Core Viewpoint - Amicus Therapeutics (FOLD) is positioned as a strong investment opportunity due to its improving earnings outlook and positive analyst sentiment [1][2]. Earnings Estimate Revisions - Analysts are increasingly optimistic about Amicus Therapeutics' earnings prospects, leading to a rising trend in estimate revisions, which is expected to positively impact the stock price [2]. - The consensus earnings estimate for the current quarter is $0.12 per share, reflecting a year-over-year increase of +20.0%, with a 15% rise in estimates over the last 30 days [5]. - For the full year, the expected earnings are $0.31 per share, indicating a year-over-year change of +29.2%, with a 33.33% increase in consensus estimates due to positive revisions [6][7]. Zacks Rank and Performance - Amicus Therapeutics has achieved a Zacks Rank 2 (Buy), indicating favorable conditions for investment based on the positive estimate revisions [8]. - Historically, stocks with a Zacks Rank 1 (Strong Buy) and 2 (Buy) have significantly outperformed the S&P 500, suggesting strong potential for Amicus Therapeutics [8]. Stock Performance - The stock has gained 12.9% over the past four weeks, driven by solid estimate revisions and positive earnings growth prospects, making it a candidate for portfolio consideration [9].

Core Viewpoint - Amicus Therapeutics (FOLD) shares have increased by 12.9% recently, with a mean price target of $16.27 suggesting a potential upside of 138.2% from the current price of $6.83 [1] Price Targets and Analyst Consensus - The average of 11 short-term price targets ranges from a low of $9.00 to a high of $22.00, with a standard deviation of $3.85, indicating variability among analysts [2] - The lowest estimate suggests a 31.8% increase, while the highest points to a 222.1% upside [2] - A low standard deviation indicates a strong agreement among analysts regarding the price movement of FOLD [9] Earnings Estimates and Market Sentiment - Analysts have shown increasing optimism about FOLD's earnings prospects, with a strong consensus in revising EPS estimates higher [11] - Over the last 30 days, the Zacks Consensus Estimate for the current year has risen by 33.3%, with no negative revisions [12] - FOLD holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimates [13] Conclusion on Price Movement - While the consensus price target may not be a reliable indicator of the extent of FOLD's potential gains, the implied direction of price movement appears to be a useful guide [14]

Core Insights - Amicus Therapeutics reported strong quarterly results for Q2 2025, exceeding expectations in both GAAP revenue and non-GAAP earnings, with GAAP revenue of $154.7 million, a 22.1% increase year-over-year, and non-GAAP EPS of $0.01, significantly better than the estimated –$0.12 [1][2] Financial Performance - GAAP revenue reached $154.7 million, up from $126.7 million a year earlier, surpassing the analyst target of $146.7 million [1][2] - Non-GAAP EPS was $0.01, compared to an estimate of –$0.12 and a previous year EPS of $0.06, reflecting an 83.3% decline year-over-year [2] - Galafold revenue was $128.9 million, a 16.3% increase from $110.8 million in Q2 2024 [2][5] - Pombiliti + Opfolda revenue increased by 62.8% to $25.8 million, up from $15.9 million a year earlier [2][6] - Non-GAAP operating expenses rose to $127.8 million, a 55.7% increase from $82.1 million in the previous year [2][8] Business Focus and Strategy - Amicus Therapeutics specializes in treatments for rare diseases, particularly genetic disorders, with key products being Galafold for Fabry disease and Pombiliti + Opfolda for Pompe disease [3] - The company is focused on broadening patient access, securing regulatory approvals, and expanding into related rare disease therapies [4] - The majority of sales are driven by Galafold, which is now approved in over 40 countries, with high global patient demand [5] Recent Developments - The company licensed U.S. rights for DMX-200, a late-stage drug for focal segmental glomerulosclerosis, paying a $30 million upfront fee [7] - Operating expenses increased due to the licensing payment and ongoing investments in research and development [8] Financial Outlook - Amicus reaffirmed its 2025 revenue growth outlook of 15–22% at constant exchange rates, with Galafold projected to grow 10–15% and Pombiliti + Opfolda expected to increase 50–65% [9] - The company anticipates gross margin to normalize in the mid-80% range for 2025 and aims for positive GAAP net income in the second half of 2025 [9] Key Monitoring Points - Investors should watch for progress in new country launches for Pombiliti + Opfolda, patient onboarding, and any changes in operating expenses or regulatory landscapes that could impact growth [10]

