Biomarin Pharmaceutical Inc at UBS Global Healthcare Virtual Conference Transcript

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

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : That's great. Super helpful. So just taking a step back and from a high level, BioMarin is traditionally being focused on enzyme replacement products, and now the company is sort of undergoing a transformation into -- like a multifaceted biotech company with gene therapy and like a nucleotide small molecules biologics. Can you just walk us through this transformation? And where you see the company going over the next 3 years, 5 years?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : That's great. So I guess, starting with hemophilia, the current Factor VIII replacement products in hemophilia A and Roche's Hemlibra already working very well for many patients. So how would you frame the unmet need that you see ROCTAVIAN addressing?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Thinking about the sort of subpopulation of hemophilia A, like, who do you think is going to benefit the most? And where do you see the greatest initial uptake?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. That's great. And as we look at the data, there is some decline in Factor VIII activity over time. How are you thinking about this? So what's the feedback you've had from prescribers and payers regarding how they'll view this? And how are you thinking about payment models, et cetera?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : And so when this gets approved, is this -- a couple of things. This is going to be a value-based model. So if the patient doesn't respond, the setup is different. And then just in terms of how you receive the payments, how are you thinking about that? Are you going to [allow] payment over time and will that vary by country?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. That's helpful. And actually, that brings me on to a question in terms of in what work are you doing in both the identifying that small percentage of patients who don't respond? And then also, at some point in the future, the efficacy will weigh in. And what work you're doing there in terms of looking options for retreatment? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 24, 2021 / 3:00PM, BMRN.OQ - Biomarin Pharmaceutical Inc at UBS Global Healthcare Virtual Conference

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. So obviously, you just received the accelerated assessment from the EMA. Can you just talk about how that interaction and process has differed from the FDA? And do you just -- what assurances or greater conviction you have that this is going to be a more predictable regulatory process?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Fantastic. So maybe shifting on to achondroplasia. So can you discuss the unmet need there? And just frame the market size and opportunity for VOXZOGO?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. And can you just talk -- give us a bit of color on the commercialization, launch preparation?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay. I guess the same question on VOXZOGO regarding just conceptually how you're doing about pricing, payment models here?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Fantastic. On the competitive side, in ACH, there are others out there. There's obviously Ascendis, Pfizer, et cetera. Could you just give us your thoughts on these competitive assets? And how you see yourselves as being differentiated?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. You touched upon margins a bit at the beginning, but maybe we could just break out a little bit. And could you just help us understand the margin progression from VOXZOGO and ROCTAVIAN, just breaking out [the team] and how we should think about that over time?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : That's very helpful. And you mentioned business development. Just to expand on that a little bit. How are you guys thinking about that now? What are -- what areas of interest to you? Any preference on deal size or stage?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. J.J., anything to add to that?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Okay Great. And I'm consciously getting towards the end of time. Maybe just a more of a high-level question. As we kind of move into this post-COVID lockdown world, what changes in efficiencies to the way that you've been operating the business are going to be here to stay, be it commercial, manufacturing. What can you tell us there?

Question: Colin Nigel Bristow - UBS Investment Bank, Research Division - Analyst : Great. Thank you. Well, I think that's pretty much at the end of our time. So J.J., Brian, thank you so much for joining us today. This has been fantastic. Thank you to everyone who's been listening in. If anyone has any follow-ups, you can reach me at colin.bristow@ubs.com. And with that, have a great day, everyone.