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Alnylam is like a **Moderna or BioNTech**, but rather than mRNA vaccines, they specialize in using RNA interference (RNAi) to create medicines that treat genetic and other complex diseases.

Think of Alnylam as a **Vertex Pharmaceuticals**, but applying RNA interference (RNAi) technology to develop groundbreaking treatments for a wider range of genetic and rare diseases.

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• ONPATTRO (patisiran): A lipid complex injection for the treatment of polyneuropathy in hereditary transthyretin-mediated amyloidosis in adults.
• GIVLAARI: A therapeutic for the treatment of acute hepatic porphyria (AHP) in adults.
• OXLUMO (lumasiran): A treatment for primary hyperoxaluria type 1 (PH1).
• Givosiran (for adolescents): An investigational treatment for acute hepatic porphyria (AHP) in adolescent patients.
• Patisiran (for cardiomyopathy): An investigational treatment for transthyretin amyloidosis (ATTR amyloidosis) with cardiomyopathy.
• Cemdisiran: An investigational RNAi therapeutic designed to treat complement-mediated diseases.
• ALN-AAT02: An investigational therapeutic for AAT deficiency-associated liver disease.
• ALN-HBV02: An investigational therapeutic aimed at treating chronic HBV infection.
• Zilebesiran: An investigational therapeutic for hypertension.
• ALN-HSD: An investigational therapeutic for NASH.
• Fitusiran: An investigational therapeutic for hemophilia and rare bleeding disorders.
• Inclisiran: An investigational therapeutic to treat hypercholesterolemia.
• Lumasiran (for advanced PH1 & recurrent renal stones): An investigational treatment for advanced primary hyperoxaluria type 1 (PH1) and recurrent renal stones.
• Vutrisiran: An investigational therapeutic in Phase 3 clinical trials for the treatment of ATTR amyloidosis.

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Alnylam Pharmaceuticals (ALNY) sells primarily to individuals (patients) who are prescribed its RNAi therapeutics by healthcare professionals. The company's major customers can be categorized by the disease areas its products address:

• Patients with Rare Genetic Diseases: This category includes individuals suffering from conditions such as hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with polyneuropathy or cardiomyopathy, acute hepatic porphyria (AHP), and primary hyperoxaluria type 1 (PH1).
• Patients with Cardio-Metabolic Diseases: This category encompasses individuals with conditions like hypercholesterolemia, hypertension, and nonalcoholic steatohepatitis (NASH), for which Alnylam is developing or commercializing treatments.
• Patients with Chronic Infectious Diseases: This category includes individuals living with chronic hepatitis B virus (HBV) infection, a therapeutic area where Alnylam is developing treatments.

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Yvonne Greenstreet, Chief Executive Officer

Dr. Yvonne Greenstreet became Chief Executive Officer of Alnylam in January 2022, having previously served as President and Chief Operating Officer from 2020 and COO from 2016. Prior to joining Alnylam, she was Senior Vice President and Head of Medicines Development at Pfizer, where she was part of the executive team leading a $16 billion division. Before Pfizer, Dr. Greenstreet spent 18 years at GlaxoSmithKline plc, serving as Senior Vice President and Chief Strategy Officer for Research and Development, and holding various other senior positions. She also practiced as a clinician before earning her MBA from INSEAD. Dr. Greenstreet has been instrumental in delivering medicines and building successful businesses across the US, Europe, and globally for over three decades.

Jeff Poulton, Executive Vice President and Chief Financial Officer

Jeff Poulton joined Alnylam as CFO in August 2019. Before Alnylam, he served as Chief Financial Officer at Indigo Agriculture, a plant microbiome company, from January 2018 to April 2019, where he supported the initial commercial scale-up and international expansion of the business. From 2003 to December 2017, Mr. Poulton held various roles of increasing responsibility at Shire Plc, culminating in his service as Chief Financial Officer and a member of Shire's Executive Committee and Board of Directors from January 2015 to December 2017. During his tenure at Shire, he also led the company's rare disease commercial operations across the US, Latin America, and Asia Pacific, as well as its global rare disease business unit. Earlier in his career, he held financial leadership positions at Cinergy Corp and PPG Industries, where he led corporate finance and business development initiatives. Mr. Poulton also served as a U.S. Navy Commissioned Officer.

