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

Good afternoon. I'm Salveen Richter, biotechnology analyst at Goldman Sachs. I'm pleased to have BioMarin here with us this afternoon. And with us, we have J.J. Bienaimé, Chairman and CEO; Hank Fuchs, President, Worldwide Research & Development; and Brian Mueller, Acting CFO. And with that, I'll turn it over to you, J.J., in the case that you want to make some opening remarks here.

So thank you, Salveen. We are very happy to participate in your June conference for the seventh year in a row, even if we cannot talk in person. So it is no coincidence that BioMarin is at a significant inflection point in 2020. We have been growing the company for many years, honing our R&D platform, advancing our manufacturing capabilities, expanding our global commercial footprint and working with the health authorities around the world to develop essential medicine for people with rare diseases. The results is that we are turning profitable this year on a GAAP basis for the first time in the company's history, and we are anticipating approval of ROCTAVIAN, the first-ever gene therapy treatment for any hemophilia indication. We are planning to submit the marketing applications for vosoritide, the first potential approved therapy to treat achondroplasia, which is the most common form of human dwarfism, in addition to numerous other pipelines initiatives that are expected to drive significant profitability and value creation in both the near term and the long term. So as you know, about a week ago or so, we shared the highlights from the 4 years of clinical data with ROCTAVIAN for the treatment of severe hemophilia A. We are so pleased that 4 years after a onetime infusion of ROCTAVIAN, patients in our study remain off prophylactic therapy and have experienced less than 1 bleed on a cumulative basis. Four years of bleed control and counting is a tremendous win for those patients, and we look forward to Dr. John Pasi presenting the full 4-year dataset on June 17 at 9:00 a.m. Eastern Time at the Virtual World Federation of Hemophilia Congress. We continue to anticipate the U.S. approval of ROCTAVIAN in the second half of the year based on our August 21 PDUFA action date. So despite the immediate challenges of the COVID-19 pandemic, BioMarin has never been in a stronger position to execute our growth strategy for a number of reasons, including high barriers to entry for our existing business, our diversified pipeline and the high likelihood of approvals for both ROCTAVIAN and vosoritide. So we announced 25% top line growth on our first quarter results call, demonstrating strong demand for our existing commercial products. We maintain our full year GAAP net income and non-GAAP income guidance, even though we slightly lowered our top line guidance. These are some of the highlights for the first half of the year. So we look forward to the remainder of 2020. We look forward to it with enthusiasm as our next 2 potential blockbusters advance over the coming months. So we'll pause there and hand it back to you, Salveen, for questions.

Great. So maybe to start here. I mean we've seen a pretty nice evolution of the company over the last 5 years. How do you see the next 5 years playing out for BioMarin?

I mean the next 5 years, I mean, we have communicated that our target goal is to reach around $5 billion of revenues by 2025, assuming that ROCTAVIAN and vosoritide are approved. We believe it's achievable on top of our existing commercial business. We believe that we'll have multiple products in the clinic. We are gearing up our R&D pipeline of late-stage preclinical opportunities to make that happen. We probably will be more active -- a little bit more active in business development, continuing to do early-stage deals, but maybe larger sales, as you know, ROCTAVIAN and vosoritide generates some significant cash flow. And we anticipate that we need the company to be significantly profitable on a GAAP basis.

And just speaking about the BD strategy here, can you speak to the decision to enter into the recent DiNAQOR collaboration? And do you see this as representative of what you might like to do in the future? And are there any other technologies or therapeutic modalities of disease areas that you're looking to enter into?

So I'll start, and then Hank can continue. I mean, the DiNAQOR transaction is great for us because it leverages our know-how of gene therapy in general, in vectors -- vector biology, major manufacturing experience and know-how. It allows us to enter into a less busy and crowded, I would say, gene therapy field or emerging cardiac gene therapies market, which is a very significant market potentially, actually larger than DMD, for instance. So -- and we believe that we partnered here with one of the leaders in the field of cardiac gene therapy. So that did make a lot of sense for us. And so we have other ideas, although we don't want to disclose everything for competitive reasons, but maybe Hank can give his perspective here on this.

