Overview of Catalyst - Catalyst focuses on orphan and rare diseases with three product candidates [1] - The first product, FIRDAPSE, has been on the market since 2019 and is used for Lambert-Eaton myasthenic syndrome, both idiopathic and cancer-associated [1] Product Candidates - The second product, FYCOMPA, is for epilepsy, which is not classified as a rare disease; however, its acquisition has benefited the company [2] - FYCOMPA's solid dose lost patent protection in late May, leading to some market competition, but the product has still overperformed [2]

Financial Data and Key Metrics Changes - The company reported a strong cash position with $689.9 million in cash and cash equivalents and no funded debt, indicating financial health and flexibility for future acquisitions [63] - The company anticipates a mid-term growth rate of 15%-20% for Firdapse as it continues to optimize patient adherence and reduce discontinuation rates [56][58] Business Line Data and Key Metrics Changes - Firdapse has been on the market since 2019 and is focused on Lambert-Eaton myasthenic syndrome (LEMS), with a strong emphasis on both idiopathic and cancer-associated LEMS [2][3] - Agamree, for Duchenne muscular dystrophy, is expected to penetrate the market further, with 95% of patients having received steroids, but only 70% currently on treatment [58] - The company has achieved 95% penetration in 100 Centers of Excellence (COEs) for DMD, with 100% penetration in the top 45 COEs that account for 80% of the steroid business [14] Market Data and Key Metrics Changes - The total addressable market for Agamree is estimated at $1.4 billion, with a conservative estimate of $1.2 billion based on current patient treatment rates [18][51] - The company noted that 90% of cancer-associated LEMS patients remain undiagnosed, highlighting a significant opportunity for growth through education and screening [29] Company Strategy and Development Direction - The company is focused on a buy-and-build strategy, acquiring products that are de-risked and near market, while also considering late-stage development opportunities [4] - The management is looking to deepen market penetration for both Firdapse and Agamree, with a systematic approach to education and care pathway changes [36][40] - The company aims to leverage its strong infrastructure to support patients in rare diseases, remaining therapeutically area-agnostic [12] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in overcoming the decline of Fycompa due to generic competition by focusing on growth in other product lines [55] - The company is optimistic about the potential for Agamree and Firdapse, citing strong patient adherence programs and ongoing education efforts [56][58] - Management acknowledged the challenging market environment but remains committed to pursuing strategic acquisitions to accelerate growth [64] Other Important Information - The company has settled three out of four patent challenges, with the remaining challenge expected to be resolved in the future, providing a clearer path for market stability [52][63] - The company has conducted over 100 assessments for potential business development opportunities since January, indicating a proactive approach to growth [62] Q&A Session Summary Question: What is the peak market opportunity for Agamree? - The company estimates the peak market opportunity for Agamree at approximately $1.4 billion, with a conservative total addressable market of $1.2 billion based on current treatment rates [18][51] Question: How is the education and screening for cancer-associated LEMS progressing? - The company is focusing on educating physicians about the undiagnosed cancer-associated LEMS patients, with significant efforts in screening and care pathway changes [29][30] Question: What is the strategy for Firdapse amidst generic competition? - The company believes it can outgrow the decline of Fycompa by optimizing patient adherence and leveraging strong educational programs [55][56]