FOLD

Management Statement and Operational Highlights

• Delivered double-digit revenue growth in Fabry and Pompe core business, with GAAP profitability and confidence in positive outlook.
• Galafold had 13% year-over-year patient growth, driven by record demand and new patient starts. It remains the treatment of choice for amenable Fabry patients, with significant unmet need in the disease.
• Pombiliti and Opfolda had a strong quarter with momentum in established and newly launched markets, supported by growing commercial demand and new patient starts. Shared new 4-year data from the PROPEL ongoing extension study for Pombiliti and Opfolda.
• Confidence that 2 commercial products will combine to generate $1 billion in sales by 2028, with Galafold's growth supported by improving diagnosis and access, and Pombiliti and Opfolda contributing to long-term performance.
• Progress with DMX-200, a first-in-class therapy for FSGS in late-stage Phase III development, with the ACTION3 pivotal study over 90% enrolled.
• Achieved GAAP profitability in Q3 and confidence in positive GAAP net income for the second half of 2025, along with a growing cash position.

Guidance

• Full year 2025 financial guidance: Total revenue growth of 15%-22%, Galafold revenue growth of 10%-15%, and Pombiliti and Opfolda revenue growth of 50%-65%, all at constant exchange rates.
• Confidence in positive GAAP net income for the second half of 2025.

Segment Performance

• Galafold: Revenue reached $138.3 million, up 12% at constant exchange rates and 15% in reported terms. Year-over-year patient demand growth was 13%, and it ended the quarter with approximately 69% of the global market share of treated Fabry patients with amenable mutations. Key drivers include finding new patients, expanding into new markets, increasing market share of treated amenable patients, and sustaining high compliance rates.
• Pombiliti and Opfolda: Q3 revenue was $30.7 million, up 42% at constant exchange rates and 45% in reporting terms. Year-to-date, it grew 59% at constant exchange rates (CER) and 61% in reported revenue. The 2025 revenue growth guidance is 50%-65% at CER. Key growth drivers include increasing net new patients, expanding prescribers, launching in new countries, generating evidence, and maintaining high compliance rates.

Risks

• No significant risks discussed in the provided transcript.

Question and Answer

• Q: Congrats on the strong performance. Now that we're into the second full year of the Pom-Op launch, I was wondering if you could talk a little bit about the overall reception to both the label for Pom-Op, especially in the U.S. and the real-world evidence that you seem to be layering in now and how that's driving prescription patterns and whether the conversations vary across treatment centers? Do any certain types of physicians or patients seem to appreciate one set of data more than others? What are you finding is encouraging the most patients to switch to Pom-Op nowadays? A: Joe, thanks a lot for the question. From a label perspective, it's largely been well received, with hopes to expand to pediatric patients. Real-world evidence is a growing part of the conversation with physicians, and the body of evidence will continue to support Pombiliti and Opfolda. Jeff highlighted different types of data important for physicians and the importance of finding the right endpoints for patients and physicians.
• Q: We have 2 on Pompe, if we may. Number one, congrats on the progress, but talk about the U.S. new patient starts in Q3 as they continue to go up relative to some of the commentary you made earlier this year for April and May? And then number two, as we think about 2026, consensus seems to expect a big inflection in Pompe revenue. Can you talk about things that you can actively do to accelerate that revenue trajectory relative to 2025? And expanding your sales force is something you're considering, particularly in the U.S.? A: Q3 was the largest ever net commercial demand for Pombiliti and Opfolda, with the U.S. being a major contributor. Progress in the U.S. includes increased prescriptions and improved time to reimbursement. To accelerate revenue in 2026, experience and real-world evidence are key, along with new countries contributing to demand, like Japan and the Netherlands.
• Q: For Galafold, in particular, you talked about the strongest patient adds since launch on a year-to-date basis. Can you expand a little bit on if that's coming from core countries? Or is that being more driven by emerging countries? A: Key countries continue to grow, driven by naive patients being diagnosed and Galafold being the standard of care for newly diagnosed naive patients with amenable mutations. There's significant growth potential due to underdiagnosis of Fabry disease, with demand stronger than anticipated before launch.
• Q: Just one on DMX-200. Is there a defined threshold for MCP-1 levels or other inflammatory markers that would make patients particularly good candidates for DMX-200? And any thoughts on FILSPARI, the FDA no longer requiring an advisory committee meeting for FILSPARI. A: Regarding FILSPARI, we're eagerly anticipating progress and hopeful for the FDA. For DMX-200, MCP-1 is a chemokine related to inflammation, and DMX-200 interrupts its signaling. Phase II showed an effect on MCP-1 levels, and Phase III will analyze results based on MCP-1 levels.
• Q: This is Joshua Fleishman on the line for Ritu. Congrats on the quarter. So what impact may BIOSECURE 2.0 have on the usability of Ireland plant PomOp product in the U.S.? And how has physician feedback changed over the last quarter on the competitive dynamic in Pompe? Are the goalposts changing from when docs feel comfortable to switch patients to PomOp? A: BIOSECURE 2.0 ensures stable supply from Ireland, with confidence in maintaining supply. Physician feedback shows growing interest in Pombiliti and Opfolda, with evidence mounting to demonstrate its benefits. The goalposts are evolving with more subtle endpoints and patient-driven factors influencing switching decisions.
• Q: This is Rick Miller on for Kristen. Just one for us on Pompe. How should we be thinking about when you could potentially receive infantile onset Pompe disease label expansion? And will this be solely contingent on the ROSELLA, trial? A: Pediatric late-onset Pompe patients (12-17) are a priority, with anticipation of a submission for label expansion mid next year. For infantile onset Pompe disease, enrollment is progressing, with switch patients nearly enrolled and naive patients in progress, likely leading to label expansion after a year-plus study.
• Q: As we look to 2026 here with the switches from Nexviazyme expected in the U.S. and more ex-U.S. countries coming along, how should we think about the commercial trajectory of PomOp versus what we've seen this year? And I guess as you think forward to kind of a steady state, how are you thinking about market share for this asset? A: Confidence in continued momentum into 2026, with early indications of strong growth next year. Pombiliti and Opfolda aims to be the leading product for Pompe patients, with a goal of over 50% market share at peak, driven by growth in markets like the U.K. and potential to expand globally.