BioMarin Pharmaceutical Q4 2023 Earnings Call Transcript

Quartr
Provided by Quartr
February 22, 2024

There are 18 speakers on the call.

Operator

Thank you for standing by and welcome to the BioMarin Pharmaceutical 4th Quarter and Full Year 2023 Conference Call. I would now like to welcome Tracy McCarty, Head of Investor Relations to begin the call. Tracy, over to you.

Speaker 1

Thank you, Mandeep. Thank you, everyone, for joining us today. 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 research and development. Results may differ materially depending on the progress of BioMarin's product program, 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 SEC such as 10Q, 10 ks and 8 ks reports. In addition, we will use non GAAP financial measures as defined in Regulation G during the call today.

Speaker 1

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.

Speaker 1

GAAP in the earnings release and earnings presentation, both of which are available on 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 and D and Brian Mueller, Executive Vice President, Chief Financial Officer Jeff Azer, Executive Vice President, Chief Commercial Officer and Greg Guyer, Executive Vice President and Chief Technical Officer are here with us to answer questions during the Q and A portion of the call. I will now turn the call over to BioMarin's President and CEO, Alexander Hardy.

Speaker 2

Thank you, Tracy, and good afternoon, everyone. Thank you all for joining us today. As I said in January and reiterate today, the opportunity at BioMarin to positively impact patients' lives through transformative therapies, while delivering value creating revenue growth and profitability to shareholders has never been more evident. Touching on today's financial results. Total revenue grew 20% in the Q4 of 2023 compared to the Q4 of 2022.

Speaker 2

And total revenue grew 15% for the full year of 2023 compared to the prior year. This is a very strong performance. On a constant currency basis, total revenue growth was 25% in the 4th quarter and 20% for the full year. Equally as important BioMarin's growth story of bottom line results. Non GAAP earnings per share increased 48% in the Q4 versus the Q4 of 2022 and increased by 36% year over year.

Speaker 2

These 2023 results along with our growth expectations as implied by our full 2024 financial guidance provided today solidifies our position as a financially self sustaining business that can grow revenue, expand operating margins and accelerate earnings per share. Brian will provide more details on the financials in a moment. So I will now turn to update you on our progress on the priorities I outlined in January. The first to accelerate and maximize the Voxergo opportunity remains front and center. As demonstrated by the Voxergo financial results today, 178% growth year over year with close to 300 new patients added in Q4.

Speaker 2

The launch in achondroplasia is on a path to blockbuster status. We were pleased that 70% of new U. S. Prescriptions in Q4 were for children under the age of 5 following FDA's age expansion approval last October. The U.

Speaker 2

S. And EU approvals last quarter allowing treatment from infancy sets FOXErgo up to be a major multiyear growth driver. Beyond having an expanded age label, FOXOgo's profile benefits from more than 1,000 patient years of long term safety and efficacy data beyond just height. We believe this substantial clinical track record will encourage families to pursue VOXOZOGO treatment as early and for as long as possible to enable maximum essential therapeutic benefit. As a result of the growing global demand for BOXROGO treatment and the scope of our extensive long term clinical data, we're seeing an increase in the breadth of our prescriber base.

Speaker 2

Our world our work to build prescribing confidence and relationships with pediatric endocrinologists in the United States has been extremely well received. With recent Voxergo uptake children with 800 plazia under 5 being twice as fast as launch uptake for children over 5, our plan to drive earlier and longer intervention with a goal of greater therapeutic outcomes is on track. We plan to build on our established leadership in achondroplasia treatment to expand into mother into multiple other statural conditions. In the Q4, BioMarin began the enrollment in the 6 month observational run-in portion of the pivotal program with FOXO go for the treatment of children with hyperchondroplasia And we are actively engaging global health authorities regarding development programs in idiopathic short stature and multiple genetic short stature pathway conditions with plans to begin pivotal studies later this year. As Hank will discuss later, we believe that there is strong proof of concept and indications beyond achondroplasia.

Speaker 2

As we expand into these indications, we hope FOXO2 treatment will empower patients and families across a spectrum of statural conditions to live their lives to the fullest. For this reason, Voxelgau acceleration, achondroplasia and expansion into other indications remains the top priority at BioMarin. The second priority is establishing the Rocktavian opportunity. As I said in January, we believe 2024 2025 will inform Rocktavian's uptake curve and long term potential. We have been very pleased with the strong and positive payer response to the value proposition associated with Rockavian and how this is translated into published payer policies and lives covered.

Speaker 2

Furthermore, we continue to be confident with the clinical profile of the product, which is evidenced by the warranty agreement, which has been equally well received in the marketplace. We have also continued to make good progress, activating the global marketplace, including the recent publishing of the Italian Octavian price. We want to reiterate, however, that the complexity of aligning the required pre infusion checklist will take time. As I outlined last month for successful Octavian treatment, we need a motivated patient, a supportive payer and a treatment site with a physician who's willing and able to use the product. For a pioneering new therapy, this isn't a surprise and is the reason why we intend to let the results do the talking for Octavian uptake.

