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Here are 1-3 brief analogies for Alnylam Pharmaceuticals (ALNY):

• The Moderna of RNAi: Alnylam is a pioneer in RNA interference (RNAi) therapeutics, developing gene-silencing drugs using advanced RNA technology, much like Moderna is known for its leadership in mRNA technology.
• The Genentech of RNAi: Similar to how Genentech was a foundational company for the modern biotechnology industry (pioneering recombinant DNA), Alnylam is a trailblazer and leader in the field of RNA interference (RNAi), creating an entirely new class of genetic medicines.
• The Tesla for genetic diseases: Alnylam is a highly innovative company developing breakthrough, platform-based RNAi treatments for serious diseases, akin to Tesla's innovative and category-leading approach in electric vehicles and clean energy.

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• ONPATTRO (patisiran): Treats hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy.
• AMVUTTRA (vutrisiran): Treats adult patients with hereditary transthyretin-mediated (hATTR) amyloidosis.
• GIVLAARI (givosiran): Treats adults with acute hepatic porphyria (AHP).
• OXLUMO (lumasiran): Treats primary hyperoxaluria type 1 (PH1) to reduce urinary oxalate levels.

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Alnylam Pharmaceuticals (ALNY) sells primarily to other companies, specifically specialty distributors and specialty pharmacies, rather than directly to individuals. These companies then facilitate the distribution of Alnylam's RNAi therapeutics to healthcare providers and ultimately to patients.

According to Alnylam's most recent 10-K filing (for the fiscal year ended December 31, 2023), no single customer accounted for 10% or more of its total net product revenues for the years ended December 31, 2023, 2022, and 2021. Therefore, Alnylam does not disclose specific major customers by name due to a lack of revenue concentration with any single entity.

However, the distribution channels for Alnylam's products typically involve major players in the pharmaceutical distribution and specialty pharmacy industries. While Alnylam has not identified any of these specific companies as individually representing 10% or more of their revenue, the types of companies they sell through include:

• Specialty Pharmaceutical Distributors: These companies purchase pharmaceuticals directly from manufacturers and distribute them to hospitals, pharmacies, and other healthcare providers. Major players in this sector include:
  • AmerisourceBergen Corp. (Symbol: ABC)
  • Cardinal Health, Inc. (Symbol: CAH)
  • McKesson Corporation (Symbol: MCK)
• Specialty Pharmacies (often part of larger healthcare or PBM organizations): These pharmacies specialize in dispensing high-cost, high-touch medications that require specific handling and patient support. They often work directly with patients and healthcare providers. Examples of companies operating specialty pharmacies include:
  • CVS Health Corporation (Symbol: CVS) (operates CVS Specialty)
  • UnitedHealth Group Incorporated (Symbol: UNH) (operates OptumRx Specialty Pharmacy)
  • Cigna Group (Symbol: CI) (operates Evernorth Specialty Pharmacy)

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• AGC Biologics
• Croda International Plc (CRDA.L)
• Evonik Industries AG (XTRA: EVK)
• Nitto Denko Corporation (TYO: 6988)
• Thermo Fisher Scientific Inc. (TMO)

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Yvonne Greenstreet, MD, MBA - Chief Executive Officer

Dr. Yvonne Greenstreet joined Alnylam in 2016 as Chief Operating Officer, was promoted to President and COO in 2020, and was appointed Chief Executive Officer in late 2021. She brings over 25 years of experience in the biopharmaceutical industry, focusing on strategy, innovation, and bringing transformative medicines to patients globally. Before joining Alnylam, she served as Senior Vice President and Head of Medicines Development at Pfizer from 2011 to 2013, where she was part of the executive team leading a $16 billion division. Prior to Pfizer, she spent 18 years at GlaxoSmithKline (GSK) in various roles of increasing responsibility, including Senior Vice President and Chief of Strategy for Research and Development, and Chief Medical Officer for Europe. She trained as a physician, earning her medical degree from The University of Leeds in the UK, and holds an MBA from INSEAD Business School in France.

