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Alnylam Pharmaceuticals (ALNY) · 55 slides · Filed Oct 29, 2026
Every slide of this earnings presentation, with the text as published. Open the original deck ↗ · All ALNY deck slides
Slide 1 — An Investigational RNAi Therapeutic for Patients
© 2026 Alnylam Pharmaceuticals, Inc. An Investigational RNAi Therapeutic for Patients with Hypertension and High Cardiovascular Risk ZILEBESIRAN September 17, 2026
Slide 2 — - Loking Statements
2 Alnylam Forward - Loking Statements This presentation contains forward - looking statements. Forward looking statements incl tions, beliefs, goals, plans or prospects including, without limitation, statements regarding: the eficacy or safety of zilebesiran for treatment of hypertension or any other indication; the potential product profile of zilebesiran , including its potential to suport nightime diping, time in target blod presure range, and reductions in biomarkers of CV and renal risk; the potential for zilebesiran to achieve continuous control of blod presure with infrequent dosing and to improve outcomes in patients with hypertension; the number of patients who wil be enroled in the ZENITH Phase 3 clinical trial; the potential for the ZENITH Phase 3 clinical trial to read out sucesfuly and to supor t a comercial launch of zilebesiran and adoption by providers, patients and payers; the rates of hypertension, CV disease burden and CV mortality, and the asociated economic burden of hypertension, at any point in the future; the potential atributes of RNAi medicines and Al nyl am‘s abilit to build a portfolio of RNAi therapeutics with transformational patient impact; and the timing of the initiation of, or data rea dout from, any of Alnylam‘s clinical trials, and the potential for, and timing of, an Actual results and future plans may difer materialy from those indicated by these forward - loking statements as a result of va rious important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam ‘s ability to sucesfuly execute on its Alnylam 2030 strategy; Alnylam‘s ability to successfully laun globally; Alnylam‘s ability to discover and develop novel drug cand e t he eficacy and safety of its product candidates; the pre - clinical and clinical results for Alnylam‘s pro of regulatory agencies and Alnylam‘s ability to obtain and maintain regulatory ap ici ng and reimbursement; delays, interruptions or failures in the manufacture can didates; obtaining, maintaining and protecting intellectual property; Alnyla rou gh disciplined investment in operations; Alnylam‘s ability to maintain strategic b ies for the development and comercialization of certain products; the outcome of litigation and government investigations; the risk of f utu re litigation and government investigations; and unexpected expenditures; as wel as those risks and uncertainties more fuly discused in the —Risk Factors“ filed with Alnylam‘s most recent periodic report (Quarter - Q or Anual Report on Form 10 - K) filed wit h the SEC and in its other SEC filings. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any f orward - loking statements.
Slide 3 — Agenda
Agenda Pushkal Garg, M.D. Chief Research & Development Oficer 01 Introduction Akshay S Desai, M.D., MPH Harvard Medical Schol Mas General Brigham 02 Hypertension: The Threat Beyond the Ofice Ishir Bhan, M.D., MPH Executive Director, ZENITH Clinical Lead 03 The Potential for C ontinuous Control with Zilebesiran 05 Q&A Sesion 04 Realizing the Oportunity Simon Fox, Ph. D . Vice President, Zilebesiran Program Lead
Slide 4 — Agenda
