Financial Data and Key Metrics Changes - Fourth quarter revenue totaled $213 million, and full year 2024 revenue was $807 million, exceeding guidance [8][31] - Cash, cash equivalents, and marketable securities totaled approximately $1.1 billion as of December 31, 2024, compared to $877 million as of December 31, 2023 [35] Business Line Data and Key Metrics Changes - DMD franchise revenue for the full year 2024 was $547 million, with Translarna net product revenue of $94 million and Emflaza net product revenue of $50 million in the fourth quarter [32][33] - The company achieved all clinical and regulatory milestones on schedule, including four FDA approval applications submitted [12][11] Market Data and Key Metrics Changes - The US represents the largest opportunity for Sepiapterin, with approximately 17,000 PKU patients, most of whom are not on medical treatments [23] - The estimated prevalence of Friedreich's ataxia in the US is about 6,000 patients, with one-third being pediatric [27] Company Strategy and Development Direction - The company plans to support 2025 commercial launches and continue investing in R&D platforms while exploring business development opportunities [10][11] - Anticipated milestones for 2025 include the global launch of Sepiapterin and potential launch of Vatiquinone in the US [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's strong financial position and ability to reach cash flow breakeven without raising additional capital [10][11] - The management highlighted the excitement surrounding Sepiapterin due to its potential to address significant unmet needs in the PKU patient population [15] Other Important Information - The company received a $1 billion upfront payment from Novartis as part of the PTC518 collaboration, with potential for up to $1.9 billion in development and sales milestones [13][35] - The company is preparing for multiple new product launches throughout the year, leveraging its experienced global commercial infrastructure [29] Q&A Session Summary Question: What is the expected impact of diet liberalization for PKU patients? - Management highlighted that over 97% of patients in the feed protocol can liberalize their diet, with many achieving or exceeding the recommended daily allowance of protein [40][41] Question: What is the market opportunity for Friedreich's ataxia? - Management expressed excitement about the opportunity to provide therapy for all FA patients, including pediatric patients, and emphasized the strong safety profile of the treatment [45][46] Question: What is the visibility on the Translarna review process? - Management indicated that the review is ongoing, and while they do not expect a formal PDUFA date, they anticipate more information in the first half of the year [57] Question: How should investors frame expectations for the PIVOT-HD update? - Management stated that the update will include both stage 2 and stage 3 patients, with a focus on biomarker data and clinical outcome scales [68][69] Question: How is the company preparing for the Sepiapterin launch? - Management noted that they are actively engaging with key stakeholders and preparing for a strategic launch sequence targeting access for PKU patients [22][24]