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]

Financial Performance - Total revenue for Q1 2025 was $125 million, a 15% increase at CER (constant exchange rates)[4,56] - Galafold revenue for Q1 2025 reached $104.2 million, representing a 14% increase[10,12] - Pombiliti + Opfolda revenue for Q1 2025 was $21 million, a 92% increase at CER[18] - The company expects total revenue to surpass $1 billion in FY 2028[5] - The company is guiding for total revenue growth of 15% to 22% for FY 2025[60] Product Performance and Growth - Galafold is the only approved oral treatment for Fabry disease, holding a 69% share of treated amenable patients[9,10] - The company anticipates Galafold revenue growth of 10% to 15% for FY 2025[4,15,60] - Pombiliti + Opfolda revenue growth guidance is updated to 50% to 65% for FY 2025[5,20,60] - Approximately 2,730 individuals have been treated with Galafold as of the end of 2024[10] DMX-200 and Strategic Initiatives - The company has an exclusive U S license agreement with Dimerix for DMX-200, a Phase 3 program for FSGS (Focal Segmental Glomerulosclerosis)[4,34,76] - The upfront investment for DMX-200 is $30 million, with potential success-based milestone payments up to $560 million and tiered royalties[38] - DMX-200 targets a market of over 40,000 people in the U S with FSGS, a rare and fatal kidney disease[37]

[Executive Summary](index=1&type=section&id=Executive%20Summary) Amicus Therapeutics reported strong Q1 2025 performance with double-digit revenue growth, driven by key products, and expanded its pipeline with DMX-200, reaffirming its goal for GAAP profitability in H2 2025 [First Quarter 2025 Performance Overview](index=1&type=section&id=1.1%20First%20Quarter%202025%20Performance%20Overview) Amicus Therapeutics achieved significant double-digit revenue growth in Q1 2025, driven by strong performance from Galafold and Pombiliti + Opfolda, with the company on track for GAAP profitability in H2 2025 Q1 2025 Key Financial Data | Metric | Q1 2025 | Q1 2024 | | :----------------------- | :------------- | :------------- | | Total Revenue | **$125.2M** | $110.4M | | Year-over-Year Growth (CER) | **15%** | - | | GAAP Net Loss | **$(21.7)M** | $(48.4)M | | GAAP Loss Per Share | **$(0.07)** | $(0.16) | | Non-GAAP Net Income (Loss) | **$9.0M** | $(4.6)M | | Non-GAAP Income (Loss) Per Share | **$0.03** | $(0.02) | [Strategic Developments & Outlook](index=1&type=section&id=1.2%20Strategic%20Developments%20%26%20Outlook) Amicus expanded its portfolio by in-licensing DMX-200 for a rare kidney disease, aligning with its strategy to leverage existing commercial infrastructure, and updated 2025 revenue guidance while reaffirming H2 2025 GAAP profitability - DMX-200 In-Licensing: Secured exclusive U.S. commercialization rights for Dimerix's Phase III DMX-200, a therapy for FSGS, a rare and fatal kidney disease[1](index=1&type=chunk)[4](index=4&type=chunk)[8](index=8&type=chunk) - 2025 Total Revenue Growth Guidance: Adjusted to **15-22% growth at Constant Exchange Rates (CER)**[1](index=1&type=chunk)[9](index=9&type=chunk) - GAAP Profitability: Reaffirmed expectation to achieve **GAAP profitability in the second half of 2025**[1](index=1&type=chunk)[3](index=3&type=chunk)[9](index=9&type=chunk) [First Quarter 2025 Financial Results](index=1&type=section&id=First%20Quarter%202025%20Financial%20Results) Amicus Therapeutics demonstrated strong Q1 2025 financial performance with significant revenue growth, reduced GAAP net loss, and a positive non-GAAP net income, while maintaining a stable cash position [Total Revenue](index=1&type=section&id=2.1%20Total%20Revenue) Total revenue reached $125.2 million in Q1 2025, achieving 15% operational growth at constant exchange rates (CER) despite a 1% foreign exchange headwind Total Revenue (Q1 2025) | Metric | Amount | | :--- | :--- | | Total Revenue | **$125.2M** | | Operational Growth (CER) | **15%** | | Foreign Exchange Headwind | $1.4M (1%) | [Product-Specific Revenue](index=1&type=section&id=2.2%20Product-Specific%20Revenue) Both Galafold and Pombiliti + Opfolda achieved significant revenue growth in Q1 2025, with Pombiliti + Opfolda showing particularly strong growth, though both were impacted by order timing and UK rebates [Galafold® Net Product Sales](index=1&type=section&id=2.2.1%20Galafold%C2%AE%20Net%20Product%20Sales) Galafold® net product sales showed strong patient demand, partially offset by order timing and UK VPAG rebates Galafold® Net Product Sales (Q1 2025) | Metric | Amount | | :--- | :--- | | Sales | **$104.2M** | | Year-over-Year Growth (CER) | **6%** | | Patient Demand Growth | **14%** | | 2025 Revenue Growth Guidance (CER) | **+10-15%** | - Strong patient demand was partially offset by order timing and the UK VPAG rebate impact[6](index=6&type=chunk) [Pombiliti® + Opfolda® Net Product Sales](index=2&type=section&id=2.2.2%20Pombiliti%C2%AE%20%2B%20Opfolda%C2%AE%20Net%20Product%20Sales) Pombiliti® + Opfolda® net product sales saw significant growth, though Q1 sales were impacted by patient initiation timing in new launch countries and UK VPAG rebates Pombiliti® + Opfolda® Net Product Sales (Q1 2025) | Metric | Amount | | :--- | :--- | | Sales | **$21.0M** | | Year-over-Year Growth (CER) | **92%** | | 2025 Revenue Growth Guidance (CER) | **+50-65%** (previously +65-85%) | - Q1 sales were impacted by patient initiation timing in new launch countries and the UK VPAG rebate, with patient initiation benefits in new markets expected to be more concentrated in the second half of the year[8](index=8&type=chunk) [Operating Expenses](index=2&type=section&id=2.3%20Operating%20Expenses) GAAP operating expenses decreased 2.5% year-over-year to $121.5 million in Q1 2025, while non-GAAP operating expenses increased 10.4% to $94.5 million, reflecting the DMX-200 upfront license fee Operating Expenses (Q1 2025) | Metric | Q1 2025 | Q1 2024 | | :----------------------- | :------------- | :------------- | | GAAP Operating Expenses | **$121.5M** | $124.6M | | Year-over-Year Change | **-2.5%** | - | | Non-GAAP Operating Expenses | **$94.5M** | $85.6M | | Year-over-Year Change | **+10.4%** | - | [Net Income (Loss) and EPS](index=2&type=section&id=2.4%20Net%20Income%20(Loss)%20and%20EPS) Amicus significantly reduced GAAP net loss to $21.7 million ($0.07 per share) in Q1 2025, compared to $48.4 million ($0.16 per share) in Q1 2024, achieving non-GAAP net income of $9.0 million ($0.03 per share) this quarter Net Income (Loss) and EPS (Q1 2025) | Metric | Q1 2025 | Q1 2024 | | :----------------------- | :------------- | :------------- | | GAAP Net Loss | **$(21.7)M** | $(48.4)M | | GAAP Loss Per Share | **$(0.07)** | $(0.16) | | Non-GAAP Net Income (Loss) | **$9.0M** | $(4.6)M | | Non-GAAP Income (Loss) Per Share | **$0.03** | $(0.02) | [Cash Position](index=2&type=section&id=2.5%20Cash%20Position) As of March 31, 2025, total cash, cash equivalents, and marketable securities slightly increased to $250.6 million from $249.9 million on December 31, 2024 Cash, Cash Equivalents, and Marketable Securities | Date | Amount | | :--- | :--- | | March 31, 2025 | **$250.6M** | | December 31, 2024 | $249.9M | [Corporate Updates & Strategic Initiatives](index=2&type=section&id=Corporate%20Updates%20%26%20Strategic%20Initiatives) Amicus advanced its strategic goals through Pombiliti + Opfolda market expansion, diversified its supply chain, in-licensed DMX-200 for a rare kidney disease, and reiterated its long-term revenue target of over $1 billion by 2028 [Pombiliti + Opfolda Market Expansion & Approvals](index=2&type=section&id=3.1%20Pombiliti%20%2B%20Opfolda%20Market%20Expansion%20%26%20Approvals) Pombiliti + Opfolda was selected as the preferred treatment for late-onset Pompe disease in the Netherlands, with commercial treatment expected