11/17/2025Find out which top-rated stocks have just earned a spot... BREAKING: Stocks Bounce But Tumble For Week Among big winners in the stock market this year, Alnylam Pharmaceuticals (ALNY) has climbed amid increasing uptake of its chief drug, Amvuttra. The stock did retreat from its all-time high of 495.55 in the wake of the company's strong third-quarter results. However, even after accounting for the recent pullback, the biotech stock has soared about 96% in 2025. Alnylam is… Related news Alnylam, Mon ...

Alnylam Pharmaceuticals (NasdaqGS:ALNY) 2025 Conference November 19, 2025 06:30 AM ET Company ParticipantsYvonne Greenstreet - CEOTolga Tanguler - Chief Commercial OfficerOperatorHi, everyone. My name is Maury Raycroft, and I'm one of the biotech analysts at Jefferies. It's with great pleasure that I'd like to welcome the Alnylam management team. We've got Yvonne Greenstreet, the CEO, and Tolga Tanguler, the Chief Commercial Officer. Thanks so much for joining us today. Maybe to start off, it's been an exci ...

Summary of Alnylam Pharmaceuticals Conference Call Company Overview - **Company**: Alnylam Pharmaceuticals (NasdaqGS:ALNY) - **Event**: UBS Healthcare Conference - **Date**: November 10, 2025 Key Highlights from the Conference Call Financial Performance - **Third Quarter Earnings**: Alnylam reported $300 million in cardiomyopathy (CM) revenue for Q3, doubling the $150 million from Q2, leading to an upgraded full-year guidance by $275 million [6][11][24] - **Five-Year Goals**: The company has achieved its five-year goals set for 2025, including: - 500,000 patients on RNAi therapeutics - Six commercial products launched - 20 clinical programs - 40% compound annual growth rate (CAGR) in top-line growth - Non-GAAP profitability [10][11] Product Launch and Market Dynamics - **Cardiomyopathy Launch**: The launch has seen strong early momentum, with successful access to 170 health systems and first-line access to payers, primarily in a Medicare population (80%) [15][18][29] - **Pricing Strategy**: Alnylam anticipates a mid-single-digit year-over-year price decline for the TTR business in 2025, while volume growth is expected to offset the price decline [24][26] Pipeline and Future Goals - **2030 Goals**: The company plans to announce new goals at the JPMorgan conference, focusing on TTR leadership, pipeline progression, and financial profile evolution [12][14] - **Next-Generation Products**: Zilebesiran is expected to achieve over 95% TTR reduction with a twice-annual dosing regimen, with ongoing studies in both cardiomyopathy and hereditary polyneuropathy [32][33] Competitive Landscape - **Market Positioning**: Alnylam is focused on first-line positioning against competitors, with expectations of expanding the market pie rather than just taking market share [30][45] - **Combination Therapy**: The company is preparing for a potential shift towards combination therapies as Tafamidis goes generic, leveraging data from ongoing studies [45][46] Early-Stage Pipeline - **Mivelsiran**: Targeting amyloid precursor protein for Alzheimer's and cerebral amyloid angiopathy, with promising early results [48][49] - **Huntington's Disease**: The company is optimistic about its differentiated approach targeting both full-length and variant proteins [50][52] - **ALN-6400**: Aiming to be a universal hemostatic agent, with initial focus on hereditary hemorrhagic telangiectasia [54] Additional Insights - **Value-Based Agreements**: Alnylam has implemented value-based agreements with payers to ensure patient compliance and reduce costs associated with non-compliance [29] - **Patient Enrollment**: The company is confident in its ability to enroll patients in ongoing studies without significant impact from competitive dynamics [36] This summary encapsulates the key points discussed during the conference call, highlighting Alnylam Pharmaceuticals' financial performance, product launches, pipeline developments, and strategic positioning in the market.

