BioMarin Pharmaceutical Inc. (BMRN) Earnings Call Transcript & Summary

Good morning, and thank you for joining us on day 2 of Guggenheim genomic medicines at our Disease Conference. I am Debjit the therapeutic analyst at Guggenheim. My privilege to host Brian Mueller, BioMarin's Executive VP and Chief Financial Officer. Thank you for your time today, Brian.

Thanks, Debjit. Appreciate you having us.

Awesome. So before we get started with the Q&A, do you want to spend a minute just walking people through BioMarin now that the company needs any introduction.

Yes, absolutely. Thanks for the opportunity. I've got some opening remarks. I'd love to share, really appreciate the opportunity to speak to you today and happy to provide a high-level update on the company. As you know, BioMarin is a best-in-class genetic disease company at an inflection point of transformative growth. Starting with our solid profitable IRA insulated and growing base business of enzyme products, we built the foundation for value creation, generating nearly $2 billion in annual revenue from that enzyme business as well as positive operating cash flows. Some of these enzyme products have been on the market for many years and have built in barriers to entry, including the high cost of goods due to the complexity of manufacturing the medicines as well as the small patient populations. Our high-touch patient support is another barrier to entry since these products are the only ones approved and available around the world. Beyond building the strong base business, we've been thrilled to see rapid patient uptake of our most recently approved product, Voxzogo for the treatment of children with achondroplasia. Contributions from Voxzogo 2022 drove full year revenues of $2.1 billion and position us for meaningful top and bottom line growth again in 2023. Recent company news includes the acceptance by the FDA of our supplemental NDA to expand the use of Voxzogo to children under the age of 5 since we're already approved for children 5 and older in the U.S. We look forward to a PDUFA action date later this year for a potential age expansion in the U.S. Recall that in January, European health authorities validated our application for the expanded use of Voxzogo for children under the age of 2 since Voxzogo is already commercially available in Europe for children 2 years and older. We expect to hear from European health authorities on that label expansion in the second half of this year as well. This is great news for families with very young children as we believe the earlier treatment begins, the better the outcome. We're seeing significant uptake of Voxzogo in Japan, where it is approved for children of all ages from newborn through 18 years old. The demand for Voxzogo around the world has exceeded even our high expectations. And as a result, has led to our double-digit top and bottom line growth expectations for full year 2023. It's been a spectacular launch. And if these age label expansions come to fruition, 1,000 additional families will have access to Voxzogo the only improved medicine that treats the underlying genetic cause of achondroplasia. With Roctavian the only approved gene therapy for the treatment of hemophilia A we plan to provide an update on the European launch progress on our upcoming first quarter call later this month. In the U.S., we also look forward to the outcome of our PDUFA action date at the end of June. We have been preparing for the U.S. launch of Roctavian since 2020. So suffice it to say, we're ready to go. We're hopeful that people with severe hemophilia A will have a new treatment choice available should FDA approve the product later in June. It's a very exciting time at BioMarin and we look forward to doing our good work for those who benefit from targeted genetic medicines only now for the treatment of larger patient populations we operate in 78 countries with integrated world-class capabilities, including manufacturing, R&D, regulatory and commercial. We're poised to deliver another record-setting year of growth and profitability from our wholly owned portfolio of 7 marketed products. And I'll turn it over to you for questions, Debjit.

Thanks for the intro Brian. And let's start with your enzyme replacement business. As you mentioned, it's sort of insulated from the IRA, where do you see the franchise about in a decade? Mid-single-digit growth sustained into 2025, is that sort of the right way to think about it?

