Q3 2024 Biomarin Pharmaceutical Inc Earnings Call

Bailey: Thank you for standing by. My name is Bailey and I will be your conference operator today. At this time, I would like to welcome everyone to the BioMorning Fulcke Pharmaceuticals 3rd Quarter 2024 conference call.

Bailey: All lines have been placed on mute to prevent any background noise.

Bailey: After the speaker's remarks, there will be a question and answer session.

Bailey: If you would like to ask a question during this time, simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question, again press star and one. I would now like to turn the call over to Traci McCarty, Group Vice President of Investor Relations. You may begin.

Traci Mccarty: Thank you, Operator. To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of Biomarin Pharmaceutical, Inc., including expectations regarding Biomarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development.

Traci Mccarty: Results may differ materially depending on the progress of Biomarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors. And those factors detailed in Biomarin's filings with

Traci Mccarty: with the Securities and Exchange Commission, such as 10-Q, 10-K, and 8-K reports.

Traci Mccarty: In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today.

Traci Mccarty: These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to, financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation.

Traci Mccarty: both of which are available in the investor relations section of our website.

Speaker Change: On the call from Biomarine Management today are Alexander Hardy, President and Chief Executive Officer, Brian Mueller, Executive Vice President, Chief Financial Officer, Kristen Hubbard, Executive Vice President, Chief Commercial Officer, and Greg Freyberg, Executive Vice President, Worldwide R&D.

Traci Mccarty: I will now turn the call over to Biomarine's President and CEO, Alexander Hardy.

Alexander Hardy: Thank you, Traci, and good afternoon, everyone. Thank you for joining us today for our third quarter 2024 earnings call.

Alexander Hardy: The strategic and operational decisions we have made over the last nine months are driving strong performance.

Alexander Hardy: And I'm pleased to report another quarter of record financial results, with revenues reaching $746 million, marking a robust 28% increase compared to the same period in 2023.

Alexander Hardy: As outlined in InVEST today, we're implementing our new corporate strategy focused on innovation, growth, and value commitment.

Alexander Hardy: During the quarter, we made significant progress on each of these pillars, resulting in strong performance.

Alexander Hardy: We are structuring the company around new business units in skeletal conditions, enzyme therapies, and Roktavian to drive accountability, deliver stronger revenue growth, and improve efficiency.

Alexander Hardy: while reaching a growing number of patients around the world.

Alexander Hardy: We have set this company on the path to stronger performance, and these results are evidence of our progress.

Alexander Hardy: Today, I'm also delighted to welcome Greg Freyberg and James Sabre to Biomarin.

Alexander Hardy: With Greg and James in role, and the addition of Kristen Hubbard earlier this year, we have incredible strength and depth in our leadership team, with the right combination of scientific and business acumen.

Alexander Hardy: I'm confident that with these leaders in place, we are positioned to deliver the new strategy and innovate across all aspects of our business.

Alexander Hardy: Greg and Kristen will provide their updates on the call in a moment.

Alexander Hardy: Moving to the results in the quarter. Strong financial performance was driven by a 50% revenue growth in Voxogo for the treatment of achondroplasia.

Alexander Hardy: This growth was supported by a significant level of new patient starts driven by increased penetration in the U.S. and new geographies that leveraged our broad geographic footprint and capabilities.

Alexander Hardy: The growth is also being driven by our expanded indication into infants and the increasing evidence of Voxogo's benefits to the health of patients beyond height.

Alexander Hardy: As our global launch of VOXERGO for achondroplasia continues to gain momentum worldwide, we have confidence in our sustained leadership across our skeletal conditions franchise over the long term.

Alexander Hardy: Our proven capabilities of diagnosing, treating, facilitating market access, and building the achondroplasia therapeutic area, provide Biomarin the unique ability to sustainably lead this global market.

Alexander Hardy: The five key reasons supporting our confidence in growing and maintaining the achondroplasia market are

Alexander Hardy: First, a potential five-year commercial lead including the largest database in achondroplasia of long-term durable efficacy in greater than 6,000 patient years of safety.

Alexander Hardy: and an emerging data set on benefits beyond height, including quality of life.

Alexander Hardy: Second, global approvals from infancy and our plans to continue to deeply penetrate this market segment along with complicated potential switch market dynamics.

Alexander Hardy: The US and EU markets comprise approximately 32% of the global total addressable patient population.

Alexander Hardy: and therefore the majority, 68%.

Alexander Hardy: is more insulated from mid-term competitive threats because of our global capabilities and market leadership.

Alexander Hardy: Third, our global commercial medical and market access capabilities built over 20 years as just described.

Alexander Hardy: Fourth, the prioritization and acceleration of BMN 333, a long-acting CNP that leverages the same CNP as FOXOGO.

Alexander Hardy: And fifth, our plans to vigorously defend our intellectual property.

Alexander Hardy: In summary, we maintain our confidence in the long-term growth potential of our Skeletal Conditions franchise.

Alexander Hardy: Beyond skeletal conditions, our global enzyme therapies portfolio continue to perform well and grow in the third quarter.

Alexander Hardy: with 27% revenue growth in Q3 and 13% year-to-date growth.

Alexander Hardy: Q3 enzyme therapy revenue benefited from increased aldurazine revenue as we delivered a substantial amount of product to Sanofi during the quarter.

Alexander Hardy: Excluding the algerozyme revenue, the enzyme therapies still grew at close to 10% year-to-date.

Alexander Hardy: We're starting to deploy the new initiatives outlined at Investor Day, and these, together with a focus from the business unit structure, give us confidence that we can grow this portfolio even faster than historical rates.

Alexander Hardy: We're pleased today to raise for you guidance at the midpoint.

Alexander Hardy: for all items driven by our strong ongoing execution and focus on performance.

Alexander Hardy: Alongside record revenues, we have driven considerable profitability expansion, with year-to-date non-GAAP EPS growing more than three times faster than revenue.

Alexander Hardy: As a result of our focused cost transformation program and leveraged revenue growth, we continue to drive substantial expansion of our operating margin.

Alexander Hardy: Our strong financial performance reinforces our confidence in reaching our mid-term and long-term outlook provided we invest today.

Alexander Hardy: Today, we are reaffirming our long-term guidance and outlook, which include achieving approximately $4 billion in total revenues by 2027.

Alexander Hardy: reaching 40% non-gap operating margin starting in 2026 and growing to the low mid 40% range over time.

Alexander Hardy: generating more than 1.25 billion in operating cash flow per year starting in 2027.

