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

As Gena mentioned, I'll start with just a few slides to orient you with the company, and then we'll -- I'll be brief so we can get into the Q&A. Briefly, forward-looking statements. I'll be making comments on BioMarin's future business prospects. So I definitely encourage you to review all of our disclosures in our SEC filings, including risk factors in our most recently filed Form 10-K. I'll also comment on some non-GAAP financial measures. These are not meant to be reviewed in isolation or as a substitute for GAAP and are provided as supplemental information to highlight some of our operating performance. So BioMarin is a best-in-class genetic disease company at an inflection point of transformative growth. Captured here are the 3 cornerstones to our investment thesis that I'll review with you briefly. It all starts with this solid, profitable IRA insulated and growing base business of mostly enzyme-based therapies that generate nearly $2 billion of revenue as well as positive operating cash flows. Some of these products have been on the market for many years, and they are high barriers to entry, which results in lower risk competition -- lower risk competition and long-lived revenue streams. And then on top of that base business, BioMarin is going through the process of launching 2 of our largest market opportunity products in the company's history. VOXZOGO is a first treatment approved for achondroplasia and BioMarin's strongest global launch to date. Last year was the first full year of global launch, and we reported $169 million of revenue in that first full year. ROCTAVIAN is poised to revolutionize treatment for severe hemophilia A. It's the first gene therapy in that indication. It's approved in Europe and launch is underway and under review in the U.S. So with those 2 larger market opportunity products on top of that, base business, we have an opportunity to leverage our global infrastructure and all the capabilities that we've built over the years, which should result in larger profit margin expansion over time. And we're not done there. The third key aspect for the future is our best-in-class innovation capabilities. We've got an industry-leading R&D teams, 8 internally developed approved products fully integrated capabilities and 2 decades of genomic science experience during this time where the pace of genomic research and science is at an all-time high. We got this exceptional track record, too, whether it be our INDs to approval or Phase III studies to approval is industry-leading. We recently reported our fourth quarter and full year 2022 results. This included record revenues of $2.1 billion for the full year 2022. Rapid uptake of VOXZOGO for achondroplasia. I mentioned $169 million, but also noteworthy is in our guidance for 2023. At the midpoint of the range for VOXZOGO, revenues are more than doubling. European commercial launch is underway. I'm sure we'll get into that, Gena. We submitted the 3-year Phase III clinical trial results for ROCTAVIAN to the FDA, and that's under review with a PDUFA date of June 30 of this year and solid double-digit revenue growth and bottom line growth as well. This next page summarizes our 2022 actuals against our 2023 guidance. You see here this healthy growth in the base enzyme product business as well as the contributions from VOXZOGO and ROCTAVIAN. This is helping to drive margin improvement down the P&L here, where you see our GAAP net income, GAAP EPS as well as our non-GAAP income are all growing substantially in our 2023 guidance. Long-term revenues on top of this base business, we expect will be fueled by these, again, large market opportunities. We believe that each VOXZOGO and ROCTAVIAN have the potential to be billion-dollar opportunities. And with continued growth in the base business, plus ROCTAVIAN and VOXZOGO, and this would include U.S. approval of ROCTAVIAN anticipated later this year. We expect that we can grow our revenues to $4 billion to $5 billion by the middle of the decade. And this would be constituted by roughly 50% of the base business and 50% of VOXZOGO and ROCTAVIAN together. Last week, we announced that the FDA has accepted our supplemental NDA to expand the use of VOXZOGO to younger children under the age of 5. VOXZOGO is approved in the U.S. today for children greater than 5 years of age. European authorities are also reviewing our application to expand access to VOXZOGO to children under 2 years of age as the product there is approved for children over 2. With those age-label expansions, we have the opportunity to add 1,000 additional patients to what is already a large achondroplasia market. We're pleased to have over 1,000 patients in over 30 countries already using VOXZOGO. and look forward to the 2023 label expansion opportunities with a PDUFA date of October in the U.S. and later in the year, we expect to hear from the European authorities. ROCTAVIAN is also a very large market opportunity with over 13,000 patients in our global footprint. The launch is underway in Germany. We also are going through the pricing and reimbursement process in Italy and France, and we'll hope to generate revenues there by the end of the year. And I mentioned that the U.S. is under review with the June 30 PDUFA. Noteworthy on the U.S. progress is that ICER has issued their conclusion that ROCTAVIAN is valuable to the health care system at a price of $2.5 million. Our payer launch is underway and we'll be ready to launch ROCTAVIAN should it be approved this summer. The FDA has also completed their on-site manufacturing -- pre-licensure inspection of our manufacturing facility in Novato, California, that was back in December. There's some clinical site inspections ongoing this month. Perhaps just to keep moving and get to Q&A. I'll be brief here, but the same CNP pathway that's utilized by VOXZOGO for achondroplasia. We believe has the opportunity to impact other genetic short statures and with similar criteria as the achondroplasia VOXZOGO clinical program that could be significantly larger than achondroplasia, including hypochondroplasia and these other diseases noted here on the right, but it's a potential for over 600,000 additional patients. We've got the largest pipe -- early-stage pipeline in company history. I'll be brief here and just ask you to stay tuned for our R&D Day planned for September 12 in New York City for more details on our early-stage research. Again, against this track record of 5 products approved in the last 8 years. I mentioned the long-term revenue growth that should allow us to achieve substantially higher profit margins over time will aspire a large biopharma type profit margins. And that's across the P&L leverage across cost of goods sold, R&D and SG&A. So our focus is to deliver on this base indication revenue, aggressively pursue life cycle management initiative for VOXZOGO and ROCTAVIAN, accelerate our clinical research and balance our desire to grow profits with reinvesting in the business through a very sound high shareholder creating capital allocation strategy. I'll pause there. Now with Gena.