It is. It is. So just as a backdrop, with the continued success of the Voxzogo launch hoped over the next few years, as well as hopeful approval and successful launch of Roctavian in the U.S. and the launch ramping in Europe. When we say that we have a plan and would have a goal of getting to $4 billion to $5 billion of revenue by the middle of the decade. That's basically comprised of 50% roughly of this base business and 50% Voxzogo and Roctavian which, again, depending on time and each of the respective potential launches could happen at different times. So we've discussed both Voxzogo -- each Voxzogo and Roctavian as being $1 billion-plus opportunities. So on the base business, if we think we can get to roughly $2.5 billion or by the middle of the decade, it's exactly that. Mid to -- in some years, depending on purchasing patterns and the product, it could be high single-digit growth. As I've mentioned and as you're aware, some of the enzyme replacement therapies been on the market for almost 10 years or more now. Also another key contributor to the growth is Palynziq. Just recall that Palynziq was launched just a few years ago. And while the adult PKU population in the U.S. is dispersed and there are several folks in that population that are not in the clinic. There's a number of adult PKU patients that are in the clinic and in some cases, may have been taking Kuvan previously. So that's a large market opportunity that we're still pursuing in the U.S. So we think Palynziq is a good runway still. And that's a key part of that base business growth.

So you mentioned Palynziq, do you think the disruption that you saw here in COVID and a year post-COVID has that started to normalize given the sort of lag time between initial patient dosing until they come to sort of get titrated to the right level before you can start billing?

Yes. Great question. Thanks. So what we observed during the pandemic -- during the core shutdown and then it was interesting, we observed even as the rest of the world started to open up these PKU clinics were late to open up. And that's why we continue to struggle with maintaining our initially pre-COVID planned Palynziq new patient start rates. And in observing that drove a strategy to identify alternative prescriber bases and mechanisms for new Palynziq patient starts. Different these PKU clinics. PKU clinics are often small practices within larger genetic centers. So when we observed that some of these clinics were slow to open, it was really due to some of the physicians being available in-person, perhaps just a couple of days a month. As you can imagine, they might be seeing their highest priority, most severe patients and Palynziq new patient starts weren't at the top of the list. So that -- and observing that is what drove the strategy to pursue some of these alternative prescribers. We think that endocrinologists could be the good strategy and we've had some success there. It's just going to take some time in the U.S. because that's a pretty wide prescriber base, but these are folks that already prescribed pharmaceutical therapies. Another note is that Palynziq has a REMS, which means that the first dose needs to be overseen by a medical professional. So we also started a treatment, started home program where a nurse can visit the home to help through that REMS process. So these strategies because it's a pivot in sort of the cover point and the new patient start process takes some time, but we are seeing some progress there, and that's why we're still positive about the large opportunity.

Got it. I know you sort of will update the Street during the earnings call on Roctavian launch. So maybe we can just touch base a little bit on the PDUFA extension. And from a labeling perspective, do you expect the FDA to include the 3-year data or the 5- or 6-year data that you currently have from a durability perspective?

Yes. Good question. With the major amendment, not surprisingly being triggered by that large volume of Phase III data that we released in January. I have to reserve commenting on the specifics because we're in review and that would qualify as sort of labeling review discussion, and we are in a quiet period at the moment. Suffice it to say that should the PDUFA date may have -- would have stayed in March and if there wasn't an in-depth review then it probably would have been unlikely that the 3-year data would have been on the label, but we'll ask you to stay tuned on that one, but it's a great question. And it was great to see that the 3-year data stayed consistent with the 2-year data in terms of safety and efficacy.

Got it. There was something of expectation that you could potentially launch or expand beyond Germany in the first quarter. Is that still on the cards or that sort of move because Germany is sort of delayed.

We've started -- yes, thanks. Good question. We've started the pricing and reimbursement process for other countries in Europe. And again, big picture, where we're in a bit of a quiet period here being outside of the first quarter and having our earnings call in just a few weeks here at the end of the month. So asked you to stay tuned for a more robust update. But I can't say we didn't necessarily expect the actual formal pricing and reimbursement processes outside of Germany that complete and launch in Q1. These processes typically take a year or more. I'm talking about France and Italy, Spain here. So our expectation from the beginning was that some of those additional European countries wouldn't come online until later in the year.

Got it. And the final price in Germany as and when it does get cemented. Do you think it's going to be in line with where we -- where you originally thought it was going to be?

