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.

PRINCETON, N.J., June 03, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced the publication of a post-hoc analysis of data from the ERT-experienced cohort of the PROPEL study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) in Muscle and Nerve. In this new publication, based on a within group effect-size analysis, subjects who switched from alglucosidase alfa to cipa+mig achieved improvements or stability in most of the outcomes me ...

PRINCETON, N.J., May 30, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that management will participate in upcoming presentations at the following investor conferences in June. Jefferies Global Healthcare Conference 2025 in New York, NY, on Wednesday, June 4, 2025, at 11:40 a.m. ETGoldman Sachs 46th Annual Global Healthcare Conference 2025 in Miami, FL, on Tuesday, June 10, 2025, at 2:00 p.m. ET A live audio webcast of each presentation can also be accessed via the investors se ...

Summary of Amicus Therapeutics Conference Call Company Overview - **Company**: Amicus Therapeutics - **Industry**: Biotechnology, specifically focusing on rare diseases and therapies Key Points and Arguments Macro Environment and Regulatory Impact - Recent executive orders, particularly the most favored nation (MFN) pricing, could impact Amicus's revenue structure, with approximately 60% of Galafold's revenue coming from outside the US and 40% from within, including Medicare and Medicaid exposure [4][5][6] - Tariffs are not expected to significantly affect Amicus in 2025 due to existing inventory and a transition from China to Ireland for manufacturing [7][8][10] Product Performance and Market Dynamics - **Galafold**: Expected to surpass $500 million in sales this year, driven by growth in the US, Germany, and the UK, with a focus on diagnosing underdiagnosed Fabry disease [15][16][20] - The company is leveraging AI to identify potential Fabry patients through medical records, which could enhance patient diagnosis and treatment [17][18] - The amenability rate for Galafold is projected to increase to over 50% in new patient diagnoses, enhancing market share [20][21] Growth Strategy - Future growth will primarily come from diagnosing new patients rather than switching existing patients from other treatments [19][24] - There is a significant pool of diagnosed but untreated patients, estimated at 6,000 to 7,000, which presents an opportunity for growth [24][25] Pompe Disease Treatment - **Pompe Disease**: The company anticipates a revenue growth guidance of 50% to 65%, with April showing the strongest demand since launch [31][32] - The company is expanding into new reimbursed markets like Italy and the Netherlands, with a focus on existing markets for growth [33][34] - Transitioning patients from existing therapies to Amicus's products is expected to accelerate as more data becomes available [38][39] Acquisition Strategy - Amicus is pursuing a cautious acquisition strategy, focusing on in-licensing products that align with its existing portfolio, such as DMX 200, which targets FSGS [53][54][58] - The acquisition of DMX 200 is seen as a strategic move to leverage existing infrastructure and address a significant unmet medical need in the US market [58][59] Financial Outlook - The company aims to achieve GAAP profitability while managing risks associated with new acquisitions and product launches [60] Additional Important Insights - The company is focused on balancing shareholder interests with growth opportunities, avoiding over-dilution while pursuing strategic investments [55][56] - There is a strong emphasis on educating physicians about the benefits of switching to Amicus's therapies, particularly in the context of progressive diseases like Pompe [47][48] This summary encapsulates the key discussions and insights from the Amicus Therapeutics conference call, highlighting the company's strategic direction, product performance, and market opportunities.

About Amicus Therapeutics Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company's website at www.amicusrx.com, and follow on X and LinkedIn. ...

Core Viewpoint - Amicus Therapeutics reported disappointing first-quarter 2025 earnings, with adjusted earnings per share of 3 cents, missing the consensus estimate of 8 cents, and revenues of $125.2 million, which also fell short of expectations [1][2]. Financial Performance - Adjusted earnings per share for Q1 2025 were 3 cents, compared to a loss of 2 cents per share in the same quarter last year [1]. - Revenues reached $125.2 million, reflecting a 13% year-over-year increase on a reported basis and a 15% increase on a constant-currency basis, but missed the consensus estimate of $136 million [1][4]. - Galafold sales amounted to $104.2 million, up 6% year-over-year at constant currency, but also missed the estimate of $113 million [4]. - Net product sales of Pombiliti + Opfolda were $21 million, below the consensus estimate of $23 million [5]. Market Reaction - Following the earnings release, Amicus shares declined by 13% on May 1, 2025 [2]. Guidance and Future Outlook - For full-year 2025, management revised revenue growth expectations to 15-22% on a constant exchange rate, down from the previous guidance of 17-24% [9]. - Galafold revenue growth is still expected to be in the range of 10-15% at constant exchange rates [9]. - Pombiliti + Opfolda revenue growth is now projected at 50-65% at constant exchange rates, reduced from 65-85% [9]. - Total adjusted operating expenses are anticipated to be between $380-$400 million, up from the previous guidance of $350-$370 million [10]. - The company aims to achieve positive GAAP net income in the second half of 2025 and targets over $1 billion in total sales by 2028 [10]. Strategic Developments - Amicus has licensed exclusive rights for Dimerix's phase III program, DMX-200, for FSGS treatment, with an upfront payment of $30 million and potential milestone payments of up to $560 million [11][12]. - A licensing agreement with Teva Pharmaceuticals was signed to resolve a patent lawsuit, preventing Teva from selling a generic version of Galafold in the U.S. until January 2037 [13].

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 (Address of Principal Executive Offices) (Zip Code) (609) 662-2000 (Registrant's Telephone Number, Including Area Code) Securities registered pursuant to Section 12(b) of the Act: For the t ...

Amicus Therapeutics (FOLD) came out with quarterly earnings of $0.03 per share, missing the Zacks Consensus Estimate of $0.08 per share. This compares to loss of $0.02 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -62.50%. A quarter ago, it was expected that this pharmaceutical company would post earnings of $0.10 per share when it actually produced earnings of $0.09, delivering a surprise of -10%.Over the last four quarters ...

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]