BioMarin Pharmaceuticals Inc 12th Annual Cantor Fitzgerald Global Healthcare Conference

Thank you for having us.

I will kick us off. For those of you following us, we are BioMarin. We are a leading rare disease biotechnology company. We operate specifically in genetically defined conditions. We have nine commercial products that are spread across our two business units, so skeletal conditions and metabolic conditions. Very recently, we announced the acquisition of Amicus, and with Amicus, that gave us two high-growth commercial products. One is Galafold in Fabry disease, which we just announced peak revenue estimates at $1.4 billion in the mid-2030s. The second being POMBILITI and OPFOLDA, which we call PomUp, in Pompe disease, which we have also announced expectations of $1.2 billion at peak in the mid to late 2030s. A really exciting acquisition, a huge strategic fit for us to really strengthen and diversify our portfolio. At the quarter, we announced very strong results, 20% growth year-over-year.

Really what we are excited about is the fact that we have got four products in our portfolio now that have the potential to be above $1 billion. Our first of which will be VOXZOGO, which in our 2026 full year guidance, we are saying that we will be at $1 billion to $1.05 billion is the range that we have given for the full year. Our focus as a company is really about driving top-line growth, about expanding our margins, and importantly, about generating cash flow. Because with that, we can then continue to invest in our pipeline. We have recently announced another acquisition for a pipeline asset, ALE-001, that Greg, I will give it over to you to talk a little bit about our portfolio.

Yeah. From the portfolio standpoint, a lot of news, again, fast-moving at BioMarin. We just revealed our data in hypochondroplasia, which again was published yesterday and presented in Europe at the ESPE meeting. Very excited. That result would provide us with another first in disease indication. This is for VOXZOGO in beyond achondroplasia and hypochondroplasia. Data really outperformed what we were expecting. We are thrilled about that opportunity. Similarly, we have been rebuilding our pipeline as well with a variety of different assets. Along with the Amicus acquisition came a molecule that we call BMN 820 or DMX-200. This is for focal segmental glomerulosclerosis. That is a study that will have a phase III card turning over in 2028. Similarly, we have two other pipeline assets now.

The recently acquired and closed last week, ALE-001 from Alesta Therapeutics for hypophosphatasia, as well as, of course, we have got our in-house created molecule, BMN 333, as a next generation asset for CNP. Again, looking for superiority over VOXZOGO. Both of those will have data to be revealed next year.

Yeah. As we have already talked a little bit about, driving top-line growth is an important component of our strategy, and these assets help us to do so. It is really exciting to think about what we can bend the curve on in terms of the treatment of Fabry disease as well as Pompe disease with both of these. But as you have mentioned, Olivia, there is the financial components here. And we really do see this as a huge accelerator. So what we talked about at the quarter were the fact that we are going to recognize about $220 million in synergies that will come in 2028. This gives us substantial EPS accretion in 2027.

And this is an area that, again, we are really looking to be able to use the cash flows that will come from this so that we can continue to allocate a lot of capital into growing our pipeline, investing in innovation, and so on. So we think that this has been a real accelerant on many fronts.

Yeah. VOXZOGO, I think we can talk about it a little bit in horizons in the different disease areas, right? We have achondroplasia, which I will get to, but also hypochondroplasia in the future. Then longer term, and maybe you will speak to this, Greg Friberg, looking out at BMN 333, which is our long-acting CNP that we look to develop the best-in-class CNP, which I know you will get to. But going back to kind of the near-term growth and achondroplasia in particular, as you have said, and as you have alluded to, Olivia Brayer, we did just reach a settlement with Ascendis Pharma. We think that this is very important both for BioMarin, but also importantly for the patients. This settlement allows us to get 20% royalty on net sales in the U.S. and 18% royalty on net sales in the EU, South Korea, and Brazil.

Really what this does is this enables us to capture value now. It is retroactive to any commercial sales. But also importantly, I think it enables us to clear a little bit of the overhang on VOXZOGO and really share in not only a percent for any patients that might switch off of VOXZOGO, but also importantly for any market growth that might start as a result of YUVIWEL now being on the market. We will now get to share in that and not just compete for it. So that at least helps us to clear that overhang. But importantly, thinking about VOXZOGO as a growth driver. We are confident in the growth going into next year with VOXZOGO, and that is in part based on how we are growing in achondroplasia.