Alexander Hardy: targeting a mid-teen compound annual growth rate for total revenues through 2034.

Alexander Hardy: developing potential treatments for skeletal conditions that represent a greater than $5 billion revenue opportunity over time. And as a reminder, this $5 billion opportunity assumes only a very modest penetration into the skeletal conditions indications.

Alexander Hardy: In summary, our third quarter performance highlights the ongoing execution of our corporate strategy under Biomarine's new operational plan to achieve sustained and significant returns.

Alexander Hardy: These results should demonstrate our commitment to superior performance resulting from our laser focus on making an impact on the life of each patient we serve.

Alexander Hardy: I would like to thank our employees around the world for all of the work they've done during the third quarter to contribute to this progress and to their commitment to continuing to evolve the way we work to support the new strategy.

Speaker Change: Thank you for your attention. I will now turn the call over to Brian to provide an overview of our financial highlights for the quarter.

Brian Mueller: Thank you, Alexander.

Brian Mueller: Please refer to today's press release summarizing our financial results for full details on the third quarter of 2024.

Brian Mueller: including reconciliations of GAAP to non-GAAP financial measures.

Brian Mueller: All third quarter 2024 results will be available in our upcoming Form 10-Q, which we expect to file in the coming days.

Speaker Change: As Alexander just shared, our record-setting performance that generated 746 million dollars of total revenue drove robust growth year over year.

Speaker Change: Voxoko was a key driver of revenue growth, increasing 54% year over year, and reaching $190 million in the third quarter.

Speaker Change: Please recall that Q2 VoxZilgo revenues benefited from order timing of approximately $20 million, and this timing dynamic negatively impacted Q3 revenues.

Speaker Change: Turning to our enzyme therapies, which include vimazine, naglozyme, alderozyme, brinura, and palenzeque, this portfolio contributed $509 million of combined net product revenues in the third quarter, representing a 27% year-over-year increase.

Speaker Change: Aldurazine was a large driver of revenue growth, primarily due to the timing of order fulfillment to Santa Fe.

Speaker Change: as we recognize revenues when the product is released and control is transferred to Santa Fe who is responsible for selling alderazine into the market.

Speaker Change: We estimate that the impact of timing on Alderazyme revenue in Q3 is approximately $20 to $30 million.

Speaker Change: Nagelsheim grew 21% year-over-year, demonstrating increasing demand and benefiting from some order timing.

Speaker Change: Palanzec revenues increased 15% year-over-year driven by the strong patient uptake in the U.S., our largest market for Palanzec.

Speaker Change: Looking ahead, we are confident in the continued strong commercial performance of our portfolio, driven by VoxOgo and the enzyme therapies.

Speaker Change: As a result, we are narrowing our full year 2024 revenue guidance to between $2.79 billion and $2.825 billion, thereby raising the midpoint, representing approximately 16% year-over-year growth at the midpoint.

Speaker Change: From an operating expense standpoint, GAAP R&D expense was $185 million in the third quarter, and GAAP SG&A expense was $253 million.

Speaker Change: SG&A expense on a GAAP basis includes $45 million of restructuring expenses that we adjust out of non-GAAP income.

Speaker Change: And after that adjustment, SG&A expense decreased slightly year over year, with R&D expense also declining year over year.

Speaker Change: Overall operating expenses are in a transition phase and trended lower in Q3 as we start to realize the savings from the various portfolio and organizational actions taken this year.

Speaker Change: We expect operating expenses to begin to increase going forward as we accelerate our commercial growth strategies and advance the R&D pipeline, albeit at a significantly lower expense growth rate than revenue growth, which will drive operating margin improvement over time.

Speaker Change: Biomarin has executed well in 2024. Our Q3 GAAP diluted earnings per share increased 162 percent over Q3 last year to 55 cents in the quarter.

Speaker Change: Our Q3 non-GAAP diluted earnings per share nearly doubled compared to Q3 2023, totaling $0.91 in the quarter.

Speaker Change: We achieved a non-gap operating margin of 28% for the third quarter, driven by a combination of robust revenue growth and our commitment to operational efficiency.

Speaker Change: It is important to note that these results have been achieved during a year of significant transformation for the company, including the previously mentioned portfolio prioritization decisions and the ongoing execution of Fomarin's enterprise-wide reorganization.

Speaker Change: Biomarin's strong execution on profitability in Q3 2024 also generated significant operating cash flows.

Speaker Change: totaling 221 million dollars in Q3.

Speaker Change: an increase of 63% compared to the third quarter of last year.

Speaker Change: We also settled the $495 million of convertible debt maturity in cash during Q3 as planned.

Speaker Change: This was the first time that Biomarin managed a convertible debt maturity without issuing a new convertible instrument.

Speaker Change: thereby retiring approximately 4 million potentially diluted shares and returning that value to shareholders.

Speaker Change: Total cash and investments at the end of Q3 2024 were approximately $1.5 billion, and we concluded the quarter with the balance sheet in an even stronger position as we continue on the trajectory to generate increasing operating cash flow into the future.

Speaker Change: As mentioned, given our strong year-to-date execution, we've raised our guidance at the midpoint across all items for 2024.

Speaker Change: in addition to the aforementioned revenue guidance raised.

Speaker Change: We now guide to a non-gap operating margin of 26.5% to 27.5%.

Speaker Change: representing a 7.6 percentage point expansion at the midpoint versus 2023.

Speaker Change: We have also increased our non-GAAP diluted earnings per share guidance to between $3.25 and $3.35, aligning with our goal to grow profitability faster than revenues.

Speaker Change: Well, we are expecting year-over-year revenue and profitability growth in the fourth quarter of 2024. It is important to reiterate that Q3 benefited from the Alderzheim timing previously forecasted in Q4.

Speaker Change: and to my comments a moment ago on operating expenses.

Speaker Change: We expect higher operating expenses in Q4 versus Q3 2024, also in line with historical Q4 trends.

Speaker Change: As a reminder, Biomarin's dynamic global business continues to experience quarter-to-quarter fluctuations, and we point to our full-year guidance and annual results as the best measures of our performance and growth.

Speaker Change: In summary, as we implement the REVAMP strategy, we drove strong results for the third quarter and year to date.

Speaker Change: This performance, along with our increased guidance for the year and reaffirmation of BioMarin's long-term outlooks, underscore our confidence in delivering high growth and superior returns in 2024 and beyond.

Speaker Change: I will now turn the call to Kristen to discuss the commercial dynamics behind our strong quarterly performance.

Speaker Change: That's it.

