Summary of Amicus Therapeutics Conference Call Company Overview - **Company**: Amicus Therapeutics (NasdaqGM:FOLD) - **Focus**: Developing transformative therapies for rare diseases, particularly Fabry disease and Pompe disease [4][5] Key Products and Financial Performance - **Galafold**: - Approved for Fabry disease, on the market for nearly 10 years in Europe and 8 years in the U.S. - Achieved over $500 million in revenue for the first time this year, with a growth rate of 10%-15% [5][6] - The global market for Fabry disease is estimated at $2.4 billion, up from $1 billion previously [10] - **Pombiliti and Opfolda (POMOP)**: - Approved for Pompe disease, launched in the last couple of years. - Expected revenue growth of 50%-65% this year [5][6] - Projected to generate over $100 million in sales this year [23] Market Dynamics and Growth Drivers - **Fabry Disease Market**: - Initial estimates of incidence were one in 50,000 live births; recent studies suggest it may be closer to one in 3,000 [10] - The diagnosed untreated population has grown from 5,000 to 6,000 patients [10] - Increased awareness and availability of genetic testing are driving patient identification [11][12] - **Patient Identification Initiatives**: - Collaborations with healthcare systems (e.g., NHS in the U.K.) to identify undiagnosed patients [13][14] - Use of AI and partnerships (e.g., with OM1) to enhance patient finding [15][19] Competitive Landscape - **Galafold vs. Competitors**: - Concerns about Sanofi's upcoming phase three data for their substrate reduction therapy (SRT) were addressed, noting that Galafold's mechanism is different and may not be directly impacted [21][22] - **Pombiliti Market Position**: - The company is seeing increased patient switches from competitor products, with a significant uptick in the last two quarters [26][29] - The U.S. market is showing strong momentum, with the company gaining market share in the U.K. and other European regions [27][32] Pipeline and Future Opportunities - **FSGS Program**: - Amicus has licensed DMX-200 for FSGS, a rare kidney disease, with potential for significant market opportunity [36][38] - The FDA has indicated that proteinuria could serve as a primary endpoint for the ongoing phase three study, which is promising for regulatory approval [39][40] - **Regulatory Strategy**: - Plans to engage with the FDA regarding the study endpoints and potential accelerated approval paths [40][41] Financial Position and Strategic Focus - **Cash Position**: - The company has over $260 million in cash and is GAAP profitable, indicating a strong financial foundation [52] - **Business Development Strategy**: - Focus on late-stage, de-risked assets with potential for revenue growth, particularly in the Fabry and Pompe disease spaces [52] Conclusion - Amicus Therapeutics is positioned for continued growth with strong product performance, strategic initiatives to identify and treat more patients, and a promising pipeline in rare diseases. The company is committed to maintaining financial discipline while pursuing opportunities for expansion and profitability [52][53]

Core Insights - Amicus is a rare disease-focused biotech company with three programs, each having a potential market opportunity of $1 billion, currently at different development stages [1] - The company is expected to turn profitable in the second half of the year and maintains a strong cash position [1] Product Performance - Galafold, the first approved product for Fabry disease, was launched in 2016 and is projected to achieve 10% to 15% growth this year, with sales exceeding $500 million [2] - Pombiliti, Opfolda, is in the early stages of its launch after receiving approval in 2023, indicating potential for future growth [2]