to begin in Q2 2025, and also received regulatory approvals in Canada and Australia, with further expansion planned - Netherlands Agreement: Pombiliti + Opfolda selected as the preferred treatment for adult patients with late-onset Pompe disease in the Netherlands, with commercial treatment expected to begin in **Q2 2025**[8](index=8&type=chunk) - Regulatory Approvals: Received regulatory approvals for adult LOPD patients in **Canada** and **Australia**[8](index=8&type=chunk) - Market Expansion: Anticipates a regulatory decision in **Japan** this year and plans to launch in up to **10 new countries** in 2025, covering over 650 LOPD patients[8](index=8&type=chunk) [Manufacturing and Supply Chain](index=2&type=section&id=3.2%20Manufacturing%20and%20Supply%20Chain) Amicus entered a commercial manufacturing and supply services agreement with Sharp Sterile to produce Pombiliti drug product in the U.S., further diversifying the Pombiliti supply chain - Manufacturing Agreement: Partnered with Sharp Sterile for the production of Pombiliti drug product in the U.S[8](index=8&type=chunk) - Strategic Objective: Further diversify the Pombiliti supply chain[8](index=8&type=chunk) [DMX-200 In-Licensing Agreement](index=2&type=section&id=3.3%20DMX-200%20In-Licensing%20Agreement) Amicus secured exclusive U.S. commercialization rights for Dimerix's Phase III DMX-200, a first-in-class therapy for FSGS, a rare and fatal kidney disease with significant market potential, leveraging Amicus's rare disease commercial infrastructure - Licensing Agreement: Acquired exclusive U.S. commercialization rights for Dimerix's Phase III program, **DMX-200**[4](index=4&type=chunk)[8](index=8&type=chunk) - Product Characteristics: DMX-200 is a first-in-class therapy for FSGS, a rare and fatal kidney disease with no approved therapies and significant market potential[4](index=4&type=chunk)[8](index=8&type=chunk) - Strategic Fit: Perfectly aligns with the company's strategy to leverage its rare disease commercial infrastructure, adding a third program with blockbuster market potential to the portfolio[4](index=4&type=chunk) [Long-Term Financial Goals](index=2&type=section&id=3.4%20Long-Term%20Financial%20Goals) Amicus is committed to significant long-term revenue growth, projecting over $1 billion in total sales by 2028, driven primarily by the continued growth of Galafold and Pombiliti + Opfolda - Long-Term Revenue Target: Anticipates total sales to exceed **$1 billion by 2028**[8](index=8&type=chunk) - Growth Drivers: Primarily driven by the continued growth of **Galafold** and **Pombiliti + Opfolda**[8](index=8&type=chunk) [2025 Financial Guidance](index=3&type=section&id=2025%20Financial%20Guidance) Amicus updated its 2025 financial guidance, adjusting total revenue and Pombiliti + Opfolda growth projections while maintaining Galafold's outlook and reaffirming the target for GAAP profitability in the second half of the year [Updated Guidance Overview](index=3&type=section&id=4.1%20Updated%20Guidance%20Overview) Amicus updated its 2025 financial guidance to include the DMX-200 upfront license fee and all anticipated operating expenses, with total revenue growth guidance adjusted downwards, while Galafold's guidance remained unchanged and Pombiliti + Opfolda's was also revised - Guidance Inclusion: Includes the upfront license fee for the DMX-200 in-licensing and all other anticipated operating expenses[9](index=9&type=chunk) [Guidance for Key Metrics](index=3&type=section&id=4.2%20Guidance%20for%20Key%20Metrics) The company adjusted total revenue growth guidance to 15-22% CER (previously 17-24%) and Pombiliti + Opfolda growth to 50-65% CER (previously 65-85%), while Galafold revenue growth guidance remains 10-15% CER, gross margin around 80%, and non-GAAP operating expenses increased to $380-400 million due to the DMX-200 upfront payment, with GAAP net income still projected