That has been our goal in these complicated negotiations with the German health care authorities over the last few months. The primary complicated topic has been the design and negotiation and execution of these robust outcomes-based agreements where we can offer protection of the value of Roctavian's durability and freedom from current standard of care prophylaxis over time. But because the -- because we wanted to -- because our goal is to have this comprehensive both price and value proposition tied to the OBA. Our goal over time has been to negotiate a price that -- during this free pricing period, which ended March 15th, that would approximate the final German price. That's not always the issue with launches in Germany. Many companies have utilized their free pricing period to set these ascertain price that is expected to be lowered, in some cases, significantly in the final negotiation so we would expect that the price of Roctavian during the free pricing period would be similar to the final price.

And do you have 2 hold-ups on the private side in Germany have those resolved -- or it's still ongoing at this point?

Yes. Thanks. I know that's a topic on everyone's mind. It's a great question, but I'll have to ask you again just to wait for the earnings call in a few weeks since we're kind of in a quiet period on all those material items.

Okay. If you did not choose to go with the outcomes-based agreement, do you think the whole process would have been easier?

Well, you can look at Voxzogo in terms of -- in our prior launch history in Germany, which is typically the first European market to pursue because of this free pricing period and early access. But the key difference with Roctavian as you know, is the outcomes-based agreement. And we believe that's important mechanically we would have been able to price and sell Roctavian. But we think it's very important that payers fully have that protection of the long-term value of Roctavian. So for us -- and it's important to the payers to having the outcomes-based agreements in place commensurate with commercial patient dosing was always a priority.

And given the European experience to date, would you pursue an OBA in the United States or given that it's a fragmented sort of a reimbursement set up, it doesn't help much with the payers.

Yes, thanks. Great question. So it's -- we believe it's still important because if Roctavian pricing is intended to capture the value of the product, which is again, freedom from the current costly standard of care of prophylaxis over a several year period. It's still important even in the U.S. that we offer that same value protection to payers. However, we're not anticipating the same level of complexity in the U.S. as we've experienced in Germany. And the reason is we plan to offer the similar terms and protection of that outcomes-based agreement in the U.S. but in the form of a warranty that will come with the product and commercial dosing. So you can envision standard terms and conditions that describe how bleeds are defined and what -- returning to standard of care looks like what's the proportional financial reimbursement or liability from BioMarin maybe depending on -- when and if the point in time where the patient resumes their standard of care, you could picture that framework, which fundamentally is similar to what we're planning around the rest of the world, but issued in the form of standard terms and conditions with Roctavian so that we don't have to negotiate each of those on an individual payer basis. We've used the analogy of the expected Roctavian warranty to a car warranty. So if you purchase your car, it comes with this warranty. It's very valuable to you because it protects the value of those most expensive complicated parts of the car, the engine, et cetera. But when you go to buy your car, you don't negotiate the warranty. It's standard issues. So for that reason -- and we'll still have other critical elements of the U.S. launch to work through getting on formulary until we have a J code, we'll be going through the medical exception process, et cetera. So there still will be key elements to navigate during the launch. But we're not expecting that individual warranty or outcomes-based agreement negotiations will be one of them.

Got it. And unfortunately, I want to ask this, but you probably wouldn't answer. Have you seen any material uptick in terms of interest that you had sort of -- in the U.S. about 300 inquiries on Roctavian as of the end of -- during our full year call in roughly 10 patients going through an antibody screening in Germany. Has there been sort of an uptick on that -- those numbers?

I can share, again, as you noted, we'll make sure to give a robust update here in just a few weeks on the Q1 call. But I can share that over this period of time where Roctavian has been approved in Europe that we are continuing to see interest from both patients and physicians. We're seeing an increase in the number of patients undergoing the diagnostic testing, for example, testing negative to antibodies to the AAV5 vector, healthy liver screening, pre dosing genetic gene therapy counseling with the patient. So we're seeing interest and we're seeing an increase in that activity.

