BioMarin Pharmaceuticals Inc Morgan Stanley 24th Annual Global Healthcare Conference

Thanks very much, Sean. Thanks very much for having us. Thanks to Morgan Stanley for hosting this. I just wanted to say a few things. We mainly want to hear the questions that you have and folks in the room. I'm very happy with our execution, and how we're delivering over the last several quarters. To be able to perform strongly with the delivery of the business, at the same time integrating major acquisition simultaneously, it's really exciting and it's a testament to the organization. I think with the Amicus integration, we've added two additional significant growth drivers, further strengthening the growth profile of the business. We're showing that BioMarin is a rare disease company at scale. We've delivered, obviously, efficiency and profit and growth in profit margins. Now with Amicus, that translates to additional cash flow.

We have this capability across all of the parts of our enterprise from research development, manufacturing to commercialization when it comes to rare disease. We have the balance sheet to act on that. Even as we're integrating Amicus, we also just obviously announced and closed an additional deal to bring in Alesta, ALE1, into our pipeline. We're really excited about that. We're going to continue doing these sorts of deals of strengthening our pipeline, all positioned towards growth in the long run, leveraging our capabilities, and it's really an exciting time at BioMarin.

It is really all, but it is mainly the first two. I would say that the diversification of our growth was a sort of secondary benefit. That was not our primary objective. We believe in VOXZOGO's growth, with the pending hypochondroplasia approval, should the FDA approve it. We think that is in good shape and we are continuing looking at the second quarter, even with a competitor now in the market in the U.S. for a second quarter. We grew at obviously 20% in terms of patient growth. We really felt that Amicus was both value-creating on the top line and on the bottom line. I think you can see from our second quarter update that we are starting to realize that. Projecting a $1.2 billion peak for POMPILI and a $1.4 billion peak for GALAFOLD in our hands, that is more than our additional geographic footprint.

It is the depth of our capabilities across those geographies. We really like what we saw as we got into the due diligence and then the integration planning. On top of it, as a rare disease company operating at scale, the synergies on the bottom line, $200 million of non-GAAP expense savings. It does all of those three things, but I think the very cool thing is particularly on that top line, you do not see that many deals which offer a higher peak and a faster ramp. I think you can be confident that BioMarin's capabilities, we can realize those additional revenue.

Excellent. I will start off and hand over to you, Brian. With regard to the synergies, I think you can feel also very confident around those. As you know, biotech P&Ls from an expense standpoint is very much on the headcount, disproportionately headcount. We made very quickly. I was very impressed. The execution of the organization in terms of the integration planning and then implementation, we very quickly communicated to all the Amicus employees their future employment status. There were about 505 Amicus employees. We are retaining in the long run about 192 of those.

Therefore, those decisions are made, and those savings will be realized. Who of the 192 were retained? Obviously, as we talked about, a very important part of this deal thesis is synergistic on the top line. We wanted to maintain and then add to that growth rate on the revenue line for GALAFOLD and POMP. We primarily retained the commercial and the medical affairs team, so the customer-facing teams. In fact, we will be, and we are investing even more in driving the growth of GALAFOLD and POMP. The bulk of the savings, on the other hand, were on the G&A side, about 70% savings in G&A. The remainder was in R&D, where there were duplicate R&D functions. Maybe hand over to Brian for the next part.

Yeah. Thanks. Yeah. Thanks, Alexander.

Thanks for the question, Sean, and thanks for having us.

You are welcome. First, it was very important, given the strong strategic rationale for the transaction, that we develop an integration plan to fully realize the potential value from the transaction, and Alexander's already touched on some of those revenue and the OpEx synergies.

To be clear, as Alexander touched on, these are not high-level plans. We're executing in terms of the employees that we'll be retaining or not. Those folks have been notified. At the operational level, process by process and department by department, we've got operational cut-over plans. The integration itself will take some time through this year Sure even into next year because some of the relevant systems and processes that we're cutting over are qualified and regulated, and that takes some time.

That's why we point to 2028 as the first full year of realizing all of the synergies because, again, most of the synergies will be realized in 2027.