BioMarin Pharmaceutical Inc at TD Cowen Healthcare Conference Summary

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

Question: Philip Nadeau - TD Cowen - Analyst : <_ALACRA_META_ABSTRACT>Alexander, I'll hand it to you. Can you give a brief state of the company overview, biggest strengths, biggest challenges? And what does BioMarin need to achieve to drive shareholder value over the next 12 to 24 months?

Question: Philip Nadeau - TD Cowen - Analyst : Can you discuss your long-term vision for BioMarin. It was partially laid out through the guidance that was given at last year's analyst meeting in terms of the long-term financial targets. Maybe outline those, but then also, what is your long-term strategic goals for the company?

Question: Philip Nadeau - TD Cowen - Analyst : What role will business development play in the long-term strategy? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 04, 2025 / 2:50PM, BMRN.OQ - BioMarin Pharmaceutical Inc at TD Cowen Healthcare Conference

Question: Philip Nadeau - TD Cowen - Analyst : What type of assets would be of interest to you? Would you buy a marketed product? Would it be early stage? What do you think its bet with your current portfolio? And similarly on risk profile? Do you look more at the lower risk into the spectrum, high risk? How do you think about building portfolio externally?

Question: Philip Nadeau - TD Cowen - Analyst : Perfect. Last week or the week before, you provided 2025 revenue guidance, $3.1 billion to $3.2 billion, that's 9% to 12% growth. Can you talk about the pushes and pulls in that guidance. What could drive upside? What are the risks stating?

Question: Philip Nadeau - TD Cowen - Analyst : And maybe one more general question before diving into some of the franchises. In terms of operating margins, your guidance is 32% to 33%, I believe, in 2025, going to 40% over time. It seems like based on our model, that 32% to 33% assumes modest growth in OpEx in 2025. Is that reasonable? And are you able to get that leverage on the business because of the reorganization of the business units? Or what has permitted you to grow revenue while not really increasing expenses at least this year.

Question: Philip Nadeau - TD Cowen - Analyst : Perfect. Shifting to VOXZOGO, where investors are very keenly focused. Guidance for this year is $900 million, $950 million. That's growth of 22% to 29%. Very healthy growth, but a bit of a deceleration from prior years. Where are we in the VOXZOGO launch, which territories are driving growth, which ones are more mature?

Question: Philip Nadeau - TD Cowen - Analyst : I think investors are very keenly focused on competition as I'm sure you're aware. What gives you confidence that you can grow in the face of competition? Well, you can grow the VOXZOGO franchise in the face of (inauidble)

Question: Philip Nadeau - TD Cowen - Analyst : It seems like the biggest potential benefit of the competitors is convenience, whether it's oral or less frequent injection. How does convenience factor into the treatment decision in achondroplasia in the context of some of those other health issues you talked about as well as long-term safety and efficacy data. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MARCH 04, 2025 / 2:50PM, BMRN.OQ - BioMarin Pharmaceutical Inc at TD Cowen Healthcare Conference

Question: Philip Nadeau - TD Cowen - Analyst : In your opening remarks, you talked about the other indications, the size of the markets as well as the development program. Can you maybe dive into that in a bit more detail? Where is VOXZOGO in its development plan in like hyperchondraplasia and the other potential indications and what are the size of those.

Question: Philip Nadeau - TD Cowen - Analyst : Perfect. Maybe in the last minute, just turning to the earlier stage pipeline, BMN 351 has some proof-of-concept data coming up in the back half of this year. Can you discuss how that molecule is different than the other skipping agents? And what BioMarin would consider proof of concept in the upcoming data.

Question: Philip Nadeau - TD Cowen - Analyst : Perfect. With that, I think we're out of time. Thank you.