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]

Financial Data and Key Metrics Changes - The company reported an 11% growth in both portfolio receipts and royalty receipts, indicating strong business momentum [4][22] - Return on invested capital was 15.7% and return on invested equity was 22.9% for the last 12 months [6][25] - Portfolio receipts for Q3 2025 reached $814 million, reflecting an 11% increase [22] Business Line Data and Key Metrics Changes - The company maintained strong growth driven by key products such as Voranigo, Tremfya, and the Cystic Fibrosis Franchise [22] - The development stage pipeline expanded to 17 therapies, with multiple pivotal readouts expected in the near future [7][15] Market Data and Key Metrics Changes - The company raised its full-year 2025 top line guidance to between $3.2 billion and $3.25 billion, representing growth of approximately 14-16% [9][20] - Milestones and other contractual receipts are now expected to be around $125 million, up from $110 million previously [20] Company Strategy and Development Direction - The company aims to be the premier capital allocator in life sciences, focusing on innovative therapies with high patient impact [4][31] - The strategy includes a therapy area agnostic investment approach, allowing for diversification across various disease areas [10][17] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing demand for funding life sciences innovation, highlighting the potential for annualized total shareholder returns in the mid-teens over the next five years [31] - The company is excited about growth opportunities in China, particularly in out-licensing to multinationals [39] Other Important Information - The company deployed $1 billion in capital for value-creating royalty transactions in Q3 2025, totaling $1.7 billion for the first nine months [6][26] - Share repurchases amounted to $1.15 billion in the first nine months of 2025 [26] Q&A Session Summary Question: Impact of biotech M&A and interest rates on royalty-driven deal activity - Management noted that the uptick in M&A activity does not significantly impact their operations, as large pharma companies require capital, which the company provides [38] Question: Thoughts on the China opportunity - Management is optimistic about growth in China and is actively building relationships to capitalize on this market [39] Question: Frequency of collaborations with large pharma - Management believes the use of royalties to fund trials is becoming mainstream, indicating a growing opportunity in this area [41] Question: Insights on the Amvuttra deal and competition - Management expressed confidence in the Amvuttra investment despite potential competition from Nucresiran, citing a broad range of scenarios considered [60] Question: Updates on the LP(a) space and trial designs - Management acknowledged differences in trial designs and remains optimistic about the prospects of their investments in this area [90] Question: Updates on obexelimab and its market potential - Management highlighted the strong phase 2 data for obexelimab, validating its potential in treating autoimmune diseases [92]

Q3 2025 Financial Results November 5, 2025 Forward Looking Statements This presentation has been prepared by Royalty Pharma plc (the "Company"), is made for informational purposes only and does not constitute an offer to sell or a solicitation of an offer to buy securities. The information set forth herein does not purport to be complete or to contain all of the information you may desire. Statements contained herein are made as of the date of this presentation unless stated otherwise, and neither the deliv ...

Group 1 - The core viewpoint is that small nucleic acid drugs are expected to become the third major category of drugs, following small molecules and antibody drugs, due to their advantages such as targeting traditionally non-druggable targets and long-lasting effects [1] - The breakthrough of GalNAc technology in 2014 established a successful paradigm for liver-targeted delivery, significantly activating the development of the small nucleic acid drug industry [1] - The global small nucleic acid drug industry is currently achieving commercial profitability, breakthrough in indications, advancements in extrahepatic delivery, and dual-target breakthroughs [1] Group 2 - From a commercialization perspective, drugs like Amvuttra and Leqvio show significant potential, with global leader Alnylam expected to achieve profitability this year [2] - The years 2025-2026 are critical for indication breakthroughs, with weight loss indications expected to reach proof of concept (POC) and CNS indications likely to begin exploration [2] - Technological advancements in extrahepatic delivery are emerging, with TRiM and AOC delivery methods showing promise, and Avidity's AOC drug Del-zota expected to be commercially available by 2026 [2] Group 3 - Chinese small nucleic acid drug companies are demonstrating global competitiveness, with an accelerated growth in research pipelines [3] - Companies like Bewang and Ruibo have established business development (BD) transactions with multinational corporations (MNCs), showcasing the global competitiveness of Chinese small nucleic acid drugs [3] - The focus of Chinese small nucleic acid drug pipelines is expanding from cardiovascular diseases and hepatitis B to include weight loss and CNS indications, with potential for first-in-class (FIC) and best-in-class (BIC) drugs [3]