Group 1 - Amicus Therapeutics (NASDAQ: FOLD) is gaining momentum in the commercialization of its approved drug candidates, POMBILITI and OPFOLDA, aimed at treating patients with late-stage conditions [2] - The company is part of the Biotech Analysis Central service, which provides in-depth analysis of various pharmaceutical companies and includes a model portfolio of small and mid-cap stocks [2] Group 2 - The Biotech Analysis Central service offers a library of over 600 biotech investing articles and live chat features to assist healthcare investors in making informed decisions [2] - The subscription for the Biotech Analysis Central service is priced at $49 per month, with a yearly plan available at a discounted rate of $399, reflecting a 33.50% discount [1]

Core Insights - Amicus Therapeutics reported adjusted earnings of 1 cent per share for Q2 2025, missing the Zacks Consensus Estimate of 2 cents, compared to a loss of 6 cents per share in the same quarter last year [1][6] Financial Performance - Revenues for Q2 2025 totaled $154.7 million, representing a 22% year-over-year increase on a reported basis and an 18% increase at constant exchange rates, surpassing the Zacks Consensus Estimate of $148 million [2][6] - Galafold sales reached $128.9 million, up 12% year-over-year at constant exchange rates, although it fell short of the Zacks Consensus Estimate of $133 million [4][6] - Sales of the newly approved combination drug Pombiliti + Opfolda amounted to $25.8 million, exceeding both the Zacks Consensus Estimate of $24 million and the internal model estimate of $25 million [4][6] Growth Drivers - The revenue increase was attributed to higher sales from Galafold and additional revenues from Pombiliti + Opfolda, with ongoing studies aimed at expanding treatment labels for both Fabry and Pompe diseases [3][6] - The company maintained its 2025 revenue growth guidance of 15-22% at constant exchange rates, with Galafold expected to grow by 10-15% and Pombiliti + Opfolda by 50-65% [8] Operating Expenses and Cash Position - Adjusted operating expenses rose 56% to $127.8 million in Q2 2025, primarily due to a $30 million upfront payment for licensing rights to DMX-200 [7] - As of June 30, 2025, Amicus had cash, cash equivalents, and marketable securities totaling $231 million, down from $250.6 million as of March 31, 2025 [7] Future Outlook - The company expects to achieve positive GAAP net income in the second half of 2025 and aims to exceed $1 billion in total sales by 2028 [9] - Amicus has licensed exclusive rights for the commercialization of DMX-200, a potential first-in-class treatment for FSGS, with the pivotal phase III study on track for full enrollment by the end of 2025 [10]

PART I. FINANCIAL INFORMATION [Item 1. Consolidated Financial Statements and Notes (unaudited)](index=3&type=section&id=Item%201.%20Consolidated%20Financial%20Statements%20and%20Notes%20(unaudited)) This section provides unaudited consolidated financial statements, highlighting product sales growth, increased net loss for the quarter, and decreased cash [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) Total assets increased to **$815.3 million** by June 30, 2025, while cash and equivalents decreased to **$158.7 million** Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $158,702 | $213,752 | | Total current assets | $538,007 | $504,736 | | Total Assets | $815,303 | $785,033 | | 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 | [Consolidated Statements of Operations](index=6&type=section&id=Consolidated%20Statements%20of%20Operations) Net product sales grew to **$154.7 million** in Q2 2025, but increased R&D expenses led to a **$24.4 million** net loss Quarterly Statement of Operations (in thousands, except per share data) | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | Net product sales | $154,688 | $126,669 | | Gross profit | $139,471 | $115,408 | | Research and development | $60,848 | $24,683 | | Selling, general, and administrative | $84,543 | $73,576 | | Loss from operations | $(9,474) | $14,967 | | Net loss | $(24,420) | $(15,697) | | Net loss per share | $(0.08) | $(0.05) | Six-Month Statement of Operations (in thousands, except per share data) | Metric | H1 2025 | H1 2024 | | :--- | :--- | :--- | | Net product sales | $279,937 | $237,072 | | Gross profit | $253,022 | $212,244 | | Research and development | $88,687 | $53,012 | | Selling, general, and administrative | $176,370 | $161,605 | | Loss from operations | $(17,426) | $(12,754) | | Net loss | $(46,106) | $(64,116) | | Net loss per share | $(0.15) | $(0.21) | [Consolidated Statements of Cash Flows](index=8&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operating activities increased to **$18.8 million** for H1 2025, resulting in a **$54.7 million** net cash decrease Cash Flow Summary for Six Months Ended June 30 (in thousands) | Activity | 2025 | 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(18,768) | $(7,004) | | Net cash used in investing activities | $(38,639) | $(14,440) | | Net cash used in financing activities | $(12,100) | $(14,166) | | **Net decrease in cash** | **$(54,701)** | **$(37,720)** | | Cash at end of period | $162,015 | $212,357 | [Notes to Consolidated Financial Statements](index=9&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) Notes detail business operations, accounting policies, product revenue growth, and the **$30 million** Dimerix licensing agreement - The company markets Galafold® and Pombiliti® + Opfolda® for rare diseases, approved in major global markets[33](index=33&type=chunk) - An exclusive license agreement with Dimerix for DMX-200 involved a **$30 million** upfront payment and up to **$560 million** in potential milestones[33](index=33&type=chunk)[65](index=65&type=chunk) Net Product Sales by Product and Geography (in thousands) | Product/Region | Q2 2025 | Q2 2024 | H1 2025 | H1 2024 | | :--- | :--- | :--- | :--- | :--- | | **Total Galafold® sales** | **$128,872** | **$110,817** | **$233,116** | **$210,176** | | - Galafold® U.S. | $53,600 | $42,770 | $93,766 | $76,885 | | - Galafold® Ex-U.S. | $75,272 | $68,047 | $139,350 | $133,291 | | **Total Pombiliti® + Opfolda® sales** | **$25,816** | **$15,852** | **$46,821** | **$26,896** | | - Pombiliti® + Opfolda® U.S. | $10,730 | $7,368 | $20,557 | $10,628 | | - Pombiliti® + Opfolda® Ex-U.S. | $15,086 | $8,484 | $26,264 | $16,268 | | **Total net product sales** | **$154,688** | **$126,669** | **$279,937** | **$237,072** | - Inventories increased to **$154.9 million** by June 30, 2025, from **$118.8 million** at year-end 2024, supporting commercial growth[62](index=62&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=19&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial performance, strategic progress, and liquidity, highlighting commercial growth and the DMX-200 licensing deal [Overview and Strategy](index=21&type=section&id=Overview%20and%20Strategy) The company's strategy focuses on developing and commercializing rare disease therapies, expanding its pipeline with DMX-200 - The company's strategy involves developing and delivering high-quality rare disease medicines through various means[82](index=82&type=chunk) - Pipeline advancement includes licensing exclusive U.S. commercialization rights for DMX-200, a Phase 3 candidate for FSGS[81](index=81&type=chunk)[88](index=88&type=chunk) - Continued focus remains on Fabry and Pompe disease franchises, with ongoing discovery for next-generation therapies[82](index=82&type=chunk)[90](index=90&type=chunk) [Consolidated Results of Operations](index=23&type=section&id=Consolidated%20Results%20of%20Operations) Q2 2025 net product sales increased **22%** to **$154.7 million**, while R&D expenses surged due to a **$30.0 million** license payment Q2 2025 vs Q2 2024 Financial Comparison (in thousands) | Metric | Q2 2025 | Q2 2024 | Change | | :--- | :--- | :--- | :--- | | Net product sales | $154,688 | $126,669 | $28,019 | | Research and development | $60,848 | $24,683 | $36,165 | | Selling, general, and administrative | $84,543 | $73,576 | $10,967 | | Net loss | $(24,420) | $(15,697) | $(8,723) | - The **$36.2 million** increase in Q2 R&D costs was primarily due to the **$30.0 million** upfront Dimerix license payment[93](index=93&type=chunk) H1 2025 