BioMarin Pharmaceuticals Inc 2026 Q2 Earnings Call

Please- Thank you, operator, and thank you all for joining us today.

To remind you, this non-confidential presentation contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc., including expectations regarding BioMarin's financial performance, commercial products, and potential future products in different areas of therapeutic research and development. Results may differ materially depending on the progress of BioMarin's product programs, actions of regulatory authorities, availability of capital, future actions in the pharmaceutical market, and developments by competitors, and those factors detailed in BioMarin's filings with the Securities and Exchange Commission such as 10-Q, 10-K, and 8-K reports. In addition, we will use non-GAAP financial measures as defined in Regulation G during the call today.

These non-GAAP measures should not be considered in isolation from, as substitutes for, or superior to financial measures prepared in accordance with U.S. GAAP, and you can find the related reconciliations to U.S. GAAP in the earnings release and earnings presentation, both of which are available in the investor relations section of our website. Please note that our commentary on today's call will focus on non-GAAP financial measures unless otherwise indicated. Moving to slide three and introducing BioMarin's management team joining today's call, Alexander Hardy, Chief Executive Officer, Cristin Hubbard, Chief Commercial Officer, Greg Friberg, Chief R&D Officer, and Brian Mueller, Chief Financial Officer. I will now turn the call over to Alexander to provide our quarterly highlights.

Alexander? Thank you, Traci, and thank you all for joining us today.

Starting on slide five, BioMarin delivered a standout second quarter, combining strong growth to nearly $1 billion in revenue with the successful close and integration of Amicus while delivering on milestones that strengthen our leadership in genetic conditions. Our strong performance demonstrates both the value creation of our portfolio and the continued execution of our commercial organization executing at scale, integrating meaningfully accretive assets and continuing to innovate, enabling us to bring important medicines to people living with rare diseases as we enter an exciting new phase of growth. Second quarter highlights start with 20% year-over-year total revenue growth, accelerated by a more diversified portfolio and setting up an even stronger second half of 2026 to be fueled by full third and fourth quarter GALAFOLD and POMBILITI and OPFOLDA contributions and sustained demand across our other products.

Turning to VOXZOGO, double-digit revenue growth in both the U.S. and international markets led us to increase VOXZOGO's full year guidance, putting it on a path to become BioMarin's first $1 billion product. In its first quarter with a U.S. competitor, the continued revenue growth demonstrates our ability to defend VOXZOGO's leadership position. Building on this momentum, we are pleased to share that we have submitted our sNDA for the approval of VOXZOGO to treat hypochondroplasia based on strong pivotal data shared during the quarter. We will provide an update on the submission as part of our third quarter results. Turning to Amicus, as anticipated, when we announced the acquisition, this deal demonstrates that BioMarin can leverage our scale and capabilities to rapidly integrate high-growth assets to maximize value creation. Today, we provide estimated peak revenue for GALAFOLD of $1.4 billion and for POMBILITI and OPFOLDA of $1.2 billion.

Together, these innovative therapies, combined with significant cost synergies expected to reach approximately $220 million annual run rate in 2028, are anticipated to drive substantial EPS accretion and operating cash flow. Most importantly, we look forward to bringing GALAFOLD and POMBILITI and OPFOLDA to more patients with Fabry and Pompe disease worldwide. Briefly on our pipeline, which continues to build momentum. We recently added BMN 820, formerly DMX-200, an exciting new late-stage pipeline opportunity resulting from the Amicus acquisition that Greg will expand upon in a moment. I'm also impressed by the speed at which we submitted the sNDA for VOXZOGO for the treatment of hypochondroplasia. The speed of our submission reflects the benefits of our investments in AI capabilities and sets the new standard for how BioMarin will execute going forward. Together, these results tell a clear story.

