross to net item in the U.S. is 340 rebates at 23% and then traditional planned discounting in Italy. So, I'll stand by prior comments on that.
Operator
I will now turn the call back to CEO, Alexander Hardy, for closing remarks.
Alexander Hardy
Thank you very much, everybody, and thank you for joining our call for all your questions. We're very proud of the quarter and the strong growth, both in revenue and profitability that BioMarin has achieved this quarter and very excited about the outlook and raising our guidance. on both the top and the bottom line. And we're also very excited about the progress we've made so far this year with the priority prices we set out in January. A reminder of those priorities: One was to accelerate and maximize the VOX
Transcript from August 5, 2024
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