Kristen Hubbard: Thank you, Brian. As you have heard, we are proud of our commercial performance, demonstrating continued momentum in the third quarter.

Kristen Hubbard: The VOXOGO results underscore the significant opportunity ahead in achondroplasia, where we have identified a total addressable patient population, or TAP, of 24,000 patients across BioMarin's global commercial presence.

Kristen Hubbard: To provide some context on our expanding global leadership in achondroplasia, we've been pleased with the number of new patient starts, especially for the youngest patients who can benefit most from the longer Voxogo treatment.

Kristen Hubbard: Supportive of this benefit, we expect that new global guidelines will soon be published based on early real-world experience in clinical practice that recommends children with achondroplasia be referred as soon as diagnosis is suspected to enable early initiation of treatment.

Kristen Hubbard: These guidelines reinforce our belief that beginning treatment soon after diagnosis provides the optimal outcome for children with achondroplasia, including for benefits beyond height and the comorbidities associated with the condition.

Kristen Hubbard: Now since Foxogo's approval in 2021, we've been extremely gratified to be able to provide families with the only approved therapy that treats the underlying cause of achondroplasia.

Kristen Hubbard: Through our ongoing global expansion, supported by a growing dossier of supportive safety and efficacy data, we aspire to reach every family interested in Vox Zoba therapy.

Kristen Hubbard: As we expand our global leadership in achondroplasia, we're pleased with the momentum behind Voxodo Awareness.

Kristen Hubbard: We have continued to add significant levels of new commercial patients, with over 3,800 children being treated with VoxZova worldwide at the end of the third quarter of 2024, compared to over 2,300 children as of the same period last year.

Kristen Hubbard: This represents approximately 65% growth year-over-year.

Kristen Hubbard: Our global commercial operations are yielding significant new patient starts, including in Europe, the Middle East, and Latin America.

Kristen Hubbard: We're committed to leveraging our global commercial capabilities to expand VoxOvo into more than 20 additional markets by 2027.

Kristen Hubbard: We estimate that approximately 90% of the global achondroplasia total addressable patient population is outside of the U.S., which, considering BioMarin's ability to leverage its established global commercial footprint, is a significant competitive advantage.

Kristen Hubbard: Moving to dynamics in the United States, we are seeing robust patient growth across all ages, with the majority of new patients starts during the quarter from children under five years old.

Kristen Hubbard: The expanded VOXOGO age label from infancy, approved late last year in the U.S., continues to be a major driver of new patient starts.

Kristen Hubbard: And finally on VoxOgo, consistent with BioMarin's prior product launches, VoxOgo has now reached a level of global commercialization where it's appropriate to focus on revenue rather than patient numbers as the key metric of growth.

Kristen Hubbard: As we launch into dozens of countries across multiple continents, patient-level data by country becomes unreliable or even unavailable. So as a result, with Voxoga reaching its third full year of its global launch, this is the last quarter in which we will share quarterly patient growth. We will move forward using revenues as the key performance indicator.

Kristen Hubbard: Now turning to our enzyme therapies, 27% revenue growth during the quarter was driven by robust demand across the portfolio.

Kristen Hubbard: While Q3 benefited from order timing dynamics, particularly for Alderazyme, we also saw strong patient uptake across the brands that Biomarin commercializes, especially in Palantzig.

Kristen Hubbard: New patient additions in the U.S. and a strong ongoing launch in Japan drove 15% year-over-year revenue growth in Palanzee during the quarter.

Kristen Hubbard: Looking forward, we expect continuing growth of the enzyme therapy portfolio as we implement new initiatives over the coming quarters outlined in Investor Day.

Kristen Hubbard: A few examples include focused investments in high-yield patient finding initiatives, selective geographic expansion, and patient support programs enabling long-term adherence to therapies.

Kristen Hubbard: We believe these additional initiatives will drive long-term compound annual growth rate in the high single digits for the enzyme therapies.

Kristen Hubbard: In our Octavian business, we generated $7 million in revenue during the quarter, supported by three new patient starts in Italy and three in the U.S.

Kristen Hubbard: Recall that Roktavian price is weight-based, so revenue per patient can fluctuate accordingly.

Kristen Hubbard: So to add color to the three countries we are focused on, let's start with the U.S.

Kristen Hubbard: The U.S. sites that have dosed patients have increased confidence in their ability to successfully negotiate single case agreements and reimbursement.

Kristen Hubbard: In Italy, patients have been treated across multiple regions thus far and going forward, as new sites are added across the country, we expect to see increased patient uptake.

Kristen Hubbard: And in Germany, we continue to work with additional sub-insurers to facilitate access for patients, and we will provide further updates when we have progress to share.

Kristen Hubbard: So, in conclusion, our strong commercial performance in the third quarter, combined with the promising outlook for the remainder of 2024, has enabled us to raise our full-year revenue guidance.

Kristen Hubbard: We are excited about the opportunities ahead and are confident in our ability to deliver exceptional value to our patients and stakeholders around the world.

Speaker Change: Thank you so much for your attention. I'll now turn the call over to Greg to provide an R&D update. Greg?

Greg Freyberg: Thank you, Kristen. I'm happy to speak with you on my first quarterly results call since joining the company in September.

Greg Freyberg: Since joining as the lead for World Wide R&D, I've been energized by the team's commitment to innovation and their emphasis on execution.

Greg Freyberg: I look forward to leading this organization as we target 11 high-impact launches by 2034. These include two by 2027, those being Voxogo for hypochondriplasia and Palanzec for adolescence.

Greg Freyberg: A few activities to highlight from the quarter. I'm pleased to announce that our pipeline is progressing well, including our canopy clinical program, evaluating the expanded potential of oxogo in children with hypochondroplasia and with other genetic skeletal conditions.

Greg Freyberg: For hypochondroplasia, we completed the enrollment target for the Phase III-eligible patients in our observational study. After six months of observation, we anticipate that these very patients will feed directly into our Phase III Voxogo interventional study.

Greg Freyberg: to complete its enrollment in the first half of 2025.

Greg Freyberg: We can therefore reaffirm our expectation for pivotal data for the hypochondroplasia phase 3 program in 2026 and an approval in 2027 if those data are supportive.

Greg Freyberg: are Phase 2 canopy studies.

Greg Freyberg: including one in idiopathic short stature and the second basket study in Noonan syndrome, Turner syndrome, and Shocks deficiency. Each remain on track.

Greg Freyberg: We're looking forward to advancing these programs, and we expect Phase 2 data readouts in 2026.