Speaker 2

We do expect patients to be treated with Octavian in 2020 4 as implied by its inclusion in our 2024 total revenue guidance. In the meantime, we will continue our work to activate the global marketplace and look forward to reporting Rocktavian revenues on a quarterly basis. The 3rd priority is our focus on the most productive R and D assets, those with transformational benefits for patients and high commercial potential. I've been spending a lot of time with the R and D team to understand the unique profile of each pipeline asset currently under development. I've been impressed by the level of innovation and expertise in developing transformational therapies.

Speaker 2

Keeping with our ambitious financial goals, we intend to hold a very high bar in terms of disciplined spend and prioritization of the most impactful medicines. To that end, we're undertaking a strategic portfolio review to determine which pipeline assets will advance and which will not. A complete update on prioritized R and D assets, those with the highest potential patient impact and highest potential value creation for shareholders will be communicated at our Investor Day later in 2024. In the meantime, we have a number of promising candidates advancing and Hank will provide an update on those in a moment. And lastly, our 4th priority is increasing profitability faster than originally planned.

Speaker 2

Our 2023 results and our full year guidance for 2024 both demonstrate our transition to growing profitability and significant operating leverage. Our full year 2024 guidance reflects double digit revenue growth regardless of RockTabin contributions and non GAAP earnings per share growing faster than revenues. OpSogo is expected to be a major driver of year over year growth and is reflected in our streamlined guidance. Total revenues guidance item, which includes all BioMarin commercial products. Non GAAP operating margin is a new line item heading to BioMarin's 2024 full year guidance, primarily because it reflects our focus on leverage across the P and L and an anticipated margin expansion this year, as well as providing you with a clear line of sight into our business performance.

Speaker 2

We believe these streamlined full year guidance items will allow you to track our financial progress as we strive to achieve the 4 strategic priorities I just described. So in summary, it's truly an exciting time at BioMarin and I see tremendous opportunity to create value for patients and for shareholders. The entire leadership team is hard at work on shaping the future corporate strategy, which will include views on R and D and capital allocation, as well as setting and taking steps to achieve ambitious long term financial targets. The entire organization is mobilized and approaching this work with a sense of urgency. We will be making significant progress that we will want to share externally on a timely basis.

Speaker 2

That could be incrementally and at the Investor Day for which we are evaluating the specific timing. Please stay tuned for additional updates. Thank you for your attention. And I will now turn over the call to Hank to provide an update on key R and D highlights. Hank?

Speaker 3

Thank you, Alexander, and good afternoon, everyone. The R and D organization is gratified by the productivity achieved on behalf of those who benefit from our innovative therapies. As we take BioMarin into the future and align our drug development capabilities with our ambitious financial goals, we will continue to prioritize the high impact medicines for patients. What will change is the level of spend and the rigor of the criteria used to determine which assets advance. As Alexander mentioned, we are undertaking a strategic assessment of our portfolio with the goal of ranking, prioritizing and then advancing only those assets that represent the highest value to patients.

Speaker 3

The assessment will evaluate the amount of time and investment required to determine proof of concept through growth through global approvals as well as the market opportunity and competitive landscape for each asset. The higher bar is designed to expedite development, increase the probability of success and improve cost effectiveness. We look forward to sharing the results of the strategic assessment at our Investor Day later in 2024. Touching briefly on a few clinical updates, we intend to open the treatment study of the pivotal trial with oxo go for hypochondriplasia in the middle of this year. Results from Doctor.

Speaker 3

Dawber's 52 week Phase 2 study with oxego in hyperchondroplasia were recently posted to the ACMG website ahead of his oral presentation at the meeting in Toronto next month. We are encouraged to see an increase in the 12 month annualized growth velocity of 1.81 centimeters in Boxogo treated children treated with hypochondriplasia and a safety and sustained durability profile observed in achondroplasia at 6 months 12 months. These data provide further support for our imminent plans to initiate the treatment phase of our pivotal program in hypochondriplasia expected to begin in mid-twenty 24. Regarding our development programs in idiopathic short stature and multiple genetic short stature pathway conditions, we are in discussions with global health authorities with plans to begin pivotal studies later this year. We look forward to Doctor.

Speaker 3

Dawber's ISS update at the Pediatric Endocrine Society meeting in May. BioMarin is securing our leadership position for treating multiple genetic central conditions and we look forward to updating you on our progress across the 3 pivotal programs just described. Turning briefly to other assets in the early stage pipeline. We're pleased to share that the Phase 2 proof of concept study of BMN-three fifty one for the treatment of Duchenne muscular dystrophy is open for enrollment. The 52 week study will enroll 18 boys with Duchenne muscular dystrophy with the potential to expand enrollment as needed and is designed to assess both dystrophin levels and functional measures.

Speaker 3

BMN349 is an early bioavailable small molecule designed for the treatment of alpha-one antitrypsin deficiency liver disease. Our nonclinical studies indicate 349 enables secretion of the mutant protein and prevents its polymerization in liver cells, which if unchecked drives the progression of liver disease. The single ascending dose study in healthy human volunteers is progressing with no safety signals observed thus far. We look forward to providing an update on our other pipeline assets following the strategic assessment described by Alexander. Thanks for your attention.