BioMarin is executing at scale, raising guidance, outperforming on the Amicus integration with substantial combined peak revenue potential ahead, and advancing pivotal pipeline data towards VOXZOGO's next indication. As we enter the second half of 2026, BioMarin is stronger, more diversified, and better positioned than ever to lead in rare disease and deliver for patients worldwide. Now on slide six. The addition of Amicus transforms BioMarin's growth trajectory through the mid-2030s with a combined peak revenue potential of $2.6 billion from GALAFOLD and POMBILITI and OPFOLDA, and significant cost synergies layering in along the way. We expect meaningful non-GAAP EPS accretion, expanding operating margins, and stronger operating cash flow, powering our next phase of growth. Moving now to slide seven and starting with GALAFOLD, a growing product in a growing market.

From a $522 million base in 2025, we project peak revenue of $1.4 billion by the mid-2030s, supported by two complementary growth drivers. First, we see significant opportunity to expand diagnosis and treatment. Leveraging BioMarin's proven diagnostic capabilities, our goal is to more than double the number of U.S. patients treated with GALAFOLD. We plan to do this through scaling AI-enabled patient identification initiatives, expanded genetic testing, newborn screening, and family cascade screening, helping more amenable patients access treatment earlier. Second, we see meaningful opportunity to expand market penetration globally. With GALAFOLD already established in 40 countries, we intend to deepen penetration within existing markets while selectively expanding into new geographies, leveraging BioMarin's global commercial infrastructure to accelerate access and broaden reach. Together, these drivers are expected to support approximately 10% CAGR from 2027 to 2032. Turning to slide eight. POMBILITI and OPFOLDA is at an earlier stage in its commercial journey compared to GALAFOLD.

We estimate $1.2 billion in peak revenue by the mid to late 2030s, growing at a greater than or equal to 20% CAGR from 2027 to 2032. We expect growth to be driven primarily by increased patient switching and global market expansion, complemented by continued improvements in diagnosis and treatment rates. We believe switching will be supported by growing awareness amongst healthcare providers and patients of the expanded body of real-world evidence demonstrating the benefits of POMBILITI and OPFOLDA on disease outcomes. At the same time, we plan to leverage our diagnostic capabilities to identify and support treatment of additional eligible patients across our global footprint. These growth drivers are expected to be further strengthened by planned expansion into more than 20 additional markets over time. Turning to slide nine. We are pleased with the significant cost synergies identified, which we expect to contribute to substantial EPS accretion beginning next year.

We anticipate approximately $220 million of synergies to be fully realized in 2028, representing a roughly 50% reduction from Amicus's 2025 non-GAAP operating expenses of $432 million. These synergies are weighted towards G&A, which makes up more than 70% of the total, with the remainder coming primarily from R&D. As planned, we retained Amicus's commercialization team to ensure patient continuity and supplement our global capabilities. These synergies, combined with peak revenue, align with the value creation we anticipated when we announced the deal last year and demonstrate our ability to successfully integrate large accretive assets that strengthen our growth profile. Turning to slide 10. By accelerating our financial profile with the addition of accretive assets that benefit from our proven global expertise serving patients with genetic conditions, the Amicus acquisition sets the stage for our next phase of growth.

With peak targets of $1.4 billion for GALAFOLD and $1.2 billion for POMBILITI and OPFOLDA, these revenues, combined with $220 million in anticipated annual cost synergies fully realized in 2028, support substantial expected non-GAAP diluted EPS accretion beginning in 2027 and a significant increase in operating cash flow. GALAFOLD and POMBILITI and OPFOLDA combined are expected to reach over 60% non-GAAP operating margin by 2030. At the same time, we believe our rapid integration and growth plans will enable us to de-leverage approximately one year sooner than initially communicated. This quarter reinforces what sets BioMarin apart.

We are the leading rare disease company operating at scale, with a proven integration capability to maximize the value of high-growth assets. We look forward to updating you on our progress scaling GALAFOLD and POMBILITI and OPFOLDA as we enter the next exciting phase of BioMarin's growth. I will now turn the call over to Cristin for the commercial update.