Summary of Amicus Therapeutics Conference Call Company Overview - **Company**: Amicus Therapeutics (NasdaqGM:FOLD) - **Focus**: Rare disease therapeutics with a unique position in the biotech sector, targeting first or best-in-class medicines [3][4] Key Products and Financials - **Galafold**: - Approved for Fabry disease since 2016 - Expected growth of 10%-15% in 2025, with over $500 million in sales [3][4] - Currently holds a 65% market share among treated amenable patients, with potential to increase to 85%-90% in established markets [5][6] - **Pombiliti (Opfolda)**: - Launched in 2023, showing 50%-65% growth, projected to exceed $100 million in sales [4][5] - Strong patient demand in the U.S. and expansion into new countries [8][9] - **DMX-200**: - Targeting Focal Segmental Glomerulosclerosis (FSGS), a rare kidney disease with no approved treatments [4][29] - Positioned as a potential blockbuster with a unique mechanism of action [29] - **Financial Position**: - Projected revenue of over $600 million in 2025, aiming for $1 billion by 2028 [4][5] - Currently holds over $260 million in cash, with positive cash generation of over $30 million in Q3 [4][5] Market Dynamics and Growth Drivers - **Galafold**: - Growth driven by increased patient diagnoses and treatment initiation [5][6] - Newborn screening initiatives and family cascade testing are significant tailwinds [6][12] - The total diagnosed Fabry patient population has grown from 10,000 to nearly 20,000, with potential for over 100,000 undiagnosed patients [14] - **Pombiliti**: - Strong momentum in new patient additions, particularly from patients previously on Nexviazyme [8][11] - Geographic expansion with eight new countries added this year, aiming for ten total [9][18] Competitive Landscape and Pricing Strategy - **Pricing Strategy**: - Amicus maintains a pricing philosophy focused on access, pricing below competitors and committing to not raise prices above the Consumer Price Index (CPI) [19][20] - No significant pricing erosion observed in the U.S. or ex-U.S. markets [19][20] - **Market Share**: - Achieved over 40% market share in the U.K. for Pombiliti, with expectations for similar performance in other markets [19] Regulatory and Development Updates - **Intellectual Property (IP)**: - Galafold has patent protection extending into the 2040s, with ongoing litigation against generic filers [23][24] - Anticipated resolution of IP trials in the second half of next year [24] - **DMX-200 Development**: - Enrollment for the Action 3 study is over 90% complete, with top-line data expected in two years [37][38] - Potential for accelerated approval discussions based on one-year data [39] Upcoming Catalysts - Continued patient addition and revenue growth for Galafold and Pombiliti [40] - Updates on Galafold IP resolution and Pombiliti's pediatric label expansion expected in mid-2026 [41] - Completion of DMX-200 enrollment and potential updates on FDA discussions in the first half of next year [41]

Core Insights - Investors should consider adding stocks with strong liquidity to their portfolios, as liquidity indicates a company's ability to meet short-term obligations and supports business expansion [1][2] - Four top-ranked stocks recommended for investment include EverQuote, Inc. (EVER), Amicus Therapeutics, Inc. (FOLD), Lam Research Corporation (LRCX), and Cboe Global Markets, Inc. (CBOE) [2][9] Liquidity Measures - Current Ratio: Measures current assets against current liabilities; a ratio below 1 indicates more liabilities than assets, while a range of 1-3 is ideal [4] - Quick Ratio: Indicates a company's ability to pay short-term obligations, with a desirable ratio of more than 1 [5] - Cash Ratio: The most conservative measure, focusing on cash and equivalents relative to current liabilities; a ratio greater than 1 is desirable but may indicate inefficiency [6] Screening Parameters - Asset Utilization: A measure of efficiency, calculated as total sales over the last 12 months divided by the average total assets; companies with a ratio higher than their industry average are considered efficient [7][8] - Growth Score: A proprietary score added to ensure that liquid and efficient stocks also have solid growth potential [8] Company-Specific Insights - **EverQuote, Inc. (EVER)**: Reported revenues of $173.9 million, a 20% year-over-year increase; projected fourth-quarter revenues between $174-$180 million [12][13] - **Amicus Therapeutics, Inc. (FOLD)**: Revenues of $169.1 million, a 19% year-over-year increase; expects total revenues to grow by 15-22% in 2025 [14][15] - **Lam Research Corporation (LRCX)**: Reported revenues of $5.32 billion, a 28% increase year-over-year; projects second-quarter revenues of $5.2 billion [16][17] - **Cboe Global Markets, Inc. (CBOE)**: Achieved record revenues of $605.5 million, a 14% year-over-year increase; raised 2025 organic revenue growth target to low double-digit to mid-teens [18][19]