Pushkal Garg, Executive Vice President, Chief Research and Development Officer

Dr. Pushkal Garg joined Alnylam in 2014 to lead the Clinical Development organization, was promoted to Chief Medical Officer in 2017, and then appointed Executive Vice President, Chief Research and Development Officer in June 2025 to oversee all R&D and Medical activities. Prior to Alnylam, Dr. Garg was Vice President, Global Clinical Research, Immunoscience at Bristol-Myers Squibb (BMS), where he strategically led the Immunoscience franchise and oversaw the development of multiple clinical assets. He also held various roles at Millennium Pharmaceuticals, overseeing the clinical development of therapeutics for inflammatory disorders.

Kevin Fitzgerald, Executive Vice President and Chief Scientific Officer

Dr. Kevin Fitzgerald joined Alnylam in 2005 as Associate Director of Research and has served in roles of increasing responsibility, becoming Chief Scientific Officer and Executive Vice President, Head of Research in May 2019. He has 24 years of drug discovery experience and has led the company's RNAi delivery efforts, contributing to two clinically validated modes of siRNA delivery and the development of Alnylam's RNAi therapeutic novel class of medicines. Prior to 2005, Dr. Fitzgerald spent seven years at Bristol-Myers Squibb.

Tolga Tanguler, Executive Vice President and 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. He has been instrumental 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 a wide range of therapeutic areas, from early development to product launch and growth management.

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Key Business Risks for Alnylam Pharmaceuticals (ALNY)

1. Clinical Development and Regulatory Approval Risk: Alnylam Pharmaceuticals' future success is highly dependent on the successful completion of clinical trials and subsequent regulatory approval for its investigational RNAi therapeutics. The company has a substantial pipeline, including several late-stage assets like patisiran for ATTR amyloidosis with cardiomyopathy, zilebesiran for hypertension, and vutrisiran for ATTR amyloidosis. Failures in clinical trials, unexpected safety concerns, or delays in obtaining regulatory approvals for these or other pipeline candidates could significantly hinder the company's growth prospects and future revenue streams.
2. Intense Competition and Market Access Challenges: While Alnylam has successfully commercialized products for rare diseases (ONPATTRO, GIVLAARI, OXLUMO), its pipeline is expanding into larger, more competitive therapeutic areas such as hypertension (Zilebesiran) and hypercholesterolemia (Inclisiran). In these markets, Alnylam faces established pharmaceutical companies with significant resources and existing market share, which could lead to challenges in achieving market penetration, securing favorable reimbursement terms, and differentiating its products.
3. Intellectual Property Protection and Patent Expiry: Alnylam's business model relies heavily on the exclusivity provided by its intellectual property, particularly patents covering its RNAi technology platform and individual therapeutic products. The expiration of patents for its marketed products or key pipeline assets, or challenges to their validity by competitors, could lead to the introduction of generic or biosimilar versions. This would result in significant pricing pressure and erosion of revenue, similar to a "patent cliff" scenario experienced by other pharmaceutical companies.

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Alnylam Pharmaceuticals (ALNY) operates in several therapeutic areas with significant addressable markets for its main products and pipeline. The addressable market sizes for their key offerings are as follows:

ONPATTRO (patisiran) and Vutrisiran (for Transthyretin-Mediated Amyloidosis)

• The global market for transthyretin amyloidosis treatment was valued at approximately USD 6.94 billion in 2024 and is projected to grow to USD 20.35 billion by 2033.
• North America represented over 46.3% of this market in 2024.
• The cardiac manifestations of transthyretin amyloidosis (ATTR-CM) segment accounted for USD 4.7 billion in revenue in 2023.
• RNA "silencers," such as patisiran and vutrisiran, are expected to grow at a compound annual growth rate (CAGR) of 29.60% through 2031.

GIVLAARI (givosiran) for Acute Hepatic Porphyria (AHP)

• The global acute hepatic porphyria treatment market was estimated at USD 4.6 million in 2025 and is projected to reach USD 7.6 million by the end of 2035, growing at a CAGR of 8.2% from 2026 to 2035.
• North America is anticipated to hold a 48% market share by 2035.