Well, I think in addition, what DiNAQOR saw in us is a company who is able to produce oversized transgenes in abundance, target beyond the liver and achieve high levels of expression. All things in the last year we've made public with you. But for cardiac gene therapy, considerations of biodistribution and protein expression are going to become really important. And in fact, I think as you get outside of liver expression of proteins, the ability to target tissues, control gene expression and get adequate expression of the protein of interest is going to be the coin of the realm. So I think they saw in BioMarin, the first company who was really poised to break out of just their specific gene therapy because of these additional technological attributes.

And maybe we can just expand on that and talk about what's coming from the next-generation outlook when we look at capsids and then how you seek to kind of evolve with regard to the manufacturing capabilities that you have as well.

Well, we have had quite a bit of active research in the area of capsids, and our primary focus has been to be -- is to come up with -- there've been really 2 co-primary focuses. One is to come up with non-seroprevalent capsids that could be used for, for example, redosing or for patients who are pre-immune, if we can't solve the problem any other way. And a second fundamental approach that we've been undertaking is to look at novel capsids to improve on tissue tropisms, and I think we've made progress in both areas. And I think some of the proof points are, for example, we've -- PAH gene for PKU is ready to go into the clinic. HAE gene expression in vivo has been -- in preclinical studies has been optimized and to remind people we've gotten several orders of magnitude, extra protein expression out of our HAE system compared to anything that's ever been published or presented. So real powerful proof point on how much gene expression we can achieve. And then in terms of novel distribution of capsids, we showed at our R&D Day some novel capsids that had different tissue tropisms, better muscle delivery. And so I think that we're laying the tracks of the proof points to show that our technology is muscular in regard to going into other tissues and achieving higher levels of expression.

Got it. And J.J., at our CEO Conference in January, I think you mentioned that BioMarin was undervalued in your view, just given the base business, the upcoming launches, the pipeline and the manufacturing facilities. Do you still feel similarly as you look to the story in the context of what's [indiscernible]

So I guess, based on the increase in the stock price since the conference, I guess, it was right. So we -- it all depends on the probability you ascribe to valrox -- I mean, ROCTAVIAN's approval and vosoritide approval and then commercial success. So on a -- on the last 12 months trading revenues or multiple of revenues for any biotech companies above $1 billion is only second to Vertex. So then we could say, well, a nice perspective. So we are not undervalued. But then it all depends on how successful we are with the launch of ROCTAVIAN and vosoritide. And then, in this respect, we could be undervalued. So we know we are managing the company for the long term. We have growing revenues, significant cash position, a growing pipeline. The company is not for sale. And I think if someone wants to buy some top line [indiscernible], there are other companies where -- that are trading at a lower multiple of sales that could generate some top line revenues for an acquirer at a lower price than BioMarin.

And so with regard to achieving GAAP profitability in 2020, how should we think about the margins here in both the short and long term? And how is that impacted by the 2 upcoming launches?

Brian, do you want to take that one?

