Financial Data and Key Metrics Changes - Total revenue for Q2 2025 reached $154.7 million, a 22% increase compared to Q2 2024, with an 18% growth at constant exchange rates [24] - Galafold revenue was $128.9 million, up 12% at constant exchange rates and 16% in reported terms [10] - Non-GAAP net income for Q2 2025 was $1.9 million, compared to $18.5 million in Q2 2024 [26] Business Line Data and Key Metrics Changes - Galafold saw a 13% year-over-year patient growth, with over 69% global market share for treated Fabry patients with amenable mutations [6][10] - Pombility and Upholda revenue reached $25.8 million, up 58% at constant exchange rates, with the U.S. contributing approximately 42% of revenue [14][15] - The company expects Pombility and Upholda to achieve a combined revenue growth of 50% to 65% for 2025 at constant exchange rates [16] Market Data and Key Metrics Changes - The U.S. market remains a significant driver, with over 1,000 patient starts for Galafold since launch [11] - The company launched Pombility and Upholda in six new countries in Q2 2025, expanding its market presence [18] - The untreated population for Fabry disease is estimated to be significantly larger than previously believed, indicating substantial market opportunity [13] Company Strategy and Development Direction - The company aims to achieve GAAP profitability in the second half of 2025, maintaining financial discipline while expanding its product portfolio [9] - Strategic partnerships, such as with Dimerix for DMX200, are key to addressing unmet needs in rare diseases [7][21] - The company is focused on expanding into new markets and increasing patient access, with plans for further geographic expansion in 2026 [85] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory for the remainder of 2025, citing strong demand for Galafold and the potential of Pombility and Upholda [5][6] - The company highlighted the importance of real-world evidence in driving physician and patient adoption of its therapies [33] - Management remains optimistic about the long-term potential of its products, particularly in underdiagnosed populations [13] Other Important Information - The company reiterated its full-year financial guidance for 2025, expecting total revenue growth of 15% to 22% [27] - Cash, cash equivalents, and marketable securities were $231 million as of June 30, 2025, down from $250 million at the end of 2024 [26] Q&A Session Summary Question: What clinical assessments resonate with physicians and KOLs for switching from Nexviazyme to Pombility? - Management indicated that physicians look for stability or improvement in patient outcomes, including biomarkers and quality of life measures [32][34] Question: Update on the additional manufacturing facility in Ireland? - The company expects the Irish facility to come online in the latter half of 2025, which will help meet global demand [38][40] Question: Confidence in IP position and potential for settlement? - Management remains confident in the strength of its IP and the potential for favorable settlements, reinforced by previous agreements [46][48] Question: Timelines for the new U.S. manufacturing process and impacts on COGS? - The U.S. drug product manufacturing facility is progressing, with timelines not yet specified, but the company is optimistic about its overall manufacturing strategy [52][54] Question: Insights on patient starts in ex-U.S. markets? - The company reported strong market share growth in the UK and Germany, with ongoing launches in new countries [62][64] Question: Drivers for patient switches at the two-year mark? - Management noted that switches are influenced by patient outcomes and physician assessments, with a focus on proactive treatment strategies [68][70] Question: Regulatory alignment with the FDA on proteinuria for DMX200? - The FDA has indicated that proteinuria can serve as a primary endpoint, which is a positive development for the program [74][80]

Company Performance - Amicus Therapeutics reported quarterly earnings of $0.01 per share, missing the Zacks Consensus Estimate of $0.02 per share, and down from $0.06 per share a year ago, representing an earnings surprise of -50.00% [1] - The company posted revenues of $154.69 million for the quarter ended June 2025, surpassing the Zacks Consensus Estimate by 4.87%, compared to year-ago revenues of $126.67 million [2] - Over the last four quarters, Amicus Therapeutics has surpassed consensus EPS estimates just once and topped consensus revenue estimates two times [2] Stock Performance - Amicus Therapeutics shares have lost about 34.9% since the beginning of the year, while the S&P 500 has gained 8.2% [3] - The current status of estimate revisions translates into a Zacks Rank 4 (Sell) for the stock, indicating expected underperformance in the near future [6] Future Outlook - The current consensus EPS estimate for the coming quarter is $0.11 on revenues of $164.66 million, and $0.32 on revenues of $621.22 million for the current fiscal year [7] - The outlook for the industry, specifically the Medical - Biomedical and Genetics sector, is currently in the bottom 41% of over 250 Zacks industries, which may impact stock performance [8]