positive in H2 2025 2025 Updated Financial Guidance | Metric | Previous Guidance | Updated Guidance | Change | | :------------------------ | :---------------- | :--------------- | :----- | | Total Revenue Growth (CER) | 17% to 24% | **15% to 22%** | ↓ | | Galafold Revenue Growth (CER) | 10% to 15% | **10% to 15%** | No Change | | Pombiliti + Opfolda Growth (CER) | 65% to 85% | **50% to 65%** | ↓ | | Gross Margin | Around 80% | **Around 80%** | No Change | | Non-GAAP Operating Expenses | $350M to $370M | **$380M to $400M** | ↑ (includes $30M upfront license fee) | | GAAP Net Income | Positive in H2 2025 | **Positive in H2 2025** | No Change | [Product Information](index=3&type=section&id=Product%20Information) This section provides detailed information on Galafold and Pombiliti + Opfolda, including their mechanisms of action, U.S. indications, usage, and important safety information for patients and healthcare providers [About Galafold®](index=3&type=section&id=5.1%20About%20Galafold%C2%AE) Galafold (migalastat) is an oral pharmacological chaperone for adults with Fabry disease who have treatable GLA variants, stabilizing dysfunctional enzymes to clear disease substrate accumulation, and is approved in over 40 countries worldwide - Mechanism of Action: An oral pharmacological chaperone for alpha-galactosidase A (alpha-Gal A)[14](index=14&type=chunk) - Indication: Used to treat adult patients with Fabry disease who have a treatable alpha-galactosidase A (GLA) gene variant[14](index=14&type=chunk)[15](index=15&type=chunk) - Global Approvals: Approved in over **40 countries** worldwide, including the U.S., EU, UK, and Japan[14](index=14&type=chunk) [U.S. Indications and Usage](index=3&type=section&id=5.1.1%20U.S.%20Indications%20and%20Usage) Galafold is indicated for adults with Fabry disease who have a treatable alpha-galactosidase A (GLA) gene variant, with continued approval contingent on verification of clinical benefit in confirmatory trials - Galafold is indicated for adult patients with Fabry disease who have a treatable alpha-galactosidase A (GLA) gene variant, as confirmed by an in vitro assay[15](index=15&type=chunk) - This indication is approved under accelerated approval based on reduction in kidney interstitial capillary globotriaosylceramide (KIC GL-3) substrate, with continued approval potentially contingent upon verification and description of clinical benefit in confirmatory trials[16](index=16&type=chunk) [U.S. Important Safety Information](index=5&type=section&id=5.1.2%20U.S.%20Important%20Safety%20Information) Important safety information for Galafold includes common adverse reactions and considerations for use in special populations like pregnant women and patients with severe renal impairment - Most Common Adverse Reactions: The most common adverse reactions (≥10%) include headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia[17](index=17&type=chunk) - Use in Specific Populations: Clinical data on Galafold use in pregnant women are insufficient to inform drug-associated risks for major birth defects and miscarriage; it is not recommended for patients with severe renal impairment or end-stage renal disease requiring dialysis[17](index=17&type=chunk) [About Pombiliti® + Opfolda®](index=5&type=section&id=5.2%20About%20Pombiliti%C2%AE%20%2B%20Opfolda%C2%AE) Pombiliti + Opfolda is a two-component therapy for late-onset Pompe disease, comprising cipaglucosidase alfa-atga (a bis-M6P-enriched rhGAA) and oral enzyme stabilizer miglustat, designed to enhance high-affinity M6P receptor uptake and reduce enzyme activity loss - Composition: Consists of cipaglucosidase alfa-atga (a bis-M6P-enriched rhGAA) and the oral enzyme stabilizer miglustat[18](index=18&type=chunk) - Mechanism of Action: Designed to enhance high-affinity M6P receptor uptake while preserving the enzyme's ability to be processed into its most active form, and to reduce the loss of enzyme activity in the bloodstream[18](index=18&type=chunk) [U.S. Indications and Usage](index=5&type=section&id=5.2.1%20U.S.