I covered Amicus Therapeutics, Inc. (NASDAQ: FOLD ) two years ago, when it was waiting for approval for the Pombiliti® (cipaglucosidase alfa-atga) + Opfolda® (miglustat) program in Pompe Disease. That approval happened a month after myAbout the TPT serviceThanks for reading. At the Total Pharma Tracker, we offer the following:-Our Android app and website features a set of tools for DIY investors, including a work-in-progress software where you can enter any ticker and get extensive curated research material ...

Core Points - The U.S. Court of Appeals for the D.C. Circuit has stayed a district court's permanent injunction, allowing President Trump to remove Hampton Dellinger from the U.S. Office of Special Counsel [1] - Dellinger announced he will end his lawsuit following the circuit court's ruling, which affirms the President's authority to remove top executive branch officials [1][3] - The New Civil Liberties Alliance (NCLA) supports the D.C. Circuit's decision, emphasizing the President's constitutional authority to remove executive officials [1][4] Summary by Sections Legal Authority - The Constitution grants the President broad authority to remove executive branch officials, as confirmed by the Supreme Court in the 2020 Seila Law case [2] - The removal authority is essential for the President to ensure that laws are faithfully executed, and there are no constitutional limits on this power [2][3] Court Rulings - A federal district court judge overstepped by attempting to bar the President from firing a top executive branch official, which is a violation of the President's Article II authority [3] - The D.C. Circuit's reversal of the district court's decision is seen as a correction of judicial overreach [3] NCLA Statements - NCLA representatives expressed satisfaction with the D.C. Circuit's decision, highlighting the importance of maintaining the separation of powers [4][5] - The organization criticized the district court's attempt to dictate who serves in the Executive Branch, reaffirming that executive power resides with the President [4]

Core Insights - Amicus Therapeutics will participate in several investor conferences in March 2025, indicating active 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 patient focus and innovation [3] - The company aims to advance a pipeline of first- or best-in-class medicines, showcasing its commitment to addressing unmet medical needs in rare diseases [3] Upcoming Events - The company will present at the TD Cowen 45th Annual Health Care Conference in Boston, MA, on March 4, 2025, at 9:50 a.m. ET [4] - Additionally, Amicus Therapeutics will participate in the Leerink Partners 2025 Global Healthcare Conference in Miami, FL, on March 10, 2025, at 10:00 a.m. ET [4]

Core Viewpoint - Amicus Therapeutics reported mixed financial results for Q4 2024, with adjusted earnings missing estimates but revenues showing significant year-over-year growth driven by product sales, particularly from Galafold and the newly approved Pombiliti + Opfolda [1][2][3]. Financial Performance - Adjusted earnings for Q4 2024 were 9 cents per share, below the Zacks Consensus Estimate of 10 cents, compared to 1 cent per share in the same quarter last year [1]. - Revenues for Q4 totaled $149.7 million, reflecting a 30% increase year over year, slightly exceeding the Zacks Consensus Estimate of $149 million [2]. - Galafold sales reached $127.5 million in Q4, up 20% year over year at constant currency, but fell short of the Zacks Consensus Estimate of $141 million [5]. - Net product sales of Pombiliti + Opfolda were $22.2 million, showing a sequential growth of 5.2%, narrowly missing the Zacks Consensus Estimate of $22.3 million [6]. Full-Year Results - For the full year 2024, Amicus generated revenues of $528.3 million, a 33% increase year over year [9]. - The company reported adjusted earnings of 24 cents per share, a significant improvement from a loss of 13 cents per share in the previous year [9]. 2025 Guidance - Amicus reiterated its revenue guidance for 2025, expecting total revenues to grow between 17%-24% on a constant exchange rate [10]. - Revenue from Galafold is anticipated to grow by 10%-15% at constant exchange rates in 2025, while Pombiliti + Opfolda is expected to see growth of 65%-85% [10]. - Total adjusted operating expenses are projected to be between $350 million and $370 million [10]. Cash Position - As of December 31, 2024, Amicus had cash, cash equivalents, and marketable securities totaling $249.9 million, slightly up from $249.8 million as of September 30, 2024 [8]. Patent Litigation Settlement - In October 2024, Amicus signed a licensing agreement with Teva Pharmaceuticals, resolving a patent lawsuit regarding Galafold, preventing Teva from selling a generic version until January 2037 [12][13].

