Biomarin Pharmaceutical Inc at Canaccord Genuity Growth Conference (Virtual) Transcript

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

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Great. Thank you, Brian. So I'd like to start off by talking a little bit about ROCTAVIAN. Your hemophilia A gene therapy. You have a PDUFA date coming up later this month. And maybe we can just start here and talk about overall the data you've generated for this program in hemophilia A. And really try to also dig into the 4-year results you presented very recently. Why are these data important to patients, physicians and payers when you start to think about the value that ROCTAVIAN brings to this community? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. AUGUST 13, 2020 / 5:00PM, BMRN.OQ - Biomarin Pharmaceutical Inc at Canaccord Genuity Growth Conference (Virtual)

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Great. And maybe we can dive in a little bit. You've mentioned or previously spoken about an expected eligible U.S. population of about 2,400 or 2,500 patients at launch. Can you help us understand which patients this number includes? Which patients it may not include? And who do you think, out of this eligible patient population -- what kind of patient might be an early adopter when you think about the launch?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Got it. And just digging into some of your comments around HEMLIBRA. Able to be dosed as infrequently as monthly, and Roche has really announced a pretty high U.S. patient share. Maybe you could talk a little bit about what some of the advantages, on the safety and efficacy side, for a Factor VIII gene therapy might be in your view, producing the protein itself?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : All right. So do you expect the, I guess, early adopter population for HEMLIBRA to be different than the early adopter population for Factor VIII gene therapy?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Great. And just digging in a little bit more to some of those competitive dynamics that you might face at launch. If approved, ROCTAVIAN might be competing for patients against other gene therapy trials as well. I guess how would you see being a commercial-approved therapeutic as -- versus entering into another AAV gene therapy trial? I guess how do you see that dynamic playing out? And what advantage does, I guess, the ROCTAVIAN complete data package provide for when you're going out and marketing this drug?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : That makes sense. And you mentioned earlier that you're talking to clinicians, most of them seem very aware of AAV gene therapy, and ROCTAVIAN and implications as well. How -- when you think about preparing for launch, I guess, especially in this COVID-19 era, what are some of the goals you have around getting sites ready, educating them? And how much work do you think you need to do on that awareness side? Or right now, are clinicians primarily coming to you asking for you to educate them and prepare them for a potential launch?

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : And just 1 on pricing to follow that up. You've talked a bit about how an outcomes-based pricing model might be -- there might be an opportunity to implement 1 in the U.S. How would that affect, I guess, your launch, especially given your commentary about payers being a potential bottleneck

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : Great. I want to talk about all the other stuff you guys have going on, too. But maybe we can start with BMN 307. You're anticipated to dose patients this quarter. Can you just talk a little bit about the trial design, some of the endpoints you've chosen? And what do you view as a bar for success to be competitive in this market? You've been long-established in the PKU market. What is the...

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : And then you also announced that 331 is an IND-enabling study. And you just announced this deal with DiNAQOR around rare genetic cardiomyopathies. How should we think about kind of the platform beyond ROCTAVIAN and vosoritide and 307 that you anticipate will begin to drive long-term value.

Question: Michelle Lim Gilson - Canaccord Genuity Corp., Research Division - Analyst : All right. Great. Thank you so much for all of your insights today. I think we're out of time here. But I really appreciate you guys coming here and telling us more about BioMarin and all of the exciting developments you guys have going on this year. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. AUGUST 13, 2020 / 5:00PM, BMRN.OQ - Biomarin Pharmaceutical Inc at Canaccord Genuity Growth Conference (Virtual)