Got it. Then moving on to -- letting Roctavian this side, the sort of corporate goal of $4 billion to $5 billion by mid-decade, right? Now you talked about $2.5 billion -- $2.5 billion on the ERT franchise, clear line of sight to $1 billion plus on Voxzogo. But I think the Street is having a hard time making that leap of faith from $3.5 billion to $4 billion, $4.5 billion, right? And that's where the Roctavian piece comes in. Is there anything that you can add to or help us cross that hurdle?

Yes. Well, importantly, as you know, when you did some of the math there, when we talk about that $4 billion to $5 billion by the middle of the decade, that does assume both the launch in Europe for Roctavian which is underway as well as approval and launch in the U.S. So that is a key element to the growth. We do think we'll be able to get to a substantial level, perhaps not in that range. But as you noted with that mid- to high single-digit growth in the base business and continued solid ramp of Voxzogo, we could get closed. But by all means, we would expect their need to have contributions -- material contributions from Roctavian to get to that mid-decade goal for certain.

And so if you get to like $3.5 billion, what would your margins look like versus if you get to $4.5 billion?

Yes, great question. What we do have the ability to do, and we've shown this over the last couple of years as we set our goal and achieved it of reaching GAAP profitability and increasing operating income or non-GAAP income. Over the last couple of years. We've shown that we do have the ability to control our investment both in the future of the business, current business through both capital and operating expense governance. So recognizing that we're profitable, expecting to be profitable today. That's a key element of how we are managing operating expenses today, commensurate with the expected top line growth. So what I'd say is if that growth were slower, then we reinvest more slowly in the business and throttle back. With that being said, if the P&L ramps and we get to that $4 billion to $5 billion revenue by the middle of the decade. Fair to say that, that's more revenue that we would continue to drop most of to the bottom line so we would see higher operating margins in that scenario. But we're committed to GAAP profitability. And even if the ramp is slower than that $4 billion to $5 billion mid-decade trajectory, I would expect that we would be able to modulate our investment in the business, operating expenses and still grow margins and profitability over that time period just at a slower rate.

Got it. Voxzogo is obviously off to a great start, very early, right? I mean it's 1,000-odd patients on treatment as of the end of last year. How high do you think the adoption could get?

Yes. Well, again, with Voxzogo being the only approved therapy for the underlying cause of achondroplasia and for BioMarin, this being a much larger market opportunity than we've had with our other products which has also launched successfully. And where we built markets, that's the other unique thing to Voxzogo, where we're getting a lot of leverage from our historical commercial experience, where many of our other products have been the first approved therapy for the indication. So what that means is when you go out to market, we literally need to build that market, and we're doing the same for Voxzogo and achondroplasia. So that will take time. We're very pleased with the first full year of launch success and the expected growth in 2023. But with a market of 18,000 patients in the current label, it's a little less than that because of the under 5 in the U.S. and the under 2 in Europe that I mentioned, but total market opportunity should we be able to expand the label is 18,000 patients, so what that means with that roughly 1,200 patients that we shared at the end of February that were on commercial Voxzogo, we're less than 10% penetrated today. So just as an example, if that 18,000 patients represents a potential global market of about $3 billion penetrating just 1/3 of that, call it, 30% gets you to that $1 billion revenue. And so when we talk about the opportunity for Voxzogo to be $1 billion plus, that's just a question of how much further we get beyond that 1/3 penetration. But we're already seeing rapid uptake, interest from families and physicians. So we have reason to believe that we can continue the momentum.

And given that it's sort of a weight-based dosing, has the initial population being on the older side and as you expand the label 0 to 5 or 0 to 2 in the U.S. and 0 to 2 in Europe. You think the -- how should we sort of model the impact on price?