Jeff Poulton, MBA - Chief Financial Officer

Jeff Poulton joined Alnylam as Chief Financial Officer in August 2019. Before his tenure at Alnylam, he was the Chief Financial Officer at Indigo Agriculture, a plant microbiome company, from January 2018 to April 2019, where he supported the company's initial commercial scale-up. From 2003 to December 2017, Mr. Poulton held several positions of increasing responsibility at Shire Plc, ultimately serving as Chief Financial Officer and a member of Shire's Executive Committee and Board of Directors from January 2015 to December 2017. While at Shire, he also directed the company's rare disease commercial operations in the US, Latin America, and Asia Pacific, as well as its global rare disease business unit. Earlier in his career, he held financial leadership roles at Cinergy Corp and PPG Industries and served as a Commissioned Officer in the United States Navy. He holds a BA in Economics from Duke University and an MBA in Finance from the Kelley School of Business at Indiana University.

Pushkal Garg, MD - Chief Research & Development Officer

Dr. Pushkal Garg joined Alnylam in 2014 to lead the Clinical Development organization and was promoted to Chief Medical Officer in 2017. In June 2025, he was appointed Executive Vice President, Chief Research and Development Officer, overseeing all R&D activities. Prior to Alnylam, Dr. Garg was Vice President, Global Clinical Research, Immunoscience at Bristol-Myers Squibb (BMS), where he led the Immunoscience franchise and oversaw the development of clinical assets across rheumatology, gastroenterology, nephrology, and transplantation.

Kevin Fitzgerald, PhD - Chief Scientific Officer

Dr. Kevin Fitzgerald serves as the Chief Scientific Officer at Alnylam Pharmaceuticals. He is an inventor on over 50 patents related to Alnylam's programs and has authored more than 50 papers in scientific journals. He has led multiple programs from discovery through preclinical development, regulatory submissions, and early clinical development, including Alnylam's inclisiran program. Before 2005, Dr. Fitzgerald spent seven years at Bristol-Myers Squibb.

Tolga Tanguler, MBA - Chief Commercial Officer

Tolga Tanguler joined Alnylam in January 2021 as Executive Vice President and Chief Commercial Officer, bringing over 20 years of experience in the global biopharmaceutical industry. At Alnylam, he has played a significant role in scaling the company globally, expanding its commercial presence to over 60 markets, and tripling the number of patients served and product sales within three years. Mr. Tanguler has led commercialization efforts across various therapeutic areas, from early development to product launch and growth management.

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The key risks to Alnylam Pharmaceuticals (ALNY) business are its regulatory and legal challenges, the intense competitive landscape, and its product concentration risk.

1. Regulatory and Legal Challenges: Alnylam Pharmaceuticals faces significant risks from an evolving regulatory landscape, including changes in healthcare reforms, pricing, reimbursement, and insurance coverage. A particularly pressing near-term risk is a subpoena received from the U.S. Attorney's Office concerning pricing practices for its main products. This represents a serious legal, financial, and reputational risk for the company, introducing significant uncertainty. The high list price of Alnylam's drug Amvuttra, at approximately $480,000 per year, could also lead to access challenges and potential pushback from payers. Non-compliance with intricate requirements of U.S. governmental pricing programs, such as the 340B Drug Pricing Program, also poses a material business risk.
2. Intense Competition: The biopharmaceutical industry, particularly the RNA interference (RNAi) therapeutics sector where Alnylam operates, is highly competitive. Alnylam faces challenges from numerous companies, including both established pharmaceutical giants and emerging biotech firms like Silence Therapeutics, Moderna, BioNTech, Wave Life Sciences, Roche, Pfizer, BridgeBio Pharma, Amgen, Gilead Sciences, Vertex Pharmaceuticals, Regeneron Pharmaceuticals, Biogen, and Ionis Pharmaceuticals. This competition includes rival products, some of which are generics, that could impact the commercial success of Alnylam's marketed drugs. The transthyretin-mediated (ATTR) amyloidosis cardiomyopathy (ATTR-CM) market, where Alnylam's Amvuttra competes, is rapidly expanding with multiple products vying for market share.
3. Product Concentration Risk: Alnylam's financial health and revenue stream are heavily reliant on a single therapeutic area, specifically its TTR (transthyretin-mediated amyloidosis) franchise, with Amvuttra being a significant driver of revenue. While the company is working to diversify its pipeline, this concentration creates a vulnerability. Should a competitor like Pfizer's Vyndaqel/Vyndamax gain significant market share, or if new gene-editing therapies become viable, Alnylam's top-line revenue could face substantial pressure. Concerns about "TTR franchise momentum" have also been raised, particularly following reports of slowing sales from a key rival in the third quarter.