Speaker 3

Thanks for your support. And I will now turn the call over to Brian for financial

Speaker 4

quarter and full year 2023, including reconciliations of GAAP to non GAAP financial measures. I will provide my comments on a GAAP basis for 2023 results and will comment on key financial updates and guidance for 2024. All 2023 financial results will be available in our upcoming Form 10 ks, which we expect to file within the next few days. In the Q4 of 2023, BioMarin generated strong total revenue of $646,000,000 representing 20% year over year growth, including Kuvan and 26% growth, excluding Kuvan. As expected, the strength of our enzyme products generated more than $1,700,000,000 of net product revenues for the full year 2023, an increase of 4% year over year.

Speaker 4

Looking more closely at performance in the 4th quarter, Boxogo revenues of 140 $6,000,000 represented 118 percent quarter over quarter growth. We've previously discussed the supply constraint on Voxogo revenue in 2023. Our plans for supply to satisfy forecasted commercial demand around the middle of this year remain intact and our 4th quarter revenue result being higher than guidance was driven by some incremental supply that became available late in the quarter. Double digit revenue growth of both Vimizim and Palynziq in the 4th quarter was partially offset by anticipated lower Kuvan revenues and were also an important contributor to total revenues exceeding $2,400,000,000 for the full year 2023 and in line with our expectation. R and D expenses in the Q4 were $206,000,000 up $34,000,000 year over year, primarily due to increased early stage research activities as well as increased activity in our clinical program.

Speaker 4

SG and A expense in the Q4 was $275,000,000 up $29,000,000 from last year. Please note, we had a number of unique expenses during the Q4 of 2023, including impairment charges related to the discontinuation of our 1st generation Voxogopen, the devaluation of the Argentinian peso, along with incremental G and A expense related to corporate governance matters and leadership transitions. Together these items combined to total over $40,000,000 in Q4 2023. Moving to the bottom line, GAAP net income for the Q4 was $20,400,000 contributing to $168,000,000 of GAAP net income for the full year, representing GAAP diluted earnings per share of $0.87 per share. GAAP net income for the 4th quarter was also impacted by the recognition of a loss on an impairment recorded in the other income and expense line.

Speaker 4

I will note that on taxes similar to the Q3, we recognized some incremental discrete tax benefits in the 4th quarter that lowered our effective tax rate for the full year. We continue to project on a consolidated global level an effective tax rate in the low 20s for 2024 and beyond. Non GAAP income for the Q4 was $95,000,000 and $405,000,000 for the full year, representing full year non GAAP diluted EPS of $2.08 per share and growth over 36% over 2022. As we move into 2024, please note that there are select changes in BioMarin's P and L geography becoming effective in 2024. We are expecting to classify foreign currency revaluation and other income and expense going forward instead of its prior classification within SG and A expense.

Speaker 4

And idle plant time related costs will be classified in cost of sales going forward instead of their prior classification within SG and A expense. These changes, which again are classification only and do not impact the bottom line, are being made to provide greater clarity on our core operating results as well as to better align with industry peers. Details of the revised presentation of idle plant costs and foreign exchange gains and losses for previously reported 2023 2022 periods to conform with the presentation that we'll use going forward are available on our website. As Alexander mentioned, for 2024, we are guiding to total revenues, non GAAP operating margin and non GAAP diluted earnings per share. For the full year 2024, total revenues, we expect between $2,700,000,000 $2,800,000,000 which at its midpoint represents approximately 14% growth compared to 2023.

Speaker 4

To provide some additional context, since this is the first time that we are not guiding to specific brand revenues, our 2024 revenue guidance is driven by continued strong growth in Voxogo with expansion into younger age groups being a key driver of anticipated 2024 new patient starts, mid to high single digit growth across the 5 brands in our ERT portfolio and offset by the continued decline of Kuvan due to the loss of market exclusivity. While Rocktavian is definitively included in our total revenue forecast, the revenue contribution from RockTavian is relatively modest and risk adjusted based on our recent experience. Going forward, we will let the actual RockTavian results speak to the performance. Also one comment specific to the Q1 of 2024, that we are expecting a Q4 to Q1 dynamic with respect to order timing and volumes, whereby Q4 included some modest incremental revenue that will impact Q1 growth. We stand behind full year 2024 total revenue guidance provided today and we expect most of the twenty 24 total revenue growth to be weighted to the second half of the year.

Speaker 4

With respect to non GAAP operating margin for the full year 2024, we are guiding to between 23% 24%, which represents 4 percentage points of expansion versus the 2023 non GAAP operating margin of 19.4%. This is inclusive of our G and A reclassifications that I just mentioned. Over the last 3 years, including through our 2024 guidance, our non GAAP operating margin is projected to increase by approximately 13 percentage points from approximately 10% in 2021 to the 23.5 midpoint of our 2024 guidance. This demonstrates our commitment to growing profitability. However, it is not the end of the story as we are working through our cost efficiency and resource allocation priorities to help set ambitious long term financial targets to be communicated this year.