Greg Freyberg: At the 16th Annual International Skeletal Dysplasia Society meeting in September, BioMarin and our external research partners contributed to eight presentations, including four orals, discussing the value of Voxogo and the burden of illness in achondroplasia and related disorders.

Greg Freyberg: These data included showing that children with achondroplasia treated with Voxoga experienced meaningful improvements beyond height, such as gains in health-related quality of life, and maintained bone strength while increasing bone length.

Greg Freyberg: Researchers also presented encouraging data from ongoing investigator-led studies in children with other genetic skeletal conditions.

Greg Freyberg: These included hypochondroplasia, Noonan syndrome, and with genetic variants often associated with idiopathic short stature, such as aggrecan deficiency and heterozygous NPR2 mutations.

Greg Freyberg: Our Phase 3 program with Palanzec and adolescents age 12 to 17 has completed enrollment and is expected to read out in the first half of 2025. This will allow for a potential supplemental NDA filing mid-year 2025.

Greg Freyberg: We believe this supplement for adolescents could offer patients and their families the opportunity to address the burdens of fetal ketonuria, as well as the dietary restrictions early on, and thus facilitate an easier transition to independent adult living.

Greg Freyberg: With our earlier stage pipeline, I am pleased to share that BMN351 for Duchenne's muscular dystrophy, a potential best-in-class therapy, has completed enrollment of our first cohort, six patients, with initial proof-of-concept data expected internally in quarter two of 2025. This will be in the form of muscle dystrophin levels after 25 weeks of dosing.

Greg Freyberg: with BMN 349, an oral therapeutic for alpha-1 antitrypsin deficiency associated liver disease. We have completed the single ascending dose study in healthy volunteers and expect to begin the multiple ascending dose study by the end of the year.

Greg Freyberg: Finally, I'm happy to share that Biomarin's long-acting C-type natriuretic peptide BMN333 remains on track for initiation of the first in human study in early 2025.

Greg Freyberg: I look forward to future opportunities to communicate our progress across the R&D organization over the coming quarters, and to personally meeting many of you at an upcoming investor event.

Greg Freyberg: Thank you for your continued support, and we will now open the call to your questions. Operator?

Speaker Change: Thank you. At this time I would like to remind everyone in order to ask a question press star and the number one on your telephone keypad.

Speaker Change: And we will take our first question from Phil Nadeau with T.D. Cohen. Your line is open.

Phil Nadeau: Good afternoon, thanks for taking our questions.

Phil Nadeau: just two for us, one on VoxOgo, one on business development. On VoxOgo, you noted that patient starts were primarily among the patients under the age of five here in the U.S. What does that say about the maturity of the launch in the U.S.? Are we approaching a period where new patient starts will largely be an incident population, or is there still a lot of the prevalent population out there that's untreated in the U.S. and could go on therapy?

Speaker Change: The second on business development, James Welcome, congrats on the new position and great article on BioSentry over the weekend. Curious, based on the presentation you noted, you wanted to target deals less than $1.5 billion. What phase of development would be ideal for BioMarin at this stage in the pipeline's maturity? Thanks.

Speaker Change: Yeah, I'll take the first question. Thanks so much for it. So with regard to VoxOgo growth well into our third year here of the launch, you mentioned the the call-out on the age of patients being treated. So that is true that with the label expansion that we had in the U.S. only last year, we are seeing the majority of new patient starts in the zero to five year old age group.

Speaker Change: So that is more for the maturity of when that label was expanded in the U.S.

Speaker Change: In other markets where we have really high penetration and some of our early strategic markets where we have patient level data, we're seeing that many of the new starts are in the zero to two population. So to your point, that's where we really do start to see that incident market be created where we have higher penetration.

Speaker Change: Thank you very much.

Alexander Hardy: Hi Phil, this is Alexander. I'll take the question on business development. James is not on the Q&A today.

Alexander Hardy: With regard to business development, we will.

Alexander Hardy: be focusing, as you say, on transactions less than 1.5 billion in...

Alexander Hardy: transaction size.

Alexander Hardy: we'll have two areas of focus that James will be prioritizing. One is really leveraging our scientific right to win our development.

Alexander Hardy: Regulatory Manufacturing and Commercial Footprint

Alexander Hardy: across the globe in approximately 80 countries to identify potential deals. He's also working with research in terms of identifying earlier transactions in our pipeline.

Alexander Hardy: to bolster our early research efforts and pipeline. So those are the two focuses that James has. He's just into his first, ending his first month in the role and you'll hear more from him in the future. Perfect, thanks for taking our questions.

Speaker Change: Your next question comes from the line of Salveen Richter with Goldman Sachs. Your line is open.

Salveen Richter: Good afternoon. Thanks for taking our questions. Could you just walk through the development strategy here for BMN333 and how quickly you can move into other indications beyond achondroplasia with the long-acting CNP? And then separately, with regard to your...

Salveen Richter: IP strategy here and in the context of Ascendus and the long-acting CNP, just help us understand you know where you stand on your IP and just the strategy on the forward here with regard to your franchise.

Speaker Change: Thanks for watching!

Speaker Change: This is Greg Freiburg. I'll take the first question. With regard to BMN333, of course, we're quite excited to be able to bring that into patients, and we should be entering the clinic early next year. The strategy behind the molecule, of course, is to elongate the half-life, and we're

Speaker Change: I'm confident that the linker technology and the binding technology, again, are tried and true, and we'll be able to do that.

Speaker Change: What that can afford, of course, is one of two things. The less exciting would be less frequent administration. But the more exciting aspect is the one I just want to focus on for a moment, which is if you can change the PK profile of this near-native CNP.

Speaker Change: and you can increase the delivery of that.

Speaker Change: that hormone into the body in a safe way.

Speaker Change: then we could think about actually increasing not only the pharmacokinetic exposure to CNP, but also the pharmacodynamic effect in our preclinical models.

Speaker Change: would suggest that there is more to be gained there with regard to activity and bone lengthening.

Speaker Change: The first set of data is going to give us a lot of importance.

Speaker Change: clues as to whether or not the PK profile is playing out the way that we had anticipated. That's data that we should have in-house next year. The Healthy Volunteer Study is going to give us a good read on that PK profile. Depending upon the results there, we could go in a variety of directions.

Speaker Change: We do believe, as you point out nicely, that this is a pathway that should be able to address

Speaker Change: genetic skeletal disorders beyond simply achondroplasia and hypochondroplasia, and the teams are working very diligently to come up with scenarios.

Speaker Change: if that profile plays out the way that we believe it may to accelerate that approval. More to come there, but I think you've highlighted very nicely that there are opportunities here to accelerate the program and we'll be looking for those actively.