Yes. Great question. Thanks. So first of all, we have seen uptake from Voxzogo in all ages within the label. I remember our commercial team sharing that some physicians in the early days of the launch actually prioritized some of their older patients, some of these teenagers because they recognize that the window for treatment for those patients was shorter, because they were nearing the end of their growth plate closure. So we have seen uptake across the board. As I mentioned, we believe that treating early can lead to the best outcomes over time. But we've seen uptake across all age groups. Interestingly, while dosing for Voxzogo is weight band dosing, that increases with different bands of body weight. The pricing is less variable than with other products, including some of the enzyme replacement therapies and similar biologics on the market that would be sold at a simple per vial basis. And I'm talking about other products here. So a patient double the size might consume milligram per kilogram dosing at double the volume and therefore, double the price of revenue. While Voxzogo does have weight band dosing, most of the pricing is actually fixed. So what that might mean is some different gross margins for BioMarin, but from a pricing standpoint, you're not going to see this high variability of patients that are heavier being high in the range of a per patient revenue. And obviously, with infants, they're lighter or young children, so they'd be consuming less. So this relatively not fixed, but stable pricing across these weight band doses was the strategy for Voxzogo. It's similar for Palynziq, by the way.

Got it. And given that this is -- Voxzogo is the current crown jewel of the portfolio, how are you thinking about life cycle management and keeping competition update.

Well, competition, we know that was a hot topic in the news over the last few weeks. We look forward to seeing Phase III data from any competitors in understanding regulator thoughts on competitor programs. As we've noted, it's difficult to do comparisons of products that -- in young children where inherently growth rates are highly variable in young children, placebo comparators are important larger end and patient numbers are important. So we'll watch competition, of course, but it feels early days for any of the competitors. Life cycle management, though, we're very excited about last year, Dr. Dauber and Children's National Hospital in Washington, D.C. He's running an investigator-sponsored study of Voxzogo in other genetic short statural indications. And last summer, he released an interim cut of his data, which was a subset of his patients at 6 months, showing the annualized growth rates. And it looks like there is a positive impact of Voxzogo in those indications. So we immediately began to develop a strategy and development plan around indication expansion for Voxzogo. We're looking forward to Dr. Dauber's more robust set of update, more patients and additional duration here sometime this year. But in the meantime, again, we've developed -- we've got a development plan that we'll be discussing with regulators here in the near future. So I'd ask you to stay tuned for more details from us later in the year, including our R&D Day planned in September but these are much larger -- as much as I talked about, achondroplasia being a much larger indication for BioMarin. Some of these genetic short statural indications in total are significantly larger than achondroplasia. So potential long-term market growth opportunity beyond just this large indication in achondroplasia itself.

And after this call, we are hosting 2 physicians talking about achondroplasia and everything beyond. So there will be a perfect segue to that. So one last question then of the pipeline assets currently, what would management point to as the next big drug from BioMarin outside of the approved or soon to be approved indication.

Yes. We're pleased to have more assets in early development than any other time in the company's future. I shared in my opening remarks how these therapies like Voxzogo and Roctavian represent the pivot of our strategy to larger indications where we can still adhere to our core research principles of being highly transformative in severe disorders, but in larger patient populations around the same time as when we rebooted our early-stage research engine, both internally and in licensing a number of early-stage assets. So we're looking forward to filling out that early-stage clinical pipeline over the next couple of years. The 2 products that are currently in the clinic, BMN 331 for HAE and 255 for chronic kidney disorder. Those are 2 where we're interested in sharing more details at the R&D Day. I think if I had to say what has a large opportunity, BMN 255 has the opportunity to treat what is potentially a larger patient population in that chronic kidney disorder or liver disease. There's another product on the market today that utilizes a similar pathway. We shared some data where we've seen some early signals that our compound could actually be more potent and in the same pathway, but slightly more potent and a potentially larger indication opportunity. So that's one where it could be, again, in this pivoted strategy of larger disorders. This could fit those criteria pretty well.

Awesome Brian, I think unfortunately we have run out of time, actually run out of time a while ago, but we can talk to BioMarin for learning on time here. I appreciate your time, Brian. Thank you so much, and have a wonderful day.

Take care. Thanks for having us.