Amicus Therapeutics(FOLD) - 2025 Q1 - Quarterly Results

Executive Summary 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 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 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][4][8] - 2025 Total Revenue Growth Guidance: Adjusted to 15-22% growth at Constant Exchange Rates (CER)[1][9] - GAAP Profitability: Reaffirmed expectation to achieve GAAP profitability in the second half of 2025[1][3][9] First Quarter 2025 Financial Results 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 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 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 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] Pombiliti® + Opfolda® Net Product Sales 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] Operating Expenses 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 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 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 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 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] - Regulatory Approvals: Received regulatory approvals for adult LOPD patients in Canada and Australia[8] - 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] Manufacturing and Supply Chain 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] - Strategic Objective: Further diversify the Pombiliti supply chain[8] DMX-200 In-Licensing Agreement 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][8] - 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][8] - 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] Long-Term Financial Goals 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] - Growth Drivers: Primarily driven by the continued growth of Galafold and Pombiliti + Opfolda[8] 2025 Financial Guidance 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 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] Guidance for Key Metrics 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 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® 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] - Indication: Used to treat adult patients with Fabry disease who have a treatable alpha-galactosidase A (GLA) gene variant[14][15] - Global Approvals: Approved in over 40 countries worldwide, including the U.S., EU, UK, and Japan[14] U.S. Indications and Usage 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] - 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] U.S. Important Safety Information 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] - 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] About Pombiliti® + Opfolda® 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] - 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] U.S. Indications and Usage 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] Safety Information 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] - Contraindications: POMBILITI combined with Opfolda is contraindicated during pregnancy due to potential embryo-fetal toxicity[20] - Most Common Adverse Reactions: The most common adverse reactions (≥5%) include headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia[20] Company Information 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 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] - Strategic Focus: To advance and expand a best-in-class rare disease pipeline[21] Non-GAAP Financial Measures Explanation 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] - 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] Forward-Looking Statement 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] - 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] Contact Information 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] - Media Contact: Diana Moore, Head of Global Corporate Affairs & Communications, dmoore@amicusrx.com, (609) 662-5079[25] Financial Statements 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 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 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 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) |