Financial Data and Key Metrics Changes - For the full year 2024, total revenues grew by 23.5% year over year to $491.7 million, exceeding previous guidance [6][24] - Full year net product revenue reached $489.3 million, a 23.4% increase over 2023, driven by the successful launch of AGAMRI [6][24] - In Q4 2024, total revenues and net product revenues reached $141.8 million, an increase of 28.3% and 30% over Q4 2023, respectively [7][24] - GAAP net income for 2024 was $163.9 million, or $1.38 per basic share, a 130% increase year over year [28] - Non-GAAP net income for 2024 was $276.3 million, or $2.33 per basic share, representing a 95% increase year over year [29] Business Line Data and Key Metrics Changes - Firdapse's net product revenue for 2024 was $306 million, an 18.4% increase year over year [25] - AGAMRI generated $46 million in net product revenue from its launch on March 13, 2024, through year-end [25] - Fycampa's net product revenue for 2024 was $137.3 million, slightly down from $138.1 million in 2023 due to changes in gross to net arrangements [25][30] Market Data and Key Metrics Changes - The company ended 2024 with a cash position of $517.6 million and no debt, enhancing its capacity for strategic investments [8][33] - The company expects 2025 total revenue to be between $545 million and $565 million [7] Company Strategy and Development Direction - The company is focused on a buy and build approach for long-term growth, actively evaluating rare disease opportunities [13] - The international strategy includes building a sustainable network of out-licensing partners and targeting markets where products can enhance access to care [14] - The company aims to leverage its rare disease expertise and operational excellence to drive sustained growth [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued growth of Firdapse, expecting organic growth to exceed 15% in 2025 [20] - The company is committed to navigating market and regulatory challenges while adapting to industry changes [15] - Management highlighted the importance of patient engagement services and strong relationships with healthcare communities [8] Other Important Information - The company secured a favorable resolution to the Teva patent litigation, reinforcing Firdapse's intellectual property strength [10] - The SUMMIT study for AGAMRI is underway, aiming to provide real-world data on its long-term benefits [11] Q&A Session Summary Question: Can you provide more color on new patient adds and the low discontinuation rate for Firdapse? - Management noted over 500 pipeline leads for LEMS patients, contributing about 50% to new enrollments each month, with a steady discontinuation rate of 15% [40][41] Question: What is the plan for replacing revenue from Fycampa in 2026? - Management remains open to orphan and differentiated products, including potential opportunities in epilepsy [44] Question: What indications are being pursued for AGAMRI? - The focus is on further characterizing AGAMRI for its immunosuppressive capabilities in rare diseases [50] Question: How does the clarity on Firdapse IP protections shape investment? - Should a full ten-year opportunity arise, the company would consider aggressive investments in the oncology side and other research opportunities [96][97] Question: What is the expected impact of generic erosion on Fycampa? - Management expects some price and volume erosion post-patent expiry but believes the product will maintain a considerable market share [80][100]

Financial Data and Key Metrics Changes - For the full year 2024, total revenues grew by 23.5% year over year to $491.7 million, exceeding previous guidance [6][25] - Full year net product revenue reached $489.3 million, a 23.4% increase over 2023, driven by the successful launch of AGAMRI [6][25] - In Q4 2024, total revenues and net product revenues reached $141.8 million, an increase of 28.3% and 30% over Q4 2023, respectively [7][25] - The company ended the year with a cash position of $517.6 million and no debt, enhancing its capacity for strategic investments [8] Business Line Data and Key Metrics Changes - Firdapse's net product revenue for 2024 was $306 million, an 18.4% increase year over year [26] - AGAMRI generated $46 million in net product revenue from its launch on March 13, 2024, through year-end [26] - Fycompa's net product revenue for 2024 was $137.3 million, slightly down from $138.1 million in 2023 due to changes in gross to net deductions [26][30] Market Data and Key Metrics Changes - Firdapse has maintained 14 consecutive quarters of 15% or more growth year over year, reflecting strong market demand [9] - AGAMRI has gained traction with 93% of the top 45 DMD centers of excellence submitting enrollments [18] - VICOMPA's full year 2024 net product revenue reached $137.3 million, surpassing guidance [19] Company Strategy and Development Direction - The company is focused on a "buy and build" approach for long-term growth, actively evaluating rare disease opportunities [13] - Catalyst is building a sustainable network of out-licensing partners and seeking markets where its products can enhance access to care [14] - The company aims to navigate market and regulatory challenges while leveraging its rare disease expertise for sustained growth [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued revenue growth, forecasting total revenue for 2025 to be between $545 million and $565 million [8] - The company is committed to protecting the long-term value of its franchise amid ongoing patent litigation [10] - Management highlighted the importance of patient engagement services and strong relationships with healthcare communities [8][9] Other Important Information - The company reported a GAAP net income of $163.9 million for 2024, a 130% increase year over year [29] - Non-GAAP net income for 2024 was $276.3 million, representing a 95% increase from 2023 [30] - Research and development expenses decreased significantly in 2024 due to a one-time expense in 2023 [32] Q&A Session Summary Question: Can you provide more color on new patient adds and the low discontinuation rate for Firdapse? - Management noted over 500 pipeline leads for LEMS patients, contributing about 50% to new enrollments, with a steady discontinuation rate of 15% [40][42] Question: What is the plan for replacing revenue from Fycompa in 2026? - Management remains open to orphan and differentiated products, including potential epilepsy opportunities, but is focused on the orphan space [44][45] Question: What indications are being pursued with AGAMRI? - The company is further characterizing AGAMRI for its immunosuppressive capabilities in rare diseases [52] Question: How does the company view gene therapy opportunities? - Management is cautious about gene therapy, focusing on products that are close to market and have a clear need [53][55] Question: What is the expected impact of gross to net changes for Firdapse in 2025? - The company anticipates a gross to net change of 3% to 3.5% for 2025, influenced by the Inflation Reduction Act [61] Question: What factors are expected to drive demand for AGAMRI in 2025? - Management expects steady transitions from both prednisone and Emflaza segments, with a focus on accelerating enrollments mid-year [68] Question: How well has Firdapse penetrated the LEMS market? - Management did not provide specific penetration percentages but indicated strong market presence and growth potential [108]