BioMarin Pharmaceutical Inc at Evercore ISI HealthCONx Conference Summary

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

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : <_ALACRA_META_ABSTRACT>So going to start big picture. Obviously, there's been a lot of changes with BioMarin over the last year. Alexander took over as CEO. It's a new corporate strategy. You've talked about cost reductions, added a number of new executives sort of across the Board. Can you kind of describe the -- go through some of the key changes that have taken place and how you think this is setting the company up for success going forward? Brian Mueller - Biomarin Pharmaceutical Inc - Chief Financial Officer, Executive Vice President - Finance Thanks so much, Cory. It's great to be here. We are very excited about BioMarin's path forward, and you touched on many of the key elements. I'll unpack those a bit more for you. But we believe that after the work this year, we've got the right strategy, team and growth objectives to generate long-term value for patients and shareholders. You're right. Alexander, just recently, within the last few days, I believe, reached his one-year anniversary with the company. It's been a busy year. We completed a full strategic review of the enterprise. This was top to bottom, end-to-end across the business, resulting in a revamped corporate strategy, organization and operating model changes and compelling long-term financial targets. That includes a new team. So Cristin's here with us. She was most recently the Head of Global Product Strategy at Roche and also spent a significant amount of time at Genentech. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 04, 2024 / 3:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Evercore ISI HealthCONx Conference Hank Fuchs, our former Head of Research and Development for 15 years, recently retired from the company. His successor, who's been on board a couple of months now, is Greg Friberg. He had a long 18-year career at Amgen, going across several roles. Most recently, he was the Head of Medical for the Rare Disease business unit at Amgen. And then the most recent team member to join the leadership team is James Sabry, former Head of Partnering at Roche. And he also had a longtime career at Roche and Genentech, longtime biopharma career, both in partnering, founding companies, and CEO. So back to the reorganization in terms of the high-level strategy, we are focused -- the strategy is focused on innovation, growth and value commitment. From an operating model standpoint, we are organizing the company into business units. This is new for BioMarin. We have eight internally developed and approved products generating close to $3 billion in revenue. We previously were organized as a portfolio company, but there's enough opportunity to accelerate, prioritize, focus, optimize what our individual sort of therapeutic areas, if you think about it that way. Those are skeletal conditions, enzyme therapies, and then a small business unit for Roctavian, the gene therapy for severe hemophilia A. The revamped corporate strategy has been -- we've been successful in executing on it already this year. We're generating record revenues, record profitability, and issued some compelling long-term financial guidance. We're expecting to reach $4 billion of total revenue in 2027, a non-GAAP operating margin of 40% in 2026, growing into the low to mid-40% from there. That's a more than doubling of our non-GAAP operating margin in 2023, which was a little over 19%. And then long term, on revenue, beyond the $4 billion in 2027, we do expect that we can continue to grow revenue at a mid-teen -- low- to mid-teen CAGR through 2034. That financial growth is also resulting in significant cash flow generation. Our long-term financial guidance includes $1.25 billion of operating cash flow in 2027. That level of cash flow accumulation, plus a recently healthier balance sheet after paying down a convertible debt maturity, opening up a $600 million revolving credit line, gives us ample firepower to be able to pursue a more focused BD, business development strategy than BioMarin has done in the past. Again, James recently joining us. So we believe that these are very compelling, rare defined in this sector for a company of our size, financial targets and growth strategy elements. We're very excited, and we're off to a good start.

