Amsterdam, the Netherlands, August 26, 2025 – Scenic Biotech, a pioneer in modifier therapies for severe diseases, today announced a license and research agreement with Alnylam Pharmaceuticals, Inc. (NASDAQ: ALNY) to leverage Scenic's Cell-Seq platform for discovering novel targets for RNAi therapeutics. "Collaborating with the leading innovator in RNAi therapeutics represents significant validation of our Cell-Seq platform's unique ability to connect cellular pathways to actionable drug targets," said Vin ...

Summary of Alnylam Pharmaceuticals (ALNY) FY Conference Call - August 12, 2025 Company Overview - Alnylam Pharmaceuticals was founded in February 2002 and is recognized as a global leader in RNA interference technology, which has led to the development of a new class of medications [4][5] - The company has established a robust pipeline with six approved medications and aims to drive long-term growth through three pillars: leadership in transthyretin amyloidosis (ATTR), growth through innovation, and outstanding commercial performance [5][6] Core Points and Arguments Leadership in ATTR - The recent approval of Ambutra for ATTR cardiomyopathy is expected to set a new standard of care, addressing a significant unmet need in a rapidly growing patient segment [5][6] - Ambutra's clinical profile and the growing patient population present a strong franchise opportunity for Alnylam [5] Commercial Performance - Alnylam reported $492 million in revenues for the quarter, exceeding consensus estimates by 34% [8] - The launch of Ambutra has been successful, with half of the priority healthcare systems adding it to their formularies within five to six weeks of launch [12] - The company anticipates continued growth through increased diagnosis rates and treatment penetration among existing patients [15] Patient Demographics and Treatment Dynamics - Globally, there are approximately 300,000 patients affected by ATTR cardiomyopathy, with about 80% remaining undiagnosed [14] - In the U.S., around 150,000 patients are affected, with an estimated 10,000 new patients presenting annually [16] - There is a significant opportunity to treat patients who are stabilizer progressors, with about 45,000 patients globally on stabilizers, and 30-50% of them may continue to progress [19] Competitive Landscape - Alnylam positions Ambutra as the first RNAi silencer approved for both manifestations of ATTR, emphasizing its unique mechanism of action [21][22] - The company is focused on establishing Ambutra as a first-line treatment option, leveraging its robust clinical data package [24] - Future competition from gene editing technologies is acknowledged, but Alnylam is advancing its next-generation RNA silencer, Neuquesiran, which could provide significant benefits [28][29] International Expansion and Regulatory Considerations - Alnylam has received regulatory approvals in Europe, Brazil, and Japan, with international launches expected to ramp up in 2026 [30][31] - The company is monitoring the Most Favored Nation (MFN) policy but continues to move forward with its launch processes [31][33] Pipeline Developments - The Zalbeceran program targets hypertension, addressing a significant unmet need as up to 80% of patients on therapy do not meet blood pressure targets [35][36] - Alnylam is planning to start a cardiovascular outcomes trial for Zalbeceran later this year [41] - The company is also advancing its Melvisiran program targeting cerebral amyloid angiopathy (CAA), a condition with no approved therapies [44][45] Future Outlook - Alnylam is focused on internal growth and innovation, while remaining open to external opportunities that align with its strategic goals [54] Other Important Content - The company emphasizes the importance of adherence to treatment regimens, particularly in rapidly progressing conditions like ATTR and hypertension [27][36] - Alnylam's approach to treatment includes a focus on patient quality of life and functional capacity, which resonates with both physicians and patients [23]

Investors might want to bet on Alnylam Pharmaceuticals (ALNY) , as earnings estimates for this company have been showing solid improvement lately. The stock has already gained solid short-term price momentum, and this trend might continue with its still improving earnings outlook.The rising trend in estimate revisions, which is a result of growing analyst optimism on the earnings prospects of this RNA interference drug developer, should get reflected in its stock price. After all, empirical research shows a ...

One other Medical stock that has outperformed the sector so far this year is Alnylam Pharmaceuticals (ALNY) . The stock is up 70.8% year-to-date. For Alnylam Pharmaceuticals, the consensus EPS estimate for the current year has increased 41.2% over the past three months. The stock currently has a Zacks Rank #2 (Buy). To break things down more, ADC Therapeutics SA belongs to the Medical - Biomedical and Genetics industry, a group that includes 490 individual companies and currently sits at #143 in the Zacks I ...

