BioMarin Pharmaceuticals Inc Wells Fargo 21st Annual Healthcare Conference

Thanks for inviting us. Awesome.

I'll start. Okay. We are really excited at BioMarin. As you've mentioned, we've had a lot of updates over the last year, and I'd say the last couple of years. I know you'll get into some of the R&D updates, but some of the things that we're really excited about is that when we look at our established business units, so we have two of them, one which we used to call enzyme therapies, but since the acquisition of Amicus, we're now calling it metabolic conditions. We have a lot of exciting activity therein. This was historically a durable franchise- Right If you think about it.

We had five products that we consider in that, four of which were first in disease, and these are products that we've seen 9% CAGR over the last three years, going up to $2.1 billion at full year last year.

This was things that are not coming from new launches. We have NAGLAZYME, launched in 2005, hit $500 million last year. We have VIMIZIM, launched in 2014, $800 million last year. We have a really durable franchise there. We had the addition of Amicus. We closed this past April.

Right. Really exciting growth story that I know that we will likely get into, but we were able to talk a little bit more about the opportunity in our Q2 call.

This is where we are talking about the potential. These are two commercial fast-growing products that we have real potential to expand on, growing to possibly $1.4 billion for GALAFOLD and Fambril, and $1.2 billion at peak, in the mid 2030s, late 2030s for POMBILITI and OPFOLDA in Pompe. Really exciting growth areas for us, not to mention what we have going in skeletal conditions, which I will touch on quickly. We did just recently announce, just to start it off, a settlement with Ascendis Right which I think helps to clear some of the competitive overhang.

which I'm sure we'll get into. This is also an exciting area for growth for us in terms of VOXZOGO. We had 14% revenue growth at the quarter, 20% patient growth Q2 year-over-year, but also growing sequentially, and just a really exciting time where we're seeing great growth coming out of the younger population for VOXZOGO, but also importantly, we're seeing global expansion of this brand. Very exciting times. Then we've added some things to our pipeline that I know you'll get into, Greg.

Okay. I'll hand it over to you.

Yeah, no. Hot off the press is, of course, this morning we were able to show the world the totality of our hypochondroplasia data.

Really excited about that, again, the opportunity for really a first-in-disease therapy for VOXZOGO as an extension. The results exceeded our expectations, and again, not only on the efficacy side, but the safety came out quite nice, and again, that currently has been submitted to the FDA for review. Similarly, we're looking across our portfolio and really trying to restock our pipeline.

That involves both internal as well as external innovation sources. We most recently closed a deal with Alesta Therapeutics for an HPP oral therapy.

Happy to get into if we Sure have additional questions.