Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) ranks among the best rebound stocks to buy right now. On March 2, Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) outlined its growth strategy at the TD Cowen 46th Annual Health Care Conference, emphasizing significant revenue expansion and ongoing innovation in RNAi therapies. The company expects revenue of around $4.4 billion to $4.7 billion by 2026, indicating strong growth and a 25% CAGR through 2030. It also aims for 30% operating margins, which might rise to ...

PresentationTolga TangulerExecutive VP & Chief Commercial Officer Good morning, everyone, and thank you for joining us today. I'm Tolga Tanguler, Chief Commercial Officer at Alnylam, and I'm thrilled to be hosting today's webinar to update you on our leadership journey in delivering transformative therapies for patients living with ATTR amyloidosis. This past Friday marked one year since AMVUTTRA was approved in the U.S. for patients with ATTR cardiomyopathy, a pivotal milestone for Alnylam that has launch ...

Alnylam Pharmaceuticals (NasdaqGS:ALNY) Update / briefing March 24, 2026 09:30 AM ET Company ParticipantsChristine Akinc - Chief Corporate Communications OfficerJohn Kennedy - SVP and TTR Franchise Commercialization LeadMark Soued - SVP, Head of U.S., and TTR LeadSameer Bansilal - VP of Global Medical Affairs and Head of TTR Disease AreaTolga Tanguler - Chief Commercial Officer and EVPTolga TangulerGood morning, everyone, and thank you for joining us today. I'm Tolga Tanguler, Chief Commercial Officer at Al ...

Alnylam Pharmaceuticals FY Conference Summary Company Overview - **Company**: Alnylam Pharmaceuticals (NasdaqGS:ALNY) - **Event**: FY Conference on March 02, 2026 Key Industry Insights - **Market Growth**: The treatment category for the disease is under-diagnosed and under-treated, with a current treatment rate of approximately 20%-25% [3][12]. The market has been growing at about 40% annually over the past few years, driven by the introduction of multiple therapies [3][10]. - **Access to Treatment**: Over 90% of patients currently have access to AMVUTTRA as a first-line therapy, indicating strong market access [13][31]. The company anticipates this access will remain stable through 2026 [13][21]. Core Product Insights - **AMVUTTRA**: - The product is positioned as the treatment of choice for a progressive fatal disease, with a focus on increasing its market share in the first-line treatment segment [14][21]. - Revenue guidance for the AMVUTTRA franchise is projected to be between $4.4 billion and $4.7 billion for 2026, representing an 83% growth at the midpoint [21][22]. - The company aims for a compound annual growth rate (CAGR) of 25% through 2030, driven primarily by the TTR franchise [21][22]. Competitive Landscape - **Market Competition**: The entry of additional competitors is seen as beneficial for overall market growth, as it raises awareness and treatment rates for the under-diagnosed population [58][59]. - **WAINUA Threat**: The company acknowledges the potential competitive threat from WAINUA but believes its advantages in treatment efficacy and administration frequency will help maintain its market position [58][61][70]. Pipeline and Future Developments - **Next-Gen Products**: The company is developing a next-generation product with a potential twice-a-year dosing schedule, aiming for deeper knockdown of TTR levels [90][94]. - **Huntington's Disease**: A phase 1B trial is underway, targeting exon 1 of the Huntington's gene, with expected data on safety and efficacy by the end of the year [171][174]. - **Cardiometabolic Franchise**: The pipeline includes products targeting blood pressure and diabetes, with phase 3 trials ongoing for zilebesiran and GRAB fourteen [134][136][138]. Financial Outlook - **Profitability Goals**: Alnylam aims for 30% operating margins through 2030, with a commitment to reinvest 30% of revenues into R&D [200][201]. The company is focused on balancing profitability with pipeline expansion [188][190]. - **Long-Term Strategy**: The company plans to diversify its business beyond TTR, with a goal of having at least two new therapies in the market by 2030 [205][206]. Additional Considerations - **Payer Policies**: The company has established 26 payer policies with no significant changes affecting access to AMVUTTRA, indicating a stable reimbursement environment [31][32]. - **Market Dynamics**: The majority of AMVUTTRA patients are Medicare beneficiaries, with a split between fee-for-service and Medicare Advantage plans [35][36]. This summary encapsulates the key points discussed during the conference, highlighting Alnylam Pharmaceuticals' strategic focus, market positioning, and future growth prospects.