Core Viewpoint - Alnylam Pharmaceuticals reported strong second-quarter earnings and raised its 2025 sales guidance, indicating positive momentum in its product launches and overall financial performance [1][3]. Financial Performance - The company reported adjusted earnings of 32 cents for Q2 2025, a decrease from 56 cents year-over-year, but significantly better than analysts' expectations of a loss of 79 cents [1]. - Quarterly sales reached $773.69 million, reflecting a 17% year-over-year increase and surpassing the consensus estimate of $638.55 million [1]. Product Revenue - Global net product revenues for Amvuttra and Onpattro were $492 million and $53 million, respectively, while Givlaari and Oxlumo generated $81 million and $47 million in revenue for the same quarter [2]. - The launch of Amvuttra for ATTR-CM has been particularly successful, with approximately 1,400 patients treated by June 30, 2025, contributing to an estimated $150 million increase in revenue [4]. Guidance and Analyst Outlook - Alnylam raised its fiscal 2025 sales guidance from $2.7 billion-$3 billion to $3.3 billion-$3.55 billion, exceeding the consensus of $2.89 billion [3]. - Analysts from Chardan Research and William Blair expressed positive outlooks, with Chardan highlighting the strong start of Amvuttra's launch [3][6]. Analyst Ratings and Price Forecasts - Various analysts have upgraded their price forecasts for Alnylam, with Chardan raising its target from $325 to $400, and UBS increasing its forecast from $403 to $550 [5][8]. - Morgan Stanley maintained an Equal-Weight rating while increasing its price forecast from $312 to $405, and Piper Sandler raised its target from $304 to $449 [7].

Core Insights - Alnylam Pharmaceuticals reported second-quarter 2025 earnings of $0.32 per share, significantly surpassing the Zacks Consensus Estimate of a loss of $0.03, primarily due to increased revenues from its lead drug, Amvuttra [1][11] - Total revenues for the quarter reached $773.7 million, exceeding the Zacks Consensus Estimate of $673 million, and reflecting a 17% year-over-year increase [2] - The company's net product revenues were $672.2 million, a 64% increase year-over-year, driven by strong demand for Amvuttra, Givlaari, and Oxlumo [3] Revenue Breakdown - Alnylam's net product revenues from Amvuttra amounted to $492 million, representing a 114% increase year-over-year, aided by expanded label use and patient switches from Onpattro [8][11] - Givlaari generated sales of $80.8 million, a 30% year-over-year increase, while Oxlumo recorded revenues of $46.9 million, up 15% year-over-year [9] - Net revenues from collaborators were $61.5 million, down 73% from the previous year, primarily due to a significant milestone payment received in the prior quarter [4] Financial Guidance - Alnylam raised its 2025 product revenue guidance to a range of $2.65 billion to $2.8 billion, up from the previous range of $2.05 billion to $2.25 billion [11][16] - The guidance for net revenues from collaborations and royalties remains unchanged at $650 million to $750 million [16] Research and Development - Adjusted R&D expenses increased by 11% year-over-year to $274.1 million, reflecting costs associated with new studies in partnership with Roche [13] - Adjusted SG&A expenses rose by 26% year-over-year to $261.2 million, driven by higher employee compensation and marketing investments related to Amvuttra [14] Cash Position - As of June 30, 2025, Alnylam's cash, cash equivalents, and marketable securities totaled $2.86 billion, an increase from $2.63 billion as of March 31, 2025, primarily due to net cash inflows from operating activities [15]

Core Insights - Alnylam Pharmaceuticals reported a revenue of $773.69 million for the quarter ended June 2025, reflecting a year-over-year increase of 17.3% and a surprise of +14.96% over the Zacks Consensus Estimate of $673 million [1] - The company's EPS was $0.32, down from $0.56 in the same quarter last year, with an EPS surprise of +1166.67% compared to the consensus estimate of -$0.03 [1] Revenue Breakdown - Product revenues were $672.21 million, exceeding the average estimate of $540.14 million by analysts, representing a year-over-year increase of +63.9% [4] - Royalty revenue reached $39.98 million, surpassing the average estimate of $32.74 million, with a year-over-year change of +78.5% [4] - Net Product Revenue from Oxlumo was $46.87 million, slightly above the estimated $46.64 million, marking a +15.4% change year over year [4] - Net Product Revenue from Givlaari was $80.85 million, exceeding the average estimate of $72.59 million, reflecting a +30.1% year-over-year increase [4] - Net Product Revenue from Amvuttra was $491.95 million, significantly higher than the estimated $365.41 million, showing a +113.8% change compared to the previous year [4] - Net Product Revenue from Onpattro was $52.24 million, below the average estimate of $45.89 million, indicating a -32.4% year-over-year decline [4] Research Collaborator Revenues - Net revenues from research collaborators totaled $61.5 million, falling short of the average estimate of $90.07 million, representing a -73% change year over year [4] - Other net revenues from research collaborators were $10.69 million, exceeding the estimated $3 million, with a remarkable +872.4% change compared to the previous year [4] - Net revenues from Regeneron Pharmaceuticals were $32.54 million, significantly lower than the estimated $17.5 million, reflecting an -84.3% change year over year [4] - Net revenues from Roche were $18.27 million, surpassing the average estimate of $12 million, with a +10.7% year-over-year change [4] Stock Performance - Alnylam's shares returned +3% over the past month, outperforming the Zacks S&P 500 composite's +2.7% change [3] - The stock currently holds a Zacks Rank 3 (Hold), indicating potential performance in line with the broader market in the near term [3]

