Ligand Pharmaceuticals 2025 Investor Day Summary Company Overview - **Company**: Ligand Pharmaceuticals (NasdaqGM:LGND) - **Event**: 2025 Investor Day held on December 09, 2025 Core Industry Insights - **Industry**: Biopharmaceuticals, focusing on royalty financing and commercialization of biotech products - **Market Dynamics**: The biopharmaceutical industry is experiencing significant growth, with a notable increase in royalty financing, which is less than 10% of total funding but rapidly expanding Key Financial Highlights - **2022 to 2025 Growth**: - Core revenue expected to grow from $108 million in 2022 to $230 million in 2025, and projected to reach $430 million by 2030 [22] - Royalty revenue is anticipated to grow nearly 40% in 2025, driven by products like Filspari, Ohtuvayre, and Capvaxive [23] - Adjusted EPS expected to be in the range of $7.40-$7.65 per share for 2025, reflecting a 31% growth [24] - **Long-term Outlook**: - Long-term royalty growth rate revised to 23% CAGR from 2025 to 2030, up from the previous 22% [29] - Significant contributions expected from commercial-stage products and development-stage programs [30] Strategic Initiatives - **Portfolio Expansion**: - Strong portfolio with 12 commercial-stage products and over 80 development-stage programs [5] - Focus on high unmet clinical needs and diversified product offerings [10] - **Investment Strategy**: - Emphasis on royalty monetization and project finance to create tailored investment opportunities [11] - Recent investments include Pelthos and Castle Creek, focusing on high-value assets [41][42] Operational Efficiency - **Cost Management**: - Operating expenses reduced to less than half of previous levels since 2022, contributing to increased profitability [6] - Cash operating profits increased by approximately 50% year-over-year [24] Product Development and Market Positioning - **Key Products**: - Filspari and Ohtuvayre are expected to be major revenue drivers, with Filspari projected to grow approximately 75% and Ohtuvayre by 150% in 2026 [27] - Zelsuvmi, a new product for treating molluscum contagiosum, is positioned as a unique therapeutic option with no direct competition [17] Team and Management - **Investment Team Growth**: - Investment team projected to grow from 3 to 18 members by 2026, enhancing deal origination capabilities [34] - **Leadership**: - Strong management team with extensive experience in the biopharmaceutical sector, ensuring effective execution of strategies [14] Risks and Considerations - **Market Risks**: - The biopharmaceutical industry is inherently risky, with potential for volatility in operating expenses and market acceptance of new products [9] - **Regulatory Environment**: - Ongoing changes in healthcare policies may impact product development and market dynamics [16] Conclusion - Ligand Pharmaceuticals is positioned for significant growth through a diversified portfolio, strategic investments, and a strong focus on high unmet clinical needs. The company aims to leverage its operational efficiencies and robust financial outlook to deliver value to investors and patients alike.

Summary of Amicus Therapeutics Conference Call Company Overview - **Company**: Amicus Therapeutics (NasdaqGM:FOLD) - **Focus**: Developing transformative therapies for rare diseases, particularly Fabry disease and Pompe disease [4][5] Key Products and Financial Performance - **Galafold**: - Approved for Fabry disease, on the market for nearly 10 years in Europe and 8 years in the U.S. - Achieved over $500 million in revenue for the first time this year, with a growth rate of 10%-15% [5][6] - The global market for Fabry disease is estimated at $2.4 billion, up from $1 billion previously [10] - **Pombiliti and Opfolda (POMOP)**: - Approved for Pompe disease, launched in the last couple of years. - Expected revenue growth of 50%-65% this year [5][6] - Projected to generate over $100 million in sales this year [23] Market Dynamics and Growth Drivers - **Fabry Disease Market**: - Initial estimates of incidence were one in 50,000 live births; recent studies suggest it may be closer to one in 3,000 [10] - The diagnosed untreated population has grown from 5,000 to 6,000 patients [10] - Increased awareness and availability of genetic testing are driving patient identification [11][12] - **Patient Identification Initiatives**: - Collaborations with healthcare systems (e.g., NHS in the U.K.) to identify undiagnosed patients [13][14] - Use of AI and partnerships (e.g., with OM1) to enhance patient finding [15][19] Competitive Landscape - **Galafold vs. Competitors**: - Concerns about Sanofi's upcoming phase three data for their substrate reduction therapy (SRT) were addressed, noting that Galafold's mechanism is different and may not be directly impacted [21][22] - **Pombiliti Market Position**: - The company is seeing increased patient switches from competitor products, with a significant uptick in the last two quarters [26][29] - The U.S. market is showing strong momentum, with the company gaining market share in the U.K. and other European regions [27][32] Pipeline and Future Opportunities - **FSGS Program**: - Amicus has licensed DMX-200 for FSGS, a rare kidney disease, with potential for significant market opportunity [36][38] - The FDA has indicated that proteinuria could serve as a primary endpoint for the ongoing phase three study, which is promising for regulatory approval [39][40] - **Regulatory Strategy**: - Plans to engage with the FDA regarding the study endpoints and potential accelerated approval paths [40][41] Financial Position and Strategic Focus - **Cash Position**: - The company has over $260 million in cash and is GAAP profitable, indicating a strong financial foundation [52] - **Business Development Strategy**: - Focus on late-stage, de-risked assets with potential for revenue growth, particularly in the Fabry and Pompe disease spaces [52] Conclusion - Amicus Therapeutics is positioned for continued growth with strong product performance, strategic initiatives to identify and treat more patients, and a promising pipeline in rare diseases. The company is committed to maintaining financial discipline while pursuing opportunities for expansion and profitability [52][53]

