Financial Data and Key Metrics Changes - In Q3 2025, the company generated $157 million in revenue, a 17% increase year over year, and for the first nine months, revenue totaled $740 million, reflecting a 55% increase compared to the prior year [23][24] - The company raised its 2025 financial guidance, now expecting total revenue between $875 million and $900 million, an increase of $50 million from previous guidance [25][26] - Operating loss is anticipated to be between $275 million and $300 million for the full year, with a cash balance expected to exceed $2.1 billion [25][26] Business Line Data and Key Metrics Changes - Tringolsa reported $32 million in net product sales, a nearly 70% increase quarter over quarter [14][23] - Royalty revenues increased by approximately 13% to $76 million in Q3, driven by contributions from Spinraza and Waylivra [24] - Donzera's launch is showing strong early adoption, with initial prescriptions being filled shortly after approval [19][20] Market Data and Key Metrics Changes - The U.S. prophylactic HAE market is well established, with approximately 20% of patients switching treatments annually, indicating a significant opportunity for Donzera [20] - The severe hypertriglyceridemia (SHTG) market is estimated to have over 1 million high-risk patients in the U.S., with Olzarsen positioned to address unmet needs in this population [16][17] Company Strategy and Development Direction - The company is focused on executing its strategy with two independent launches underway and plans for two additional launches next year [27][22] - The advancing wholly owned late-stage pipeline and a robust partnered portfolio are expected to drive sustained growth [7][26] - The company aims to achieve cash flow breakeven by 2028, emphasizing long-term value creation [26] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued success of Tringolsa and the upcoming launches of Olzarsen and Zilganersen, highlighting the strong momentum across the business [5][27] - The management team is optimistic about the potential for multi-billion dollar revenue from the new product launches and the overall pipeline [7][25] Other Important Information - Richard Geary, Chief Development Officer, will retire at the end of the year, marking his last earnings call with the company [28] Q&A Session Summary Question: Thoughts on the launch curve for Olzarsen in SHTG - Management indicated strong interest from healthcare providers and plans to target approximately 20,000 HCPs covering around 360,000 patients [30][32] Question: Concerns regarding acute pancreatitis events in Olzarsen studies - Management reassured that the data is groundbreaking and that the acute pancreatitis outcomes will be detailed at the upcoming AHA presentation [36][38] Question: Peak revenue potential for Donzera and Olzarsen - Management expects peak sales for Donzera to exceed $500 million and for Olzarsen to exceed $1 billion [39] Question: Pricing strategy for Olzarsen - Management is still working on pricing and expects to provide more clarity next year [44][45] Question: Early prescriptions for Donzera - The launch is going well, with both switching patients and newly diagnosed patients contributing to early prescriptions [70][72]

Financial Data and Key Metrics Changes - In Q3 2025, the company generated $157 million in revenue, a 17% increase year over year, and $740 million for the first nine months, reflecting a 55% increase compared to the prior year [23][24] - Tringolsa reported $32 million in net product sales, representing a nearly 70% increase quarter over quarter [13][23] - Royalty revenues increased by approximately 13% to $76 million in Q3, driven by contributions from Spinraza and Waylivra [24] - The company raised its 2025 financial guidance, now expecting total revenue between $875 million and $900 million, an increase of $50 million from prior guidance [25][26] Business Line Data and Key Metrics Changes - Tringolsa's launch is gaining momentum, with a significant increase in prescribers and patient identification efforts [15][16] - Donzera, approved for hereditary angioedema, is seeing strong early adoption, with patients switching from prior therapies and treatment-naive patients starting on Donzera [18][19] - Olzarsen showed a significant reduction in triglycerides and acute pancreatitis events, positioning it for a strong launch next year [9][10] - Zilganersen demonstrated a disease-modifying effect in Alexander disease, with plans for an independent launch next year [10][11] Market Data and Key Metrics Changes - The company anticipates targeting approximately 20,000 healthcare providers (HCPs) for Olzarsen, covering around 360,000 patients with severe hypertriglyceridemia (SHTG) [32] - The U.S. prophylactic HAE market is well established, with many patients dissatisfied with current therapies, indicating a significant opportunity for Donzera [19][66] Company Strategy and Development Direction - The company is focused on executing its commercial strategy for Tringolsa and Donzera while preparing for upcoming launches of Olzarsen and Zilganersen [22][28] - The pipeline includes multiple late-stage programs, with expectations for four key launches from the partnered pipeline by the end of 2027 [6][12] - The company aims to achieve cash flow breakeven by 2028, driving long-term value creation [26][28] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing momentum across the business, with strong performance from independent launches and a robust pipeline [4][28] - The company is optimistic about the potential of Olzarsen and Zilganersen to address significant unmet medical needs [10][11] - Management highlighted the importance of patient identification and education efforts to maximize the impact of new therapies [15][16] Other Important Information - The company plans to submit a new drug application for Zilganersen in Q1 2026 and is initiating an expanded access program in the U.S. [11] - The company has a strong balance sheet, expecting to end the year with over $2.1 billion in cash [26] Q&A Session Summary Question: Thoughts on the launch curve for Olzarsen in SHTG - Management indicated strong interest from HCPs and plans to target approximately 20,000 HCPs covering 360,000 patients, expecting strong uptake based on phase 3 data [32] Question: Concerns regarding acute pancreatitis events in Olzarsen studies - Management reassured that the data is groundbreaking and will be presented at AHA, with no significant concerns regarding acute pancreatitis events [36] Question: Peak revenue potential for Donzera and Olzarsen - Management expects peak sales for Donzera to exceed $500 million and for Olzarsen to exceed $1 billion [38] Question: Pricing strategy for Olzarsen - Management is still working on pricing and expects to provide more clarity next year [40] Question: Early prescriptions for Donzera - Management reported strong early adoption, with switches from existing therapies and newly diagnosed patients [66]