Financial Data and Key Metrics Changes - Alnylam reported total net product revenues of $672 million for Q2 2025, representing a 64% year-over-year growth [9][37] - The TTR franchise generated $544 million in revenues, reflecting a 77% increase year-over-year [8][11] - The company increased its total net product revenue guidance for 2025 from a range of $2.05 billion to $2.25 billion to a revised range of $2.65 billion to $2.8 billion, a 27% increase at the midpoint [9][42] Business Line Data and Key Metrics Changes - The TTR franchise in the U.S. saw combined sales of ONPATTRO and Ambutra rise by 80% compared to Q1 2025, driven primarily by the launch of Ambutra [14][16] - The rare franchise, including GIVLAARI and OXLUMO, delivered $128 million in combined Q2 sales, up 24% year-over-year [12][11] - The U.S. TTR franchise grew 125% compared to 2024, primarily due to the demand from the ATTR Centimeters launch [15][11] Market Data and Key Metrics Changes - International markets are expected to contribute to Ambutra's revenues in the second half of 2025, with launches in Germany and Japan [15][27] - The U.S. TTR franchise achieved $383 million in Q2 2025, indicating robust growth dynamics [16][11] - The company has yet to recognize any ATTR Centimeters revenue internationally, but anticipates strong growth as international launches commence [15][11] Company Strategy and Development Direction - Alnylam focuses on three core elements: TTR leadership, growth through innovation, and strong financial performance [6][7] - The company aims to establish long-term leadership in TTR and expand its pipeline of RNAi therapeutics [6][9] - Alnylam is committed to disciplined capital allocation and sustainable growth, with a focus on patient and customer centricity [28][9] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong early performance of Ambutra and its potential for sustainable growth [9][28] - The company highlighted the importance of real-world evidence generation and the development of next-generation RNAi therapeutics [27][9] - Management remains optimistic about achieving its Alnylam Pizza Fit by 25 goals, reinforcing its position as a top-tier biotech company [10][9] Other Important Information - The FDA granted fast track designation to nucrisiran for ATTR Centimeters, enhancing its development prospects [9][31] - Alnylam's gross margin on product sales was 79% for the quarter, down from 84% in 2024, primarily due to increased royalties [39][37] - The company ended the quarter with cash, cash equivalents, and marketable securities of $2.9 billion, an increase from $2.7 billion at the end of 2024 [41][37] Q&A Session Summary Question: Can you provide details on the patient profiles for Ambutra? - Management noted broad uptake across first-line patients and stabilizer progressors, with a healthy mix emerging [48][49] Question: How are you thinking about net price for Ambutra moving forward? - The expectation is for a mid-single-digit reduction in net price for Ambutra in 2025 compared to 2024 [56][57] Question: Was there any bolus effect in cardiomyopathy scripts in Q2? - Management confirmed solid commercial execution and emphasized that results are not just a flash in the pan, expecting continued sustainable growth [60][61] Question: Can you comment on the criteria physicians are using for stabilizer progressors? - Clinical evidence suggests that 30% to 50% of patients on stabilizers may progress, and physicians are using a variety of factors to determine treatment [81][82] Question: How should we think about the rate of new patient starts per quarter? - Management expects both categories of patients to continue growing, with a balanced uptake between new patients and stabilizer progressors [97][98]

