[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].

PRINCETON, N.J., July 21, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that the Company will host a conference call and live audio webcast on Thursday, July 31, 2025, at 8:30 a.m. ET to discuss financial results for the second quarter ended June 30, 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 ...

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]

Core Insights - Amicus Therapeutics has received approval from Japan's Ministry of Health, Labour and Welfare for Pombiliti (cipaglucosidase alfa) in combination with Opfolda (miglustat) to treat adult patients with late-onset Pompe disease [1][4]. Company Overview - Amicus Therapeutics is a biotechnology company focused on developing high-quality medicines for rare diseases, emphasizing patient dedication and innovative treatments [9][10]. Product Details - Pombiliti + Opfolda is a two-component therapy designed for increased uptake into muscle cells, with Pombiliti being a recombinant human GAA enzyme and Opfolda serving as an enzyme stabilizer [3][5]. - The therapy is indicated for adult patients with late-onset Pompe disease who weigh 40 kg or more and are not improving on current enzyme replacement therapy [6]. Clinical Study - The approval was based on clinical data from the Phase 3 pivotal study (PROPEL), which included both ERT-naïve and ERT-experienced participants [4]. Market Approval - With this approval, Pombiliti + Opfolda is now authorized in multiple regions including the U.S., E.U., U.K., Canada, Australia, Switzerland, and Japan [4]. Disease Background - Late-onset Pompe disease is an inherited lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase, leading to glycogen accumulation and progressive muscle weakness [8].

Core Viewpoint - The U.S. Supreme Court ruled that individuals adversely affected by FDA orders can challenge the agency in court, affirming the right of retailers to contest FDA decisions that impact their sales [1][2][3]. Group 1: Legal Context - Under the Family Smoking Prevention and Tobacco Control Act (TCA), manufacturers must obtain FDA approval to sell certain vaping products, such as R.J. Reynolds' "Vuse" e-cigarettes [2]. - The FDA denied R.J. Reynolds' application, which led to a ban on the sale of Vuse e-cigarettes by retailers who were previously allowed to sell them while the application was pending [2]. - Retailers argued that the FDA's decision adversely affected them by resulting in lost sales, leading to a petition for review in the U.S. Court of Appeals for the Fifth Circuit [2]. Group 2: Court Rulings - The Fifth Circuit ruled that retailers have standing to challenge the FDA's decision, rejecting the FDA's argument that only the manufacturer could be considered "adversely affected" under the TCA [2]. - The Supreme Court affirmed the Fifth Circuit's judgment, reinforcing the broad interpretation of "adversely affected" as it relates to judicial review of agency actions [3]. Group 3: Implications of the Ruling - The ruling prevents the FDA from narrowing the scope of judicial review provisions under the TCA and other statutes, allowing those harmed by agency actions to seek relief in federal court [3]. - The decision is seen as a significant affirmation of the ability for individuals and entities to challenge federal regulations, even if they were not directly involved in the agency's decision-making process [4].

Core Viewpoint - The Association of American Physicians and Surgeons (AAPS) filed an amicus brief in the U.S. Supreme Court challenging Colorado's ban on conversion therapy for minors, arguing it violates free speech rights of licensed counselors [1][2]. Group 1: Legal Context - Colorado and many blue states restrict therapists from assisting teenagers with gender dysphoria and same-sex attractions, while allowing support for transgender transitions [2]. - The Supreme Court case focuses on whether licensed counselors have the right to provide gender support therapy, rather than the therapy's effectiveness [2]. Group 2: First Amendment Rights - AAPS argues that professionals such as physicians and therapists should retain their First Amendment rights post-licensure, which they had prior to obtaining their licenses [3]. - The brief emphasizes that government censorship of therapists constitutes viewpoint discrimination, violating the First Amendment [2][4]. Group 3: Implications of the Case - AAPS urges the Supreme Court to invalidate Colorado's ban, which could lead to the negation of similar laws in approximately half the states [5]. - The brief references Justice Clarence Thomas's dissent regarding viewpoint-based discrimination in counseling minors about gender dysphoria [4].