Core Insights - Amicus Therapeutics reported a revenue of $149.71 million for the quarter ended December 2024, reflecting a year-over-year increase of 30.1% [1] - The earnings per share (EPS) for the quarter was $0.09, a significant improvement from -$0.11 in the same quarter last year [1] - The revenue fell short of the Zacks Consensus Estimate by 0.13%, while the EPS was 10.00% below the consensus estimate of $0.10 [1] Financial Performance Metrics - Geographic net product sales outside the U.S. were $87.26 million, which is 21.4% higher year-over-year but below the average estimate of $94.04 million [4] - U.S. geographic net product sales reached $62.44 million, exceeding the average estimate of $55.85 million and representing a 44.6% increase compared to the previous year [4] - Net product sales for Pombiliti and Opfolda totaled $22.21 million, slightly below the average estimate of $22.35 million [4] - Net product sales for Galafold were reported at $127.50 million, which is lower than the average estimate of $140.98 million but shows a year-over-year increase of 19.6% [4] Stock Performance - Over the past month, shares of Amicus Therapeutics have returned +0.2%, while the Zacks S&P 500 composite increased by +2.6% [3] - The stock currently holds a Zacks Rank 2 (Buy), indicating potential for outperformance in the near term [3]

Financial Performance - Net product sales increased by $128.9 million to $528.3 million in 2024, driven by growth in Galafold and the launch of Pombiliti + Opfolda[330]. - Net product sales for 2024 reached $528,295, an increase of 32.4% compared to $399,356 in 2023[391]. - Gross profit for 2024 was $475,352, reflecting a gross margin of approximately 90%[391]. - The net loss attributable to common stockholders for 2024 was $56,106, a significant improvement from a loss of $151,584 in 2023[391]. - The net loss for the year ended December 31, 2024, was $56,106 thousand, a decrease from the net loss of $151,584 thousand in 2023 and $236,568 thousand in 2022, indicating a 63.0% improvement year-over-year[399]. - The Company reported a total income (loss) before income taxes of $(28.8) million for 2024, an improvement from $(150.1) million in 2023[497]. Expenses - Cost of goods sold as a percentage of net product sales rose to 10.0% in 2024 from 9.3% in 2023, primarily due to inventory write-offs[330]. - Research and development expenses decreased by $43.0 million to $109.4 million, mainly due to reduced clinical spending following the commercial launch of Pombiliti + Opfolda[331]. - Selling, general, and administrative expenses increased by $48.1 million to $323.4 million, attributed to higher personnel costs for commercial launch activities[331]. - The total operating expenses for 2024 were $450,476, slightly higher than $439,241 in 2023[391]. - The total stock-based compensation expense for 2024 was $84,905 thousand, slightly lower than $86,077 thousand in 2023[399]. - The Company incurred approximately $32.7 million in royalty expenses under the GSK collaboration agreement for the year ended December 31, 2024[506]. Cash Flow and Financing - Cash, cash equivalents, and marketable securities totaled $249.9 million as of December 31, 2024[343]. - Net cash used in operating activities was $33.9 million in 2024, a decrease from $69.1 million in 2023[344][345]. - The company issued 1,732,204 shares through its ATM program, generating net proceeds of $19.6 million in 2024[341]. - The company prepaid the Senior Secured Term Loan due 2026, recognizing a loss on extinguishment of debt of $13.9 million[333]. - Net cash provided by financing activities for the year ended December 31, 2023 was $61.7 million, primarily reflecting $387.4 million from a Senior Secured Loan due in 2029[349]. - The company raised $19,566 thousand from the issuance of shares in connection with at-the-market offerings in 2024, compared to $63,108 thousand in 2023[399]. Assets and Liabilities - Total assets as of December 31, 2024, were $785,033, a slight increase from $777,880 in 2023[389]. - Total liabilities decreased to $590,988 in 2024 from $617,706 in 2023, a reduction of 4.3%[389]. - Stockholders' equity increased to $194,045 in 2024, up from $160,174 in 2023, reflecting a growth of 21.1%[389]. - The company had an accumulated deficit of $2.7 billion as of December 31, 2024, and anticipates continuing to incur losses into the second half of 2025[403]. - The company reported accrued expenses and other current liabilities of $127.3 million as of December 31, 2024, a decrease from $144.2 million in 2023[464]. Research and Development - Total research and development costs for 2024 were $109.4 million, down from $152.4 million in 2023[331]. - Research and development expenses decreased to $109,362 in 2024 from $152,381 in 2023, a reduction of 28.2%[391]. - The company expects to incur significant costs in the foreseeable future primarily due to research and development expenses, including clinical trials[350]. - Research and development costs are expensed as incurred, primarily related to personnel and consulting fees, with no specific figures provided for 2024[426]. Equity and Shares - The weighted-average common shares outstanding increased to 304,380,502 in 2024 from 295,164,515 in 2023[391]. - The total number of potentially issuable shares excluded from the computation due to anti-dilutive effects was 34,290 thousand for the year ended December 31, 2024[510]. - The Company has reserved up to 14,228,754 shares for issuance under the Amended and Restated 2007 Equity Incentive Plan as of December 31, 2024[471]. Debt and Interest - The company has a net carrying value of long-term debt of $390.1 million as of December 31, 2024, compared to $387.9 million in 2023, reflecting a slight increase[486]. - The Senior Secured Term Loan due 2029 has a principal amount of $400 million with an effective interest rate of 12.0% for 2024, down from 12.8% in 2023[491]. - Interest expense for the Company was $47.4 million in 2024, slightly decreasing from $47.6 million in 2023[491]. Commitments and Contingencies - The company has binding minimum purchase and manufacturing commitments totaling $179.7 million, with $102.7 million and $77.0 million expected in 2025 and 2026, respectively[354]. - The Company has a contingent milestone payment of $4.7 million due to GSK as of December 31, 2024[505]. Tax and Regulatory - The gross unrecognized tax benefits increased to $13.8 million in 2024 from $4.9 million in 2023, indicating a significant rise in uncertain tax positions[499]. - Deferred tax assets totaled $617.2 million in 2024, with a valuation allowance of the same amount, reflecting uncertainty regarding the realization of these assets[500]. - The Company has U.S. federal research and experimentation credit carryforwards of approximately $19.1 million, expiring between 2030 and 2043[502]. - The Company has state research and development tax credit carryforwards of $13.5 million, subject to a full valuation allowance[502]. - The Company does not expect the OECD's BEPS Pillar 2 rules to apply until it meets the minimum global revenue threshold[504]. Miscellaneous - The Company operates in one business segment focused on advanced therapies for rare diseases, with performance evaluated based on net product sales and net loss[441]. - The Company entered into a non-exclusive license with Teva allowing them to market a generic version of Galafold in the U.S. starting January 30, 2037[508].