Financial Performance - Q2 2025 total revenue reached $154.7 million, an 18% increase at CER (Constant Exchange Rates)[61] - Non-GAAP net income for Q2 2025 was $1.9 million[61] - The company expects to surpass $1 billion in total revenue in FY 2028[6] - The company reiterates FY 2025 total revenue growth guidance of 15% to 22% at CER[65,67] Product Performance - Galafold Q2 2025 revenue was $128.9 million, a 16% increase in reported sales growth[10] - The company anticipates Galafold revenue growth of 10% to 15% for FY 2025 at CER[4,65,67] - Pombiliti + Opfolda Q2 2025 revenue was $25.8 million, a 58% increase at CER[22] - The company anticipates Pombiliti + Opfolda revenue growth of 50% to 65% for FY 2025 at CER[5,25,65,67] Strategic Initiatives - The company acquired U S licensing of DMX-200 Phase 3 Program[4] - The company aims to deliver positive GAAP net income during H2 2025[65,67]

[Q2 2025 Financial Results and Corporate Updates](index=1&type=section&id=Amicus%20Therapeutics%20Announces%20Second%20Quarter%202025%20Financial%20Results%20and%20Corporate%20Updates) Amicus Therapeutics reports strong Q2 2025 financial results and significant corporate advancements [Financial Performance](index=1&type=section&id=Second%20Quarter%202025%20Financial%20Highlights) 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](index=4&type=chunk) [Business and Operational Highlights](index=1&type=section&id=Corporate%20Updates) 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](index=2&type=chunk) - 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](index=13&type=chunk) - 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](index=2&type=chunk)[13](index=13&type=chunk) [2025 Financial Guidance](index=3&type=section&id=2025%20Financial%20Guidance) 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](index=3&type=section&id=Financial%20Position) 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](index=6&type=chunk) 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](index=4&type=section&id=Product%20Portfolio%20Overview) Overview of Amicus Therapeutics' key product offerings, Galafold and Pombiliti + Opfolda, detailing their indications and mechanisms [Galafold](index=4&type=section&id=About%20Galafold) 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](index=15&type=chunk) - The most common adverse reactions (≥10%) include headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia[16](index=16&type=chunk) [Pombiliti + Opfolda](index=5&type=section&id=About%20Pombiliti%20%2B%20Opfolda) 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](index=18&type=chunk) - 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](index=19&type=chunk) [Appendix: Detailed Financial Statements](index=10&type=section&id=Appendix%3A%20Detailed%20Financial%20Statements) Comprehensive financial statements including consolidated operations, balance sheets, and non-GAAP reconciliations for Q2 2025 [Consolidated Statements of Operations](index=10&type=section&id=TABLE%201%20Amicus%20Therapeutics%2C%20Inc.%20Consolidated%20Statements%20of%20Operations) 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](index=11&type=section&id=TABLE%202%20Amicus%20Therapeutics%2C%20Inc.%20Consolidated%20Balance%20Sheets) 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](index=12&type=section&id=TABLE%203%20Amicus%20Therapeutics%2C%20Inc.%20Reconciliation%20of%20Non-GAAP%20Financial%20Measures) 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 |

Core Insights - Amicus Therapeutics reported strong second quarter growth of 18% in total revenue, marking the seventeenth consecutive quarter of double-digit gains at constant exchange rates (CER) [3][4] - The company anticipates reaching GAAP profitability in the second half of 2025 and projects total revenue to exceed $1 billion by 2028 [3][4] Financial Performance - Total revenue for Q2 2025 was $154.7 million, up 18% at CER, with a currency tailwind contributing an additional $5 million or 4% [4] - Galafold generated revenue of $128.9 million, reflecting a year-over-year increase of 16% or 12% at CER [5][12] - Pombiliti + Opfolda revenue reached $25.8 million, representing a year-over-year increase of 63% or 58% at CER [5][12] - Total GAAP operating expenses for Q2 2025 were $148.9 million, an increase of 48% compared to $100.4 million in Q2 2024 [5][12] Product Insights - Galafold is an oral pharmacological chaperone for the treatment of Fabry disease, approved in over 40 countries [13] - Pombiliti + Opfolda is a two-component therapy for late-onset Pompe disease, recently granted regulatory approval in Japan for adult patients [16][12] Future Guidance - The company reiterates its 2025 financial guidance, expecting total revenue growth of 15% to 22%, with Galafold revenue growth projected at 10% to 15% and Pombiliti + Opfolda revenue growth at 50% to 65% [7] - Gross margin is expected to be in the mid-80% range, with non-GAAP operating expenses projected between $380 million and $400 million [7] Corporate Developments - The ACTION3 study of DMX-200 is on track for full enrollment by year-end, with the company having entered a licensing agreement with Dimerix for U.S. commercialization [12][3] - Cash, cash equivalents, and marketable securities totaled $231.0 million as of June 30, 2025, reflecting a decrease from $249.9 million at the end of 2024 [12]

"Congress never authorized SEC to build this unconstitutional panopticon for which the people most harmed— innocent American investors—must pay. Nor would Congress ever pass such intrusive legislation costing 'hundreds of millions of dollars' more than SEC's top estimate. This kind of excess is why the Founders placed the power of the purse in the legislature, and the legislature alone. Decisions about government surveillance and financial exaction first must not violate the Constitution and second may only ...