Sure. Yes, thanks. Salveen, great question. And as J.J. mentioned, this is the first year of expected GAAP profitability for the company, for the first time in our history is part of the transformation. And by the way, we reached non-GAAP profitability a couple of years ago, and that's expected to grow significantly this year. But we definitely expect that ROCTAVIAN and vosoritide, if they are approved and we launch, will drive significant margin improvement over the years. We've already been on a steady margin improvement journey, which is part of what drives our GAAP profitability this year. And might be helpful, I'll take you through a couple of the line items, just to put it in perspective. So first of all, cost of goods sold. Our gross margins have been around 20% or 80% -- about 20% cost of goods sold. And that's driven by our legacy base business of the enzyme replacement therapies. These are recombinant human enzymes made in a mammalian cell culture process, very complex manufacturing and, therefore, higher cost than perhaps some of our larger peers. But we are expecting that ROCTAVIAN and vosoritide will have lower cost of goods sold. And so as those products ramp in revenue, we'll start to see overall margin improvement with cost of goods sold perhaps settling in the mid-teens area. I should also note, we've got a number of internal cost of goods sold reduction initiatives even on the base business. R&D., we want innovation to continue to fuel the future of the company. So we will increase R&D spending on an absolute dollar basis. But again, as revenue starts to ramp up, we'll see that come down as a percentage of revenues. We think that even if R&D settles in at, say, 25% of revenues, that's enough to fund a robust pipeline, but also drop more profits to the bottom line. Sales and marketing, I think that's a leverage story. We built a global sales and marketing organization that supports our originally planned $2 billion of revenue today. And it's that same commercial infrastructure that we will use to launch ROCTAVIAN and vosoritide. Now granted, those are unique disease spaces. So some initial incremental investment will be required for those launches. So we may not see the margin improvement initially as we're launching those products because you have to spend the sales and marketing dollars before you get the meaningful revenues. But over time, by all means, we'll see margin improvement there. We think our target for sales and marketing is dropping into the teens as well over time. And then G&A is another leverage story. We've invested a lot in G&A to support the infrastructure company we have today, and that's what we'll leverage for the future. And we'll see G&A come down at the percentage of sales as well. So across the board, we're going to see margin improvement with ROCTAVIAN and vosoritide, and it all drops to the bottom line.

Thanks, Brian. So moving to the commercial products here, with the COVID-19 pandemic still ongoing, could you just talk about the disruption or impact here to your base business? And just your comfort with the guidance that you've provided in the context of this?

I could do it, but Brian, you're on a roll. So why don't you take that one on too?

Okay. Sure. Yes, I'll start. So when we updated our guidance in April, we adjusted the top line by about 5%, $100 million, and the larger portions of that were Vimizim and Naglazyme, where those are, again, weekly, typically in-clinic infusions. And as we -- as COVID interruption impacted the health care system, whether it be the reprioritization of health care resources, folks just staying at home or other circumstances, we did see some interruption in patient infusions. Many parts of the world, home infusions were already in place. So we saw minimal to no disruption there. And so we did expect that the hardest hit quarter would be Q2, and we are experiencing that. But importantly, the assumptions that we made when we updated guidance are holding up. As you can imagine, the serious nature of the diseases that our therapies treat and the criticality of our therapies, the folks involved, the stakeholders are working hard to maintain continuity of supply whether that be the physician, the patient families or the patient support network. So where parts of the world were not equipped already for home infusion, they've been working hard to enable home infusions. And we've seen some traction there in increasing the level of home infusions. There's also been a bit of delay in new patient starts, especially those that happened in the clinic. That's where you saw a reduction in our Palynziq guidance because we are still launching that product. But again, as we see things opening up and clinics opening up, we'll hopefully get back on track. But importantly, the assumptions that we made are holding out.

And this -- with regard to Palynziq and, I guess, and Kuvan in the PKU gene therapy that's entering the clinic here, how do you think about the dynamics that's going to play out in the PKU space given a generic coming to market potentially later this year?

So the generic is only for the U.S. market, which is our largest market for Kuvan. So we -- if you look at our PKU franchise since we launched Palynziq, despite the cannibalization in part of Kuvan with Palynziq, our PKU franchise has actually been going up. So obviously, in the U.S., starting in Q4, there should be erosion of our Kuvan business. But -- and there could be -- our projections are like -- so in 2021, there's probably going to be a minor reduction of our PKU franchise in terms of worldwide revenues, but we believe that, thanks to the Palynziq growth, that would be more than compensating the loss of the Kuvan business. We will actually see a growth of our PKU franchise starting in 2022. Now regarding the gene therapy, if you look at the state of the market in terms of the penetration of the patients, the adult PKU patients, especially, that's what we're talking about for Palynziq and for gene therapy at least initially, most of the adult PKU patients are not treated today. So there is still a significant room for growth. Some of it will be captured by Palynziq, some of it because it's much easier to use will be captured by gene therapy. So we believe that gene therapy will actually expand the market just like Palynziq has been expanding the market over time.