AT THE FOREFRONT OF THERAPIES FOR RARE DISEASES FY24 Results Conference Call & Webcast February 19, 2025 Forward-Looking Statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, co ...

Financial Data and Key Metrics Changes - Total revenue for Amicus Therapeutics in 2024 reached $528 million, representing a 32% year-over-year growth, or 33% on a constant currency basis [10][63] - Galafold revenue was $458 million, reflecting an 18% growth year-on-year, or 19% at constant exchange rates [11][25] - Non-GAAP net income for 2024 was $74 million, compared to a non-GAAP net loss of $39 million in 2023 [65] Business Line Data and Key Metrics Changes - Galafold continued to show strong performance with over 2,700 patients living with Fabry disease on the therapy by the end of 2024 [10] - Pombiliti and Opfolda generated over $70 million in revenue globally, marking significant growth from approximately $12 million in 2023 [40][20] - The company anticipates Galafold revenue growth of 10% to 15% in 2025, while Pombiliti and Opfolda are projected to grow by 65% to 85% at constant exchange rates [22][66] Market Data and Key Metrics Changes - The global market for Fabry disease has expanded, with the number of diagnosed patients increasing from 10,000 at launch to 18,000 by 2024 [12] - The treated market for Fabry disease has more than doubled to 12,000 patients globally [13] - In the Netherlands, approximately 125 patients with late-onset Pompe disease are expected to be treated, highlighting the market's significance [91] Company Strategy and Development Direction - Amicus aims to achieve total revenue growth of 17% to 24% in 2025, with a focus on expanding its portfolio in rare diseases [21][66] - The company is committed to advancing ongoing studies to broaden labels and strengthen scientific leadership in Fabry and Pompe diseases [22] - Amicus plans to leverage its global infrastructure to support the launch of Pombiliti and Opfolda in up to 10 new countries in 2025 [54] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in Amicus's position to achieve its vision of becoming a leading rare disease biotechnology company [9] - The company highlighted the importance of continued commercial execution and financial discipline to support long-term growth [72] - Management noted that the Fabry market is expected to grow robustly, driven by improved diagnosis and medical education [30] Other Important Information - The company achieved full-year non-GAAP profitability in 2024 and aims for positive GAAP net income in the second half of 2025 [21][68] - Amicus is focused on maintaining high compliance and adherence rates, exceeding 90% for its therapies [14][114] - The company is exploring opportunities for additional assets to build upon its existing portfolio [130] Q&A Session Summary Question: Insights on the switch patient population for Pom-Op - Management noted that physicians are excited about Pombiliti and Opfolda, but some prefer to wait one to two years before switching patients from first-generation therapies [76][80] Question: Details on the prevalence of Pompe disease in the Netherlands - Management indicated that the Netherlands has a higher diagnosed population for Pompe disease, with expectations to start dosing patients by the end of Q1 or early Q2 [84][87] Question: FX exposure and hedging strategies - Management clarified that they do not traditionally hedge FX exposure due to unpredictability, but noted that a 1% change in exchange rates could impact revenues by approximately $4 million [97][100] Question: Update on second source manufacturing for Pom-Op - Progress is being made on establishing a second manufacturing site in Ireland, with commercial product expected to enter the supply chain in Europe by the second half of 2025 [106][108] Question: Transition of EAP patients to commercial therapy - Approximately 30 patients on clinical studies are expected to transition to commercial treatments in the first half of 2025 [122][124] Question: Future growth strategy via R&D - Management highlighted plans to leverage existing infrastructure for future growth and explore early-stage discovery efforts in the Fabry and Pompe space [128][130]

