Loading...
01/11 12:00
Loading...
Access earnings results, analyst expectations, report, slides, earnings call, and transcript.
The earnings call highlights strong financial performance, with significant revenue growth and a shift to GAAP profitability. Despite increased COGS, net income improved, and cash reserves grew. The Q&A section revealed positive reception of product labels and strategic market expansion plans. However, vague responses on revenue acceleration and supply stability pose some concerns. Overall, the positive financial results, optimistic guidance, and strategic initiatives outweigh these concerns, suggesting a positive stock price movement.
Galafold Revenue Revenue reached $138.3 million, up 12% at constant exchange rates and up 15% in reported terms. Year-over-year, the underlying growth in patient demand increased by 13%. Reasons for growth include record demand, robust new patient starts, and a growing global market share of treated Fabry patients with amenable mutations.
Pombiliti and Opfolda Revenue Third quarter revenue reached $30.7 million, up 42% at constant exchange rates and up 45% in reported terms. Year-to-date, Pombiliti and Opfolda has grown 59% at CER and 61% in reported revenue. Reasons for growth include strong sales growth, high patient demand, and increasing market penetration in both established and new markets.
Total Revenue Total revenue for Q3 2025 was $169.1 million, a 19% increase over the same period in 2024. At constant exchange rates, revenue grew 17%. Reasons for growth include strong performance of Galafold and Pombiliti and Opfolda.
Cost of Goods Sold (COGS) COGS as a percentage of net sales was 12% for Q3 2025 compared to 9% in the same period last year. The increase is attributed to the hybrid year for Pombiliti and Opfolda COGS as previously expensed inventory was worked through during the first three quarters of 2025.
GAAP Net Income GAAP net income in Q3 2025 was $17.3 million or $0.06 per share compared to a net loss of $6.7 million or $0.02 per share in Q3 2024. This improvement is attributed to strong revenue growth and financial discipline.
Non-GAAP Net Income Non-GAAP net income in Q3 2025 was $54.2 million or $0.18 per share compared to $30.8 million or $0.10 per share in Q3 2024. Reasons for improvement include strong revenue growth and operational efficiency.
Cash Position Cash, cash equivalents, and marketable securities were $263.8 million as of September 30, 2025, compared to $249.9 million as of December 31, 2024. This represents a $32.8 million increase during the third quarter, attributed to positive cash generation.
Galafold: Delivered 13% year-over-year patient growth, driven by record demand and robust new patient starts. Revenue reached $138.3 million, up 12% at constant exchange rates and 15% in reported terms. Achieved approximately 69% global market share of treated Fabry patients with amenable mutations. Sustained compliance and adherence rates above 90%.
Pombiliti and Opfolda: Revenue reached $30.7 million, up 42% at constant exchange rates and 45% in reported terms. Strong sales growth and high patient demand observed. Reimbursed in 15 countries, with new markets including Japan, Belgium, Ireland, and Luxembourg. Full-year revenue growth guidance of 50%-65% reiterated.
Geographic Expansion: Pombiliti and Opfolda reimbursed in 15 countries, including new markets like Japan, Belgium, Ireland, and Luxembourg. Strong execution in newer launch markets such as Switzerland, Italy, Czech Republic, Portugal, and the Netherlands.
Financial Performance: Achieved total revenue of $169.1 million in Q3 2025, a 19% increase year-over-year. Positive GAAP net income of $17.3 million in Q3 2025, compared to a net loss in Q3 2024. Cash position increased to $263.8 million as of September 30, 2025.
Operational Efficiency: Maintained gross margin in the mid-80s and achieved positive cash generation in Q3 2025. Non-GAAP operating expenses increased but remained within guidance.
Pipeline Development: Advanced DMX-200, a first-in-class therapy for FSGS, with the pivotal Phase III ACTION3 trial over 90% enrolled and on track for full enrollment by year-end. FDA alignment on proteinuria as the primary endpoint for approval.
Scientific Evidence: Presented new 4-year data from the PROPEL study, showing stability or improvement in muscle function and strength for Pombiliti and Opfolda. Real-world evidence and independent studies further support differentiation of these therapies.