%20Indications%20and%20Usage) Pombiliti + Opfolda is indicated for adult patients with late-onset Pompe disease weighing ≥40 kg whose current enzyme replacement therapy has not improved their condition - POMBILITI combined with OPFOLDA is indicated for adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing **≥40 kg** who are not improving on their current enzyme replacement therapy (ERT)[19](index=19&type=chunk) [Safety Information](index=6&type=section&id=5.2.2%20Safety%20Information) Safety information for Pombiliti + Opfolda includes warnings for hypersensitivity and infusion-related reactions, contraindications during pregnancy, and common adverse reactions - Warnings: Include risks of hypersensitivity reactions (including anaphylaxis), infusion-associated reactions (IARs), and acute cardiopulmonary failure in susceptible patients[20](index=20&type=chunk) - Contraindications: POMBILITI combined with Opfolda is contraindicated during pregnancy due to potential embryo-fetal toxicity[20](index=20&type=chunk) - Most Common Adverse Reactions: The most common adverse reactions (≥5%) include headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia[20](index=20&type=chunk) [Company Information](index=6&type=section&id=Company%20Information) This section outlines Amicus Therapeutics' mission, explains the use of non-GAAP financial measures, includes forward-looking statements, and provides essential contact information for investors and media [About Amicus Therapeutics](index=6&type=section&id=6.1%20About%20Amicus%20Therapeutics) Amicus Therapeutics is a global, patient-focused biotechnology company dedicated to discovering, developing, and delivering novel, high-quality medicines for rare diseases, while advancing and expanding a best-in-class rare disease pipeline - Company Mission: To discover, develop, and deliver novel, high-quality medicines for rare diseases[21](index=21&type=chunk) - Strategic Focus: To advance and expand a best-in-class rare disease pipeline[21](index=21&type=chunk) [Non-GAAP Financial Measures Explanation](index=6&type=section&id=6.2%20Non-GAAP%20Financial%20Measures%20Explanation) This press release includes non-GAAP financial measures to provide investors and management with supplementary information for evaluating operating performance and trends, facilitating period-over-period comparisons, by excluding certain GAAP items deemed non-core to operations - Purpose: To provide supplementary information for evaluating operating performance and trends, and to facilitate period-over-period comparisons[22](index=22&type=chunk) - Exclusions: Typically exclude certain GAAP items that management believes do not impact core operations and do not meet the definition of non-recurring items under GAAP[22](index=22&type=chunk) [Forward-Looking Statement](index=7&type=section&id=6.3%20Forward-Looking%20Statement) This press release contains forward-looking statements regarding product regulatory and pricing approvals, commercialization, manufacturing, supply, financing plans, collaboration with Dimerix, and projected revenue and cash position, which are subject to risks and uncertainties that may cause actual results to differ materially - Scope: Includes potential regulatory and pricing approvals for products, commercialization plans, manufacturing and supply plans, financing plans, collaboration with Dimerix, and the company's projected revenue and cash position[23](index=23&type=chunk) - Risk Disclaimer: Forward-looking statements may be inaccurate and are subject to inherent risks and uncertainties in the company's business, with actual results potentially differing materially from those in this press release[23](index=23&type=chunk) [Contact