Thank you, Brian, for a comprehensive overview. So maybe I'll just start with VOXZOGO. We know that the competitor, BridgeBio reported data. I know you did comment here, the VOXZOGO could be multibillion-dollar market opportunity there. So maybe just walk us through the -- how do you see the competitive landscape? And how would you assuming it remains to be seen the clinical profile, right, the Phase III remains to be seen. But if, let's say, assuming 2027, they will be able to launch, how do you -- what would be your defensive mechanisms?

Yes, thanks, maybe I'll come back to that. Just to start with comments on developing therapies for achondroplasia. First of all, it's really early days for any potential competing product. As we have observed in our own achondroplasia programs, growth rates of children can be extremely variable. So -- and can be overestimated without an understanding of baseline growth or control. So despite a small early-stage open-label experiment in the end, what matters to patients, families and physicians is sound science and substantial evidence of safety and efficacy to determine a clinical benefit. Also key is understanding how durable an effect in intervention will have until more data accumulate. Durability is an important consideration in the conditions that so adversely affect the biology of the growth plate. And this is why the FDA was keen to see both a 2-year placebo comparison as well as confirmatory data in final adult height. We're pleased to have aligned with the FDA on a pathway to full approval with final adult height. And in the meantime, pleased to be able to be launching the product globally. I mentioned the rapid uptake, sales in over 30 markets, over 1,000 commercial patients on the drug. So -- and this gets back to your question on defensive strategy. I believe the #1 defensive strategy is just to continue to penetrate this market and get more experience with VOXZOGO in the commercial marketplace. We're continuing to generate long-term data. We released addition-- we're planning to share it at ACMG this week, additional long-term data from our Phase II and Phase III studies where we're seeing a sustained benefit. Because you got to think if you're a family and your child has been on VOXZOGO for years and you're seeing clinical benefits switching also seems like it would be difficult to do when you're getting the benefits of VOXZOGO.

Yes. I agree. But on the other hand, if the -- I totally agree this early data, we don't know. And then lots of lack of clarity on the data, patient characteristics, age. So there are many open questions. But what are you -- so what would be the defensive mechanism? What is the actually comes in, we're able to show slightly better than and [indiscernible] the feedback basically. If you take your similar clinical profile, remember this oral drug and this is subcu daily and most doctors and the patients they are willing to switch. So if that, say, worst-case scenario happened and what BioMarin would do, like do you have a second gen in development? And then what is the clinical profile you're looking for so that say, 3, 4 years down the road when the real competitor comes in, you can maintain your franchise.

Well, first of all, years away with more data to come. So that's why our #1 strategy is just to maximize the launch of VOXZOGO and continue to generate long-term data. We do have life cycle management initiatives, including a long-acting internal CNP development program. But again, I might also offer that in a large market like this, especially with the track rerecord and success that BioMarin has had for literally over 2 decades. It is understandable, first of all, that a company that we're going to start to see competition based on our success record. We believe that as a stable and trusted 25-year-old company that will just continue to support patients, physicians around the world. But these are large markets, and there could be additional players at some point. I mentioned the revenues that VOXZOGO has generated to date from just 1,000 -- this market for BioMarin is still less than 10% penetrated. And we've got a product that's generating at the midpoint of our 2023 guidance, $350 million this year. So we're focused on that.