Speaker Change: And Salveen, with regard to your question around IP, I'm not going to elaborate further on IP beyond what we said on it yesterday. Simply put, we're confident in the intellectual property we have and we plan to vigorously defend our intellectual property.

Traci Mccarty: And your next question comes to the line of Jessica Fye with JPMorgan. Your line is open.

Jessica Fye: Hey guys, good afternoon. Thanks for taking my questions. First on the P&L, COGS looks elevated this quarter. The press release makes mention of the impact of Roctavian inventory reserves.

Jessica Fye: How big was that and would gross margin have been in line with

Jessica Fye: more recent quarters without that effect. And then maybe following up on Sylvain's question for 333, I think you've talked about single ascending dose data in 2025. Is it possible we could get multiple dose data next year as well? Or if not, should we think of that in 26? Thank you.

Brian Mueller: Hi Jeff, this is Brian. I'll take the first question on COGS and margin. Good pickup, cost of goods sold here in Q3-24 at about 25% of revenue and a 75% gross margin.

Brian Mueller: That is a click down from where we've been trending close to 80 percent two things going on there this quarter first is that

Brian Mueller: You know, uh...

Brian Mueller: bolus of Al Juris I'm revenue that we had that we commented on in the prepared remarks so

Brian Mueller: Alderzheim Revenue. Again, Santa Fe is responsible for marketing to third parties.

Brian Mueller: We recognize revenue when we supply bulk inventory to Santa Fe as delivered and control is transferred. So, had a number of lots delivered in Q3, and because we're recognizing

Brian Mueller: roughly half of the Alderazyme revenue according to the the revenue split with Sanofi. That means that Alderazyme is our lowest margin product, you know, roughly half or less of the gross margin we earn across the rest of the portfolio. So, you know,

Brian Mueller: high rate of alderozyme revenue, lower gross margin, that's going to be dilutive to margins in the quarter. And then secondly, we did have some inventory reserves here in Q3, mostly related to some gene therapy raw materials that, because we paused Roctavian manufacturing, we took a reserve on before they expired.

Brian Mueller: and nothing else going on those are just those those two items account for roughly 5% which would clip put us close to the 80 79 80 percent that we've been tracking to this year

Speaker Change: Thanks. Thanks, Jessica, and this is great for our viewers.

Speaker Change: No, roughly half and half between those two. Okay. Thank you.

Speaker Change: Thanks.

Speaker Change: and Jessica. With regard to BMN 333, the single ascending dose study of course is in Healthy Volunteers. Right now what we're committing to in 2025 is that that is the PK data that we'll have in-house. Of course the data we are very eager to gather would be in patients and would be to see dose ranging and multiple ascending doses. As of right now what we're committed to publicly is the 2025 data point and we'll keep you updated as we move along.

Speaker Change: Thank you.

Speaker Change: Your next question comes from the line of Paul Mateus with Stifle. Your line is open.

Paul Mateus: Thanks very much for taking my question. As it relates to your reiteration of guidance, I think the inference that many analysts and investors have made is that if you're willing to or I guess confident in reiterating your guidance in the face of

Paul Mateus: and maybe some surprising increased competition in achondroplasia. You must have thought originally that your guidance issued at the Investor Day was fairly conservative. Is that the right inference? And if so, on what points did you feel like your initial guidance was conservative? Or on what points do you feel like the street is missing the big picture in your long-term vision around the size of these markets and your penetration? Thank you.

Paul Mateus: Thanks, Paul. This is Brian. I'll start and then I'll see if Alexander has anything more to add.

Paul Mateus: we would not describe the long-term guidance as conservative.

Paul Mateus: What we have said is that when the new competitor data showed up, which was different than what most of us had seen, you know, leading up to that announcement, we took that additional data, went back and worked it through our models.

Paul Mateus: We shared on Investor Day that our long-term revenue guidance did already have a competition assumption. We didn't go into detail, but fair to say it was based on data available to the world at the time, which was not that most recent data.

Paul Mateus: And so when we went back and reworked our assumptions around, you know, both competitive share as well as, frankly, other levers available to us across the portfolio and within BoxOgo,

Paul Mateus: We were able to absorb and confidently reaffirm the long-term guidance. So, again, we would not call it conservative, but we are confident in where we landed after that follow-on analysis.

Speaker Change: And what I would add, Paul, is that if you go for the period into the longer term, beyond

Speaker Change: at the 27th, we're talking about and we're reaffirming our targeting in teens.

Speaker Change: Kader.

Speaker Change: and the five billion potential for skeletal conditions, you know, obviously that very much factors in also, you know, the tap in these further indications beyond achondroplasia. And just to reiterate, you know, we've assumed very modest penetration levels.

Speaker Change: 10.

Speaker Change: forecasts that underpin that outlook. And, you know, additional competitors, should they declare a development plan in those indications, you know, they will be contributing to the growth of those markets, which we would share with them.

Speaker Change: in some proportion. And then the last thing I would say is of course all of this excludes intellectual property and our confidence in our intellectual property position, which I've already talked to.

Speaker Change: All right.

Traci Mccarty: Your next question comes from the line of Ellie Merrill with UBS. Your line is open.

Ellie Merrill: Hey guys, thanks for taking the question. Can you give us a bit more color on what's driving the increase in the ERT business growth? I think it's your confidence that this can continue to grow in the high single digits for years to come. I think you mentioned some initiatives you're working on, if you can maybe elaborate a bit more on what these entail. Thanks.

Kristen Hubbard: Hi Ellie, thanks for the questions Kristen. So yes, great question on our ERT business. So as we had said at Investor Day and Stand Firm Behind, we anticipate high single-digit growth.

Speaker Change: for the years to come in that business. And primarily driven right now, there's a lot coming from the Palanzec markets, or the business there that you saw in the numbers. We have 15% growth in Palanzec, and a lot of that is being driven primarily in the U.S. market.

Speaker Change: where we are seeing really great uptake not only for new patient starts.

Speaker Change: that see the highly beneficial impact that Palin-Z can have, both from an efficacy in lowering feed levels, as well as a liberalization on their diet, so they don't need to do medical nutrition. That's driving a lot of new patient starts, but we're also focused on picking up on any patients who have discontinued therapy. And so we're seeing really great uptake there.

Speaker Change: So that's the Palanzec business, but on the MPS-CLM-2 areas and our global footprint of over 80 countries.

Speaker Change: for approximately 80 countries.