Speaker 4

And for non GAAP diluted earnings per share, we expect between $2.60 $2.80 per share for the full year 2024, which at the midpoint represents 30% growth over last year. As we move into the next chapter of BioMarin, we are executing on our growth strategy with impressive performance driven by Voxelgo and achondroplasia in pursuit of new indication, a durable and growing enzyme products business and the potential to realize the value of our key. Thank you for your continued support and we'll now open up the call to your questions. Operator?

Operator

The floor is now open for your questions. Our first question comes from the line of Salveen Richter with Goldman Sachs. Please go ahead.

Speaker 5

Good afternoon. Thanks for taking my question. I just wanted to better understand your EPS guidance for 2024 and what that assumes? Are there some OpEx and overall R and D cuts that are basically baked into the assumptions there? And help us also understand that you do your strategic portfolio review of the R and D program, what the criteria is for further investment in advancement?

Speaker 5

Thank you.

Speaker 4

Yes. Thanks, Helane. This is Brian. Appreciate the question. So first of all, we're pleased that the non GAAP EPS guidance midpoint implies 30% growth over this year and this year as noted was over 30% growth over 2022.

Speaker 4

There are a few items that impact the year over year non GAAP analysis. One is those some of those tax benefits that I mentioned in 2023. So now that tax since the beginning of 2023 is in our non GAAP income To the extent those benefits don't recur, that would be a headwind, if you will, to 2024 non GAAP. There's also some unique I mentioned the unique SG and A charges in 2023. We've got some incremental SG and A in 20 24.

Speaker 4

We're implementing a new ERP system, which is commanding some G and A resources in 20 24. So those are some of the dynamics. And to the part of your question about how much of our strategic savings or cost efficiency opportunities are included, just a modest amount of the known items at this point that we were already working on. We are embarking on the cost efficiency and operating model efficiency exploration as we speak. This is going to be part of the more ambitious financial target setting for the future to be communicated later this year.

Speaker 4

So once you get to stay tuned on that part, but I will say that there's not a substantial amount of savings related to that in this guide.

Speaker 3

Hi, Sabine. In response to the second question about prioritization of the R and D portfolio, we are undertaking a strategic assessment of our portfolio with a goal of ranking, prioritizing and then advancing only those assets that represent the highest value to patients and shareholders. The assessment will include considerations of time and money involved to prove concept or to advance to global approvals as the case may be, as well as consideration on market opportunity and competitive landscape. And the concept of having a very high bar for going forward is to foster expedited development. When things happen early that are impressive and important subsequent development and registration and market access happens much more quickly.

Speaker 3

And so with those higher bars, we can also see, that subsequent events have higher probability of success and also contribute to improving the cost effectiveness of programs. We look forward to sharing the results of the strategic investments later in 2024.

Operator

Our next question comes from the line of Akash Tewari with Jefferies. Please go ahead.

Speaker 6

Hey, thanks so much. Alexander, in the past, Biomin spent a lot of time and resources building up its gene therapy capabilities, but we haven't really seen that translate to shareholder value. For example, it looks like you're spending almost identical amounts on Boxoga and RockTavian, but with different results. What are your thoughts on potentially divesting your gene therapy platform or even RockTavian in order to cut costs and focus the story? Is that option out of the question in your mind?

Speaker 6

Thanks.

Speaker 2

Thanks very much for the question. As I mentioned in terms of our priorities, we're still very much at the early stage with RockTavian. We really want to establish the opportunity there. We are seeing progress in terms of market access, market activation. So we wanted to see that run through 2024, we also think probably into 2025 is when we'll be able to really determine.

Speaker 2

In the meantime, we're spending wisely and cautiously supporting the launch, but we're very mindful of the return on investment that we're currently seeing from RockTavian. At the same time, we referenced in this conversation today, strategic review. So we're also thinking about the future strategy of BioMarin. We're looking at the R and D portfolio. We're looking at our own experience with gene therapy, the scientific challenges, regulatory market access, market uptake and also taking note of what's happening in the world outside BioMarin's walls.

Speaker 2

And as part of that, we'll assess do we have the right exposure to gene therapies in our pipeline. Is it something we want to double down on, stay the course or take a lower exposure to gene therapies. So we'll continue to keep you updated. We'll provide more information at our Investor Day later this year.

Operator

Our next question comes from the line of Jessica Fye with JPMorgan. Please go ahead.

Speaker 1

Great. Good afternoon. Thanks for taking my questions. A couple on Voxogo. First, on the financial side, you mentioned 4Q revenue, I think you're seeing sort of broadly 4Q revenue reflected modest incremental revenue that would impact 1Q growth.

Speaker 1

Can you talk to whether the 4Q Box Sogo number specifically benefited from any channel inventory changes or lumpy international orders? And if so, just quantify what was out of the ordinary? And then on the pipeline side, can you do basket trials with multiple indications for some of these additional potential Voxogo development areas? How likely is the FDA or other regulators to sign off on something like that type of approach? Thank you.

Speaker 4

Hey, Jess, it's Brian. Thanks. I'll take the first part of your question. Good question. And the way I respond is nothing excessive with respect to the channel and nothing broad across the marketplaces.