Information](index=8&type=section&id=6.4%20Contact%20Information) Contact information is provided for investor relations and media inquiries - Investor Contact: Andrew Faughnan, Vice President, Investor Relations, afaughnan@amicusrx.com, (609) 662-3809[25](index=25&type=chunk) - Media Contact: Diana Moore, Head of Global Corporate Affairs & Communications, dmoore@amicusrx.com, (609) 662-5079[25](index=25&type=chunk) [Financial Statements](index=9&type=section&id=Financial%20Statements) This section presents the unaudited consolidated statements of operations, balance sheets, and reconciliations of GAAP to non-GAAP financial measures for the specified periods [Consolidated Statements of Operations](index=9&type=section&id=7.1%20Consolidated%20Statements%20of%20Operations) This section presents the unaudited consolidated statements of operations for the three months ended March 31, 2025, and 2024, detailing net product sales, cost of sales, operating expenses, and net loss Consolidated Statements of Operations (Unaudited, in thousands) | Metric | March 31, 2025 | March 31, 2024 | | :----------------------------------- | :------------- | :------------- | | Net Product Sales | **$125,249** | $110,403 | | Cost of Sales | 11,698 | 13,567 | | Gross Profit | 113,551 | 96,836 | | Total Operating Expenses | 121,503 | 124,557 | | Operating Loss | (7,952) | (27,721) | | Net Loss Attributable to Common Stockholders | **$(21,686)** | $(48,419) | | Net Loss Per Share Attributable to Common Stockholders (Basic and Diluted) | **$(0.07)** | $(0.16) | [Consolidated Balance Sheets](index=10&type=section&id=7.2%20Consolidated%20Balance%20Sheets) This section provides the unaudited consolidated balance sheets as of March 31, 2025, and December 31, 2024, outlining assets, liabilities, and stockholders' equity Consolidated Balance Sheets (Unaudited, in thousands) | Metric | March 31, 2025 | December 31, 2024 | | :----------------------------------- | :------------- | :---------------- | | Total Current Assets | **$510,799** | $504,736 | | Total Assets | **$789,839** | $785,033 | | Total Current Liabilities | **$152,890** | $148,702 | | Total Liabilities | **$596,281** | $590,988 | | Total Stockholders' Equity | **$193,558** | $194,045 | [Reconciliation of Non-GAAP Financial Measures](index=11&type=section&id=7.3%20Reconciliation%20of%20Non-GAAP%20Financial%20Measures) This section provides a reconciliation of GAAP to non-GAAP operating expenses and net income (loss) for the three months ended March 31, 2025, and 2024, detailing adjustments such as stock-based compensation, depreciation, amortization, and restructuring charges Reconciliation of Non-GAAP Operating Expenses (Unaudited, in thousands) | Metric | March 31, 2025 | March 31, 2024 | | :----------------------------------- | :------------- | :------------- | | Total GAAP Operating Expenses | **$121,503** | $124,557 | | Total Non-GAAP Operating Expense Adjustments | 27,009 | 39,002 | | Total Non-GAAP Operating Expenses | **$94,494** | $85,555 | Reconciliation of Non-GAAP Net Income (Loss) (Unaudited, in thousands, except per share amounts) | Metric | March 31, 2025 | March 31, 2024 | | :----------------------------------- | :------------- | :------------- | | GAAP Net Loss | **$(21,686)** | $(48,419) | | Stock-Based Compensation Expense | 25,172 | 30,803 | | Depreciation and Amortization | 1,837 | 2,154 | | Restructuring Charges | — | 6,045 | | Income Tax Expense | 3,641 | 4,836 | | Non-GAAP Net Income (Loss) | **$8,963** | $(4,581) | | Non-GAAP Net Income (Loss) Per Share | **$0.03** | $(0.02) |