Core Viewpoint - Small nucleic acid drugs are expected to become the third major category of drugs following small molecules and antibody drugs, driven by their ability to target traditionally undruggable targets and their long-lasting effects [1][2]. Group 1: Mechanism and Advantages - The mechanism of small nucleic acid drugs involves three main steps: endocytosis, endosomal escape, and gene silencing [2]. - Key factors for their effectiveness include receptor selection, optimization of delivery systems to enhance endosomal escape efficiency, and chemical modifications to improve stability [2]. - Compared to other drug types, small nucleic acid drugs offer advantages such as a rich target pool, strong specificity, and long-lasting effects [2]. Group 2: Industry Breakthroughs - The global small nucleic acid drug industry is achieving multi-dimensional breakthroughs, with drugs like Amvuttra and Leqvio showing significant potential, and Alnylam expected to achieve profitability this year [3]. - The years 2025-2026 are critical for validating new indications, with weight loss indications anticipated to reach proof of concept (POC) and CNS indications expected to begin exploration [3]. - Technological advancements include breakthroughs in extrahepatic delivery methods, with Avidity's AOC drug Del-zota showing promising efficacy and expected to be commercially available by 2026 [3]. Group 3: China's Competitive Edge - Chinese small nucleic acid drug companies are demonstrating global competitiveness, with an accelerated growth in research pipelines [4]. - Companies like Bosheng and Ruibo have established business development (BD) deals with multinational corporations (MNCs), highlighting China's competitive position [4]. - The focus of Chinese small nucleic acid drug pipelines has expanded from cardiovascular diseases and hepatitis B to include weight loss and CNS indications, with potential for first-in-class (FIC) and best-in-class (BIC) drugs [4].

Core Insights - Alnylam Pharmaceuticals reported strong third-quarter results, driven by its leading drug Amvuttra, which significantly exceeded sales expectations and demonstrated robust growth in patient demand [3][10]. Financial Performance - Amvuttra generated $685 million in sales during the third quarter, surpassing the expected $622 million and more than doubling from $259 million in the same period last year [3]. - Total sales across all products reached $851 million, reflecting a 103% increase and beating forecasts of $791 million [10]. - The company anticipates total product sales for the year to be between $2.95 billion and $3.05 billion, marking a 10% increase at the midpoint compared to previous projections [10]. Product and Market Position - Amvuttra is the only approved treatment for transthyretin amyloidosis in patients with both polyneuropathy and cardiomyopathy, with only 20% of patients diagnosed [7]. - The drug competes with offerings from Pfizer, BridgeBio, and Ionis Pharmaceuticals, but Alnylam's targeted approach has positioned it favorably in the market [5][6]. Growth Drivers - The company identified 170 health systems that prescribe approximately 80% of TTR medicines, ensuring that 90% of patients can access Amvuttra treatment within a 10-mile radius [8]. - A significant portion of U.S. patients are receiving insurance coverage for Amvuttra, further supporting its market penetration [8]. Future Outlook - Alnylam is developing a next-generation drug, nucresiran, which aims to further reduce problematic TTR protein levels and could be administered every six months [11]. - The company expects to launch nucresiran for cardiomyopathy patients around 2030, depending on clinical trial outcomes [12].

Core Viewpoint - Alnylam Pharmaceuticals reported strong third-quarter earnings, but the stock is trading lower due to unmet investor expectations regarding Amvuttra sales, despite significant year-over-year growth in revenue and earnings [1][2][7]. Financial Performance - The company reported adjusted earnings of $2.90 for Q3 2025, a significant improvement from a loss of $0.50 in the same quarter last year, surpassing analyst estimates of $0.75 [2]. - Quarterly sales reached $1.25 billion, representing a 149% increase year-over-year, exceeding the consensus estimate of $977.79 million [2]. Revenue Breakdown - Total net product revenues increased by 103% (99% at constant currency), primarily driven by Amvuttra's growth due to increased patient demand, particularly among patients with ATTR amyloidosis with cardiomyopathy in the U.S. [3]. - Amvuttra sales surged by 165% to $685.30 million, while Onpattro revenue decreased by 22% to $39.07 million. Oxlumo sales increased by 31% to $52.83 million, and Givlaari sales reached $73.87 million, up 4% year-over-year [4]. Guidance and Future Outlook - Alnylam raised its fiscal 2025 sales guidance from $3.3 billion-$3.55 billion to $3.6 billion-$3.8 billion, compared to the consensus of $3.53 billion [6]. - Total TTR net product revenues (Amvuttra and Onpattro) are now expected to be $2.48 billion-$2.53 billion, an increase from the previous guidance of $2.175 billion-$2.275 billion [6]. Market Reaction - The stock experienced an 8.05% decline, trading at $442.81, attributed to high investor expectations for Amvuttra sales, which fell short of the anticipated range of $695 million to $700 million [7].