Alnylam Pharmaceuticals (NasdaqGS:ALNY) FY Conference January 12, 2026 12:00 PM ET Company ParticipantsYvonne Greenstreet - CEOTolga Tanguler - Chief Commercial OfficerPushkal Garg - Chief Research and Development OfficerJeff Poulton - CFOConference Call ParticipantsJessica Fye - Managing Director and Senior Biotechnology AnalystJessica FyeGood morning, everyone. My name's Jess Fye, biotech analyst at JPMorgan, and we're continuing the 44th annual JPMorgan Healthcare Conference this morning with Alnylam. Th ...

Investment Rating - The industry investment rating is "Outperform" compared to the market, indicating an expected performance greater than the CSI 300 index by more than 5% over the next six months [49]. Core Insights - Alnylam has shown strong revenue growth, with total revenue reaching $1.249 billion in Q3 2025, a year-over-year increase of 149%. Net product revenue was $851 million, up 103%, and revenue from collaborations and royalties surged by 393% to $398 million [2][7]. - The TTR product line is experiencing rapid growth, with Q3 2025 revenue of $724 million, a 135% increase year-over-year. The company has raised its full-year revenue guidance for TTR products to between $2.475 billion and $2.525 billion [16][29]. - Alnylam's research pipeline is robust, with seven drugs in Phase 3 clinical trials, including Nucresiran, which is expected to offer a better dosing regimen. The company is also exploring siRNA therapies in chronic disease areas [38][40]. Summary by Sections 1. Q3 2025 Performance Overview - In Q3 2025, Alnylam's total revenue was $1.249 billion, reflecting a 149% increase from the previous year. The net product revenue was $851 million, up 103%, while collaboration and royalty revenues reached $398 million, a 393% increase [2][7]. 2. Rapid Growth of TTR Products - The TTR product revenue for Q3 2025 was $724 million, marking a 135% year-over-year increase. The company has adjusted its full-year revenue guidance for TTR products to $2.475 billion to $2.525 billion, up from the previous estimate of $2.175 billion to $2.275 billion [16][29]. 3. Rich R&D Pipeline - Alnylam has a diverse R&D pipeline with seven drugs in Phase 3 trials. Nucresiran, a new generation TTR product, is expected to provide a more effective dosing schedule. The company is also advancing siRNA therapies in chronic disease management [38][40].

Summary of Alnylam Pharmaceuticals Conference Call Company Overview - Alnylam Pharmaceuticals is a leading RNA interference (RNAi) company, recognized for its innovative approach based on Nobel Prize-winning science. The company has successfully launched six marketed products and has a robust clinical pipeline with over 20 programs [2][4]. Key Focus Areas 1. **TTR Leadership**: - Alnylam aims to establish leadership in transthyretin (TTR) therapeutics, particularly with the launch of Amvuttra for cardiomyopathy. The company believes Amvuttra has the potential to become the standard of care [3][4]. - The next-generation program, Nucresiran, is expected to provide long-term durability for the TTR franchise, extending its impact into the 2040s [3]. 2. **Innovation and Pipeline Growth**: - Alnylam is focused on expanding its pipeline, which has the potential for three to four blockbuster medicines. The company emphasizes the high probability of success from its R&D efforts [3][4]. 3. **Financial Performance**: - The company is committed to disciplined capital allocation and aims to achieve sustainable non-GAAP profitability. Alnylam has seen growing revenues and is building a foundation for long-term growth [4][6]. Amvuttra Launch Insights - The launch of Amvuttra has exceeded expectations, with rapid access to 170 healthcare systems that account for 80% of TTR amyloidosis prescriptions. This early success allowed the company to raise revenue guidance [10][11]. - Patient volume demand has doubled, indicating strong market acceptance and effective execution by the commercial team [11][12]. - The company reports a balanced utilization of Amvuttra across both first-line and second-line treatment settings, with a significant share of first-line prescriptions [20][21]. Market Dynamics - There is a significant underdiagnosis issue in TTR cardiomyopathy, with only 20% of an estimated 150,000 patients in the US diagnosed. Alnylam sees opportunities for improved diagnosis rates due to advancements in diagnostic tools [31][32]. - The company is successfully penetrating community centers, which now contribute over half of its business, indicating a broad acceptance of Amvuttra [34]. Future Developments - Nucresiran is in two Phase 3 studies, with expectations for improved patient outcomes due to higher TTR knockdown rates and a more convenient dosing schedule [42][44]. - Alnylam is also advancing its pipeline in other areas, including Huntington's disease and hypertension, with promising early results [48][49]. Conclusion - Alnylam Pharmaceuticals is positioned for significant growth with its innovative RNAi therapeutics, particularly through the successful launch of Amvuttra and the upcoming Nucresiran. The company is focused on expanding its market presence, improving patient outcomes, and achieving financial sustainability [51].