And as you think about the 2 upcoming launches with ROCTAVIAN and vosoritide, at a high level, can you just lay out your expectations here? And how much of a contributor are they to your previously stated goal of $5 billion in net revenue in 2025?

Probably slightly more than half based on the current business. And again, there's the Kuvan erosion, there's going to be growth of Palynziq. So about -- it's about 50-50, roughly. So a pretty significant contribution.

So moving to ROCTAVIAN here. So you just -- you commented on the 4-year durability. We know that will be presented at WFH, but you provided some highlights in a press release. Could you just recap the 3-year data that was demonstrated in the 4-year highlights and how you find this to be clinically and commercially meaningful in terms of the expression levels and to ABR?

I might have missed something in your question, Salveen, so come back to me if I didn't get it quite right. But what we showed is 4 years after the 6e dose and 3 years after the 4e dose, that all patients remain off of prophylaxis. 6 of the 7 high-dose patients and 5 of the 6 low-dose patients remain free of joint bleeds -- treated joint bleeds -- treated bleeds. And importantly, the only 2 people that experienced spontaneous bleeds experienced them in target joints, not in nontarget joints. And at a frequency that was lower than their pretreatment baseline, at a frequency that would be considered a good prophylactic Factor VIII level outcome. And in their case, without the benefit of prophylactic therapy. So even our lowest responders, 3 and 4 years later, have had a fantastic therapeutic outcome. And I think the importance of the 3-year mark for the lower dose group is going back to the 201/301 data where some have expressed a concern that maybe the 301 product launch is a little bit lower. Even if the launch is lower, durability can be expected to be longer than 3 years. And at this point, actually, given where the way the curve looks like and given the fact that we have hemostatic efficacy at fairly low levels of factor, it could be a long time before patients need to resume factor prophylaxis. So we're very pleased, and so is the community.

And again, there will be more details, including Factor VIII levels, presented a week and a day from now at WFH. It's not that far away.

And then as you near this August 21 PDUFA date, could you just comment on what gating factors are -- still are there ahead of the PDUFA and commercialization? With regard to the U.S. facility, we know it's pending FDA inspection. So are there any alternative ways, I guess, for the FDA to certify that plant via virtual means?

Well, we're in the middle of a mesh with the FDA. Any given day, it's like back and forth. But what I would say is that in regards to major metrics, the agency is actually ahead of their PDUFA mandated regulatory metrics in terms of time line. So really, what remains are inspections and completion of reviews. They have all the information on file that they need to complete their reviews. And so we're working very closely with them to keep things on track. Obviously, they're very busy on their end. It's not like they didn't have anything to do before all this happened. So we continue the collaboration.

And then can you just talk about disease education and the targeting of the 150 hemophilia transplant centers in the U.S. ahead of the launch? What are the most important steps that need to be done here?

I mean it's -- I mean, most important step is to educate actually the clinicians -- I mean, the patients and the clinicians on gene therapy because even hematologists, obviously, they know they're experts in hematology, but some of them don't know that much about gene therapy and vectors and AAV and preexisting antibodies and that kind of stuff. So there's a lot of education we're doing, and then trying to prepare them for how to administer ROCTAVIAN, which is not going to be very complex, by the way. It's going to be like a 3-hour or so infusion. There's no need for a preconditioning regimen or that kind of stuff. This is way, way simpler than ex vivo gene therapy and not that different from what they're doing today by infusing replacement Factor VIII. So we are already interacting. Our sales force is interacting with those hemophilia centers. Actually, all our sales reps who are -- the vast majority of them have significant experience in hemophilia and have established relationship with hemophilia centers. And of course, we also have been interacting with the payers in the past few months to also educate them and prepare them for the launch, and the interaction so far has been very positive. I mean, the main thing that has changed with the shelter-in-place is that a lot of these interactions that were supposed to take place face-to-face are now taking place with Zoom or video conferences, which is a little different. They're not sometimes not as good, but we're doing the best we can and, again, preparing the centers for the ROCTAVIAN launch.

