Amicus Therapeutics(FOLD) - 2025 Q2 - Quarterly Results

Q2 2025 Financial Results and Corporate Updates Amicus Therapeutics reports strong Q2 2025 financial results and significant corporate advancements Financial Performance Amicus reported strong Q2 2025 total revenue growth, driven by product sales, despite a widened GAAP net loss due to increased operating expenses Q2 2025 Revenue Performance (YoY) | Product | Q2 2025 Revenue (in millions) | Q2 2024 Revenue (in millions) | Reported Growth | Growth at CER | | :--- | :--- | :--- | :--- | :--- | | Total Net Product Revenues | $154.7 | $126.7 | 22% | 18% | | Galafold | $128.9 | $110.8 | 16% | 12% | | Pombiliti + Opfolda | $25.8 | $15.9 | 63% | 58% | Q2 2025 Profitability Metrics | Metric | Q2 2025 (in millions) | Q2 2024 (in millions) | | :--- | :--- | :--- | | GAAP Net Loss | ($24.4) | ($15.7) | | GAAP Loss Per Share | ($0.08) | ($0.05) | | Non-GAAP Net Income | $1.9 | $18.5 | | Non-GAAP Income Per Share | $0.01 | $0.06 | - Total GAAP operating expenses increased by 48% to $148.9 million, and non-GAAP operating expenses rose 56% to $127.8 million. Both figures include the $30 million upfront payment for the U.S. licensing agreement of DMX-200[4] Business and Operational Highlights The company anticipates exceeding $1 billion in revenue by 2028, with key regulatory approvals and clinical trial progress - The company is positioned to achieve GAAP profitability in the second half of 2025 and anticipates exceeding $1 billion in total revenue in 2028[2] - Pombiliti + Opfolda received regulatory approval in Japan for adult LOPD patients and the company is on track for launches in up to 10 new countries in 2025[13] - The ACTION3 Phase 3 study of DMX-200, a treatment for the rare kidney disease FSGS, is on track for full enrollment by the end of the year. Amicus acquired exclusive U.S. commercialization rights from Dimerix[2][13] 2025 Financial Guidance Amicus reiterates full-year 2025 financial guidance, projecting continued revenue growth and GAAP profitability in H2 2025 Full-Year 2025 Guidance | Metric | Guidance | | :--- | :--- | | Total Revenue Growth | 15% to 22% | | Galafold Revenue Growth | 10% to 15% | | Pombiliti + Opfolda Revenue Growth | 50% to 65% | | Gross Margin | Mid 80% | | Non-GAAP Operating Expenses | $380M to $400M | | GAAP Net Income | Positive during H2 2025 | Financial Position Amicus maintained a solid financial position with $231.0 million in cash, reflecting strategic investments in DMX-200 licensing - Cash, cash equivalents, and marketable securities totaled $231.0 million at June 30, 2025. The current cash position reflects the $30 million upfront payment made in Q2 for the U.S. licensing of DMX-200[6] Key Balance Sheet Items (as of June 30, 2025) | Account | June 30, 2025 (in millions) | December 31, 2024 (in millions) | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $158.7 | $213.8 | | Total current assets | $538.0 | $504.7 | | Total Assets | $815.3 | $785.0 | | Liabilities and Stockholders' Equity | | | | Total current liabilities | $167.6 | $148.7 | | Long-term debt | $391.3 | $390.1 | | Total liabilities | $611.0 | $591.0 | | Total stockholders' equity | $204.3 | $194.0 | | Total Liabilities and Stockholders' Equity | $815.3 | $785.0 | Product Portfolio Overview Overview of Amicus Therapeutics' key product offerings, Galafold and Pombiliti + Opfolda, detailing their indications and mechanisms Galafold Galafold is an oral pharmacological chaperone for adults with Fabry disease, approved globally for amenable GLA gene variants - Indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant[15] - The most common adverse reactions (≥10%) include headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia[16] Pombiliti + Opfolda Pombiliti + Opfolda is a two-component therapy for adult late-onset Pompe disease patients not improving on current ERT - Indicated for adult patients with late-onset Pompe disease (weighing ≥40 kg) who are not improving on their current enzyme replacement therapy (ERT)[18] - The therapy carries a boxed warning for hypersensitivity reactions, infusion-associated reactions (IARs), and risk of acute cardiorespiratory failure in susceptible patients. It is contraindicated in pregnancy[19] Appendix: Detailed Financial Statements Comprehensive financial statements including consolidated operations, balance sheets, and non-GAAP reconciliations for Q2 2025 Consolidated Statements of Operations Summary of Amicus Therapeutics' unaudited consolidated statements of operations for the three and six months ended June 30, 2025 Consolidated Statements of Operations (Unaudited, in thousands) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | | | 2025 | 2024 | 2025 | 2024 | | Net product sales | $154,688 | $126,669 | $279,937 | $237,072 | | Gross profit | $139,471 | $115,408 | $253,022 | $212,244 | | Total operating expenses | $148,945 | $100,441 | $270,448 | $224,998 | | Loss from operations | ($9,474) | $14,967 | ($17,426) | ($12,754) | | Net loss attributable to common stockholders | ($24,420) | ($15,697) | ($46,106) | ($64,116) | | Net loss per share — basic and diluted | ($0.08) | ($0.05) | ($0.15) | ($0.21) | Consolidated Balance Sheets Overview of Amicus Therapeutics' unaudited consolidated balance sheets as of June 30, 2025, and December 31, 2024 Consolidated Balance Sheets (Unaudited, in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | $158,702 | $213,752 | | Total current assets | $538,007 | $504,736 | | Total Assets | $815,303 | $785,033 | | Liabilities and Stockholders' Equity | | | | Total current liabilities | $167,647 | $148,702 | | Long-term debt | $391,322 | $390,111 | | Total liabilities | $610,999 | $590,988 | | Total stockholders' equity | $204,304 | $194,045 | | Total Liabilities and Stockholders' Equity | $815,303 | $785,033 | Reconciliation of Non-GAAP Financial Measures Reconciliation of GAAP to Non-GAAP financial measures, including operating expenses and net income, for Q2 2025 Reconciliation of GAAP to Non-GAAP Operating Expenses (Unaudited, in thousands) | | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | Total operating expenses - as reported GAAP | $148,945 | $100,441 | | Total operating expense adjustments to reported GAAP | $21,113 | $18,379 | | Total operating expenses - as adjusted (Non-GAAP) | $127,832 | $82,062 | Reconciliation of GAAP Net Loss to Non-GAAP Net Income (Unaudited, in thousands) | | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | | :--- | :--- | :--- | | GAAP net loss | ($24,420) | ($15,697) | | Adjustments (Share-based comp, D&A, etc.) | $26,352 | $34,184 | | Non-GAAP net income | $1,932 | $18,487 |