Speaker Change: What we're seeing there is that we're putting some new initiatives into place which, as you had alluded to, I mentioned at Investor Day. And what those are, are looking at high-yield diagnostic efforts in markets where we think we can really move the needle, as well as some adherence programs in a few select markets, and expansion into new geographies where we think that the access marketplace is in the right place for us to move forward.

Speaker Change: Those are the initiatives that we've mentioned we're going to start, but because of the nascent stage of those initiatives, we don't have an update to give on those yet, but we'll commit to further showing the progress that we're making in those. I've seen early data that are looking very good, but we will be updating you on those in the future.

Speaker Change: Not to mention, as we've talked a lot about, we are moving as well and transforming the business into business unit models, which we believe that that alone will really help us to drive the urgency, the accountability, and the real clarity of where we can move our resources in a quick way.

Speaker Change: So we'll be reporting on that in the earnings calls and investor days to come.

Speaker Change: right

Traci Mccarty: Your next question comes from the line of Corey Kasimov with Evercore. Your line is open.

Corey Kasimov: Hey, good afternoon guys. Thanks for taking my questions. Just two for me as well. So for Voxogo, can you comment as to how many patients from your original Pivotal Phase III trial have now reached their full adult height? And then just a clarification on the DMD program. You noted that you're gonna have the data in-house in 2Q25. Is that going to be publicly shared at that time as well? I just wasn't sure when you said it was gonna be internal. Thank you.

Corey Kasimov: Thanks, Corey. This is Greg. For the first question, we are tracking those patients from the Phase 3 program very vigorously. We're not going to release numbers with regard to how many patients have reached final adult height.

Corey Kasimov: Clearly, that is an endpoint that regulators are very interested in and that is part of our commitment for post-marketing requirements to provide that data at a reasonable time.

Corey Kasimov: Part of the challenge of course is that these patients reach that final adult height at different timing based on what age they were when they entered the study. But suffice to say we're of course following that very closely and continue to make progress year to year.

Corey Kasimov: With regard to the Deschanes muscular dystrophy program, yes, we're very excited that we will have

Corey Kasimov: muscle dystrophin data.

Corey Kasimov: from the first six patients in-house.

Corey Kasimov: mid-year. We'll be looking at that and just to take a step back for those who aren't quite as familiar, this is a molecule that's engineered to increase dystrophin levels in the muscle to levels that

Corey Kasimov: are dramatically higher than what we've seen with other molecules in the space. And there's a variety of engineering levers that were pulled to try to accomplish that, but the most important of which is addressing a novel binding domain.

Corey Kasimov: in the nucleic acid that, at least in the preclinical models, results in 30, 40, 50%.

Corey Kasimov: dystrophin levels in the rodent models at a much higher rate than what we've seen with

Corey Kasimov: with other molecules that we both synthesized and those that are available. So that data from the first cohort is gonna give us a line of sight on whether or not we can hit our target. Our target is a 10% level or higher at steady state.

Corey Kasimov: in the muscle of these boys who are on the study. Now, it's important to remember that steady state

Corey Kasimov: actually is not at 25 weeks. It may be later than that.

Corey Kasimov: But we've done some modeling, and I think...

Corey Kasimov: with our 13-week data and our 26-week biopsies.

Corey Kasimov: will have a good read by mid-year where we're headed. Depending on what that data is, it may be worthy of release, it may be giving us information that also tells us that we need to adjust the dose, but we will certainly be revealing that data when we feel that it's of a sufficient quality and volume that it would be meaningful to release.

Corey Kasimov: Beyond that, not much more to add at this point.

Speaker Change: Okay, that's very helpful, Greg. I really appreciate it.

Speaker Change: Your next question comes from the line of Kostas Bilyoris with BMO Capital Market. Your line is open.

Kostas Bilyoris: Thank you.

Kostas Bilyoris: Hello everyone, congrats on the quarter and thanks for taking our questions. Two quick from us, one on Voxogo and one on Palinzi.

Kostas Bilyoris: On Voxogo, one follow-up from a question before. Can you remind us what is the status of the transition to full approval from accelerated approval and what this may mean for the regulatory pathway of competitors that are behind you in development? And the second question on Palindzic is any thoughts around the competitive positioning, given that some oral agents may enter the market in 2025? Thank you.

Kostas Bilyoris: Thank you, Costas. This is Greg again. Why don't I take a stab at those two?

Kostas Bilyoris: With respect to Voxogo, we are committed to measuring final adult height.

Kostas Bilyoris: on our patients who were on our clinical study, our pivotal study, and we have that agreement with regulatory authorities. Beyond saying that we're vigorously collecting that data and looking forward to reaching the critical mass that the regulators are interested in, I don't have much else to add. Of course, regulators, with regard to full and accelerated approval, they always have their ability to use their judgment to decide when new agents come along, so we're very much focused on our own agent and being able to convert that to full approval for the good of, of course, the patients who are treated with it.

Kostas Bilyoris: With regard to Palin Zeke

Speaker Change: The PKU space is one that Biomarin has been in a fortunate position to be able to serve these patients for almost 20 years now, and in that time we've learned quite a bit about the space, about the patients.

Speaker Change: about the treating physicians and about what the needs of this group are.

Speaker Change: The big picture here is that these are a very heterogeneous group of patients.

Speaker Change: They have different ages. They have different needs. We know from the clinical studies that they respond differently to the therapies that are available.

Speaker Change: And because of that, we see the addition of new agents to this field actually as being good for patients. It'll give physicians more arrows in their quiver. Not every patient is created equal.

Speaker Change: a Palin-Zeek patient, for example. These are patients in our pivotal study that had fee levels that were over 1,000, for example. And this is a molecule that if you look at the...

Speaker Change: the treatment guidelines that are out there.

Speaker Change: the Palin-Zeek patient, the goal of that patient is to be able to reduce the fee levels as low as possible and you know I believe the quote in many of the guidelines is with a normalized diet. That is a very different patient than perhaps the BH4 analogs may be able to address.

Speaker Change: That being said, it's a good thing for patients that they have multiple different options, and we're anticipating that that these are the kind of

Speaker Change: advances that that patients need. In fact, we're developing a next-generation Palanzec because we believe that we can help, you know, build upon that profile as well. So, you know, from that standpoint, I think the only other objective data that's out there, there was an FDA ruling recently. One of the oral agents that was filed was given a standard review. You know, by definition, you know, the FDA has

Speaker Change: you know, they use two criteria when they define that. One is that, is this a serious illness? We believe that PKU is a serious illness by their definitions. The other criteria they use is

Speaker Change: did this new applicant meet the criteria for a significant improvement in efficacy and safety compared to available therapies? They obviously chose to give a standard review. So I think from an objective standpoint, that speaks for itself.