OXLUMO (lumasiran) for Primary Hyperoxaluria Type 1 (PH1)

• The primary hyperoxaluria treatment market across the 7MM (United States, EU4 (Germany, France, Italy, and Spain), United Kingdom, and Japan) was approximately USD 316 million in 2023.
• The United States alone accounted for approximately 89% of this 7MM market size in 2023.

Zilebesiran (for Hypertension)

• The global antihypertensive drugs market was valued at USD 25.50 billion in 2025 and is projected to reach USD 37.29 billion by 2035.
• North America led the global industry with a 38% revenue share in 2025.
• Specifically, for treatment-resistant hypertension, the market size in the 7MM (United States, EU4, United Kingdom, and Japan) was approximately USD 6,958 million in 2025 and is projected to reach USD 12,081 million by 2034.
• Projected revenue for Zilebesiran in the U.S. is expected to reach an annual total of $116 million by 2037.

Fitusiran (for Hemophilia and Rare Bleeding Disorders)

• The global hemophilia treatment market was estimated at USD 20.68 billion in 2025 and is predicted to increase to approximately USD 63.28 billion by 2035, expanding at a CAGR of 11.83% from 2026 to 2035.
• North America dominated the global market with a 45% market share in 2025.
• The Hemophilia A market size in the 7MM was USD 12,918 million in 2025 and is projected to reach USD 16,756 million by 2034.
• The Hemophilia B market across the 7MM was valued at approximately USD 3.4 billion in 2023.

Inclisiran (for Hypercholesterolemia)

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Alnylam Pharmaceuticals (ALNY) is expected to experience significant revenue growth over the next 2-3 years, driven by several key factors:

1. Continued uptake and market expansion of AMVUTTRA (vutrisiran) for ATTR amyloidosis with cardiomyopathy (ATTR-CM): AMVUTTRA received FDA approval for ATTR-CM in March 2025 and European Commission approval in June 2025, opening a substantial and lucrative market. The TTR franchise, which includes AMVUTTRA and ONPATTRO, is identified as the primary growth engine, with anticipated total TTR revenue guidance of $4.4 billion to $4.7 billion for 2026, representing an 83% growth compared to 2025. This growth is largely fueled by the strong performance of AMVUTTRA in the U.S. and its ongoing launch in international markets.
2. Sustained growth of the existing rare disease portfolio (GIVLAARI and OXLUMO): Alnylam's rare disease portfolio continues to deliver meaningful impact for patients and consistent business performance. GIVLAARI (for acute hepatic porphyria) and OXLUMO (for primary hyperoxaluria type 1) together became a $0.5 billion franchise in 2025, reflecting continued growth more than five years post-launch. This portfolio is projected to grow by 10% in 2026.
3. Strategic collaboration and milestone payments from Zilebesiran: Alnylam's partnership with Roche to develop and commercialize zilebesiran for hypertension represents a potential long-term growth driver, with substantial near-term development milestone payments anticipated. While the market arrival for zilebesiran is projected to be further out, potentially not before 2033 due to the need for cardiovascular outcomes trial (CVOT) data, the collaboration significantly reduces Alnylam's development costs and includes an upfront payment of $310 million, with potential milestones up to $2.8 billion. Zilebesiran is expected to initiate a global Phase 3 CVOT by late 2025.

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Share Repurchases

• Alnylam Pharmaceuticals repurchased approximately $34.4 million aggregate principal amount of its 1.00% Convertible Senior Notes due 2027 for a total cost of approximately $51.9 million in December 2025.
• In 2025, the company repurchased $672.2 million aggregate principal amount of its 1.00% Convertible Senior Notes due 2027.

Share Issuance

• In September 2025, Alnylam priced an upsized private offering of $575 million aggregate principal amount of 0.00% convertible senior notes due 2028.
• Alnylam's shares outstanding increased to 0.135 billion in 2025, representing a 5.51% increase from 2024.

Inbound Investments

• Alnylam recognized $300 million in milestone revenue during Q3 2025 from its collaboration with Roche, tied to the dosing of the first patient in a Phase 3 clinical trial.

Capital Expenditures

• Cash outflows for capital expenditures in 2025 were $58.7 million.
• Alnylam plans to invest $250 million to expand its manufacturing facility in Norton, Massachusetts, with the goal of creating the industry's first fully dedicated siRNA enzymatic-ligation manufacturing facility by late 2027. This investment aims to expand capacity and reduce production costs.