BioMarin Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference Summary

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

Question: Sean Laaman - Morgan Stanley - Analyst : Sure. Thanks for those comments. Maybe just to talk about the macro for a little bit. We're doing this with all the companies, so we'd love to have your view. So with China's rise in biotech innovation, how are you thinking about the competitive dynamic and does it influence your R&D and business development strategy? Yeah.

Question: Sean Laaman - Morgan Stanley - Analyst : Sure. It's rare disease in (inaudible) more or less untouched at the moment the visible levels at least anyway. So few question on the macro. So what's been the most impactful, sorry, second question on the macro, how are you currently leveraging AI or thinking about AI as a disruptive technology?

Question: Sean Laaman - Morgan Stanley - Analyst : Fantastic. Thank you. And last question on the macro piece. So what's been most impact the most impactful thing to BioMarin on the regulatory side? Is it being MFN? Is it being tariffs? Is it being changes at the FDA?

Question: Sean Laaman - Morgan Stanley - Analyst : Awesome. Thank you for such -- that was great. On to more specifics on --

Question: Sean Laaman - Morgan Stanley - Analyst : I'm right on it. Don't be fooled by the relaxed nature. Can you talk about the recent Inozyme acquisition and why it was a good acquisition? How long how long have you been evaluating it and do you see those or when do you see those revenues being a meaningful contributor to BioMarin?

Question: Sean Laaman - Morgan Stanley - Analyst : Okay, wonderful. In your Q2 you noted that the company plans to provide updates to the $4 billion FY27 revenue guidance later this year and also the '25 guidance currently does not incorporate IPR&D expenses associated with it recently with the Inozyme acquisition. Can you provide any more color on those threads? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 09, 2025 / 3:30PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference

Question: Sean Laaman - Morgan Stanley - Analyst : Thank you. Maybe to go product specific on VOXZOGO. So it's been launched in 51 countries, I think more recently, up from 49 in 1Q. Where do you stand on near term country additions and reimbursement wins and how should we measure our progress against your longer term ambitions?

Question: Sean Laaman - Morgan Stanley - Analyst : Sure. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 09, 2025 / 3:30PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Morgan Stanley Global Healthcare Conference

Question: Sean Laaman - Morgan Stanley - Analyst : And I guess we'll stand back and look at where the share price and everyone seems to think it's worth double where it's trading, at least on the sell side and the overhang seems to be VOXZOGO and what happens with [transcontinental piece], just to get your agreement on that if that's what you think that the major overhang is and then maybe sort of walk us through your competitive positioning of VOXZOGO and your early indication on an age-related basis, your geographic spread. Maybe why you might not necessarily think, there is a material risk to VOXZOGO.

Question: Sean Laaman - Morgan Stanley - Analyst : Right, so think about the, I guess maybe prevention switches, but the CANOPY clinical trial program, maybe touch on that as something that you think whether investors are focused on or not focused on, maybe that is a defense strategy if you like, if you did lose in the achondroplasia and net that off if that makes sense.

Question: Sean Laaman - Morgan Stanley - Analyst : And then maybe move to forward a little bit more longer term, but on 333, which is showing extraordinary promise with the data that you released, can you talk through getting to the registrational study and looking at sort of longer term, potential launch plans like how far are we potentially thinking?

Question: Sean Laaman - Morgan Stanley - Analyst : Wonderful. Thank you, Alex. On PALYNZIQ, so some strong numbers [18 to 25], and I believe there's some more data around on adolescents. So you potentially utilization of that data to further accelerate sales.

Question: Sean Laaman - Morgan Stanley - Analyst : Wonderful. And are there any steps dating the supplementary BLA submission in US and EU and what sort of revenue contribution would you expect from the inclusion to 12 to 17 year olds?

Question: Sean Laaman - Morgan Stanley - Analyst : Well, thank you, and I believe there's a recent approval in the PKU space to change your view on the competitive dynamic market share assumptions.

Question: Sean Laaman - Morgan Stanley - Analyst : Sort of taking a step back and looking at the investment case, so there's I think you mentioned 30% of the revenue is coming from VOXZOGO and you've got sort of balanced portfolio across the rest of the business and for biotech companies sort of cracking profitability and generating cash flow. And you know I think you've talked about (inaudible) the business into operating units, but I guess a lot going on in the R&D pipeline, but what thinking from here is invested? Do we think about enough appetite in your current R&D portfolio to suck up cash flows? Do you think about the business development opportunities? Do you think sort of mid to longer term formal capital? How do you put all those pieces together?

Question: Sean Laaman - Morgan Stanley - Analyst : Wonderful. One final question, is there anything that I didn't ask that I should have?

Question: Sean Laaman - Morgan Stanley - Analyst : At another time, but thank you gentlemen, for your time today. It's been great.