Speaker Change: Thank you.

Speaker Change: Your next question comes from the line of Joe Schwartz. Your line is open.

Joe Schwartz: Great, thanks very much. I have a question on 333 and also 390. So at Investor Day, I think you projected that 333 could come to market in 2030.

Joe Schwartz: But since your C&P patent expires in that year, that doesn't seem to give you much time to convert people to your own long acting before generics arrive. And then you'll probably also have a couple of branded competitors by then. So I'm just wondering if you can talk about what the practical benefit of 3-3-3 is expected to be on the business.

Joe Schwartz: And then likewise, you noted there that your next-gen Palin-Zick with a novel PEG could expand the PKU market a lot, but the development path you laid out suggested it would take about 10 years to get it to market. I know PEG-PAL took that long, but...

Joe Schwartz: I'm just wondering why should it take that long for BNN 390? Thanks.

Speaker Change: Thanks, Joe.

Speaker Change: I'm going to hand it off to

Speaker Change: Alexander first, and then I'll dive in. Yeah, thanks very much, Joe. Just wanted to start off with the first part of your question with regard to 333 and the rationale, given the patent on VoxOgo. Actually, we estimate our patents, our IP, will last to the mid-2030s.

Speaker Change: therefore giving us plenty of time after the the launch of 333 to convert the market over. Again, as you well know, Joe, you know, we're at the target product profile here is for a product that's more efficacious.

Speaker Change: as well as offering the convenience of weekly dosing. We think that is a sort of proposition that's required to move patients off FOXOGO. The hierarchy of needs of both physicians and caregivers is first and foremost efficacy.

Speaker Change: safety and then only those if those are improved is convenience seem to be an important. So we're very focused on that and if we're successful with that product profile with this time frame we believe we can convert our business from Boxergo. And now I'll hand it over to Greg for the rest of your

Speaker Change: question.

Greg Freyberg: Thanks, Alexander. With regard to BMN 390...

Greg: I would just comment that, you know, we're very early in its development. We're pleased that we'll be able to file an IND targeting next year. The dates that you laid out, indeed, represent similar development timelines to the predecessor molecules. I would think of those as the upper error bar. We're going to be working very diligently, not only as the profile reveals itself, but also, you know, as we learn more in the marketplace of developing Palanzec as well, to look for opportunities to accelerate that development.

Traci Mccarty: Your next question comes from the line of Akash Tewari with Jeffries. Your line is open.

Akash Tewari: Hey, thanks so much. Can you talk about the dynamics between total patient ads and incremental res for VoxOgo? You had 300 patient ads from Q2 to Q3. You had a mod at 7 million.

Akash Tewari: increase in revs. I know there was a $20 million stocking benefit, but number one,

Speaker Change: Is 300 patient ads a reasonable assumption going forward for VoxOvo growth in ACON? And then number two, how should we think about reps per patient evolving as you expand into international populations? And then just on ascendance and IP, how aggressive is your team willing to be here? You know, should we be thinking about some type of royalty payment or is the the angle Biomarin is going to pursue?

Speaker Change: It's going to be a preliminary injunction and you're aiming to stop attendance from launching the product altogether How should we think about the the how aggressive you'll be on IP? Thank you

Speaker Change: Yeah, thanks so much. I'll start with your first question, Akash, about the incremental patient ads and really the question about Voxelgo growth. So, you know, we have about 3,800 infants and children as of Q3, which is a pretty big jump versus last year in this period, which is 2,300 patients.

Speaker Change: That's 54% growth in revenues and 65% in patient ads.

Speaker Change: Now, interestingly, I think you asked the question, kind of, is that, does that seem like the right number? Is that strong growth? And I will say that we are tracking exactly the plan on where we think we should be in terms of the growth of this product well into our year of launch. Now, what I think is important is to reiterate where we think we're going from here, and that is that we had said it investor day and still stand strong behind our projected CAGR of 25% growth for VoxOgo through 2027.

Speaker Change: And really the reason to believe and the reason that we're confident in the ability to deliver on that, which we do think is strong growth for a

Speaker Change: for a product at this stage in its life cycle is really in and around the totality of the data that we have in the 6,000-plus patient years.

Speaker Change: of efficacy and safety and the notion that we have a really broad age label which as we're seeing in new patient starts this is really important because we know that treating early is absolutely critical.

Speaker Change: It's also in our ability to be in a very broad geographical reach. We talk about our 80 countries that we're in, we've already started with Voxelgoin-44 and we intend to expand to another 20 or greater countries by 2027. And this really is basing it on our expertise in our commercialization, our medical and our market access capabilities.

Speaker Change: And we're seeing, you know, we're seeing that we're able to grow this expansion at a pace that we feel is very, not only strong, but that we can continue in the years to come. So, just want to reiterate that strong character we see through 2027.

Speaker Change: Cash, just on your question on IP, I'm afraid I'm not going to elaborate further on our IP strategy or how this might play out. We'll keep you informed as and when we take action, but that is all we can share at this point.

Speaker Change: Thank you very much.

Speaker Change: Your next question comes from the line of Chris Raymond with Piper Sandler. Your line is open.

Chris Raymond: Hey, thanks for taking the question. Just on the clinical premise behind BMM 333, I hear you guys talk about improved PK and PD with a long-acting C&P that could drive better, you know, growth metrics.

Chris Raymond: But I get this question from investors, maybe walk through the differences between 333 and Transcon, CNP, and why, you know, if we haven't seen, you know, a discernible growth benefit from that asset,

Chris Raymond: Why mechanistically would 333 be different? Thanks.

Chris Raymond: Thanks, Chris. This is Greg. Let me take a stab at that. First and foremost, we are targeting a free CNP level that is higher than not only what VoxOgo can deliver from a time above threshold AUC standpoint, but higher than published data for agents external to Biomarin as well.

Chris Raymond: So, in the preclinical models, that suggests if you can increase that 50-100% that there are discernible changes in AGV in those animal models.

Chris Raymond: The premise, of course, assumes that that can be done safely. Again, our preclinical package suggests that that is the case. But then that is an experiment that we do need to test in the clinic with those ranging studies in patients.

Speaker Change: The totality of evidence here is going to be something that absolutely is key. We still believe that Voxogo is, you know, an agent that, you know, not only is first-in-class, but has the best-in-class evidence package. There's a variety of reasons why we believe that. It's beyond just the 6,000 patients of data. It also really touches on the durability of data. It's not just single-year AGV or proportionality. We see, you know, out to seven years of consistent data there.