Speaker 4

I made that comment because when we gave our full year guide, essentially Q4 guide back on the 1st November, the high end was $455,000,000 and we talked about how that high end was supply constrained. And so here we are reporting close to $470,000,000 for the quarter. So I wanted to explain that the circumstances there were some additional commercial supply that became available. So we used it to meet demand that was out there. And the point is that with respect to Q1, we don't necessarily expect or guarantee that that same dynamic can recur.

Speaker 4

So that's the Q4, Q1 dynamic. That plus decreasing Kuvan in Q1 is just an overall comment that Q1 is not going to be our strongest quarter and we're going to see most of the growth in the second half of the year.

Speaker 3

Thanks, Jessica. As regards to the concept of basket, one of the great benefits of having Doctor. Dawber do his study is we actually have really good regulatory feedback on that question already. And in fact, you could see that his study is itself a basket of things that might be formally known as subsets of idiopathic short stature or hypochondriplasia. So the concept of basketing has already been in front of the FDA and has already gained a little bit of acceptance.

Speaker 3

And having said that though, as reflected in our prepared comments, we are in the stages of finalizing our game plan after one after the last interactions with health authorities around the world and we'll communicate final design decisions when those studies come online. All that said, we're not waiting for any of that because we're also implementing our feeder studies, the baseline run-in studies that are essential for enrolling these baskets of studies and so that we can expedite the start and completion of these additional registration activities. So in sum, I think we're trying to be as clever as we can be about getting these studies underway and working with health authorities around the world to ensure that they meet the demands of regulators.

Speaker 1

Thank you.

Operator

Our next question comes from the line of Phil Nadeau with TD Cowen. Please go ahead.

Speaker 7

Thanks for taking our question. A follow-up question on Voxogo. In the past, Varmint had talked about converting Voxogo's approval from accelerated to full and that potentially changing what would be necessary for the competition to be approved. Is there any update on the process of converting FOXOZOZOZ approval to full? And at this point, would it have any impact on the regulatory requirements for the potential competition that's in pivotal studies already?

Speaker 7

Thanks.

Speaker 3

Hi, Phil. No particular update on the regulatory status or strategy or read through on competitors. But one of the things that's really exciting about update that we can report is that every so often we look at ongoing studies to assess durability of Voxogo's effect and we continue to be reassured that Voxogo unlike other growth promoting types of drugs has this remarkable benefit of sustaining its effect. And that's really one of the key features of benefit Oxego for patients. And I think it's going to be an important bar for any future innovation in this space to ensure that patients can resume a physiologically normal growth rate safely and through the entire period of their growth starting from earliest diagnosis all the way through the closure of the growth plate.

Speaker 3

So good on VoxSOGO so far.

Speaker 2

Perfect. Thank you.

Operator

Our next question comes from Geoff Meacham with Bank of America. Please go ahead.

Speaker 8

Hey, guys. Good afternoon. Thanks for the question. Another one on VoxSOGO, I know it's a centerpiece of growth and the pipeline, the major pipeline investment for the year beyond. But I wanted to ask you, what's been the impact of your commercial investments so far in terms of the pace of new starts or broaden the prescriber base looking to the second half of last year?

Speaker 8

And then what do you think about what does success look like for 2025? I'm just trying to figure out a couple of metrics that we can kind of evaluate over the course of this year for Box AUGO. Thank you.

Speaker 4

Hey, Jeff. It's Jeff here.

Speaker 9

Let me take a shot of that. So, I think we've had some good returns on our commercial investments today. You mentioned new starts. New starts were accelerated starting late last year following the label expansion as noted in the prepared remarks. Expanding the prescriber base, particularly in the United States to pediatric endocrinologists is a particular area of focus both last year and into this year.

Speaker 9

We're making good progress. And in terms of metrics, I would say we're dialing back from specific quantitative metrics on these launches and turning more towards revenue. I might suggest that with a post launch revenue base for Boxelgo in particular, the best metric I would recommend now and going forward is the revenue line.

Speaker 6

Great. Thank

Operator

you. Our next question comes from the line of Ellie Merly with UBS Financial. Please go ahead.

Speaker 10

Hey guys, thanks for taking the question. At the ISS FOXO go data update you mentioned will be coming in May. What should we expect from that in terms of the patient numbers and what you're looking to see? And then just as you look across the many different short stature conditions, how are you thinking about prioritization across the indications in which you'd move forward versus not and any threshold in terms of efficacy?

Speaker 3

Okay. As regards to Doctor. Dawber's ISS anticipated update, this is his study and so it's very much under his determination as to what he's going to update. My understanding is that he's enrolled more hypochondriplasia patients than other indications, but I think he wouldn't be reporting unless he felt that there was useful information to be reported. I think the things to be looking for really are corroboration of what I think most scientists fundamentally believe, which is that CNP as a master regulator of bone growth is going to work across a wide variety of indications.

Speaker 3

And so as we had seen with the hypochondriplasia program, so it's actually sort of the first half of the cohort at the 6 month mark that enabled FDA to gain confidence that we could go directly into Phase 3 and that's predicated on a lot of this biology and therefore, I think the important outcome of the ISS presentation at PES is going to be, are we seeing basically the same effect that we expect to see in the different types of statral disorders. And as regards to your second question, I think the prioritization process has been described, it really boils down to fairly conventional metrics around time and cost to proof of concept or to global registration, value that gets created for patients and for shareholders, with the idea of by setting a high bar for something like proof of concept, then you really only advance your winners. And by focusing on those, we hope that will expedite development and bring faster and higher value to patients and shareholders of the undertaking.