Core Insights - Amicus Therapeutics (FOLD) reported adjusted earnings of 17 cents per share for Q3 2025, surpassing the Zacks Consensus Estimate of 12 cents and up from 10 cents in the same quarter last year [1][6] - Revenues for Q3 totaled $169.1 million, reflecting a 19% year-over-year increase and exceeding the Zacks Consensus Estimate of $165 million [2][6] - The revenue growth was driven by sales of Galafold and the newly approved combination therapy Pombiliti + Opfolda, which is aimed at treating late-onset Pompe disease [2][3] Financial Performance - Galafold sales reached $138.3 million, a 12% increase year-over-year at constant exchange rates, although it fell short of the Zacks Consensus Estimate of $150 million [4][6] - Sales of Pombiliti + Opfolda amounted to $30.7 million, surpassing both the Zacks Consensus Estimate of $29.7 million and the internal estimate of $29.4 million [7] - Adjusted operating expenses rose by 15% to $95.4 million in Q3 2025, while cash and marketable securities increased to $263.8 million from $231 million as of June 30, 2025 [7] Guidance and Future Outlook - Amicus reaffirmed its 2025 guidance, projecting total revenue growth of 15-22% at constant exchange rates, with Galafold expected to grow by 10-15% and Pombiliti + Opfolda by 50-65% [8] - The company anticipates achieving positive GAAP net income in the second half of 2025 and aims to exceed $1 billion in total sales by 2028 [9] Recent Developments - Amicus licensed exclusive rights for Dimerix's phase III program, DMX-200, in the U.S., targeting FSGS, a rare kidney disease, with the pivotal ACTION3 study on track for full enrollment by the end of 2025 [10]

Amicus Therapeutics (FOLD) reported $169.06 million in revenue for the quarter ended September 2025, representing a year-over-year increase of 19.5%. EPS of $0.17 for the same period compares to $0.10 a year ago. The reported revenue compares to the Zacks Consensus Estimate of $164.76 million, representing a surprise of +2.61%. The company delivered an EPS surprise of +41.67%, with the consensus EPS estimate being $0.12. While investors closely watch year-over-year changes in headline numbers -- revenue ...

Financial Data and Key Metrics Changes - Total revenue for Q3 2025 reached $169.1 million, a 19% increase compared to Q3 2024, with a 17% growth at constant exchange rates [23] - GAAP net income for 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, marking the first quarter of positive GAAP net income in 2025 [24][25] - Non-GAAP net income for Q3 2025 was $54.2 million, or $0.18 per share, compared to $30.8 million, or $0.10 per share, in the same quarter last year [25] Business Line Data and Key Metrics Changes - Galafold revenue reached $138.3 million, up 12% at constant exchange rates and 15% in reported terms, with a year-over-year patient demand increase of 13% [10] - Pombiliti and Opfolda generated $30.7 million in revenue for Q3, a 42% increase at constant exchange rates and 45% in reported terms, with year-to-date growth of 59% at CER [14] Market Data and Key Metrics Changes - Approximately 69% of the global market share of treated Fabry patients with amenable mutations is held by Galafold [10] - The U.S. represented approximately 43% of Pombiliti and Opfolda revenue, while ex-U.S. accounted for 57% [15] Company Strategy and Development Direction - The company aims to achieve combined sales of $1 billion for its two commercial products by 2028, supported by growing diagnostic rates and patient access [7] - The company is advancing the development of DMX-200, a therapy for FSGS, with over 90% enrollment in the ACTION3 pivotal study expected to complete by year-end [8][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in sustaining double-digit revenue growth in the Fabry and Pompe core business, driven by record demand and robust new patient starts [5] - The company anticipates positive GAAP net income for the second half of 2025 and reiterated full-year financial guidance for 2025, projecting total revenue growth of 15%-22% [26][27] Other Important Information - The company highlighted the significant unmet need in Fabry disease, with over 6,000 diagnosed patients remaining untreated, indicating a larger market opportunity for Galafold [12] - The company is focused on expanding its market presence and increasing patient compliance and adherence rates above 90% [16] Q&A Session Summary Question: Reception of Pombiliti in the U.S. and real-world evidence impact - Management noted that the label for Pombiliti has been well received, with ongoing studies expected to support label expansion for pediatric patients [32] - Real-world evidence is increasingly important in discussions with physicians, and the company plans to continue supporting publications and presentations [33][34] Question: U.S. new patient starts and revenue trajectory for 2026 - Management reported significant increases in new patient starts in Q3 and expressed confidence in continued momentum into 2025 [42] - The company is focused on expanding its sales force and enhancing real-world evidence to drive revenue growth [43] Question: Galafold patient adds from core vs. emerging countries - The growth in patient adds is primarily driven by core countries, with a significant number of naive patients being diagnosed [48][49] Question: DMX-200 candidate selection based on MCP-1 levels - Management indicated that higher MCP-1 levels correlate with more robust responses to DMX-200, and they are measuring MCP-1 throughout the phase three study [54][55] Question: Impact of Biosecure 2.0 on Pombiliti usability in the U.S. - Management expressed confidence in maintaining a stable supply from the Ireland facility and highlighted the importance of providing evidence to physicians regarding Pombiliti [59][60] Question: Timing for infantile onset Pompe disease label expansion - Management anticipates a submission for adolescent patients mid-next year, with further progress expected for infantile onset patients thereafter [66][68] Question: Commercial trajectory of Pombiliti and market share expectations - Management is confident in continued growth and aims for over 50% market share at peak for Pombiliti, with a target of exceeding $1 billion in revenue [71][72]