Speaker Change: By building on that data, both with regard to growth velocity, as well as the basket of data, which is evidence beyond just growth, the wellness and health of these patients.

Speaker Change: We think that if we can take that molecule and increase the exposure, if we can do it in a safe way, that that will translate not just into increased growth velocity, but also we're going to start seeing benefits on the health and wellness of patients.

Speaker Change: and of course there's a variety of those measures that we will continue to publish on not only for Voxogo but also for the new agent.

Speaker Change: Thank you.

Speaker Change: Your next question comes from the line of Mohit Bansal with Wells Fargo. Your line is open.

Mohit Bansal: Great, thank you very much for taking my questions. Two, if I may ask...

Mohit Bansal: So, one is, what is your base case assumption around the length of trial for BMN-333 at this point? Is it a two-year control trial or a one-year control trial?

Mohit Bansal: asking because if there's any room to expedite it and then maybe launching before 2030 here. Number two, it does seem like Naglazam has done really well compared to what we have seen in prior years, year-over-year basis. So can you talk a little bit about the strategy and what has helped here, and how should we think about the product going forward? Thank you.

Speaker Change: Thanks Mohit for the question. For the phase 3 study, the development plan for BMN 333, of course there's two variables in play here. Any study, of course, needs to recruit, you know, the set of patients.

Speaker Change: and the effect size.

Speaker Change: that we would be targeting would absolutely drive not only the number of patients we need, but the amount of time it would take to recruit those patients. The profile is going to, again, reveal itself incrementally. I think the PK will be the first step. But if we believe that this is a molecule that can deliver

Speaker Change: much higher CNP exposure in the patients, and if we believe that that translates into effect sizes, that would indeed change the endpoints that we'd look for in a phase 3.

Speaker Change: There are far too many permutations to be able to go into additional details at this point of what the phase three study might look like or might not look like. But inherent in your question is, can this be accelerated?

Speaker Change: I believe there are scenarios where, if the profile plays out the way that we're anticipating, that we will have the chance to try to accelerate those timeframes.

Speaker Change: No promises today but it's something we're actively looking at and urgency to get to patients is one of our absolute core values here at Biomarin and we understand that if we have a molecule that's delivering something more valuable than even our own product we would want to bring that as quickly as we can.

Speaker Change: Thanks, Mohit. This is Brian. I'll take the Naglezyme question. I appreciate the question. Naglezyme did have a great quarter. We did include in the prepared remarks, just to be clear, that Naglezyme did benefit from some timing, that 21% growth in the quarter did have some timing in there, but nothing where we would isolate a single country order like we have in the past.

Speaker Change: I think what you're seeing with Naglazyme over time on the market for nearly 20 years is just the good case example of how we talk about the durability of this enzyme therapy portfolio and the durable growth potential. We'll see you in the next video.

Speaker Change: It's the epitome of this franchise and how we see it growing going forward.

Speaker Change: But I'd also point to, when we talk about some of the new initiatives and the promise of this business unit structure that Kristen touched on earlier, those aren't yet implemented. Those are all incremental for the future. That's what drives us to believe we can be more aspirational with the long-term enzyme therapy growth rate.

Speaker Change: Thank you.

Speaker Change: And we have reached the end of our call and this will be our last question from the line of Jenna Wang with Barclays

Jenna Wang: Thank you for squeezing me, Inc.

Jenna Wang: So, I would just ask one question regarding the BNIM 333.

Jenna Wang: Greg, you did mention that you wanted to have a higher, better efficacy, hopefully with a better exposure, but based on the Voxogo dose escalation study, there was seems there was a ceiling effect regarding the efficacy, and so what will make you confident, you know, that could be with a higher dose or higher exposure that can translate to better efficacy?

Jenna Wang: And then related questions, I think, based on the investor day, that one slide shared with investors, we did see, like, the exposure of plasma concentration almost in the one or two log or three log magnitude improvement compared to, say, Voxogl or even transconscience.

Jenna Wang: and wouldn't that, you know, frequency of dosing, you know, would that be also a very important component that you were taking into consideration?

Speaker Change: Thanks, Jenna.

Speaker Change: Your questions with regard, why don't I take first the Investor Day figure.

Speaker Change: With a molecule like BMN-333, there are two species, of course, that we need to be talking about when we look at PK. One will be the native molecule that will include the linker technology, the binding technology, and so forth. And I believe that's what was shown in yesterday, a much higher molecular weight. So again, if you're comparing the units, they will look somewhat differently.

Speaker Change: From a pull-through, what we of course care about is the liberated, the free CNP.

Speaker Change: and we, of course, from our preclinical data and in humans, will be measuring very closely.

Speaker Change: that profile of free CNP. And we do believe that we can safely, and in a way that has a different shape to the PK curve, so it would rather than be a sawtooth pattern of a shorter half-life molecule, would be able to increase AUC, again, increase time above threshold.

Speaker Change: We believe not only can we increase the exposure of CNP there But do so in a way that we could see physiologic differences as compared to Voxogo in the way that it works

Speaker Change: I think of this similar to other peptides, where insulin, for example, is one that when its given in its native form versus in a variety of constructs, you can see different biologic effects from the shapes of the curve that are more than just described by, say, Cmax and the F2.

Speaker Change: or AUC alone. And so we're absolutely excited to test this in patients. We're going into the clinic in the first...

Speaker Change: very early next year, and we should have a good read on both the native molecule, BMN333 pharmacokinetics, as well as free CNP in rapid order so that we'll have that data in-house mid-year.

Speaker Change: And at this time, I will turn the call back over to CEO Alexander Hardy for closing remarks.

Alexander Hardy: Thank you, operator. And thank you all for joining us today. As you've heard, we have the team in place this quarter to now to deliver on our new corporate strategy that you heard about at Investor Day, focused on innovation, growth, and value commitment.

Alexander Hardy: and we've made significant progress on each of these.

Alexander Hardy: Today's strong quarterly performance highlights

Alexander Hardy: the ongoing implementation of this strategy under Biomarine's new operational plan to achieve sustained and significant returns for the benefit of all stakeholders. We're looking forward to keeping you apprised as we continue to execute on these plans. Thank you very much for your attention and good day.

Speaker Change: This does conclude today's conference call. You may now disconnect.

Speaker Change: Please wait. The conference will begin shortly. Please wait. The conference will begin shortly.

Speaker Change: Thanks for watching!