Core Insights - Amicus Therapeutics reported total revenue of $125.2 million for Q1 2025, reflecting a 15% increase year-over-year at constant exchange rates (CER) [1][5][10] - The company is expanding its portfolio by in-licensing DMX-200, a Phase 3 program for a rare kidney disease, which has significant market potential in the U.S. [1][4] - Amicus is maintaining its 2025 guidance for Galafold, indicating strong underlying demand, while updating guidance for Pombiliti + Opfolda due to accelerating new patient starts in the second half of the year [1][3][9] Financial Performance - Total revenues for Q1 2025 were $125.2 million, with a 15% growth at CER, despite a currency headwind of $1.4 million or 1% [5][10] - Galafold net product sales were $104.2 million, a 5% increase year-over-year, while Pombiliti + Opfolda sales surged by 90% to $21.0 million [6][10] - The company adjusted its total revenue growth guidance for 2025 to 15-22% at CER, with Galafold growth expected at 10-15% and Pombiliti + Opfolda growth revised to 50-65% [9][10] Corporate Developments - Amicus has entered into an exclusive U.S. licensing agreement with Dimerix for DMX-200, enhancing its rare disease portfolio [4][10] - The company anticipates surpassing $1 billion in total sales by 2028, driven by the growth of Galafold and Pombiliti + Opfolda [10][23] - Regulatory approvals for Pombiliti + Opfolda have been granted in Canada and Australia, with expectations for further approvals in Japan and additional reimbursement agreements [10][10]

Core Points - Dimerix Limited has entered into an exclusive licensing agreement with Amicus Therapeutics for the commercialization of DMX-200 in the U.S. for the treatment of Focal Segmental Glomerulosclerosis (FSGS) [1][3] - DMX-200 is currently in a pivotal Phase 3 trial, ACTION3, which has shown positive interim results in reducing proteinuria without safety concerns [2][6] - Dimerix will receive an upfront payment of US$30 million and could earn up to US$560 million in success-based milestone payments, along with tiered royalties on net U.S. sales of DMX-200 [1][4] Company Overview - Dimerix is a clinical-stage biopharmaceutical company focused on developing treatments for inflammatory diseases, including kidney diseases, with DMX-200 as its lead candidate for FSGS [10] - Amicus Therapeutics is a biotechnology company dedicated to developing novel medicines for rare diseases, emphasizing patient-focused approaches [11] Financial Terms - The licensing agreement includes potential milestone payments of up to US$75 million until FDA approval, US$35 million upon first sale, and up to US$410 million in commercial sales milestone payments [4] - Dimerix is also eligible for up to US$40 million in milestone payments for future indications of DMX-200 [4] Clinical Development - The ACTION3 study is a multi-center, randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of DMX-200 in patients with FSGS [6][7] - The trial includes interim analysis points to assess proteinuria and kidney function, aiming to generate evidence for marketing approval [7] Market Context - FSGS is a rare and serious kidney disorder affecting over 40,000 people in the U.S., with no FDA-approved therapies currently available [9] - The disease is characterized by progressive scarring in the kidney's filtering units, leading to significant health complications and a high need for new treatments [9]

PRINCETON, N.J., April 21, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the Company will host a conference call and live audio webcast on Thursday, May 1, 2025, at 8:30 a.m. ET to discuss financial results for the first quarter ended March 31, 2025. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN t ...

I covered Amicus Therapeutics, Inc. (NASDAQ: FOLD ) two years ago, when it was waiting for approval for the Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) program in Pompe Disease. That approval happened a month after myAbout the TPT serviceThanks for reading. At the Total Pharma Tracker, we offer the following:-Our Android app and website features a set of tools for DIY investors, including a work-in-progress software where you can enter any ticker and get extensive curated research material ...