Agenda Pushkal Garg, M.D. Chief Research & Development Oficer 01 Introduction Akshay S Desai, M.D., MPH Harvard Medical Schol Mas General Brigham 02 Hypertension: The Threat Beyond the Ofice Ishir Bhan, M.D., MPH Executive Director, ZENITH Clinical Lead 03 The Potential for C ontinuous Control with Zilebesiran 05 Q&A Sesion 04 Realizing the Oportunity Simon Fox, Ph. D . Vice President, Zilebesiran Program Lead
Slide 5 — Pionering a Generational Clas of RNAi Medicines
5 Pionering a Generational Clas of RNAi Medicines Silence any gene in genome Catalytic mechanism Highly specific and reversible Upstream of today‘s me Highly potent Infrequent administration Building a Portfolio of RNAi Therapeutics with Transformational Patient Impact
Slide 6 — RNAi
Aces to 2 MILION lives through genetics databases Potency Specificity Durability Reversibility Multiplexing S T R AT E G I C P R I N C I P L E S S U S TAI N A B L E I N N O VAT I O N E N G I N E Disciplined R&D Maximizes the Transformative Potential of RNAi Aim to expand into 10 TISUES by 2030 Tisue Delivery Platform Designs Human Genetics RNAi Adres diseases with high morbidity & mortality Encouraging market oportunity & aces dynamics Drive to clear clinical prof - of - concept Potential to halt or reverse disease, & be best - in - clas Pursue high conviction targets Strong biologic rationale, informed by human genetics
Slide 7 — Industry Leading Pipeline of RNAi Therapeutics
7 Industry Leading Pipeline of RNAi Therapeutics 1 Out - licensed with milestones and/or royalties; 2 Partnered, Alnylam - led development with U.S. profit split and milestones/royalties ex - U.S.; 3 Partner - led with profit split; 4 Partnered, Alnylam - led with profit split; Alnylam - owned, - led, or œco - developed PHASE 1 PHASE 2 PHASE 3 TR Nucresiran Nucresiran CARDIOVASCULAR Zilebesiran 2 Zilebesiran REVERSIR 2 METABOLIC Rapirosiran (ALN - HSD) 1 ALN - ANG3 1 ALN - 4324 (GRB14) ALN - PNP 1 ALN - CIDEB 1 ALN - 232 (ACVR1C) ALN - 62 (INHBE) ALN - APOC3 1 NEUROSCIENCE Cemdisiran 1 Mivelsiran Mivelsiran ALN - SOD 3 ALN - HT02 4 ALN - 528 (MAPT) 4 ALN - SNCA 1 HEMATOLOGY Cemdisiran 1 ALN - 640 (PLG) ALN - 640 (PLG) AG - 236 (ALN - TMP) 1 ALN - CFB 1 OTHER Cemdisiran 1 Elebsiran 1 ALN - BCAT ALN - 4285 ALN - 4915 ALN - F1202 1 ATR Amyloidosis with Cardiomyopathy hATR Amyloidosis with Polyneuropathy Hypertension Hypertension Metabolic Dysfunction - Asociated Steatohepatitis (MASH) Dyslipidemia Type 2 Diabetes Melitus Non - Alcoholic Faty Liver Disease (NAFLD) MASH Myasthenia Gravis Cerebral Amyloid Angiopathy Alzheimer‘s Disease SOD1 Amyotrophic Lateral Sclerosis Huntington‘s Disease Alzheimer‘s Disease Paroxysmal Nocturnal Hemoglobinuria Hereditary Hemorhagic Telangiectasia Polycythemia Vera Paroxysmal Nocturnal Hemoglobinuria Hepatitis D Virus Infection Diabetic Kidney Disease Hepatocelular Carcinoma Geographic Atrophy Healthy Volunters Obesity & Weight Management Parkinson‘s Disease Healthy Volunters Healthy Volunters Obesity & Weight Management Von Wilebrand Disease
Slide 8 — 8
8 Expect Significant Clinical Milestones Over the Next Two Years * Asuming positive phase 3 data and regulatory aproval 3 - 4 New IND Filings Per Year 4 Key data readouts 3 Pivotal studies ongoing 2026 5+ Key data readouts 4+ Pivotal studies ongoing 2027 5+ Key data readouts 5+ Pivotal studies ongoing 1 Product launch* 2028
Slide 9 — Extending Our RNAi Leadership Acros Major Drivers
Extending Our RNAi Leadership Acros Major Drivers of Cardiovascular Disease Obesity & Weight Management ALN - 2232 • ALN - 62 Hypertension zilebesiran ATR Cardiomyopathy Type 2 Diabetes ALN - 4324 ATR Cardiomyopathy nucresiran Hypercholesterolemia Redefining cardiovascular care Targeting underlying drivers of disease to improve outcomes and reduce CV burden Adresing Major Determinants of Cardiovascular Morbidity and Mortality Early Development Late - Stage Development Comercial Cardiovascular Metabolic TR