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 ...

Core Points - The U.S. Court of Appeals for the D.C. Circuit has stayed a district court's permanent injunction, allowing President Trump to remove Hampton Dellinger from the U.S. Office of Special Counsel [1] - Dellinger announced he will end his lawsuit following the circuit court's ruling, which affirms the President's authority to remove top executive branch officials [1][3] - The New Civil Liberties Alliance (NCLA) supports the D.C. Circuit's decision, emphasizing the President's constitutional authority to remove executive officials [1][4] Summary by Sections Legal Authority - The Constitution grants the President broad authority to remove executive branch officials, as confirmed by the Supreme Court in the 2020 Seila Law case [2] - The removal authority is essential for the President to ensure that laws are faithfully executed, and there are no constitutional limits on this power [2][3] Court Rulings - A federal district court judge overstepped by attempting to bar the President from firing a top executive branch official, which is a violation of the President's Article II authority [3] - The D.C. Circuit's reversal of the district court's decision is seen as a correction of judicial overreach [3] NCLA Statements - NCLA representatives expressed satisfaction with the D.C. Circuit's decision, highlighting the importance of maintaining the separation of powers [4][5] - The organization criticized the district court's attempt to dictate who serves in the Executive Branch, reaffirming that executive power resides with the President [4]

Core Insights - Amicus Therapeutics will participate in several investor conferences in March 2025, indicating active engagement with the investment community [1][4] Company Overview - Amicus Therapeutics is a global biotechnology company dedicated to developing high-quality medicines for rare diseases, emphasizing patient focus and innovation [3] - The company aims to advance a pipeline of first- or best-in-class medicines, showcasing its commitment to addressing unmet medical needs in rare diseases [3] Upcoming Events - The company will present at the TD Cowen 45th Annual Health Care Conference in Boston, MA, on March 4, 2025, at 9:50 a.m. ET [4] - Additionally, Amicus Therapeutics will participate in the Leerink Partners 2025 Global Healthcare Conference in Miami, FL, on March 10, 2025, at 10:00 a.m. ET [4]

Core Viewpoint - Amicus Therapeutics reported mixed financial results for Q4 2024, with adjusted earnings missing estimates but revenues showing significant year-over-year growth driven by product sales, particularly from Galafold and the newly approved Pombiliti + Opfolda [1][2][3]. Financial Performance - Adjusted earnings for Q4 2024 were 9 cents per share, below the Zacks Consensus Estimate of 10 cents, compared to 1 cent per share in the same quarter last year [1]. - Revenues for Q4 totaled $149.7 million, reflecting a 30% increase year over year, slightly exceeding the Zacks Consensus Estimate of $149 million [2]. - Galafold sales reached $127.5 million in Q4, up 20% year over year at constant currency, but fell short of the Zacks Consensus Estimate of $141 million [5]. - Net product sales of Pombiliti + Opfolda were $22.2 million, showing a sequential growth of 5.2%, narrowly missing the Zacks Consensus Estimate of $22.3 million [6]. Full-Year Results - For the full year 2024, Amicus generated revenues of $528.3 million, a 33% increase year over year [9]. - The company reported adjusted earnings of 24 cents per share, a significant improvement from a loss of 13 cents per share in the previous year [9]. 2025 Guidance - Amicus reiterated its revenue guidance for 2025, expecting total revenues to grow between 17%-24% on a constant exchange rate [10]. - Revenue from Galafold is anticipated to grow by 10%-15% at constant exchange rates in 2025, while Pombiliti + Opfolda is expected to see growth of 65%-85% [10]. - Total adjusted operating expenses are projected to be between $350 million and $370 million [10]. Cash Position - As of December 31, 2024, Amicus had cash, cash equivalents, and marketable securities totaling $249.9 million, slightly up from $249.8 million as of September 30, 2024 [8]. Patent Litigation Settlement - In October 2024, Amicus signed a licensing agreement with Teva Pharmaceuticals, resolving a patent lawsuit regarding Galafold, preventing Teva from selling a generic version until January 2037 [12][13].

Core Insights - Amicus Therapeutics reported a revenue of $149.71 million for the quarter ended December 2024, reflecting a year-over-year increase of 30.1% [1] - The earnings per share (EPS) for the quarter was $0.09, a significant improvement from -$0.11 in the same quarter last year [1] - The revenue fell short of the Zacks Consensus Estimate by 0.13%, while the EPS was 10.00% below the consensus estimate of $0.10 [1] Financial Performance Metrics - Geographic net product sales outside the U.S. were $87.26 million, which is 21.4% higher year-over-year but below the average estimate of $94.04 million [4] - U.S. geographic net product sales reached $62.44 million, exceeding the average estimate of $55.85 million and representing a 44.6% increase compared to the previous year [4] - Net product sales for Pombiliti and Opfolda totaled $22.21 million, slightly below the average estimate of $22.35 million [4] - Net product sales for Galafold were reported at $127.50 million, which is lower than the average estimate of $140.98 million but shows a year-over-year increase of 19.6% [4] Stock Performance - Over the past month, shares of Amicus Therapeutics have returned +0.2%, while the Zacks S&P 500 composite increased by +2.6% [3] - The stock currently holds a Zacks Rank 2 (Buy), indicating potential for outperformance in the near term [3]