Financial Data and Key Metrics Changes - Total revenue for Q3 2025 reached $169.1 million, a 19% increase compared to Q3 2024, with a 17% growth at constant exchange rates [23] - GAAP net income for 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, marking the first quarter of positive GAAP net income in 2025 [24][25] - Non-GAAP net income for Q3 2025 was $54.2 million, or $0.18 per share, compared to $30.8 million, or $0.10 per share, in the same quarter last year [25] Business Line Data and Key Metrics Changes - Galafold revenue reached $138.3 million, up 12% at constant exchange rates and 15% in reported terms, with a year-over-year patient demand increase of 13% [10][11] - Pombiliti and Opfolda generated $30.7 million in revenue for Q3 2025, reflecting a 42% increase at constant exchange rates and 45% in reported terms, with year-to-date growth of 59% at CER [14][15] Market Data and Key Metrics Changes - The U.S. represented approximately 43% of Pombiliti and Opfolda revenue, while ex-U.S. accounted for 57% [15] - Galafold holds approximately 69% of the global market share of treated Fabry patients with amenable mutations [10] Company Strategy and Development Direction - The company aims to achieve combined sales of $1 billion for its two commercial products by 2028, supported by strong growth in patient demand and market penetration [7][8] - The company is advancing the development of DMX-200, a first-in-class therapy for FSGS, with over 90% enrollment in the ACTION3 pivotal study expected to complete by year-end [8][21] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in sustaining double-digit revenue growth in the Fabry and Pompe core business, driven by record demand and new patient starts [5][6] - The company anticipates positive GAAP net income for the second half of 2025, reinforcing its financial discipline and growth trajectory [24][26] Other Important Information - The company is focused on expanding its market presence and improving patient access, particularly in underdiagnosed populations [12][13] - The company is working on enhancing awareness and diagnosis of Fabry disease through initiatives like the Finding Fabry campaign [13] Q&A Session Summary Question: Reception of Pombiliti label and real-world evidence impact - Management noted that the label for Pombiliti has been well received, with ongoing studies expected to support label expansion for pediatric patients [32] - Real-world evidence is increasingly important in discussions with physicians, with positive feedback on durability and effectiveness from clinical trials [34] Question: U.S. new patient starts and revenue trajectory for 2026 - Management confirmed that Q3 saw the largest net commercial demand for Pombiliti and Opfolda, with significant increases in prescriptions and improved reimbursement times [42] - The company is optimistic about continued momentum into 2025, with plans to expand into new markets and enhance real-world evidence [43] Question: Galafold patient additions from core vs. emerging countries - The growth in Galafold patient additions is primarily driven by core countries, with a significant number of naive patients being diagnosed [48] Question: DMX-200 candidate selection based on MCP-1 levels - Management indicated that while there is no defined threshold for MCP-1 levels for DMX-200 candidates, higher levels may correlate with better responses [54] Question: Impact of Biosecure 2.0 on Pombiliti usability - Management expressed confidence in maintaining a stable supply from the Ireland facility, which is expected to enhance the usability of Pombiliti in the U.S. [59] Question: Timing for infantile onset Pompe disease label expansion - Management anticipates a submission for adolescent patients by mid-next year, with further expansions for younger patients and infantile onset following [66][68] Question: Commercial trajectory of Pombiliti in 2026 - Management is confident in strong growth for Pombiliti, aiming for over 50% market share at peak, with a trajectory towards $1 billion in revenue [71][72]