Speaker 11

Thanks.

Operator

Our next question comes from the line of Joseph Schwartz with Leerink Partners. Please go ahead.

Speaker 3

Great. Thank you very much and welcome, Alexander. I think I heard you say that the outcomes of the Strategic and Operating Review Committee could be communicated incrementally as well as at the Investor Day in Q3. So I was wondering if you have a sense of which updates might emerge sooner and which updates could require more time to formulate?

Speaker 2

Thanks, Joseph. Thanks very much for the question. The SoC is proceeding extremely well. We've met frequently and we're making progress on the financial guidance, long term financial guidance and the capital allocation strategy. We also have a lot of other work underway as you get a sense from cost optimization to acceleration of Voxogo even faster R and D prioritization etcetera, etcetera.

Speaker 2

And right now, we're figuring out what is the best timing to update the external world on our progress on these points. Obviously, the benefit of the Investor Day just allows us to put this all together with a strategic context of where we're going to take BioMarin into the future. At the same time, we're keen to share the information we can as soon as we can on the progress we're making. So as I said in my prepared remarks, please stand by for more information. The latest possible day will be Investor Day for all of that information.

Speaker 2

If it makes sense, we'll communicate other information sooner.

Operator

Our next question comes from the line of Robin Karnauskas with Tuohy Securities. Please go ahead.

Speaker 12

Hi, this is Nishanth. I'm on for Robin. One on Voxelgo, so I know you have spoken on competitors' oral treatment for achondroplasia. Can you give us any color anecdotally about how patients on Voxelgo or prescribers think about the orals? Is there room for both if it gets approved?

Speaker 12

Continue.

Speaker 3

We've shared robust and highly significant growth data from VOXXOGO in our clinical trials. And as I just referenced, the accumulating data on height gain is really quite positive and informative. And you'll see more of that at ACMG demonstrating these long term positive effects on growth. Importantly, also on improvements beyond height, for example, improvements in the physical domains of quality of life types of scores. And so we're really focused on is building our leadership position in the genetic statral conditions where we see an incredible potential for VOXXO, again to normalize growth, to sustain that normalization of growth through the entire growing period and having the greatest possible impact in patients' lives.

Speaker 2

Thank you.

Operator

Our next question comes from the line of Mohit Bansal with Wells Fargo. Please go ahead.

Speaker 13

Great. Thank you very much for taking my question and congrats to Alexander and welcome. Maybe one question for you. In general, what is your general thought on gene therapy as a therapeutics modality? I'm asking because a significant portion of your pipeline is investing in gene therapy and would love to understand how you think about gene therapy in general for rare diseases and what are the puts and takes there?

Speaker 13

Thank you.

Speaker 2

Thank you very much for your question. Yes, as I referenced a little bit, but let me go in a little bit more detail. We've got a very broad and exciting pipeline across multiple modalities, including gene therapies. Gene therapies offer obviously significant potential benefits for patients, transformative benefits. We have an expertise and ability to develop, manufacture and now commercialize gene therapies in multiple geographies.

Speaker 2

I said that is a unique set of capabilities that BioMarin has. As we look at our pipeline as Hank was talking about, we're going to be assessing at a program by program basis. What is the transformative potential of the program together with the commercial potential? At the same time, we're also looking at the overall strategy by Marin. What are we really, really good at?

Speaker 2

And where we're confident we're going to have a sustainable competitive advantage going forward? And that will include asking and answering the question about our exposure to gene therapy, which will then inform also the prioritization of our portfolio. So more to come on this. Please hold your question. We'll have a clearer idea of this and we'll share it at our Investor Day later in 2024.

Speaker 13

Got it. Thank you.

Operator

Our next question comes from the line of Gena Wang with Barclays. Please go ahead.

Speaker 14

Thank you. I have 2 very quick questions. The first one is regarding Voxogo. I think in your press release, you say you already treated 2,613 achondroplasia patient across 41 active markets. What is the total market opportunity in terms of achondroplasia patient numbers for these 41 active markets?

Speaker 14

And also what is the pricing dynamics there? And the second very quickly regarding RockTavian, I think the last earnings was mentioned that 6 patients were ready to be dosed in 4Q 'twenty three. Should we expect the remaining 5 patients to be dosed in Q1 'twenty four?

Speaker 4

Hi, Gina. It's Jeff here. I'm going

Speaker 9

to take that first question on Bauxhall Go patient numbers. So essentially those 41 markets represent kind of the core of the commercial footprint that we usually use to quote eligible patient numbers and market potential. And for that group of patients, that would now total about 20,000 patients eligible and that's essentially matching the lower age limit in the different labels, which are 0, 4 months or 6 months depending on where you are in the world. And in terms of the Octavian question, back to Alexander.

Speaker 2

Thanks, Gina. Yes, let me take your second question. We're going to provide details on Q1, including product revenues at our conference call in April. We're tracking progress on-site readiness at hemophilia treatment centers with payers, completed CDx tests, positive CDx tests. But clearly these metrics so far not yet correlated directly to patient update.