Okay. So since you mentioned that the penetration you think currently, you penetrate 10%. What could be peak penetration you were expecting to reach before, say, like net -- decade?

Yes. We have not taken a specific position as to peak penetration, partly because with our other rare disease therapies, we've got massive market penetration. Just a reminder, this is the case for the base business, but it's also the case for achondroplasia. We established markets by offering therapies -- first-time therapies for these severe genetic conditions that have not had a pharmaceutical alternative before we've had to go out and establish that market and in cases like NAGLAZYME or VIMIZIM for MPS VI and IV, these are deeply penetrated markets. It is unique for achondroplasia because of the rapid or the clear diagnosis or good diagnosis at or close to birth that we can characterize the market so much better with these 18,000 patients. But we talked -- I mentioned how VOXZOGO has potential to be a $1 billion-plus product. How big is the plus, I think is exactly that question. How deep will we be able to penetrate that market. But if you do the math, it's $3 billion...

10% is $300 million and $1 billion would be like maybe [ 90%. ]

Yes...

Okay. Okay. Good. And then I did saw that -- you said PDUFA could be October for -- so that means a standard review?

Yeah. It is a standard review, yes.

Okay. And do you expect AdCom?

We've had no discussions on AdCom. I wouldn't expect one. The FDA did hold an advisory committee back in 2018, not specific to VOXZOGO but around development programs in treating achondroplasia generally. VOXZOGO itself didn't have a specific advisory committee meeting and I haven't heard that for this...

Okay. Okay. Very good. So we will switch gear to ROCTAVIAN. Maybe starting with the Europe launch, I think earnings, there are quite a few surprise and especially at the beginning of this year, the conversation with investors. So maybe walk us through how does it work starting March 15. And you are still in the discussion with federal regarding the final price. Maybe walk us...

In Germany.

Yes. Sorry, yes, that's referring to Germany. And maybe walk us through, you have one payer already signed. You have 2 payers, the deal term already finalized but haven't signed and you have maybe federal price likely end of this like 4Q. And walk us through in between, how do you expect -- and you do have a 10 patients already going through the diagnosis, right? So maybe walk us through how does each step will happen and a patient get reimbursed and be able to get treated and you will be able to book revenue.

Yes. Thanks. Great question. This is a key focus area. So big picture, ROCTAVIAN approved in Europe last year. while that European approval gives you access to these European markets, pricing and reimbursement is each at the individual country payer level layer. In Germany because of their market access policies and regulations, it's typically the first priority market has been our experience in prior launches. So that's why we're starting in Germany. And what's different about ROCTAVIAN first of all, is the -- with the promise of the product being this relief from their chronic therapy, prophylactic Factor VIII over a period of time, years, the value, therefore, of the single infusion gene therapy is front-loaded and ROCTAVIAN is expected to be a high-priced therapy. But based on, again, that promise of performance and relief from their standard over care and better bleeding control, by the way, over time. So it's important for payers if they're paying a high upfront price for a product that's expected to have durability for years thereafter. It's also important for BioMarin to protect the value of ROCTAVIAN. We're negotiating these outcomes-based agreements in Germany, whereby -- while they're very complicated agreements and the operating principles are reasonably straightforward, whereby if a patient resumes their prior standard of care, prophylactic Factor VIII, at some point during this sort of -- you can think of it as a warranty period, they would be -- they would receive a pro ratable financial reimbursement from BioMarin for that unrealized value of ROCTAVIAN. By the way, in our clinical studies, we've had very few numbers of patients resume prophylaxis. So there are some other pay-for-performance type mechanisms for other therapies where either response or effectiveness is unknown and it's a high upfront price but it's not at the same scale and same level of complexity these outcomes-based agreements. And we're also negotiating with these individual sub insurers under the single national payer Germany system. So the 3 groups that Gena referred to are the statutory health insurers in Germany. The top 3 represent about 85% of covered lives. So that's why we're prioritizing the negotiations there. And frankly, it just took longer. We were in good faith in negotiations through Q4. We announced earlier this year that we did sign 1 OBA and we're expecting the other 2. And while we were negotiating a good faith and making progress, and again, these are complicated contracts in dealing with a number of officials in the German health care system. What became clear once we got into 2023 here was the importance of this March 15 date, which is the end of the "free pricing period in Germany." First of all, the free pricing period in Germany used to be 12 months. In January, new regulations became effective, where the free pricing period is now 6 months. And for us, based on our September 15 filing, that's today, March 15. And the difference for the payers, again, while we were negotiating these outcome-based agreements, the difference for the payers was that once they're on the other side of this free pricing period, if they were to pay a price for ROCTAVIAN that was higher than the final German federal price expected later this year, they would be reimbursed for that difference via a callback. And while our intention is to have the negotiated price with these individual payers now be close to the final German price, that guarantee wasn't there for the...