And when you communicate with hematologists, what is the kind of the key feedback that you get with regard to what they might want to see to put a patient on drug? Was it X levels above or bleeding rates above a certain level or at a certain threshold as you get out, just certain time point, like what is it that would be gating factors that they'd want to see?

So I'd take that one, Hank. There is a lot of marketing research on this, but...

Yes, well, that -- so that -- J.J. has made an important point. What I was going to say is, this year for me, it feels a little bit differently as we've engaged our -- the investigator community. We'll see what happens at the launch in WFH with the data because I think what I'm experiencing is, people are like, "4 years of no prophylaxis therapy, and the recent year is as good as it is?" And it look like the levels were kind of -- I mean people's attention is now way off of the factor level at this point. And it's like this conversation is now the new standard of care is going to be -- you have to be off of prophylaxis for 4 years. If you're not off of prophylaxis for 4 years, you're a set -- the bar is really high is my sense is where people are reacting to the data. But J.J., you started to mention market research.

I mean there's some marketing research we've done ourselves. Some marketing research has been done by some of your colleagues, the banks, which, in the past few weeks and months, have been very positive and going in the right direction. So even our own market research, doctors, clinicians treating hemophilia patients, U.S. and Europe, they say that they anticipate about 1/3 of their patients are severe hemophilia patients to be treated with ROCTAVIAN by the end of -- within a year or so, which is pretty positive. So -- and they're all very impressed in the recent calls, qualitatively, quantitatively, very impressed with the bleeding controls. I mean, the Factor VIII levels doesn't seem to be a major worry of them at this time, same with the payers. And I think as every year goes by, the focus on the clinical endpoint, which is BD control, is going to be taking prevalence over Factor VIII levels. And on top of it, I mean, I just read an interview by Dr. Pasi, who is our principal investigator and our key opinion leader in the field, and he himself, he say he doesn't really know the correlation between Factor VIII levels and BD control. He doesn't know also whether clinically, 1 stage or chromogenic is a better assay. So there are all those issues. But at the end of the day, the patients and the docs and the payers are going to look at bleeding episodes and Factor VIII consumptions and whether ROCTAVIAN reduces that, and we have very significant evidence that it does over at least 4 years now.

And then the WFH and ISTH and other organizations are collaborating on a hemophilia gene therapy registry. How do you think this would be utilized? And are other ways you can capitalize and track patient experiences over time and use this as a marketing tool?

Number one on that list is, I think they're very concerned to measure joint complications. I mean, I think that the initial bet of hemophilia gene therapy is that because we won't be relying on peaks and trough managements and because we'll be using physiologic correction that if you can take a child who is well prophylaxed through childhood and has very good preserved joint function that if you switch them over to gene therapy and away from prophylactic therapy, they won't develop any joint complications. So I think the first milestone that I hear people talking about is kind of 5 years from now, what's your overall joint health in the population for people who took gene therapy and didn't take gene therapy. But there are a myriad other questions that people will be interested in as well.

And then finally, with the companion diagnostics tests, how should we expect this being used initially when you launch the drug?

It's going to be fairly straightforward like any other companion diagnostic, that is, you have to get a test to verify that your -- things play out the way we think will be played out. Meaning specifically that the companion diagnostic would be approved before the -- on the PDUFA date and that the PI would reference a requirement for companion diagnostic. So you'll have to have the companion diagnostic test done. And then it may even need to be repeated depending on the time interval between you had your first companion diagnostic test and when you're in go mode. I think Jeff's talked about some of the processes that have to happen from identification of the patient to the payers' readiness level, and that can take some time. But we'll be prepared to enable patients to receive the companion diagnostic and as effortless way as possible. We have a study that we're launching a global seroprevalence study to continue to advance the science on what's known about seroprevalence. And so we'd like to enroll another 1,000 patients in hemophilia A patients and characterize their seroprevalence and they're changing their seroprevalence over time or as a function of different risk factors. And so we'll be learning a lot more about seroprevalence. And I think that will also synergize with the companion diagnostic.