Financial Data and Key Metrics Changes - Total global net product revenues for 2025 were nearly $3 billion, representing an 81% growth compared to 2024, driven by a more than doubling of revenue in the TTR franchise [25][26] - The company achieved GAAP profitability for the full year 2025 and expects to sustain profitability going forward [7][29] - Non-GAAP operating income for 2025 was $850 million, a $755 million increase compared to the previous year [28] Business Line Data and Key Metrics Changes - The TTR franchise generated $858 million in net revenues in Q4 2025, up 151% year-over-year [12] - The rare disease portfolio generated $136 million in net revenue in Q4, up 26% year-over-year [11] - GIVLAARI and OXLUMO together became a $500 million franchise in 2025, reflecting continued growth more than five years post-launch [11] Market Data and Key Metrics Changes - U.S. net revenues for the TTR franchise grew 20% compared to Q3 2025 and 222% compared to Q4 2024 [12] - International revenues grew 13% quarter-over-quarter and 47% year-over-year, indicating strong global momentum [14] - The company anticipates launching AMVUTTRA for ATTR cardiomyopathy in additional international markets throughout 2026 [14] Company Strategy and Development Direction - The company has set new five-year goals, Alnylam 2030, focusing on achieving global TTR leadership, sustainable innovation, and disciplined growth [8][9] - Plans include launching nucresiran in 2028 for polyneuropathy and 2030 for cardiomyopathy, with an aim for over 25% revenue CAGR through 2030 [10] - The company expects to invest approximately 30% of revenues in non-GAAP R&D to accelerate organic innovation and selectively access external innovation [9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the size and growth of the ATTR-CM category, noting that the majority of patients remain untreated [15] - The early momentum of AMVUTTRA is encouraging, with strong customer demand and broad access expected to drive future growth [17] - Management highlighted the importance of improving diagnosis and treatment rates to expand the category further [18] Other Important Information - The company ended 2025 with cash, cash equivalents, and marketable securities of $2.9 billion, an increase from $2.7 billion at the end of 2024 [29] - Collaboration revenue for 2025 was $553 million, an 8% growth compared to 2024, including a significant milestone payment [26] Q&A Session Summary Question: Insights on new patient additions and first-line mix for patisiran versus tafamidis - Management expressed satisfaction with the AMVUTTRA launch and noted strong fundamentals supporting continued growth [41][43] Question: Addressing choppiness in scripts and pricing dynamics - Management indicated confidence in access and pricing, with a mid-single-digit net price decline expected in 2026 [49][61] Question: Seasonality effects in Q4 and potential tailwinds for Q1 2026 - Management acknowledged typical industry seasonality but emphasized that underlying momentum remains strong [53][54] Question: Gross to net pattern over 2026 - Management confirmed a gradual mid-single-digit net price decrease expected throughout the year [59][61] Question: External innovation strategy and potential partnerships - Management highlighted a focus on complementary technologies that enhance the existing portfolio and pipeline [66][98] Question: Impact of nucresiran on operating margins - Management anticipates significant positive margin impacts post-2030 if nucresiran demonstrates a best-in-class profile [70][71] Question: Updates on the Huntington's program and trial initiation - Management is optimistic about the Huntington's program and plans to accelerate it responsibly based on initial data [78][79] Question: Prioritization of ACVR1C asset in obesity pipeline - Management explained the prioritization based on preclinical data indicating ACVR1C's potential for greater efficacy compared to other targets [86][87]