Yes. So like a final German price, where do you expect the price will be?

When? Later this year.

No, like the price.

So we decided that given the very competitive nature of severe hemophilia A therapies in Europe that we're not going to disclose...

Okay. I think previously was at net price, EUR 1.5 million -- yes.

Less than EUR 1. million.

So the final price, we will wait until end of this year, we -- okay, okay. So -- sorry, like I know we don't have too much time. I do want to get to this question. So basically, when patients -- we totally understand, I think a lot of audience is very familiar with this process. So after March 15, as all the final price, whatever the German government decided the -- from the payer perspective, the pay for performance, would that be for each individual payer discussion? Or would that be under federal discussion?

At this point in time, it's individual at the payer level. We would expect that the final German price will also have this outcomes-based agreement. So this will basically allow us to access the market faster, which by the way is a key consideration. While these have been really complicated contract negotiations that have taken longer than we expected. It is important to separate that from underlying demand.

So do you expect patient be on board after March 15 because they have a price locked end of this year, do you expect all those 2 other payers will sign after March 15?

That's the top priority...

Okay. And then do you expect also start to see meaningful patient on board, although the revenue -- final revenue you will be depending on the final price...

Yes, we're hopeful. That's where I was going with -- while this contracting process has been complicated you can separate it from the underlying demand where physicians are readying their patient populations for ROCTAVIAN. Patient eligibility testing for ROCTAVIAN is available and underway. So we're seeing uptake in that patient in physician interest and just need these outcomes-based agreements signed to truly just open the gate since the last...

The outcome-based agreement already finalized by now? Are you just waiting for them to sign or you are still in the discussion process?

We've agreed the terms and are just in the final execution phases. There's a natural sequence of events. We're turning our version in red lines around that same day, but we may not have that next meeting with one of these folks for another week or so. That's been the sort of natural sequence. But importantly, now that we're out of that free pricing period there's no financial incentive with these payers to wait any longer.

Okay. I know we're running out of time. I do want to ask the U.S. So how quick -- we now understand you have like June 30, that's a new PDUFA date. Regarding the payer discussion process, how quickly on June 30, if you've got a drug approval. And what's the status of a payer discussion in terms of the pay for performance and each program will be very complicated, how quick you can launch ROCTAVIAN and not just like able to sign the patient, we're able to get on to the drug because they will have insurance coverage.

So we -- we've been in our payer launch for the last 3 years because while the complete response letter in 2020 was a surprise and disappointing, we were ready to launch. And that's one area where we've made the most of this time is the continued preparations of the U.S. payer market. We've been approached by many large payers interested in ROCTAVIAN. We've had several of our own initiated meetings. The one key difference between our experience in Germany so far in the U.S. is that in the U.S., while we want to provide that same outcome-based guarantee, if you will. In the U.S., we plan to structure it in the form of a warranty. And so these will not be individually negotiated outcomes-based agreement. Our team has used the analogy of a warranty that comes with your car. You appreciate the value of the warranty because it protects you against some of the most expensive components in the vehicle. But when you purchase your car, you don't negotiate it. So that -- taking that off the table is one key aspect, our payer engagement as far as another. Just to be clear, we will -- until we have a J-code until we get on final formulary and coverage policies. We will need to navigate the medical exception process. We've done that before for other therapies. So we would anticipate -- and then subject to the other sort of launch logistics like getting final labels printed, product out into the market that usually takes a few weeks. And then the patient eligibility testing negative for antibodies to the AAV5 vector, that could all take a few weeks, but we'd expect ROCTAVIAN to be available within a couple of months after lunch.

And then you should be able to collect revenue basically after few months?

Yes. And then -- yes, so single dose and individual sale with revenue recognized upfront.

Sorry, we are running out of time. I know we still have quite a few questions, but...

We'll be speaking again. Thank you so much, Gena.

Thank you very much, yes. Thank you.

Thanks, everyone.

Thank you, everyone.