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)

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]