Summary of Amicus Therapeutics (FOLD) FY Conference Call - June 10, 2025 Company Overview - **Company**: Amicus Therapeutics - **Core Business**: Development of therapies for rare diseases, primarily focusing on Galafold for Fabry disease and Pombility for Pompe disease - **Sales Projections**: Expected to surpass $500 million in sales for Galafold and $100 million for Pombility in 2025 [3][4] Key Points and Arguments Financial Performance and Growth - **Galafold**: - Projected to achieve $1 billion in peak sales with a growth rate of 10% to 15% in 2025 [4] - Currently in a $2 billion global market, expected to grow to $3 billion over the next decade [8][9] - **Pompe Disease Therapies**: - Pombility and Upholda projected to achieve $100 million in sales with a growth rate of 50% to 65% [4] - Anticipated to reach $1 billion in sales in the future [4] - **New Asset - DMX200**: - Licensed from Dimerix for FSGS, a rare kidney disease, with potential blockbuster status [5] - Expected to contribute to significant revenue growth by 2028 [5] Market Position and Competitive Landscape - **Macro Environment**: - Company believes it is well-positioned to navigate macroeconomic challenges affecting the sector [7][8] - Emphasizes the undervaluation of Galafold and the potential for increased diagnoses in the Fabry disease space [8][9] - **Launch Dynamics**: - Initial launch delays due to COVID-19 impacted market entry for Pompe therapies [37] - Positive trends observed in the U.S. market with increasing patient switches from competitors [39][41] Regulatory and Operational Considerations - **Impact of Tariffs and Regulations**: - Minimal impact from tariffs due to low cost of goods for Galafold, which is manufactured in Switzerland [16][18] - Company is adapting to regulatory changes and believes it is insulated from potential negative impacts [20][22] - **FDA Dynamics**: - New leadership at the FDA is expected to support rare disease regulatory reforms, potentially benefiting Amicus [23][24] Guidance and Future Outlook - **Profitability Expectations**: - Guidance for GAAP profitability in the second half of 2025, with revenue growth projected at 15% to 22% [25][26] - Operating expenses expected to remain flat or increase slightly, allowing for positive free cash flow [27][29] - **Long-term Strategy**: - Focus on maintaining financial discipline while expanding the product portfolio [6][12] - Plans to leverage cash flow for new development opportunities in rare diseases [80] Additional Important Insights - **Market Dynamics**: - The company is seeing strong uptake in initial launch countries, with market shares reaching 20-30% in key markets [47] - New countries like the Netherlands are expected to significantly contribute to revenue through patient switches [50][52] - **Patient Diagnosis Trends**: - Increased diagnosis rates for Fabry disease due to lower costs of genetic testing and newborn screenings [64][66] - Potential for significant patient population growth as awareness and testing improve [67][70] Conclusion Amicus Therapeutics is positioned for strong growth in the rare disease market, with a focus on financial discipline and strategic expansion. The company anticipates achieving profitability in the near term while navigating macroeconomic challenges and regulatory changes. The potential for increased patient diagnoses and successful product launches underpins a positive outlook for future revenue growth.