Speaker 2

So, we're no longer going to be providing specific updates on these metrics. Once again, just to reiterate it, we're going to let the actual experience with actual commercial patient infusions, form our future expectations and we'll report at the end of the quarter about our progress this year with infusions and revenue.

Operator

Our next question comes from the line of Paul Matteis with Stifel. Please go ahead.

Speaker 15

Hi, this is Julian on for Paul. Thanks so much for taking our question. I guess just in terms of thinking about capital allocation moving forward, obviously you're reviewing the pipeline. But could you just provide a little bit of color on do you anticipate reinvesting in the pipeline perhaps after coaling programs that don't meet that high bar that you speak of for advancement? Or are you sort of more focusing on growing the commercial business for Voxogo?

Speaker 15

I guess just any color you can provide on future capital allocation for 2024 and beyond would be super helpful. Thank you.

Speaker 4

Yes. Thanks for the question. This is Brian. We'll ask you to wait a bit longer for the outcomes of our strategic review to give you details on our capital allocation strategy. I mean, we can say that internal innovation given our track record and value created in the current business is going to continue to be a priority.

Speaker 4

But we also recognize that Bauma Rin not only carries a substantial amount of total cash and investments today, but we are now cash flow positive. So we are committed to developing a capital allocation strategy that's in the interest of maximizing shareholder value. That work is in process and we will look forward very much to sharing it with you, but we'll just ask you to stay tuned until we finish the work. Thanks.

Operator

Our next question comes from the line of Vikram Per Ho with Morgan Stanley. Please go ahead.

Speaker 7

Hi, good afternoon. Thanks for taking our question. We had a follow-up question on RockTavian, a bit of a broader one. So your release mentions and you also just mentioned a couple of different areas of work that you're going through to help progress commercialization here from patient education to site readiness. But I was wondering, now that you've had a few months of experience with the process for Octavian, what do you see as the 1 or 2 specific factors or drivers of uptake that you think you have the most near term leverage with and control over that could support the trajectory here in 2024?

Speaker 9

Hi, Vikram. This is Jeff here. I'll talk a little bit about that. What we've said is that we've seen very nice signals of patient demand. For example, in the United States, much of last year we talked about the buildup of a patient funnel in the strategic market and priority market of Germany, where we had gotten good uptake of CDx testing.

Speaker 9

We've also talked about the importance of site readiness and having reimbursement approvals that kind of clear that important reimbursement gate. As Alexander noted, we haven't seen a direct correlation. So we haven't been able to say, hey, this or that or these leading indicators are good predictive indicators of patient infusion and revenue upticks. And so I think the idea from here is instead of trying to better tune that model, we're going to let the revenue, the actual revenue do the speaking for Rod Kavian.

Operator

Our next question comes from the line of Tim Lugo with William Blair. Please go ahead.

Speaker 16

Thanks for the questions. You introduced operating margin in your guidance and you have a nice step up year over year. What are the initial impressions on whether BioMarin will be able to continue to expand operating margins and eventually reach north of 40% like some of the other rare disease focused companies?

Speaker 4

Yes. Thanks a lot, Tim. Appreciate the question. It's a great one. So again, we are pleased to be able to report and deliver over the last few years kind of year on year operating margin improvement.

Speaker 4

We've been talking about it for a while and it is we're pleased to be able to actually deliver upon that. I'll give a 2 part answer to your question. On one hand, we know there's a lot of opportunity with the type of revenue growth that BioMarin has, not to mention the outcomes of the strategic work that we've talked about a lot today and the potential impact on revenue into the future, we also recognize that we've got operating model and cost efficiency opportunities. We built this global infrastructure that now we're leveraging to support the strong launch of Voxelgo, for example, in a very deliberate and organic way over the years. We've reached a lot of scale.

Speaker 4

We've got world class capabilities across manufacturing, R and D, commercial, business support, but we've been focused on growing and scaling for years. And what that means is now we have a real opportunity to optimize the business model and make the cost structure more efficient. That's the work we're doing together with the SORC. And by the way, the entire leadership team in enterprise, these are enterprise wide initiatives that the company is really rallying behind. And the second part of my question is for that reason, we'll have to ask you to stay tuned.

Speaker 4

But by all means, you could picture the work that we're doing to design what the appropriate and deliverable financial targets for long term guidance purposes will be for the company. And we'll be sure to not just share those numerically, but the rationale, the strategy and our plans to deliver upon them. So that's just going to take a bit more time. Thank you though for the question.

Speaker 16

Understood. Thank you.

Operator

Our next question comes from the line of Olivia Breyer with Cantor Fitzgerald. Please go ahead.

Speaker 17

Hey, good afternoon guys. Thank you for the question. Wanted to ask about lifecycle management for your Voxogo franchise. Obviously, you have a lot of expansion opportunities within that product. But could we see any efforts to maybe better optimize the dosing schedule or maybe any other avenues to keep that product as competitive as possible?

Speaker 17

Thank you.

