Inferra AI / Company / ALNY / Slides
Alnylam Pharmaceuticals — View the Presentation › (company IR deck)
Alnylam Pharmaceuticals (ALNY) · 69 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 — Brezosiran (ALN
© 2026 Alnylam Pharmaceuticals, Inc. Brezosiran (ALN - HT02) An Investigational RNAi Therapeutic Targeting Exon 1 of the !" #!" $%& &$!'$()'*$+,'#,&--"' October 26, 2026
Slide 2 — Agenda
Agenda Agenda Kevin Sloan, Ph.D. Vice President, Development Programs, ALN - HT02 Program Lead 02 About Brezosiran (ALN - HT02) Gil Bates, Ph.D. Profesor of Molecular Neuroscience, Co - Director of the !" #!$ %&'("&)*&)'+) !,)-'. 03 Targeting HT1a in HT Lowering Strategies 05 Q&A Sesion 04 Clinical Data and Progres Toby Ferguson, M.D., Ph.D. Senior Vice President, Neuroscience Therapeutic Area Head Pushkal Garg, M.D. Chief Research & Development Oficer 01 Introduction
Slide 3 — Disclaimer
Disclaimer Alnylam has engaged Dr. Bates as an independent academic speaking in her personal scientific capacity. She is not speaking on behalf of University Colege London or UCL Consultants today; nothing said by Dr. Bates constitutes investment advice nor should it be relied on in making investment decisions.
Slide 4 — - Loking Statements
Alnylam Forward - Loking Statements This presentation contains forward - loking statements . Forward - loking statements include statements regarding 2) 3)14/- expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding : the potential safety or eficacy of brezosiran as a treatment for !" #!' /- disease, including through the potential lowering of al HT isoforms ; the potential size of the patient population with !" #!' /- disease and the potential market oportunity for an efective treatment for !" #!' /- disease ; the potential for Alnylam to initiate a pivotal, placebo - controled eficacy study of brezosiran in adults with !" #!' /- disease in 2027 ; the potential for Alnylam to build a portfolio of RNAi therapeutics with transformational patient impact ; the transformative potential of RNAi ; and the timing of key data readouts from 2) 3)14/- clinical trials, the number of pivotal studies Alnylam wil have ongoing at any time, the number of new IND/CTA filings Alnylam wil make in any calendar year, and the potential for Alnylam to launch aditional products and the timing of any such product launches . Actual results and future plans may difer materialy from those indicated by these forward - loking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to : 2) 3)14/- ability to sucesfuly execute on its Alnylam 2030 strategy ; 2) 3)14/- ability to sucesfuly launch, market and sel 2) 3)14/- aproved products globaly ; 2) 3)14/- ability to discover and develop novel drug candidates and delivery aproaches and sucesfuly demonstrate the eficacy and safety of its product candidates ; the pre - clinical and clinical results for 2) 3)14/- product candidates ; actions or advice of regulatory agencies and 2) 3)14/- ability to obtain and maintain regulatory aproval for its product candidates, as wel as favorable pricing and reimbursement ; delays, interuptions or failures in the manufacture and suply of 2) 3)14/- marketed products or its product candidates ; obtaining, maintaining and protecting intelectual property ; 2) 3)14/- ability to manage its growth and operating expenses through disciplined investment in operations ; 2) 3)14 ability to maintain strategic busines colaborations ; 2) 3)14/- dependence on third parties for the development and comercialization of certain products ; the outcome of litigation and government investigations ; the risk of future litigation and government investigations ; and unexpected expenditures ; as wel as those risks and uncertainties more fuly discused in the 56"-7 819!',-: filed with 2) 3)14/- most recent periodic report (Quarterly Report on Form 10 - Q or Anual Report on Form 10 - K) filed with the SEC and in its other SEC filings . Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward - loking statements .
Slide 5 — Agenda
Agenda Agenda Kevin Sloan, Ph.D. Vice President, Development Programs, ALN - HT02 Program Lead 02 About Brezosiran (ALN - HT02) Gil Bates, Ph.D. Profesor of Molecular Neuroscience, Co - Director of the !" #!$ %&'("&)*&)'+) !,)-'. 03 Targeting HT1a in HT Lowering Strategies 05 Q&A Sesion 04 Clinical Data and Progres Toby Ferguson, M.D., Ph.D. Senior Vice President, Neuroscience Therapeutic Area Head Pushkal Garg, M.D. Chief Research & Development Oficer 01 Introduction
Slide 6 — Pionering a Generational Clas of RNAi Medicines
Pionering a Generational Clas of RNAi Medicines Silence any gene in genome Catalytic mechanism Highly specific and reversible !"#$%&'(&!')$*+ &%# Highly potent Infrequent administration Building a Portfolio of RNAi Therapeutics with Transformational Patient Impact
Slide 7 — Disciplined R&D Maximizes the Transformative Potential of 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 8 — Industry Leading Pipeline of RNAi Therapeutics
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 4co - 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 Brezosiran (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 Metabolic Dysfunction - Asociated Steatohepatitis (MASH) MASH Myasthenia Gravis Cerebral Amyloid Angiopathy /012#,%#"+ &3, #$ # SOD1 Amyotrophic Lateral Sclerosis 45.!,.6!'.+ &3, #$ # /012#,%#"+ &3, #$ # 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 7$"8,. '.+ &3, #$ # Healthy Volunters Healthy Volunters Obesity & Weight Management Von Wilebrand Disease
Slide 9 — * Asuming positive phase 3 data and regulatory aproval
* Asuming positive phase 3 data and regulatory aproval Expect Significant Clinical Milestones Over Next Two Years 3 - 4 New IND/CTA 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 10 — C16 Conjugates Unlocked the CNS for RNAi Therapeutics
1. Out - licensed to Regeneron with milestones and/or royalties, 2) Partnered with Regeneron, Regeneron - led with profit split, 3. Partnered with Regeneron, Alnylam - led with profit split Phase 1 Multiple - Dose Results of Mivelsiran in Early - Onset AD Preclinical Results of mivelsiran in NHPs P H A S E 1 P H A S E 2 Brezosiran (ALN - HT02) | !" #!' /-$0"-&1-& 3 ALN - 52 (MAPT) | 2);<&"4&,/-$0"-&1-& 3 ALN - SNCA | +1,7" -' /-$0"-&1-& 1 Mivelsiran | Cerebral Amyloid Angiopathy (CA) Mivelsiran | Down Syndrome - 2--'9"1!&=$2);<&"4&,/-$0"-&1-&$>0 - AD) ALN - SOD | SOD1 Amyotrophic Lateral Sclerosis (ALS) 2 C16 Conjugates Unlocked the CNS for RNAi Therapeutics Advancing a growing pipeline of investigational CNS - targeted RNAi therapeutics in colaboration with Regeneron