And also an important point is also that we selected the vectors, AAV 5, because we knew that in terms of preexisting antibodies, it would be probably the lowest one of the ones that were available. And actually in the U.S., only 20% to 25% maximum of the patients who have preexisting AAV 5 antibodies. So of like -- actually, when we're going to test about a 1,000 patients, 750 of them should actually be -- at least 750 of them should be free of AAV 5 antibodies and eligible for ROCTAVIAN treatment.

And J.J., any updates here on the payer front with regard to pricing in the U.S. and/or Europe? And any further understanding on what the models might be like? You've talked about the U.S. really looking more at onetime payments, but is Europe going to kind of pursuit to other models here.

Certainly, we've had multiple interactions with payers over the past year or so, U.S., Europe. And I would say they have been very positive interactions. We -- and obviously, they are really -- a lot of them are focused on cost offset, looking at how much these patients are costing them today, whether it's intravenous infusions of replacement factors, the cost of treating bleeding episodes, which in the U.S. is about $50,000 per bleeding episode. And so consequently, the 4-year update is very helpful here to solidify our potential pricing and pricing negotiations with payers. Actually, in the U.S., again, we will be offering from pay overtime, pay for performance. Actually, we've been working very diligently to figure out a way to come up with a real pay for performance program. And so when we launch the product, you're going to hear more about it. But we are very excited about it and we believe that can make a very big difference positively, actually, in the adoption of ROCTAVIAN, not only in Europe but also in U.S. We believe we can actually implement this in the U.S. So stay tuned on that.

And then moving to vosoritide. So we saw the top line pivotal data last year. Could you help remind us here as to the filing strategy and what needs to be done to complete the submission package?

Yes. As far as what needs to be done at this point, we've had our presubmission meetings and no additional clinical trials are required or -- and so really in report writing phase at this point. And we have targeted back half of the year for the -- Q3 for the submission. And we're feeling pretty good about the timeliness of that. Now obviously, our regulatory group is working a little extra hard because we have live submissions and review in U.S. and Europe on ROCTAVIAN. And so it will be cool. We'll be in review conceivably in both regions with 2 different assets at the same time.

And then you have your 5-year long-term data from the open-label portion of the Phase II as well as infant study data that's coming, so will you be submitting any of this? And how should we think about this data and what's clinically meaningful on this front here for both of them?

Yes, so the -- so there's a study going on. We've completed a pivotal trial on patients who are older than 5. We have initiated a study in patients who are -- and children who have achondroplasia who are under 5 years of age. And the study was designed in 3 different cohorts according to the age of the patient because it turns out that the younger you are, the higher dose of vosoritide you need. So we've completed the 2- to 5-year-old enrollment. We've completed the 6-month to 2-year-old enrollment, and we were enrolling the 0 to 6-month-old cohort. And the original plan prior to COVID was we were going to unblind that study when all 3 cohorts were fully enrolled. And so what we communicated to the agencies is that at the time of submission, you would have the safety data from cohorts 1, 2 and 3, with obviously a heavier emphasis on the amount of data in the first cohort, the 2- to 5-year-olds just because they've been on it for longer. And that the efficacy data would be submitted in a post-approval supplement. And so that's the agreements that we've reached with agencies worldwide. And it was a good thing because of COVID, it's slowed down the enrollment of that final cohort. So there won't be any efficacy data, at least since we sit here today, the plan is no efficacy data from under 5s. But in terms of the clinical significance of all of that, the FDA convened an advisory committee a couple of years ago. And this question was discussed specifically and the conclusion from the advisers was, basically, safety data is mostly what we need. We're -- if it works in children who are older than 5, it ought to work better in children who are under 5, provided it's safe.