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Emerging Threats for Alnylam Pharmaceuticals (ALNY)

The most significant clear emerging threat to Alnylam Pharmaceuticals stems from the advancement of in vivo gene editing therapies for conditions currently targeted by Alnylam's RNA interference (RNAi) drugs.

Specifically, for ATTR amyloidosis, a key indication for Alnylam's flagship products Onpattro (patisiran) and Amvuttra (vutrisiran), competing companies are developing gene-editing treatments designed to offer a potentially one-time, permanent therapeutic solution. Examples include:

• Vertex Pharmaceuticals and CRISPR Therapeutics' CTX310: An in vivo CRISPR-based gene editing therapy targeting ATTR amyloidosis.
• Intellia Therapeutics and Regeneron Pharmaceuticals' NTLA-2001: Another in vivo CRISPR-based therapy aimed at ATTR amyloidosis.

These gene editing therapies represent a potentially disruptive technology that could fundamentally alter the treatment paradigm for genetic diseases by offering a single administration to durably silence or correct the underlying genetic defect, compared to Alnylam's chronic RNAi treatments. If successful in clinical trials and commercialization, these therapies could significantly erode the market share and long-term revenue potential of Alnylam's current and pipeline RNAi products in these specific indications.

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Alnylam Pharmaceuticals (NASDAQ: ALNY) focuses on RNA interference (RNAi) therapeutics, with several commercial products addressing rare diseases. The addressable markets for their main products are as follows:

AMVUTTRA (vutrisiran)

• AMVUTTRA is approved for hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy (hATTR-PN) and transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy (ATTR-CM) in regions including the U.S., European Union, United Kingdom, Brazil, and Japan.
• The total addressable market for vutrisiran (which includes AMVUTTRA) is projected to reach approximately $2.62 billion globally by 2035, growing from an estimated $0.843 billion in 2025.
• North America represented the largest regional market for vutrisiran in 2024.
• In 2025, the vutrisiran market is expected to be around $0.67 billion.
• Analysts project AMVUTTRA sales to reach $9 billion by 2035 across both ATTR-CM and hATTR-PN indications.

ONPATTRO (patisiran)

• ONPATTRO is approved in the United States and Canada for adults with hATTR amyloidosis with polyneuropathy, and in the European Union, Switzerland, and Brazil for hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, as well as in Japan.
• Globally, hereditary transthyretin-mediated amyloidosis affects an estimated 5,500 to 38,500 individuals, with U.S. estimates for hATTR amyloidosis with polyneuropathy ranging from 104 to 3,847 cases.
• North America was the largest regional market for ONPATTRO in 2024.
• Alnylam's total TTR franchise (AMVUTTRA and ONPATTRO combined) is expected to generate net product revenues between $2.475 billion and $2.525 billion globally for 2025.

GIVLAARI (givosiran)

• GIVLAARI is designed for the treatment of acute hepatic porphyria (AHP).
• The estimated population of diagnosed AHP patients with active disease in the U.S. and Europe is approximately 3,000 individuals.

OXLUMO (lumasiran)

• OXLUMO is approved for the treatment of primary hyperoxaluria type 1 (PH1).
• The global primary hyperoxaluria treatment market, which includes OXLUMO, was valued at approximately $168.96 million in 2023 and is projected to reach about $365.49 million by 2031, with a compound annual growth rate (CAGR) of 9.8% from 2024 to 2031.
• Alnylam estimates that between 1,000 and 1,700 people in the U.S. and Europe with a confirmed PH1 diagnosis (who have not received a liver transplant) would be eligible for OXLUMO treatment.
• Europe is expected to be the dominant region in the primary hyperoxaluria treatment market, holding a 39.6% share.
• In Europe, PH1 has an estimated prevalence of 1 to 3 cases per million people.
• North America was the largest regional market for OXLUMO in 2024.

