PresentationMaxwell SkorEquity Analyst Okay. Great. I'm Max Skor, a biotech analyst with Morgan Stanley. I'm happy to be hosting Amicus Therapeutics. With us today is Sébastien Martel, Chief Business Officer; Jeffrey Castelli, Chief Development Officer. But before we dive in, I just want to note for important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representat ...

Amicus Therapeutics (NasdaqGM:FOLD) FY Conference September 09, 2025 03:20 PM ET Company ParticipantsMaxwell Skor - VP - Biotech Equity ResearchSébastien Martel - Chief Business OfficerJeffrey Castelli - Chief Development OfficerMaxwell SkorI'm Max Score, a biotech analyst with Morgan Stanley. I'm happy to be hosting Amicus Therapeutics. With us today is Sebastian Martell, chief business officer Jeff Castelli, chief development officer. But before we dive in, I just want to note for important disclosures, p ...

Long-Term Data Adds to the Growing Body of Evidence Supporting the Compelling Clinical Profile of Pombiliti® + Opfolda®PRINCETON, N.J., Sept. 08, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), today announced the presentation of new 4-year muscle function, muscle strength and biomarker endpoints from the PROPEL open-label extension (OLE) study of cipaglucosidase alfa-atga + miglustat (cipa+mig) in adults with late-onset Pompe disease (LOPD) at the International Congress of Inborn Errors of Met ...

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]

PRINCETON, N.J., Aug. 29, 2025 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD) today announced that management will participate in upcoming presentations at the following investor conferences in September. Cantor Global Healthcare Conference 2025 in New York, NY, on Wednesday, September 3, 2025, at 10:20 a.m. ETMorgan Stanley 23rd Annual Global Healthcare Conference 2025 in New York, NY on Tuesday, September 9, 2025, at 3:20 p.m. ET A live audio webcast of each presentation can also be accessed via t ...

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]