And then, again, you've talked about applicability of this drug in other diseases of short stature. So where do those programs stand?

Yes. So we have a very active collaboration with an academic investigator who put forward to us a program that we actually feel like he could move faster than we could move just because sometimes those local studies can do that. And he's been in discussion with the agency about the design of that protocol, and there's been some back and forth about that. And I think we're confident that we can get the study off the ground this year. And the fundamental principle derives from the genetics of stature, and that is to say, there are humans who have mutations in CNP that cause endogenous overexpression. And for example, one of them is 7-foot 4 inches tall. So against the polygenic background of what controls your stature, overexpression of a single gene can overcome all of that and make you 7-foot 4. So it stands to reason that if you have a specific genetic mutation that's causing a statural deficiency, say, achondroplasia, but there are a variety of other mutations that could be overcome. That use of vosoritide could overcome the negative signaling of growth of that mutation, and that's the concept that he's working on with the FDA. And I think that will go forward at the end of this year.

And with regard to the rest of the pipeline here, can you give us an update on the PKU gene therapy program and when you might be reinitiating that study?

Yes. I think the story on the PKU gene therapy program is that the clinical trial apparatus has kind of been ground to a halt due to COVID. And I think for therapies that don't have any alternative forms of care, for breakthrough therapies, for therapies that are imminently going to be licensed, they're in a different category than first-in-human studies. And so we -- the study is basically not enrolling in the moment, and we're in active discussions with our investigators. Now the thaw is starting to happen. And I do think people want to make sure that there isn't such a thing as a second wave. And as soon as institutions are ready to begin opening up enrollment, and that's going to be a very local kind of a decision, too, right? It's going to depend on where the research center is, what the hospital's capacity is, what the prevalent rate of infection is, et cetera, et cetera. So sometime, I think in the second half of the year, we're going to see some movement in the trials. And then I don't think it's different for us than any -- for our competitors, for example.

Sure. And then are you on track here with the HAE program with regard to the IND-enabling studies that you're...

Yes, yes. Yes, we're in a good position that mostly our contractors had maintained operational status. And so we've been able to progress. And we've been -- we've had access to our laboratory field so at least to finish the few experiments that have been deemed to be essential for the creation of this. And there's some facility work that had a 2-week delay that we think we can make up for in terms of production of the asset for GLP studies. So yes, on track.

So maybe one last question here. I mean, what are you most excited about in the pipeline that comes behind vosoritide and ROCTAVIAN? And is it gene therapy? Or you -- what else is coming from the non-gene therapy portfolio as well?

This is like -- even my grown children I love. And so the little babies that are in the hatch. I mean, Lon and I are just really hooked on the idea of translating genetic discoveries into transformative medicines. And so the cardiac field is right for this. I mean 500,000 hypertrophic cardiomyopathy [ subjects ], 60% of which are of genetic origin. And you go sort of organ system by organ system by organ system and you realize how many haploinsufficiency conditions there are where addition of a gene vac will attenuate disease in a considerable way. And I think, again, the story there is seroprevalence, tropisms and gene expression, and I think we've gotten muscular in all 3 areas. And so I wouldn't be surprised to see BioMarin starting to break out into other therapeutic platform areas. I wouldn't be surprised to see BioMarin develop -- branch out into other what we previously called therapeutic area spaces. When medicine was driven by organ histology and pathophysiology, therapeutic areas got to find that way. In the future, in molecular terms, it will be the molecular basis of the condition increasingly that defines the condition. And I think we're in this phase of we're trying to drive towards that. It's kind of modality independent. It's kind of therapeutic area independent. It's very much biologically driven. You can make the biggest differences in people fastest and most certainly by knowing where you are biologically. That's the story for the last 10 years of BioMarin.

Well, great. Well, thank you so much. With that, I guess, J.J. and Hank and Brian, I appreciate your time today.

Sure. Thanks, Salveen. It's always good to see you.

Thank you.

Thanks for having us. Bye.

Bye.