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : I know James has only been -- he hasn't been around for that long just yet, but what's the early feedback that you're hearing from him in terms of his excitement level for the assets that are out there and available today?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. And you mentioned in your comments upfront about the goals you've set out there, the '27 revenue guidance, the operating margin guidance. With all that, like what's the pitch you're making to investors to own the stock over the next year, over the next three years? And kind of like what are the valuation metrics that you're most focused on as you make this pitch? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 04, 2024 / 3:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Evercore ISI HealthCONx Conference

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. So let's start diving into some of the products, and not surprising, we'll start with VOXZOGO. So I think this is where the biggest disconnect exists, right, between what you guys think is going to happen with this product and where investors are right now. And so the overhang of overhangs has now become TransCon CNP. As -- assuming this reaches the market relatively near term, how does VOXZOGO grow through that? Because people don't seem to have that expectation. So can you kind of speak to that and maybe what KOLs are telling you and how you think TransCon will be incorporated into the market?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. Great. Can you remind us, roughly speaking, how much of VOXZOGO sales are currently US? And when you have this -- these projections out there for a 25%-plus growth into the future with this, how do you expect that to evolve? You talk about bringing 20 more countries online, the opportunities that are out there, rest of world, where I agree, you have a huge advantage over the competition. Can you kind of speak to the evolution of those trends? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 04, 2024 / 3:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Evercore ISI HealthCONx Conference

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. And you talked as well about sort of the life cycle with VOXZOGO and you follow on product, your once weekly. I'm curious strategically how you're thinking about this, especially as the market becomes more competitive in the future, ways to speed up the process with some of the other indications. Are there accelerated pathways you could use given the track record that's been established with VOXZOGO, things like 505(b)(2), for example, stuff like that? And would you -- are there ways to accelerate, I guess, the once a week and bring in some of these indications right off of that?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. And with 333, is -- what's the objective, right? Is this like just compete with TransCon as once a week? Is this a better VOXZOGO? I mean, based on the preclinical data that you're generating, do you think this has the chance to be more efficacious? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 04, 2024 / 3:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Evercore ISI HealthCONx Conference

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : And do you know from a regulatory point of view yet what you'll need to do in the clinic with this? Or maybe it's not a regulatory

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. All right. Fair enough. Let's switch over and we can come back and talk about -- more about that if we have time, but to cover the enzyme therapies. I mean this is another underappreciated part of the company. This high single-digit growth for this, again, not something that people are giving you credit for. It feels like looking at where consensus is. So very underappreciated. How confident are you in these numbers? Like how ambitious of a goal is the high single-digit percent growth?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. One of the products in there that is potentially facing competitive headwinds is Palynziq. How do you think about that product in the context of your guidance? And what are your expectations for competition there?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. And -- REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 04, 2024 / 3:00PM, BMRN.OQ - BioMarin Pharmaceutical Inc at Evercore ISI HealthCONx Conference

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. And then expectations across the rest of the enzyme therapy portfolio, not including Palynziq, I mean some of these products have been off patent for quite a long time, still growing. Do you have expectations for generics ever coming for any of these?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. Wanted to throw one question on the third unit product segment, Roctavian. It's amazing it never gets talked about at all anymore. You updated your strategy for it over the summer. Where do things stand with it? Do you see any sense of increasing traction here with this? Or is it kind of much the same where you were over the summer?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. Back to the pipeline, and we talked about the long-acting CNP program. I think the other asset that's starting to get a little bit of attention ahead of next year is your -- the DMD program, so BMN 351. You obviously have some pretty compelling preclinical data. What's the bar you need in this initial human proof-of-concept study to kind of validate what you've shown preclinically?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. Terrific. So we have a few minutes left. I wanted to -- I was hoping to have some time to circle back to VOXZOGO, and we do. When you talk -- you put out long-term opportunities, right? You put the patient populations out there, talk about this being a $5 billion opportunity in the future. Another competitive agent that we've all known about that's coming is still a year away from Phase 3 data to be infigratinib. What are your expectations of the potential impact infigratinib can have in this market, both for achondroplasia as well as beyond achondroplasia where they're going to look at hypochon, and we'll see if they can go beyond that or not. How does that play into your expectations and into the guidance you provided in September?

Question: Cory Kasimov - Evercore ISI Institutional Equities - Analyst : Okay. And so -- and like you said, I mean, you put out the $5 billion number, you knew about infagratinib doing that. The TransCon news came later, but you've since reiterated. So the confidence in the long-term opportunity for VOXZOGO still very much there.