Summary of Amicus Therapeutics (FOLD) Conference Call Company Overview - **Company**: Amicus Therapeutics - **Event**: Jefferies Healthcare Conference - **Date**: June 04, 2025 Key Points Business Performance and Growth - Amicus has focused on growing the Galafold and Fabry franchise, achieving mid-teen growth rates in patient numbers for Galafold, indicating sustainable growth driven by volume [3][4] - The company reported $458 million in sales last year, with a growth forecast of 15% to 22% [8] - Amicus aims to achieve GAAP profitability in the second half of 2025, having already reached non-GAAP profitability in 2024 [5] Product Updates - Galafold continues to show strong patient growth, with a guidance of 10% to 15% growth for the year [11][31] - The launch of Pombility generated $70 million in revenue last year, with strong growth in prescriptions noted in recent months [4][34] - The company is optimistic about the potential of the recently acquired Dimerix asset for FSGS, which could have blockbuster potential in the U.S. [6][47] Market Dynamics - There is a significant number of undiagnosed Fabry patients, presenting a large market opportunity for Galafold [14][16] - The company is investing in AI initiatives to identify undiagnosed patients, enhancing diagnostic capabilities [27][29] - The settlement with Teva provides Galafold with intellectual property protection for the next twelve years, reinforcing its market position [10] Competitive Landscape - Amicus is aware of the competitive dynamics with Nexviazyme, noting that switching from Lumizyme to Nexviazyme typically occurs after two years of treatment [36][38] - The company believes that positive patient feedback and outcomes will drive future market share growth for Pombility [38] Future Outlook - Amicus is exploring additional business development opportunities that align with its existing infrastructure, focusing on rare diseases [51][52] - The company is cautious about overextending its balance sheet while pursuing growth opportunities [52] Financial Considerations - The Dimerix deal involved a $30 million upfront payment, with additional milestones totaling approximately $75 million, plus royalties on sales [54][55] - The company is committed to maintaining a strong cash flow while pursuing strategic acquisitions [5][6] Additional Insights - The company is seeing a shift in treatment patterns, with more patients being diagnosed and treated earlier, particularly among females [17] - There is a growing understanding among physicians regarding Fabry disease, which is expected to drive further patient diagnosis and treatment [15][20] This summary encapsulates the key insights and strategic direction of Amicus Therapeutics as discussed during the conference call, highlighting the company's growth trajectory, product developments, and market opportunities.

Financial Data and Key Metrics Changes - Total revenue for Q1 2025 reached $125.2 million, a 13% increase year-over-year, with a 15% growth at constant exchange rates [33][34] - GAAP net loss for Q1 2025 was $21.7 million, or $0.07 per share, compared to a net loss of $48.4 million, or $0.16 per share in Q1 2024 [34] - Non-GAAP net income for Q1 2025 was $9 million, or $0.03 per share, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share in the same period last year [34] Business Line Data and Key Metrics Changes - Galafold revenue reached $104.2 million, up 6% at constant exchange rates, with new patient starts globally increasing by 14% [9][11] - Pombility revenue for Q1 2025 was $21 million, up 92% at constant exchange rates, primarily driven by the US, Germany, and Spain [13][14] - Adjusted revenue growth guidance for Pombility and Upholda was revised from 65%-85% to 50%-65% at constant exchange rates [36] Market Data and Key Metrics Changes - The US contributed significantly to Galafold's growth, with an 18% increase in sales, while UK sales were impacted by higher VPAG rebates [11][45] - The company achieved a record number of patients on Galafold in Australia following its first-line listing [10] - Revenue was recorded in seven countries, with new patient starts anticipated in four additional countries in Q2 2025 [18] Company Strategy and Development Direction - The company entered a strategic collaboration with Dimerix to commercialize DMX200, a treatment for FSGS, enhancing its late-stage pipeline [24][26] - The company is focused on diversifying its supply chain and has onshored a portion of drug product manufacturing to the US [6][19] - The long-term outlook for Galafold and Pombility remains positive, with expectations of reaching combined sales of $1 billion by 2028 [7] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving GAAP profitability in the second half of 2025, despite challenges faced in Q1 [7][36] - The management acknowledged the impact of unexpected factors on quarterly revenues but remains optimistic about sales acceleration for the remainder of the year [7][15] - The company is well-positioned to create substantial value for shareholders and deliver on its mission for patients [7][41] Other Important Information - The company reiterated its financial discipline and updated its full-year revenue growth guidance to 15%-22% from the previous 17%-24% [36] - The company is committed to maintaining high compliance and adherence rates for