Financial Data and Key Metrics Changes - Total global net product revenues for 2025 were nearly $3 billion, representing an 81% growth compared to 2024, driven by a more than doubling of revenue in the TTR franchise [25][26] - GAAP profitability was achieved for the full year 2025, with non-GAAP operating income of $850 million, a $755 million increase compared to last year [29] - Gross margin on product sales was 77% for the full year, a 4% decrease compared to 2024, primarily due to increased royalties on AMVUTTRA [27] Business Line Data and Key Metrics Changes - The TTR franchise generated $858 million in Q4, up 151% year-over-year, and $4.4-$4.7 billion in total TTR revenue guidance for 2026, representing an 83% growth at the midpoint [11][31] - The rare disease portfolio generated $136 million in Q4, up 26% year-over-year, with GIVLAARI and OXLUMO together becoming a $500 million franchise in 2025 [11][12] Market Data and Key Metrics Changes - U.S. TTR net revenues grew 20% compared to Q3 2025 and 222% compared to Q4 2024, while international revenues grew 13% quarter-over-quarter and 47% year-over-year [13][14] - The company anticipates a mid-single-digit net price decrease for AMVUTTRA in 2026, with international TTR revenue growth expected to be consistent with 2025 [31][32] Company Strategy and Development Direction - The company aims to achieve global TTR leadership and launch nucresiran in 2028 for polyneuropathy and 2030 for cardiomyopathy as part of its Alnylam 2030 goals [8][9] - The strategy includes investing approximately 30% of revenues in non-GAAP R&D to accelerate organic innovation and selectively access external innovation [9][10] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth potential of the TTR franchise, citing strong fundamentals and early momentum from the AMVUTTRA launch [41][44] - The company is focused on building a durable growth foundation through education, awareness, and diagnosis enablement to expand the ATTR-CM category [18][19] Other Important Information - The company initiated three Phase 3 studies in 2025 and expanded its clinical pipeline with four proprietary CTAs [19][20] - The proprietary enzymatic ligation manufacturing platform, Syrelis, was launched to enhance capacity and reduce costs [7][22] Q&A Session Summary Question: Insights on new patient adds and first-line mix for patisiran versus tafamidis - Management highlighted strong AMVUTTRA launch fundamentals and improved first-line access, supporting confidence in 2026 guidance [40][43] Question: Addressing choppiness in scripts and pricing dynamics - Management noted strong access for patients and a mid-single-digit net price decline integrated into the outlook [47][49] Question: Seasonality impact on Q1 and potential tailwind - Management acknowledged typical industry seasonality but emphasized that underlying momentum remains strong [52][54] Question: Gross to net pattern expectations for 2026 - Management expects a gradual mid-single-digit net price decrease throughout the year, with access discussions largely completed [59][61] Question: External innovation strategy and potential partnerships - Management indicated a focus on complementary technologies that enhance internal innovation, with a high bar for external opportunities [66][68] Question: Impact of nucresiran on operating margin - Management expressed high conviction in nucresiran's potential to drive margins to the mid-40s post-2030 due to its best-in-class profile [70][71] Question: Insights on Huntington's program and trial initiation - Management is optimistic about the Huntington's program and aims to accelerate it responsibly based on target engagement data [78][79] Question: Prioritization of ACVR1C asset in obesity pipeline - Management prioritized ACVR1C due to its potential for more potent effects compared to other targets, with results expected by year-end [86][87]