Speaker 3

Yes, thanks. That's a good question. And yes, in fact, we do plan to undertake some further dose optimization. Right now, our thinking is to try to do that in parallel with registration enabling trials. But again, details to be communicated when study designs are final after regulatory authority alignment.

Operator

Our next question comes from the line of Kostas Beluris with BMO Capital Markets. Please go ahead.

Speaker 13

Hello, everyone. Congrats on the progress. Thanks for taking our question. Given that your R and D days planned for the 3rd quarter of 2024, which is about a year after Alex took over, and likely this is when you will potentially share pipeline prioritization details. I'm wondering whether you will continue to progress all pipeline programs full steam until this Q3?

Speaker 13

Or you are going to pause the progression of some of the programs until you decide on their fate? Thank you.

Speaker 3

Thanks, Asos. I think a big part of the purpose of the early part of the year of prioritizing the R and D portfolio ties to the conversations that Brian was just having around capital allocation, that Alexander was having around longer term financial outlook. When I think about that is that it's a real it's a great thing that our company can generate cash that can be reinvested in our R and D portfolio. We have to earn that through developing assets that have value to then reapply to generate new medicines. But we have to do that in a business context.

Speaker 3

And so as soon as we can make decisions about what's going to go forward, we want to invest in expediting that and also things that are not going to go forward, we want to make sure that we don't spend money on things that are not going to go forward. So that's very much the intention and it's I think ties to Alexander's statement of how the progress of our process through to Investor Day could be potentially incrementally informed over the course of the year.

Operator

Our next question comes from the line of Jack Allen with Baird. Please go ahead.

Speaker 6

Thanks for taking our question and congratulations on all the progress made over the course of the quarter. I wanted to ask one on Boxogo. You mentioned that about 70 percent of the new U. S. Starts in the Q4 were from the younger patients and the label expansion.

Speaker 6

I guess, could you provide some more context around how penetrated the older patients are and how should we think about growth of Voxogo moving forward? Is it mainly expected that it will come from younger patients or is there still opportunity to expand uptake in the older patients in the U. S. As well?

Speaker 9

Thanks for the question, Jack. In the United States, we haven't disclosed penetration rates and we're not going to introduce that metric. Overall, with reported numbers of about 2,600 patients and you heard me quote the question to Gina earlier of an eligible patient population of 20,000. You can do the quick arithmetic on that yourself. Specifically from the United States, which is one of our largest and highest value markets, we're really encouraged by both the continued uptake in the older children, those ages 5 and up from the original approval and also the new demand from the younger patients.

Speaker 9

So I think we're pretty confident that we've got an opportunity for increased uptake there going forward.

Speaker 4

Great. Thanks.

Operator

Our next question comes from the line of David Lebowitz with Citigroup. Please go ahead.

Speaker 13

Thank you very much for taking my question. Just following up on that, with 70% of patients on Voxogo, the new scripts being young patients, is it to assume that that number would be maintained next year or should that dial back as the year goes on?

Speaker 9

Hi, David. Let me take that one on. I don't think we have enough data to specifically say that we're going to maintain that mix. But that's a one time metric intended to give you a little bit of color on how things are going in that key market since we got the younger age label expansion. But it's an important signal and I think it's a good one, good and encouraging looking forward.

Speaker 13

Thanks for taking my question.

Operator

Our final question comes from the line of Luca Eci with RBC Capital Markets. Please go

Speaker 11

ahead. Great. Thanks so much. Thanks for squeezing me in. Maybe, Alexander, at the R and D Day last year, which I appreciate was before your time, BioMarin talked quite extensively about lifecycle management for valrox, including testing in patients with neutralizing antibodies to AAV5, patients with Factor VIII inhibitors, younger patients and so on.

Speaker 11

Given the relatively slow launch, is that still the plan? Or is there a scenario which don't invest that capital, just you prioritize operational efficiency to really make the story here driven primarily by Aboxilgo, Avarzumab, the BioBox. Any color there, much appreciated. Thanks so much.

Speaker 3

We do intend to include a review of those studies in our portfolio assessment and prioritization. And so we'll communicate the plans for each of the additional potential Rockavian indications that we did outline at R and D Day that you did just list. And as we complete our portfolio prioritization process as well as our overall corporate strategy. And so we'll follow-up then.

Speaker 2

And I would just add to that, that as we've said, that R and D prioritization is looking at the potential benefit in terms of the medical impact together with the commercial potential. So we'll be taking into mind our experience with the commercial uptake of Roktavian to inform the business case around those lifecycle developments. Thank you very much.

Operator

I would now like to turn the call over to Alexander Hardy, President and CEO of BioMarin for closing remarks.

Speaker 2

Thank you very much for joining us today. As you've heard, we are really hard at work here on shaping the future corporate and R and D strategy as well as setting ambitious long term financial goals. As you've heard, these are on about accelerating revenue and also profitability. We're approaching this work with a sense of urgency and we're making significant progress. We're looking forward to sharing that with you on a timely basis.

Speaker 2

So as you heard, please stay tuned for additional updates. Thank you very much for your attention and your questions and goodbye.

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Earnings Conference Call
BioMarin Pharmaceutical Q4 2023
00:00 / 00:00