分组1 - Amicus Therapeutics reported quarterly earnings of $0.09 per share, missing the Zacks Consensus Estimate of $0.10 per share, compared to a loss of $0.11 per share a year ago, representing an earnings surprise of -10% [1] - The company posted revenues of $149.71 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 0.13%, and this is an increase from year-ago revenues of $115.08 million [2] - The current consensus EPS estimate for the coming quarter is $0.07 on revenues of $141.25 million, and for the current fiscal year, it is $0.43 on revenues of $638.94 million [7] 分组2 - The Zacks Industry Rank for Medical - Biomedical and Genetics is currently in the top 28% of over 250 Zacks industries, indicating that the industry is performing well [8] - Amicus Therapeutics has surpassed consensus EPS estimates three times over the last four quarters and topped consensus revenue estimates two times in the same period [2] - The stock has added about 3.7% since the beginning of the year, compared to the S&P 500's gain of 4% [3]

Revenue Growth - Total revenue for FY 2024 reached approximately $528 million, representing a 32% growth compared to the previous year[4] - Full-Year 2025 total revenue growth guidance is projected to be between 17% to 24%[55] - Galafold revenue growth is expected to be between 10% to 15% for FY 2025[55] - Pombiliti and Opfolda revenue growth is anticipated to be between 65% to 85% for FY 2025[55] Market Potential - The global Fabry market is expected to grow from approximately $2.1 billion in 2024 to over $3 billion by 2029, driven by improved diagnosis and treatment rates[10][11] - The global Pompe disease market is projected to grow from approximately $1.5 billion in 2024 to over $2 billion by 2029, driven by increased diagnosis and treatment[29] Patient Demographics - The number of patients treated for Fabry disease has more than doubled since 2015, with approximately 12,000 currently treated in the U.S.[13][22] - The company has identified over 100,000 undiagnosed individuals with Fabry disease in the U.S., highlighting a significant unmet need in the market[21] - The company serves over 3,000 patients living with rare diseases[52] Product Performance - Galafold continues to be the leading oral treatment for Fabry disease, capturing over 60% of the share among treated amenable patients[15] - Pombiliti and Opfolda generated $70.3 million in revenue in FY 2024, with expectations of 65-85% growth in FY 2025[49] Clinical Development - Ongoing clinical studies are focused on expanding the label for Pombiliti and Opfolda, with evidence generation supporting their differentiated mechanism of action[46] Geographic Expansion - The company anticipates launching in up to 10 new countries in 2025, expanding access to therapies for over 650 late-onset Pompe disease patients[44] - The company has expanded access to 40 patients across 16 countries as of November 2024[60] Financial Considerations - A 4% variance in USD exchange rates could negatively impact total revenue by approximately $15 million in 2025[57] - Approximately 60% of the company's revenue is generated outside the U.S.[55] Commitment to Pricing - The company has committed to never raising the annual price of its products more than consumer inflation[60] Community Engagement - As of December 31, 2023, the company has allocated $1,980,516 to U.S. community programs[60] Revenue Distribution - The average quarterly distribution of Galafold revenue over the past 5 years shows Q4 at 28%[58]