BioMarin Pharmaceutical Inc at Jefferies London Healthcare Conference Summary

The following is excerpted from the question-and-answer section of the transcript.

Question: Akash Tewari - Jefferies - Analyst : Understood. I'm actually going to start with a part of the business that I don't think gets talked about enough, but I've noticed, especially in the back half of this year, you're talking about it more, which is high single-digit growth for your ERT franchise. And I think as for a lot of investors, they're kind of skeptical, they're like, look, we all know this business, it's steadily growing and now suddenly BioMarin is talking about accelerating some of those products. Talk to me about what geographies you're really focused on in terms of accelerating growth for your ERT franchise? And are there specific products that you would call out where you feel like there's real room for upside and acceleration that maybe investors aren't seeing right now? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 19, 2024 / 9:00AM, BMRN.OQ - BioMarin Pharmaceutical Inc at Jefferies London Healthcare Conference

Question: Akash Tewari - Jefferies - Analyst : Understood. Now, maybe going -- I remember when you've kind of introduced yourself to The Street at JPMorgan last year, you were talking about, yes, we're geographically really diversified, but I want to have more of a US-centric focus. And since you've rightsized things from a cost perspective, you've gotten your team in place, Again, I feel like a lot of the questions from investors is like, well, what's happened in the past? How do things change by just changing people? How do you accelerate your US launch trajectories and specifically on VOXZOGO, where maybe the demand there hasn't been as strong as maybe your team had internally hoped for?

Question: Akash Tewari - Jefferies - Analyst : Right. Understood. Now, I think also you're going to have competition that comes out for VOXZOGO. I feel like the goal for your team is, hey, for our most important geographies, we want to make sure we're going from a prevalent to an incident population, right? So as you think about your top territories and then your kind of commercial goals over the next three years, how confident are you that, let's say, developed Europe and US will by the time you get competition, be largely an incident market and not a prevalent market.

Question: Akash Tewari - Jefferies - Analyst : Understood. Now, just thinking about competition and if it enters, the way we kind of think about it, if you're well controlled. I mean, like if compliance was an issue, you would have seen compliance be an issue of 95% compliance in the real world, and patients are well controlled on VOXZOGO. Can you -- and you keep talking about geographic diversity, and that's one of your edges. Talk to me about healthcare markets where you feel like once a patient is on VOXZOGO, it's going to be very hard from a payer perspective, from an insurance perspective to not keep them on the current product and maybe other markets where maybe it's more of a healthcare provider decision. Is there any nuance between the geographic territories that you're involved in with VOXZOGO right now?

Question: Akash Tewari - Jefferies - Analyst : Understood. In terms of your long-term guidance, and I think you get that question from investors, like the Ascendis data came out, why wouldn't they change it, right? Like why maintain the $5 billion I think even for your own team's admission, the Ascendis data was better than, I think, we expected, I think, even BioMarin expected. So why keep the $5 billion? And then number two, is there an appetite as we get into next year to perhaps give more detail about the assumptions underlying the $5 billion number that you gave out do you think that would be helpful for investors?

Question: Akash Tewari - Jefferies - Analyst : Right.

Question: Akash Tewari - Jefferies - Analyst : And I think to that point, just on achondroplasia alone, as we get into 2027, and let's say you do get competition, is BioMarin's base case modest growth, flat or a decline in the achondro-specific sales for VOXZOGO? What's kind of baked into your assumptions?

Question: Akash Tewari - Jefferies - Analyst : Understood. Maybe just more near term on VOXZOGO. In terms of patient starts, and I think I did a poor job at this, too. You had 400 patient starts kind of in Q1, but that's also when you got pediatric on the label, right? As we think about the steady-state rate of patient adds for VOXZOGO on a go-forward basis, is that kind of like 300, 350 range the appropriate metric to think about over the next several years? Or -- because I think the other kind of, I think, misunderstanding I had and The Street had was, supply is coming back online, we should see an inflection any color on, A, patient add cadence, but B, maybe how supply coming back online wasn't necessarily some huge bolus like some might have thought?

Question: Akash Tewari - Jefferies - Analyst : I could have differently, yes.

Question: Akash Tewari - Jefferies - Analyst : Makes sense. Now, I remember when you had kind of got introduced and the question you got is like you've taken a look at the pipeline, what's your kind of favorite child, right? And I think there was a perception at the time given you have expertise within DMD, it could have been the DMD corrector. But talk to me about that long-acting CNP program. And what you see there, A, from potentially bridging patients from VOXZOGO onto a next-gen program. But then B, in terms of the clinical profile for VOXZOGO, where do you feel like a long-acting formulation could actually improve efficacy, whether it's average growth velocity. But to your point, maybe other secondary markets like stenosis and proportionality?

Question: Akash Tewari - Jefferies - Analyst : Understood. Now, you're going to have healthy volunteer data on that program next year. Scientifically, it's kind of interesting because if you model Ascendis' PK versus our PK, Ascendis is kind of this slow AUC, but the Cmax for VOXZOGO is meaningfully higher, right? And I think the question becomes safety, hypertension, cardiac AEs. And I think when you look at a long-acting CNP program, that kind of sustained level of CNP elevation is kind of that open question from a safety perspective. Is that safe to do? Talk to me about how important healthy volunteer data could be in terms of derisking this program because I feel like that's something investors aren't really paying attention to.

Question: Akash Tewari - Jefferies - Analyst : Understood. And outside of average growth velocity, what -- given you've really made inroads within the achondroplasia community and their parents, what are those other secondary endpoints where you feel like a next-gen program could really differentiate and maybe investors aren't understanding?

Question: Akash Tewari - Jefferies - Analyst : Understood. Now, on the DMD corrector, I think there was -- I felt like there was a tone shift, like, okay, we were supposed to have data maybe start of next year. And now it seems like it's coming out more midyear. And I know my team and I were discussing like, is that a tone shift? Are they seeing something in the early data that's making them say we need 26-week data, not 13-week data before we reveal this to The Street. Just to kind of get this on the record. Are you seeing any data with the DMD corrector than when you made that decision? And why extend it out to 26 weeks and not 13 weeks?

Question: Akash Tewari - Jefferies - Analyst : And maybe just lastly, because 30 seconds to go, potential for accelerated approval with the data that you're going to generate on the DMD corrector program by next year, is that a possibility?

Question: Akash Tewari - Jefferies - Analyst : Understood. Given I was two minutes over on Apellis, we're going to end it on time here. Yes, he is saying, thank you. Thank you so much, Alexander. I really do appreciate it.