Financial Data and Key Metrics Changes - Alnylam reported total net product revenues of $672 million for Q2 2025, representing a 64% year-over-year growth [9][35] - The TTR franchise generated revenues of $544 million, reflecting a 77% increase year-over-year [7][35] - The company increased its total net product revenue guidance for 2025 from a range of $2.05 billion to $2.25 billion to a revised range of $2.65 billion to $2.8 billion, marking a $575 million or 27% increase at the midpoint [9][39] Business Line Data and Key Metrics Changes - The TTR franchise in the U.S. saw combined Q2 sales of ONPATTRO and Ambutra rise by 80% compared to Q1, driven primarily by the Ambutra launch [13][35] - The rare franchise, including GIVLAARI and OXLUMO, delivered $128 million in combined Q2 sales, up 24% year-over-year [12][35] - The U.S. TTR franchise grew 125% compared to 2024, primarily due to the ATTR Centimeters launch [14] Market Data and Key Metrics Changes - Approximately 1,400 cardiomyopathy patients were receiving Ambutra by the end of Q2, contributing an estimated $150 million in revenue [13][23] - International markets are expected to begin contributing to Ambutra sales in the second half of the year, with launches in Germany and Japan [8][26] - The U.S. TTR franchise achieved $383 million in Q2, representing an 80% quarter-over-quarter growth [15] Company Strategy and Development Direction - Alnylam is focusing on TTR leadership, growth through innovation, and strong financial performance as key strategic pillars [4][6] - The company aims to establish itself as a top-tier biotech firm by delivering sustainable innovation and value creation [5][10] - The launch of Ambutra is seen as a flagship commercial franchise with robust long-term growth potential [6][27] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the strong early launch performance of Ambutra and the potential for continued growth [9][39] - The company remains committed to advancing care through real-world evidence generation and the development of next-generation RNAi therapeutics [26][34] - Management highlighted the importance of disciplined execution and patient-centricity in driving long-term growth [27][39] Other Important Information - The FDA granted fast track designation to nucrisiran for ATTR Centimeters, which is expected to streamline the review process [9][31] - The gross margin on product sales was reported at 79%, down from 84% in the previous year, primarily due to increased royalties on Ambutra [37] Q&A Session Summary Question: Details on patient profiles for Ambutra - Management noted broad uptake across first-line patients and stabilizer progressors, with a healthy share of first-line patients emerging [45][46] Question: Thoughts on net price for Ambutra - The expectation is for a mid-single-digit reduction in net price for Ambutra in 2025 compared to 2024 [50][52] Question: Bolus effect in cardiomyopathy scripts - Management indicated solid commercial execution and emphasized that results are not just a flash in the pan, expecting continued sustainable growth [56][57] Question: Assumptions underpinning updated TTR franchise guidance - Management expects steady growth in both first-line and second-line patient flows, with a healthy trend in first-line use observed [65][70] Question: Criteria for stabilizer progressors - Clinical evidence suggests that one-third to half of patients on stabilizers progress, and physicians are using a variety of factors to determine eligibility for Ambutra [76][82] Question: Rate of new patient starts per quarter - Management is pleased with the initial uptake and expects both categories of patients to continue growing, though specific numbers will not be reported [91][94] Question: Payer requirements for Ambutra treatment - Broad coverage is reported across Medicare and commercial payers, with minimal step edits required for access [98][102]

Financial Performance - Total Q2 Net Product Revenues reached $672 million, a 64% increase year-over-year[17, 24] - Total TTR Net Revenues in Q2 were $544 million, representing a 77% year-over-year growth[17] - Rare Franchise Net Product Revenues in Q2 totaled $128 million, a 24% increase year-over-year[28] - Alnylam increased its 2025 Total Net Product Revenue Guidance to a range of $2650 million to $2800 million[17, 74] - The company's Q2 Non-GAAP Operating Income was $95 million[70] Commercial Highlights - U S ATTR-CM launch contributed approximately $150 million in revenue during Q2[33, 34] - There are approximately 1,400 ATTR-CM patients receiving AMVUTTRA as of June 30, 2025[17, 37, 45] - U S TTR franchise experienced 125% year-over-year growth in Q2[32] - GIVLAARI saw a 30% year-over-year growth, driven by an approximately 18% increase in global patients on therapy[29] - OXLUMO experienced a 15% year-over-year growth, driven by an approximately 16% increase in global patients on therapy[29] Pipeline Development - The TRITON-CM Phase 3 study for nucresiran in ATTR-CM has been initiated, with a targeted launch around 2030[17, 60] - Phase 1 multidose results for mivelsiran in Alzheimer's Disease were announced[17] - A Phase 1 study for ALN-4324 in Type 2 Diabetes was initiated[17]