Core Viewpoint - The market anticipates a year-over-year decline in earnings for Amicus Therapeutics despite higher revenues, with the actual results being crucial for stock price movement [1][2]. Earnings Expectations - Amicus Therapeutics is expected to report earnings of $0.02 per share, reflecting a year-over-year decrease of 66.7%, while revenues are projected to be $147.5 million, an increase of 16.4% from the previous year [3]. - The earnings report is scheduled for July 31, and better-than-expected results could lead to a stock price increase, whereas disappointing results may cause a decline [2]. Estimate Revisions - The consensus EPS estimate has been revised 10% higher in the last 30 days, indicating a collective reassessment by analysts [4]. - The Most Accurate Estimate for Amicus Therapeutics is higher than the Zacks Consensus Estimate, resulting in an Earnings ESP of +191.67%, although the stock has a Zacks Rank of 4, complicating predictions of an earnings beat [12]. Earnings Surprise History - In the last reported quarter, Amicus Therapeutics was expected to post earnings of $0.08 per share but only achieved $0.03, resulting in a surprise of -62.50% [13]. - Over the past four quarters, the company has surpassed consensus EPS estimates twice [14]. Industry Comparison - NeoGenomics, another company in the Zacks Medical - Biomedical and Genetics industry, is expected to report earnings of $0.03 per share, unchanged from the previous year, with revenues projected at $182.93 million, up 11.2% [18]. - NeoGenomics has an Earnings ESP of -22.22% and a Zacks Rank of 2, making it difficult to predict an earnings beat despite a history of surpassing estimates [19][20].

Core Viewpoint - Amicus Therapeutics will host a conference call on July 31, 2025, to discuss its financial results for Q2 2025 [1] Group 1: Conference Call Details - The conference call is scheduled for 8:30 a.m. ET on July 31, 2025 [1] - Participants must register online to access the call by phone, receiving a dial-in number and personal PIN after registration [2] - A live audio webcast and related presentation materials will be available on the company's investor website [2] Group 2: Post-Event Information - An archived webcast and accompanying slides will be accessible on the company's website shortly after the live event concludes [3] Group 3: Company Overview - Amicus Therapeutics is a global biotechnology company focused on developing high-quality medicines for rare diseases [4] - The company emphasizes patient dedication and aims to advance a pipeline of innovative medicines [4]

Core Viewpoint - Kaskela Law LLC is investigating Amicus Therapeutics, Inc. on behalf of its shareholders due to a significant decline in stock value and potential violations of securities laws or breaches of fiduciary duties by the company's officers and directors [1][2]. Group 1: Stock Performance - Year to date, shares of Amicus' stock have declined over 32% in value, trading at a current price of less than $7.00 per share [2]. Group 2: Legal Investigation - The investigation aims to determine if Amicus and/or its officers and directors violated securities laws or breached fiduciary duties in connection with recent corporate actions [2]. - Shareholders are encouraged to contact Kaskela Law LLC for additional information regarding the investigation and their legal rights [3].

Financial Performance & Growth - Total revenue for Q1 2025 reached $125 million, representing a 15% growth [6] - Galafold revenue in Q1 2025 was $1042 million, with a 14% patient demand growth [23] - Pombiliti + Opfolda revenue in Q1 2025 was $21 million, showing a 92% growth at CER [50] - The company expects to surpass $1 billion in total revenue in FY 2028 [6] Strategic Priorities & Guidance - The company aims for a total revenue growth of 15-22% at CER in FY 2025 [7, 77] - Galafold revenue is projected to grow by 10-15% at CER in FY 2025 [6, 7] - Pombiliti + Opfolda revenue is expected to increase by 50-65% at CER in FY 2025 [6, 7, 53, 77] - The company anticipates positive GAAP net income during H2 2025 [7, 77] Product & Market Overview - The global Fabry market is projected to reach approximately $3 billion by 2029 [14, 15] - The global Pompe market was approximately $15 billion in 2024 and is expected to exceed $2 billion by 2029 [42, 43] - DMX-200, a Phase 3 program for FSGS, addresses a market affecting over 40000 people in the US [67]