Financial Data and Key Metrics Changes - Total revenue and other income for Q3 2025 on a GAAP basis was $115.5 million, up from $51.8 million in the same quarter last year, with core revenue growing 68% year over year to $86.9 million [26][34] - Royalty revenue rose 47% year over year to $46.6 million, and adjusted EPS increased 68% to $3.09 [26][34] - The company raised its full-year 2025 guidance, now expecting core revenue of $225-$235 million and adjusted EPS of $7.40-$7.65 [35][36] Business Line Data and Key Metrics Changes - Royalty revenue growth was driven by strong performances from products like Travere's Filspari, Merck's Ohtuvayre, and Capvaxive, with Filspari becoming the largest royalty-generating asset [30][31] - Filspari reported Q3 sales of $90.9 million, a 155% year-over-year increase, while O2VARE sales reached $136 million, a 32% sequential increase [30][31] - Capvaxive generated $244 million in sales, an 89% sequential increase, reinforcing its market position [31] Market Data and Key Metrics Changes - The company noted strong uptake of Zelsuvmi in its early launch phase, with expectations for continued momentum [7] - Ohtuvayre is tracking ahead of initial forecasts, with Q3 sales growing 32% sequentially, and projected sales of $2 billion by 2029 [10][11] - The FDA has assigned a PDUFA date of January 13, 2026, for Filspari's second indication, FSGS, which could significantly expand its market opportunity [18] Company Strategy and Development Direction - The company has focused on building a diversified portfolio of high-margin royalties and has successfully executed a convertible debt financing to support growth initiatives [5][6] - The strategic direction set in 2022 has led to significant growth across core revenue and adjusted EPS, with a long-term royalty growth outlook of 22% CAGR [9][10] - The company is actively pursuing high-quality investment opportunities and expanding its business development team to capitalize on its robust pipeline [27][29] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strength of the commercial royalty portfolio and the potential for indication and geographic expansion of key products [36][37] - The company highlighted the positive developments in the FDA's approach to accelerating timelines for innovative treatments, which aligns with its investment strategy [16][49] - Management remains optimistic about the ongoing progress of the Pelthos team and the launch momentum of Zelsuvmi [37] Other Important Information - The company ended the quarter with approximately $1 billion in deployable capital, including cash and investments [7][27] - The Pelthos transaction resulted in a $53 million gain recognized in Q3, reflecting the strategic value of the spin-out [24][25] - The company has seen record-setting origination activity, reviewing over 130 investment opportunities in the first three quarters of the year [13] Q&A Session Summary Question: Opportunities and special situations related to Pelthos and Palvella - Management indicated a robust opportunity set in special situations, focusing on companies needing more than just capital [38][39] Question: Changes in investment opportunities and capital demands - Management noted that while the deal flow may shift with market conditions, the demand for royalty capital remains high, particularly for development-stage assets [44][45] Question: Insights on the ZELSUVMI launch and future expectations - Management provided limited additional information but indicated that more specifics would be available during the upcoming earnings call [50][51] Question: Competitive landscape for royalty assets - Management acknowledged potential future competition but noted that they have not yet seen significant changes in the competitive landscape [53][54] Question: Thoughts on the recent approval of Furoscix - Management expressed optimism about Furoscix's potential to provide a differentiated treatment option in the market [56]

Financial Performance & Guidance - Q2 2025 royalty revenue increased by 57% compared to Q2 2024, driven by Ohtuvayre, Filspari, Qarziba, Capvaxive and Vaxneuvance[3, 26] - Q2 2025 adjusted EPS grew 14% to $1.60[3, 24, 25] - The company increased revenue guidance for 2025 from $180 million-$200 million to $200 million-$225 million, a 13% increase[3, 24, 33] - Adjusted EPS guidance for 2025 was raised from $6.00-$6.25 to $6.70-$7.00 per diluted share, a 12% increase[3, 24, 33] Portfolio & Investments - Ligand has approximately 50% equity interest in Pelthos and is entitled to a 13% royalty on Zelsuvmi sales[3, 6] - The company committed $35 million in long-term capital for royalty interest in Orchestra BioMed's AVIM therapy and Virtue SAB and invested $5 million in equity private placement[3, 20, 21] - Merck is set to acquire Verona for $10 billion, which will impact Ligand's 3% royalty on Ohtuvayre[3, 4, 8] Strategic Outlook - The company anticipates a long-term royalty revenue CAGR of greater than 22%[3, 27] - Ohtuvayre peak-sales consensus updated from $1.2 billion in December 2024 to $3.4 billion in August 2025[30] - Ligand has ~$450 million in deployable capital as of June 30, 2025[3, 24]