Summary of Amicus Therapeutics (FOLD) Conference Call Company Overview - Amicus Therapeutics focuses on developing therapies for rare diseases, currently offering two commercial products: Galafold and Pombility AppFolda for Pompe disease [3][4] Financial Performance - Galafold generated over $128 million in global sales in Q2, maintaining a growth rate of 10% to 15% [4] - Pombility AppFolda achieved $26 million in global sales in Q2, aligning with a growth guidance of 50% to 65% [5] - The company is on track to achieve GAAP profitability in the second half of the year [6] Market Dynamics - The Fabry disease market has seen a significant increase in diagnosed patients, with the treated market doubling from 10,000 to 12,000 patients [10][11] - There are now 6,000 diagnosed but untreated patients, leading to a total diagnosed market of 18,000 patients [11] - The potential for Galafold is growing as more patients are identified, with estimates suggesting that 25,000 diagnosed patients could exist in the future [15] Product Insights - Galafold is established as the standard of care for patients with amenable mutations, capturing approximately 70% of the global market share for treated amenable patients [21] - The company has a large registry to support real-world evidence of Galafold's impact on patient outcomes [21] - The demand is shifting towards naive patients, with 80% of demand now coming from newly diagnosed patients [22] Pipeline Development - Amicus has licensed DMX-200 from Dimerix, targeting focal segmental glomerulosclerosis (FSGS), a rare kidney disease with no approved treatments [5][31] - There are an estimated 40,000 to 80,000 patients with FSGS in the U.S., presenting a significant market opportunity [53] - The company aims to combine DMX-200 with standard treatments to improve patient outcomes [41] Future Projections - The company anticipates that Galafold could contribute approximately 60% of projected billion-dollar sales in the coming years [24] - The Pompe disease market is expected to grow from $1.5 billion to $2 billion, with Amicus aiming for a 50% market share [57] - The company is optimistic about the potential for DMX-200 to become a billion-dollar product, especially as it explores additional indications [55] Challenges and Opportunities - The complexity of diseases like FSGS presents challenges in targeting effective treatments [35] - There is a need for improved diagnostic capabilities, particularly for underrepresented patient populations [19] - The company is focused on building its pipeline and leveraging its financial strength to support growth [62] Conclusion - Amicus Therapeutics is positioned for growth with its existing products and new pipeline developments, particularly in the rare disease space, while maintaining a strong financial outlook and commitment to patient care [61][62]

Core Argument - The U.S. Court of Appeals for the Federal Circuit ruled that President Trump's unilateral imposition of tariffs under the International Emergency Economic Powers Act (IEEPA) is unlawful, affirming that IEEPA does not grant the President authority to impose tariffs [1][2][3] Summary by Sections Legal Ruling - The Federal Circuit's decision validates the New Civil Liberties Alliance's (NCLA) argument that IEEPA does not authorize any tariffs, only defensive actions like sanctions or asset freezes [2] - The court did not explicitly rule on whether IEEPA includes any tariff power but confirmed that the extensive emergency tariff actions taken by President Trump are not authorized [1][3] Implications of the Ruling - The judgment vacated the U.S. Court of International Trade's (CIT) injunction that blocked the enforcement of the emergency tariffs, remanding the case for the CIT to decide on a nationwide injunction [3] - The government has previously argued that the legality of IEEPA tariffs should be litigated in the CIT, and now that it has lost, it should not avoid a nationwide injunction [3] Statements from NCLA - NCLA expressed satisfaction with the ruling, emphasizing that future decisions should conclude that IEEPA does not authorize any tariffs [4] - The organization highlighted that this ruling is consistent with previous court decisions that have deemed unilateral Executive Branch taxes imposed without congressional approval as unlawful [4] - NCLA called for a "universal" injunction due to the number of countries affected by the tariffs [4]

Core Insights - Amicus Therapeutics will participate in several investor conferences in September 2025, highlighting its engagement with the investment community [1][4]. Company Overview - Amicus Therapeutics is a global biotechnology company dedicated to developing high-quality medicines for rare diseases, emphasizing a patient-centric approach [3]. - The company is focused on advancing a pipeline of innovative, first- or best-in-class therapies for rare diseases [3]. Upcoming Events - The company will present at the Cantor Global Healthcare Conference on September 3, 2025, at 10:20 a.m. ET [4]. - Additionally, Amicus Therapeutics will participate in the Morgan Stanley Annual Global Healthcare Conference on September 9, 2025, at 3:20 p.m. ET [4]. - Live audio webcasts of these presentations will be available on the company's investor relations website [2].

Core Viewpoint - The Association of American Physicians and Surgeons (AAPS) is advocating for the restoration of the religious exemption to vaccination in California, arguing that the removal of this exemption is an infringement on religious liberty [1][2]. Group 1: Legal Context - California enacted SB 277 in 2016, which eliminated the religious exemption for ten vaccines and also removed the personal belief exemption [1]. - The AAPS argues that the historical context of vaccination laws, such as the 1905 Jacobson v. Massachusetts case, is not comparable to the current situation, as the previous law allowed for a small fine to avoid vaccination [3]. Group 2: Impact on Individuals - The removal of the religious exemption has resulted in Californians being denied entry to schools unless they comply with vaccine requirements, which AAPS claims disproportionately affects those with religious objections [2][4]. - The AAPS highlights that the exclusion from school can limit career opportunities for children, contrasting it with the minor penalties in historical cases [3][4]. Group 3: Religious Objections - AAPS points out that major religions, including Christianity, Judaism, Islam, Hinduism, and Buddhism, have specific objections to certain vaccine ingredients, which justifies the need for exemptions [4]. - Specific examples include the use of porcine gelatin in vaccines, which is prohibited in Orthodox Jewish and Muslim practices, and the rejection of vaccines derived from aborted fetal tissue by many Christians [5]. Group 4: Organizational Background - The AAPS is a national organization representing physicians across all specialties since 1943 and publishes the Journal of American Physicians and Surgeons [6].

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)