Regulatory and Reimbursement Challenges: The company faces challenges in securing pricing and reimbursement agreements in various countries, as evidenced by the ongoing efforts to expand access to Pombiliti and Opfolda in new markets. This could delay revenue realization and market penetration.
Market Competition: The company acknowledges competitive pressures, particularly in the Pompe disease market, where patients are switching from existing treatments like Nexviazyme to Pombiliti and Opfolda. This indicates a need to continuously differentiate their products to maintain and grow market share.
Supply Chain and Cost Management: The hybrid year for Pombiliti and Opfolda COGS, with previously expensed inventory being worked through, suggests potential challenges in managing supply chain costs and ensuring consistent gross margins.
Economic and Currency Risks: Revenue growth is reported at constant exchange rates, highlighting exposure to currency fluctuations that could impact financial performance, especially given the significant portion of revenue generated outside the U.S.
Strategic Execution Risks: The company’s ambitious goals, such as achieving $1 billion in combined sales by 2028 and launching in up to 10 new countries in 2025, require flawless execution. Any missteps could impact long-term growth and shareholder value.
Clinical Development Risks: The development of DMX-200 for FSGS, while promising, is still in late-stage Phase III trials. Any delays or failures in achieving regulatory approval could hinder the company’s pipeline expansion and future revenue streams.
Revenue Growth: The company expects to sustain double-digit revenue growth in its Fabry and Pompe core business into the years ahead. Full-year 2025 revenue growth guidance is reiterated at 15% to 22%.
Galafold Revenue: Galafold is projected to achieve 10% to 15% revenue growth in 2025, with a long-term goal of surpassing $1 billion in revenue. Key drivers include finding new patients, expanding into new markets, and sustaining compliance rates above 90%.
Pombiliti and Opfolda Revenue: Revenue growth for Pombiliti and Opfolda is expected to be 50% to 65% in 2025. The company plans to launch in up to 10 new countries in 2025 and maintain over 90% compliance rates.
Long-term Sales Target: The company reaffirms its confidence in achieving combined sales of $1 billion for its two commercial products, Galafold and Pombiliti/Opfolda, by 2028.
DMX-200 Development: The ACTION3 pivotal study for DMX-200, a therapy for FSGS, is over 90% enrolled and on track to complete enrollment by the end of 2025. The company anticipates further discussions with the FDA in early 2026.
Profitability: The company expects to achieve positive GAAP net income for the second half of 2025, though profitability may not be linear quarter-to-quarter.
The selected topic was not discussed during the call.
The earnings call highlights strong financial performance, with significant revenue growth and a shift to GAAP profitability. Despite increased COGS, net income improved, and cash reserves grew. The Q&A section revealed positive reception of product labels and strategic market expansion plans. However, vague responses on revenue acceleration and supply stability pose some concerns. Overall, the positive financial results, optimistic guidance, and strategic initiatives outweigh these concerns, suggesting a positive stock price movement.
The earnings call reveals mixed signals. Financial performance shows increased expenses and net loss, though non-GAAP income remains positive. Product development and market expansion are promising, with strong market share gains and regulatory progress. However, the decreased revenue growth guidance and increased COGS raise concerns. The Q&A highlights confidence in IP and manufacturing progress but lacks specifics on timelines and COGS impact. Overall, the sentiment is neutral, with potential for both positive and negative market reactions.
All transcripts are sourced directly from the official live webcast or the company’s official investor relations website. We use the exact words spoken during the call with no paraphrasing of the core discussion.
Full verbatim transcripts are typically published within 4–12 hours after the call ends. Same-day availability is guaranteed for all S&P 500 and most mid-cap companies.
No material content is ever changed or summarized in the “Full Transcript” section. We only correct obvious spoken typos (e.g., “um”, “ah”, repeated 10 times”, or clear misspoken ticker symbols) and add speaker names/titles for readability. Every substantive sentence remains 100% as spoken.
When audio quality is poor or multiple speakers talk over each other, we mark the section instead of guessing. This ensures complete accuracy rather than introducing potential errors.
They are generated by a specialized financial-language model trained exclusively on 15+ years of earnings transcripts. The model extracts financial figures, guidance, and tone with 97%+ accuracy and is regularly validated against human analysts. The full raw transcript always remains available for verification.