Financial Data and Key Metrics Changes - The company reported an 11% growth in both portfolio receipts and royalty receipts, reflecting strong business momentum [4][20] - Return on invested capital was 15.7% and return on invested equity was 22.9% for the last 12 months [5][23] - Portfolio receipts for the third quarter reached $814 million, with a projected full-year guidance increase to between $3.2 billion and $3.25 billion, representing growth of approximately 14-16% [25][28] Business Line Data and Key Metrics Changes - The company actively engaged in royalty transactions, deploying $1 billion in the quarter, totaling $1.7 billion for the first nine months [5][24] - Key drivers of growth included strong performance from Voranigo, Tremfya, and the Cystic Fibrosis Franchise [20] Market Data and Key Metrics Changes - The company expanded its development stage pipeline to 17 therapies, with significant potential for cumulative peak sales exceeding $36 billion [14][18] - The company noted a growing market for synthetic royalties, with $1.8 billion in synthetic royalty transactions announced in 2025, surpassing previous years [15][16] Company Strategy and Development Direction - The company aims to be a premier capital allocator in life sciences, focusing on innovative therapies with high patient impact [4][13] - The strategy includes a therapy area agnostic investment approach, allowing for diversification across various disease areas [9][16] - The company raised its full-year guidance for 2025, reflecting confidence in its diversified portfolio and ongoing capital deployment [25][28] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the uptick in biotech M&A activity, viewing it as beneficial for capital needs in the sector [34][37] - The company is excited about growth opportunities in China, particularly in out-licensing to multinationals [38] - Management emphasized a disciplined approach to capital allocation, focusing on transactions that align with their investment criteria [40][46] Other Important Information - The company repurchased 4 million shares in the quarter, totaling $1.15 billion in share repurchases for the first nine months [5][24] - The company maintains a strong financial position with cash and equivalents of $939 million and access to $2.9 billion in financial capacity [23][24] Q&A Session Summary Question: Impact of biotech M&A and interest rates on royalty-driven deal activity - Management noted that increased M&A activity does not significantly impact their operations, as large pharma companies require capital, which the company provides [34][37] Question: Updates on China strategy - Management highlighted ongoing efforts to build relationships in China, viewing it as a growth opportunity in the royalty marketplace [38] Question: Frequency of collaborations with large pharma - Management indicated that the use of royalties to fund trials is becoming mainstream, presenting a significant opportunity for the company [40][41] Question: Potential changes in risk appetite due to new launches - Management stated that while returns may fluctuate, their investment behavior remains consistent, focusing on attractive transactions regardless of market conditions [46][47] Question: Insights on the Amvuttra deal and competition - Management expressed confidence in the Amvuttra investment, highlighting a unique structure that allows for long-term royalty ownership [60][62] Question: Implications of trial outcomes for LP(a) products - Management remains optimistic about the probability of success for their LP(a) investments, despite potential challenges in trial outcomes [64][66] Question: Updates on obexelimab and market potential - Management noted that recent positive data for obexelimab validates its potential in treating autoimmune diseases, with a focus on IgG4-related disease [75][76]