Financial Data and Key Metrics Changes - Total global net product revenues for 2025 were nearly $3 billion, representing an 81% growth compared to 2024, driven by a more than doubling of revenue in the TTR franchise [24][25] - The company achieved GAAP profitability for the full year 2025 and expects to sustain profitability going forward [5][27] - Non-GAAP operating income for the full year was $850 million, a $755 million increase compared to the previous year [26] Business Line Data and Key Metrics Changes - The TTR franchise generated $858 million in net revenues in Q4 2025, up 151% year-over-year [11] - The rare disease portfolio generated $136 million in net revenue in Q4, reflecting a 26% increase compared to the same period last year [10] - GIVLAARI and OXLUMO together became a $500 million franchise in 2025, showing continued growth more than five years post-launch [10] Market Data and Key Metrics Changes - U.S. net revenues for the TTR franchise grew 20% compared to Q3 2025 and 222% compared to Q4 2024 [11] - International revenues grew 13% quarter-over-quarter and 47% year-over-year, indicating strong global momentum [12] - The company anticipates launching AMVUTTRA for ATTR cardiomyopathy in additional international markets throughout 2026 [12] Company Strategy and Development Direction - The company has set new five-year goals, Alnylam 2030, focusing on achieving global TTR leadership, sustainable innovation, and disciplined growth [6][9] - Plans include launching nucresiran in 2028 for polyneuropathy and 2030 for cardiomyopathy, with aspirations to deliver RNAi to 10 tissue types and expand the pipeline to over 40 clinical programs by 2030 [7][8] - The company aims for over 25% revenue CAGR through 2030 and a non-GAAP operating margin of approximately 30% [9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the size and growth of the ATTR-CM category, noting that the majority of patients remain untreated [14] - The early momentum of AMVUTTRA is encouraging, with strong customer demand and broad access expected to drive growth [16] - The company is focused on improving diagnosis and treatment rates to expand the category further [17] Other Important Information - The company initiated three Phase 3 studies in 2025 and expanded its clinical pipeline with four proprietary CTAs [18] - The proprietary enzymatic ligation manufacturing platform, Syrelis, was launched to enhance capacity and reduce costs [20] - The company ended 2025 with cash, cash equivalents, and marketable securities of $2.9 billion, an increase from $2.7 billion at the end of 2024 [27] Q&A Session Summary Question: Can you comment on new patient adds and the mix of first-line for patisiran versus tafamidis switches? - Management highlighted strong AMVUTTRA launch momentum and improved first-line access, supporting confidence in 2026 guidance [38][41] Question: Can you speak to the choppiness in scripts for the first quarter and pricing dynamics? - Management noted that the majority of patients have first-line access and expect a mid-single-digit net price decline in 2026, which is integrated into their outlook [44][47] Question: Have you seen any seasonality during the fourth quarter? - Management acknowledged typical industry seasonality but emphasized that underlying momentum remains strong [50][51] Question: What is the rationale for prioritizing the ACVR1C asset over others in your pipeline? - Management indicated that ACVR1C appears to be a more potent target based on preclinical work and emerging data [81][85] Question: How should we think about the impact of nucresiran on operating margins once available? - Management expects nucresiran to have a best-in-class profile, potentially driving margins to the mid-40s post-2030 [68][69]

© 2026 Alnylam Pharmaceuticals, Inc. 1 Agenda Fourth Quarter and Full Year 2025 Financial Results Welcome February 12, 2026 • Christine Akinc Chief Corporate Communications Officer Overview • Yvonne Greenstreet, M.D. Chief Executive Officer Commercial Highlights • Tolga Tanguler Chief Commercial Officer Pipeline • Pushkal Garg, M.D. Chief Research & Development Officer Financial Summary • Jeff Poulton Chief Financial Officer Q&A Session 2 Alnylam Forward-Looking Statements This presentation contains forward ...

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].

Group 1 - The small nucleic acid industry is expected to enter its first explosive growth cycle in 2025, with commercialization progress and new platform validations exceeding market expectations [1] - Alnylam's drug AMVUTTRA for treating ATTR is projected to become the first small nucleic acid drug with sales exceeding $2 billion this year [1] - Ionis's drug for treating sHTG saw a 35% stock price increase after positive data was released in September, with key phase 3 data and commercialization progress in 2026 being noteworthy [1] Group 2 - Arrowhead is focusing on weight loss and CNS projects leveraging its leading liver-external Trim platform, with the first commercial product for treating FCS, Plozasiran, expected in November [1] - The acceleration of small nucleic acid drug commercialization is driving continuous growth in demand for related raw materials and outsourced R&D [1] - The sentiment in the pharmaceutical sector is currently low, but upcoming data releases from the ASH conference, new medical insurance catalogs, and the first version of the commercial health insurance innovative drug catalog are expected to improve the outlook for innovative drugs, CXO, and the medical device industry [1] Group 3 - The vaccine ETF (159643) tracks the vaccine biotech index (980015), which selects listed companies involved in vaccine development, production, and sales, reflecting the overall performance of the vaccine sector within the biopharmaceutical industry [1] - This index exhibits high growth potential and strong specialization characteristics [1]