vs H1 2024 Financial Comparison (in thousands) | Metric | H1 2025 | H1 2024 | Change | | :--- | :--- | :--- | :--- | | Net product sales | $279,937 | $237,072 | $42,865 | | Research and development | $88,687 | $53,012 | $35,675 | | Selling, general, and administrative | $176,370 | $161,605 | $14,765 | | Net loss | $(46,106) | $(64,116) | $18,010 | [Liquidity and Capital Resources](index=26&type=section&id=Liquidity%20and%20Capital%20Resources) The company held **$231.0 million** in cash and equivalents, with **$18.8 million** net cash used in operations for H1 2025 - Total cash, cash equivalents, and marketable securities amounted to **$231.0 million** as of June 30, 2025[88](index=88&type=chunk)[102](index=102&type=chunk) - Net cash used in operations for H1 2025 was **$18.8 million**, reflecting net loss and increased inventory[103](index=103&type=chunk) - An at-the-market (ATM) equity program has **$164.2 million** remaining capacity as of June 30, 2025[101](index=101&type=chunk) - Management believes current cash is sufficient to fund operations and research programs for at least the next 12 months[110](index=110&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=27&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) No material changes to market risks or their management have occurred since December 31, 2024 - No material changes to the company's market risks have occurred since December 31, 2024[115](index=115&type=chunk) [Item 4. Controls and Procedures](index=27&type=section&id=Item%204.%20Controls%20and%20Procedures) Disclosure controls and procedures were effective as of June 30, 2025, with no material changes to internal controls - Disclosure controls and procedures were concluded effective by key officers as of the reporting period end[116](index=116&type=chunk) - No material changes to internal control over financial reporting occurred during the last fiscal quarter[117](index=117&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=27&type=section&id=Item%201.%20Legal%20Proceedings) The company is involved in Hatch-Waxman litigation for Galafold®, settling with Teva while continuing cases against others - Litigation is ongoing against Teva, Aurobindo, and Lupin concerning generic Galafold® ANDA filings[118](index=118&type=chunk) - A settlement with Teva grants a generic Galafold® license in the U.S. starting January 30, 2037, resolving their litigation[119](index=119&type=chunk) - Litigation remains active against Aurobindo, with a stay in place for the Lupin case[119](index=119&type=chunk) [Item 1A. Risk Factors](index=27&type=section&id=Item%201A.%20Risk%20Factors) No material changes to risk factors were reported compared to the prior Annual Report on Form 10-K - No material changes to risk factors were reported compared to the Annual Report on Form 10-K for fiscal year 2024[120](index=120&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=27&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities occurred, but **28,748** shares were purchased to cover tax obligations - No unregistered sales of equity securities occurred during the period[121](index=121&type=chunk) Issuer Purchases of Equity Securities (Q2 2025) | Period | Total Shares Purchased | Average Price Paid per Share | | :--- | :--- | :--- | | April 2025 | 4,374 | $6.92 | | May 2025 | 16,494 | $6.31 | | June 2025 | 7,880 | $6.04 | | **Total** | **28,748** | **$6.33** | - Purchased shares represent common stock withheld to satisfy taxes from restricted stock unit vesting[122](index=122&type=chunk) [Item 5. Other Information](index=28&type=section&id=Item%205.%20Other%20Information) No Rule 10b5-1 trading arrangement changes occurred, and the Cash Deferral Plan was frozen by the Board - No director or officer adopted, modified, or terminated a Rule 10b5-1 trading arrangement during the quarter[125](index=125&type=chunk) - The Board of Directors froze the company's Cash Deferral Plan on July 29, 2025, preventing new deferrals[126](index=126&type=chunk) [Item 6. Exhibits](index=29&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including the Equity Incentive Plan and Dimerix license agreement - Key exhibits include the 2025 Equity Incentive Plan, loan agreement amendments, and the Dimerix Biosciences license agreement[128](index=128&type=chunk)

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 |