BioMarin Pharmaceuticals Inc Citigroup’s Biopharma Back to School Summit 2026

Great. Thank you, and thank you so much for having us, Eric. So, just to take a step back, BioMarin, we're a leading global rare disease company, and we're really focused in and around genetically defined conditions. What we have now is 9 commercial therapies across how we're organized, which is in 2 business units, and that includes skeletal conditions and metabolic conditions. Now, we're a highly profitable company. We have strong revenue growth, and really, we're increasing cash flow, which is fueled in large part by the patient demand that we are creating across the 80-country footprint that we work in. We recently, in our Q2 call, announced that we've got 4 products that are tracking to blockbuster status, which we're excited about, and VOXZOGO is in the lead.

At our full year 2026 guide, we had said that VOXZOGO, the range would be $1 billion to $1.05 billion, so that'll be our first blockbuster product this year. What also happened this year that we talked quite a bit about in the Q2 call was the closing of the Amicus acquisition. What that did for us was it brought in 2 high-growth commercial products, that is GALAFOLD for Fabry, and POMBILITI and OPFOLDA, which we call PomOp for Pompe disease. What we shared in the call was that our peak potential for GALAFOLD is at $1.4 billion in the mid-2030s, and for POMBILITI and OPFOLDA, that's $1.2 billion potential in the mid to late 2030s.

As a company, after this acquisition, we really do expect substantial non-GAAP EPS accretion from these assets in 2027, and we really are generating significant cash flow from it that we can then reinvest in innovation. With that, I'll hand it over to you, Greg.

Thank you, Cristin. Everyone, can you hear me okay? Thank you for the invite. It's fantastic to be able to talk a bit about BioMarin's research and development. We are in the process of building out our pipeline, but there are a couple of highlights I want to share for you. One is actually published just yesterday, so hot off the press, our hypochondroplasia data. This is taking the CNP analog, VOXZOGO, and applying it to a related but distinct disease, hypochondroplasia. Those results actually exceeded our expectations, both from a growth standpoint as well as some of the secondary endpoints. We're very proud of that data. It's actually been submitted to the FDA, and we'll be able to update folks soon once that filing is accepted and we have a PDUFA date.

If you think back more towards the pipeline, we've had two new additions to the pipeline, one that came along with the Amicus acquisition, molecule that was referred to as DMX-200 from Dimerix. We have the U.S. rights. We call it BMN 820. This is for focal segmental glomerulosclerosis, so FSGS. It's a CCR2 oral inhibitor, and that is a phase III program running in that renal indication, which will have phase III data looking at 2-year proteinuria out in 2028. So thrilled to be able to add that in the U.S. rights to that molecule. Another hot-off-the-press update for you, we closed, just last week, acquisition of a post phase I product from Aleta Therapeutics. This is ALE-01. This is a molecule that's trying to address hypophosphatasia, but rather than being an enzyme replacement, this is more of a substrate reduction therapy.

Again, the promise there is that not only might you have an oral convenient therapy, but there may be an opportunity to reach some of the tissues that the bone-targeting biologics are unable to reach just because of their PK and their distribution. That includes muscle and joint and so forth. I'll just wrap up by saying that we've got internal innovation as well that's continuing to percolate through our pipeline. Probably the star of the show is BMN 333, which is our next generation CNP analog, more long-acting, weekly administration. Our goal there, I think we'll talk about it a little bit more, is going to be to actually deliver a greater pharmacodynamic effect than what VOXZOGO can achieve.

We have a variety of proof points, again, that tell us that we're headed in the right direction, and we have a molecule that can answer this question definitively. That's in phase II right now. We're enrolling, and it will have data available next year before we launch a seamless phase III thereafter.

Yeah, thank you for the question. Specifically to VOXZOGO, we did report in our earnings call strong growth. Revenue at 14% year-over-year, and importantly, patient demand at 20% year-over-year. That is our global numbers. What we had said is that in particular, that growth, and especially in the U.S., is being driven by exactly what you just referenced, Eric, and that is that 0 to 2 population. It is such an important population for this therapy. Most importantly, because if you look at medical consensus guidelines, it really does suggest that you treat right at the time of diagnosis, because that is where that child can then benefit the most in the long run.

We are really excited that we have the exclusive label there, and we anticipate being the only product approved in the 0 to 2 population for the next couple of years. That is an area that we really do see exciting growth coming out of. Not to mention, some of the other growth drivers are that we are continuing to expand and accelerate in certain countries globally outside the U.S. As a reminder, 75% of our revenues come from outside the U.S. There we have growth happening in countries where we are already well-established, and this is where you see the highest growth coming from that 0 to 2 population, because it is an incident population that you are catching at birth. In other countries, we are either newly launching or still expanding into those countries, and so you see growth across all age groups.

It really does come down to a kind of country-by-country look at where that growth is coming from. But in total, we are commercialized in 55 markets in our 80-country footprint, and that is an area that we will continue to grow in. So very exciting for achondroplasia. I certainly want to mention the fact that what we also announced since the quarter closed was the settlement that we reached with Ascendis in terms of getting royalties on net sales of Uvowell, which was the first competitor to launch in this space. So we have a 20% royalty on U.S. net sales and an 18% royalty in the EU, Brazil, and South Korea. What excites us most about this is that this does just kind of clear an overhang for us.

This is something that enables us to capture value right away, which our ITC proceedings would not have done. We would've been in further litigation in both the U.S. courts as well as the jurisdictions where our IP is. I think most importantly for us, we think that this is a really important moment for both patients as well as BioMarin to kind of clear this overhang and be able to move forward. That's looking at achondroplasia. Another big and exciting growth driver for VOXZOGO is in hypochondroplasia, and I know you'll speak more to this. We're anticipating a launch should the regulatory bodies approve in the coming year, so we have the team certainly gearing up around that. As we look further out, we are looking to, as Greg had mentioned, really bring forward the hopefully best-in-class long-acting CNP.

The durability of that franchise you can see over time growing.