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Expected Drivers of Future Revenue Growth for Alnylam Pharmaceuticals (ALNY)

• Continued Growth of the TTR Franchise, Especially AMVUTTRA: Alnylam Pharmaceuticals anticipates significant revenue growth from its transthyretin-related (TTR) franchise, with AMVUTTRA (vutrisiran) being a primary driver. The company has repeatedly raised its 2025 total net product revenue guidance, largely due to the strong launch performance and increasing patient uptake of AMVUTTRA in ATTR cardiomyopathy (ATTR-CM) in the U.S. and through international launches in key markets such as the EU, UK, Japan, and Brazil. The TTR franchise demonstrated 77% year-over-year growth in Q2 2025, and analysts project AMVUTTRA's revenue to reach $3.9 billion by 2026.
• Launch and Expansion of Nucresiran: The development and anticipated launch of nucresiran, a next-generation TTR silencer, are expected to fuel future revenue. Nucresiran is designed to offer greater than 95% knockdown of TTR and potentially be administered twice-annually, aiming to solidify Alnylam's leadership in the expanding ATTR-CM market. Phase 3 trials, TRITON-CM for ATTR-CM and TRITON-PN for hATTR-PN, are underway or planned to accelerate its market entry.
• Advancement of Zilebesiran for Hypertension: Zilebesiran, an RNAi therapeutic for hypertension, represents a substantial future revenue opportunity. Alnylam, in collaboration with Roche, is planning a large Phase 3 cardiovascular outcomes trial for zilebesiran, targeting a market estimated to be over $100 billion. Despite mixed Phase 2 results for KARDIA-3, the drug is still seen as having multi-billion dollar potential, with Roche aiming to start the Phase 3 ZENITH trial by the end of 2025.
• Broad Pipeline Expansion and New Product Filings: Alnylam is actively expanding its clinical pipeline, aiming to double it by the end of 2025. The company plans to file Investigational New Drug (IND) applications for at least nine new Alnylam-led RNAi programs by the end of 2025, targeting various tissue types including liver, central nervous system (CNS), adipose tissue, and muscle. This includes new clinical programs for conditions such as Huntington's disease, bleeding disorders, and mivelsiran for Alzheimer's disease, which could lead to new product launches and market expansion in the coming years.
• Steady Performance of Other Rare Disease Products: Beyond the TTR franchise, Alnylam's other rare disease products, GIVLAARI (givosiran) and OXLUMO (lumasiran), are expected to contribute to consistent revenue. These products continue to demonstrate steady performance and meaningful impact for patients, supporting overall company growth.

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Share Repurchases

• In September 2025, Alnylam repurchased approximately $637.8 million aggregate principal amount of its 1.00% convertible senior notes due 2027, with a total repurchase cost of approximately $1,105.8 million.

Share Issuance

• In September 2025, Alnylam completed an offering of $661.25 million in 0.00% Convertible Senior Notes due 2028.
• In August 2020, Alnylam raised $150 million in a Post IPO funding round.
• The number of shares issued and outstanding increased from 129,294 thousand as of December 31, 2024, to 130,977 thousand as of June 30, 2025.

Inbound Investments

• In April 2020, Alnylam entered into a $2 billion strategic financing collaboration with Blackstone, which included Blackstone's purchase of 50% of royalties on global sales of inclisiran.
• As part of the 2020 collaboration, Alnylam received up to $150 million from Blackstone Life Sciences for the development of its cardiometabolic disease programs, specifically up to $70 million for the vutrisiran HELIOS-B Phase 3 study and up to $80 million for ALN-AGT's Phase 2 and Phase 3 development.
• In April 2020, Blackstone made a $100 million equity investment in Alnylam common stock.

Capital Expenditures

• Alnylam's capital expenditures averaged $63.056 million annually from fiscal years ending December 2020 to 2024.
• Capital expenditures peaked in December 2021 at $76.372 million.
• Capital expenditures reached a 5-year low of $34.277 million in December 2024.