Financial Data and Key Metrics Changes - Total revenue for Q3 2025 reached $169.1 million, a 19% increase year-over-year, with a 17% growth at constant exchange rates [22] - GAAP net income 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, marking the first quarter of positive GAAP net income in 2025 [24] - Non-GAAP net income was $54.2 million, or $0.18 per share, compared to $30.8 million, or $0.10 per share, in the same quarter last year [24] Business Line Data and Key Metrics Changes - Galafold revenue reached $138.3 million, up 12% at constant exchange rates and 15% in reported terms, with a 13% year-over-year increase in patient demand [8][22] - Pombiliti and Opfolda generated $30.7 million in revenue for Q3, a 42% increase at constant exchange rates and 45% in reported terms, with year-to-date growth of 59% at CER [12][22] Market Data and Key Metrics Changes - Approximately 69% of the global market share of treated Fabry patients with amenable mutations is held by Galafold, with a significant opportunity for further market penetration [8] - The U.S. represented approximately 43% of Pombiliti and Opfolda revenue, while ex-U.S. accounted for 57% [13] Company Strategy and Development Direction - The company aims to achieve combined sales of $1 billion for its two commercial products by 2028, supported by strong growth in Galafold and Pombiliti and Opfolda [5][6] - The company is advancing the development of DMX-200, a late-stage therapy for FSGS, with over 90% enrollment in the ACTION3 pivotal study [6][19] Management Comments on Operating Environment and Future Outlook - Management expressed confidence in sustaining double-digit revenue growth in the Fabry and Pompe core business, driven by record demand and new patient starts [4][5] - The company anticipates positive GAAP net income for the second half of 2025 and reiterated full-year financial guidance for total revenue growth of 15%-22% [25][24] Other Important Information - The company is focused on expanding its market presence and improving patient access, with ongoing efforts to enhance awareness and diagnosis of Fabry disease [10][11] - The company has secured reimbursement in 15 countries for Pombiliti and Opfolda, with plans to launch in up to 10 new countries in 2024 [16] Q&A Session Summary Question: Can you discuss the reception of Pombiliti's label and real-world evidence? - Management noted that the label has been well received, with ongoing studies expected to support label expansion, particularly for pediatric patients [30] Question: What is the status of new patient starts in the U.S. for Pompe? - Management reported significant increases in new patient starts and improved time to reimbursement, contributing to strong growth [38] Question: How should we think about the commercial trajectory of Pombiliti in 2026? - Management expressed confidence in continued momentum and expects strong growth, aiming for over 50% market share at peak [64]

Core Insights - Amicus Therapeutics reported a total revenue of $169.1 million for Q3 2025, reflecting a 17% increase at constant exchange rates (CER) [4][3] - The company achieved a positive GAAP net income of $17.3 million, compared to a net loss of $6.7 million in Q3 2024 [7][27] - Cash position increased to $264 million, marking a $33 million rise in Q3 2025 [4][7] Financial Performance - Total revenues for Q3 2025 were $169.1 million, with a year-over-year growth of 19% reported and 17% at CER [4][5] - Galafold sales reached $138.3 million, a 15% increase year-over-year, while Pombiliti + Opfolda sales were $30.7 million, up 45% year-over-year [7][5] - Total GAAP operating expenses were $115.3 million, an 8% increase from $106.6 million in Q3 2024 [7][27] Product Performance - Galafold's net product sales for Q3 2025 were $138.3 million, with a 12% growth at CER [7][5] - Pombiliti + Opfolda saw a significant increase in sales, driven by high demand and new market entries, achieving a 42% growth at CER [7][5] - The company is on track for full enrollment in the Phase 3 study of DMX-200 by year-end 2025 [3][14] Strategic Outlook - Amicus anticipates total revenue growth of 15% to 22% for 2025, with Galafold revenue growth projected at 10% to 15% and Pombiliti + Opfolda at 50% to 65% [8][4] - The company aims to achieve sustainable double-digit growth in the coming years, with a target of surpassing $1 billion in total sales by 2028 [14][3] - Amicus continues to focus on expanding its commercial business and enhancing its product pipeline for rare diseases [22][3]