its therapies, which are crucial for long-term growth [16] Q&A Session Summary Question: What led to the higher-than-anticipated rebate for Galafold and Pombility? - Management explained that the rebate was a negotiated rate by the industry association in the UK, which was higher than expected, impacting revenue throughout the year [45][46] Question: What has changed regarding the cadence of ex-US launches and patient starts? - Management noted that reimbursement settlements led to a delay in patient starts, which are now expected to accelerate in the second half of the year [49][51] Question: Can you elaborate on the diligence process for the DMX200 licensing agreement? - Management highlighted that the diligence process involved careful review of data, market research, and discussions with physicians, leading to confidence in the asset's differentiation [58][60] Question: What is the expected timeline for the next interim analysis for DMX200? - Management indicated that the timeline for the next interim analysis could be around nine months, depending on the collaboration with Parasol and the FDA [72][74] Question: Will the VPAG rebate impact be recurring? - Management clarified that while the rebate will impact revenue this year, it is not expected to be a recurring event [86][87]

Financial Data and Key Metrics Changes - The company achieved total revenue of $125.2 million for Q1 2025, representing a 13% increase year-over-year, and a 15% increase at constant exchange rates [33] - GAAP net loss for Q1 2025 was $21.7 million, or $0.07 per share, compared to a net loss of $48.4 million, or $0.16 per share in Q1 2024 [34] - Non-GAAP net income was $9 million, or $0.03 per share, compared to a non-GAAP net loss of $4.6 million, or $0.02 per share in the same period last year [35] - Cash, cash equivalents, and marketable securities were $251 million as of March 31, 2025, compared to $250 million at December 31, 2024 [35] Business Line Data and Key Metrics Changes - Revenue for Galafold reached $104.2 million, up 6% at constant exchange rates, with a 14% increase in new patient starts globally [10] - Revenue for Pombility reached $21 million, up 92% at constant exchange rates, driven primarily by the US, Germany, and Spain [14] - The company adjusted its revenue growth guidance for Pombility and Upholda from 65%-85% to 50%-65% at constant exchange rates [36] Market Data and Key Metrics Changes - The US contributed significantly to growth, with 60% of total revenue generated outside the US and 40% from within [33] - The UK sales were impacted by higher VPAG rebates than previously anticipated, with the negotiated rate increasing from 15% to 22% [46] - The company recorded revenue in Q1 across seven countries, including the US, Germany, Austria, Spain, the UK, Switzerland, and Sweden [18] Company Strategy and Development Direction - The company entered a strategic collaboration with Dimerix to commercialize DMX200, a treatment for FSGS, enhancing its late-stage pipeline [25] - The company aims to achieve GAAP profitability in the second half of 2025 while maintaining financial discipline [9] - The company is focused on expanding its portfolio in rare diseases, particularly in neuromuscular and rare kidney diseases [92] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the long-term growth trajectory, expecting continued double-digit revenue growth in core business areas [6] - The management acknowledged challenges in the UK market due to unexpected rebate increases but remains optimistic about patient growth compensating for this [46] - The company anticipates a significant acceleration in patient starts in the second half of 2025, particularly in new launch markets [52] Other Important Information - The company is onshoring a portion of its drug product manufacturing to enhance supply chain diversification [8] - The company reiterated its commitment to maintaining high compliance and adherence rates for its therapies [13] - The company is actively enrolling in pediatric clinical trials and generating real-world data to support its therapies [24] Q&A Session Summary Question: What led to the higher-than-anticipated rebate for Galafold and Pombility? - Management explained that the rebate was a negotiated rate by the industry association in the UK, which unexpectedly increased from 15% to 22% [46] Question: What has changed regarding the cadence of ex-US launches and patient starts? - Management noted that reimbursement settlements led to delays in patient starts, which are now expected to accelerate in the second half of the year [50][52] Question: Can you elaborate on the diligence process for the DMX200 licensing agreement? - Management highlighted that the diligence included careful review of data, market research, and discussions with physicians, leading to confidence in the asset's differentiation [60] Question: What is the expected timeline for the next interim analysis for DMX200? - Management indicated that the timeline for the next interim analysis could be around nine months, depending on the collaboration with Parasol and the FDA [71] Question: How does the company plan to position Pombility against traditional ERT in new markets? - Management emphasized the importance of demonstrating the product's value proposition and differentiated data to drive adoption in new markets [90]