Regeneron Pharmaceuticals, Inc. · NASDAQ:REGN
Regeneron Pharmaceuticals — Investor Presentation (April 2026)
40 slides from Regeneron Pharmaceuticals's investor presentation, Apr 22, 2026
Slide 1: Roundtable: C5 Complement Program
ROUNDTABLE C5 Complement Program ± siRNA Antibody (Cemdisiran) (Pozelimab) APRIL 22, 2026
Slide 2: Note regarding forward-looking statements
Note regarding forward-looking statements This presentation includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. ("Regeneron" or the "Company"), and actual events or results may differ materia
Slide 3: Key pipeline programs positioned to deliver over the next few years
Key pipeline programs positioned to deliver over the next few years Late-stage opportunities spanning multiple therapeutic areas OLATOREPATIDE FIANLIMAB + CEMDISIRAN ± REGN7508 & (OLA) ± VARIOUS LIBTAYO POZELIMAB REGN9933 AGENTS LAG-3 + PD-1 BCMAxCD3 C5 siRNA ± C5 antibody Two Factor XI GIP/GLP-1, c
Slide 4: Regeneron Roundtable series providing deep dives on key programs
Regeneron Roundtable series providing deep dives on key programs Regeneron Roundtable Regeneron Roundtable Regeneron Roundtable December 10th, 2025 April 22nd, 2026 November 10th, 2025 CEMDISIRAN ± REGN7508 & POZELIMAB REGN9933 BCMAxCD3 C5 siRNA ± C5 antibody Two Factor XI antibodies allow for PNH:
Slide 5: Today's Roundtable to focus on C5 complement program
Today’s Roundtable to focus on C5 complement program Regeneron Roundtable April 22nd, 2026 CEMDISIRAN ± POZELIMAB C5 siRNA ± C5 antibody PNH: combination approach for complete C5 blockade and potentially best-in-class efficacy gMG: siRNA monotherapy delivers potentially best-in-class efficacy and co
Slide 6: Regeneron's Differentiated C5 Clinical Program
Regeneron’s Differentiated approaches to treating various complement mediated diseases Differentiated George Yancopoulos Board co-Chair • Evaluating three approaches – a C5 antibody, a C5 siRNA, C5 Clinical President Chief Scientific Officer and a first-of-its-kind combination of an antibody with a
Slide 7: Regeneron Roundtable – C5 Complement
REGENERON ROUNDTABLE – C5 COMPLEMENT C5 Program Overview and Strategy Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Geographic Atrophy (GA) Overview Generalized Myasthenia Gravis (gMG) Overview Commercial Outlook and Vision Conclusion and Q&A 7
Slide 8: Pioneering siRNA (cemdisiran) ± antibody (pozelimab) combination
Pioneering siRNA (cemdisiran) ± antibody (pozelimab) combination siRNA (cemdisiran) lowers C5 target burden while antibody (pozelimab) blocks circulating C5, enabling near-complete C5 inhibition Currently Approved OVERVIEW Cemdisiran (siRNA) Approach to C5 Inhibition Approach to C5 Inhibition In Jun
Slide 9: Differentiated therapeutic approaches to complement-mediated diseases
Differentiated therapeutic approaches to complement-mediated diseases driven by the underlying disease biology OVERVIEW Gastroenterology Hematology Neurology Ophthalmology CHAPLE Paroxysmal Nocturnal Generalized Myasthenia Geographic Disease Hemoglobinuria (PNH) Gravis (gMG) Atrophy (GA) U.S. Preval
Slide 10: Ability to modulate C5 inhibition enables tailored levels of target suppression
Annual Ability to modulate C5 inhibition enables tailored levels AAN Meeting of target suppression for different diseases Total complement hemolysis activity (CH50 assay) Some diseases (e.g., PNH) require complete C5 suppression to be OVERVIEW gMG NIMBLE Phase 3 study efficacious whereas partial blo
Slide 11: Regeneron Roundtable – C5 Complement
REGENERON ROUNDTABLE – C5 COMPLEMENT C5 Program Overview and Strategy Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Geographic Atrophy (GA) Overview Generalized Myasthenia Gravis (gMG) Overview Commercial Outlook and Vision Conclusion and Q&A 11
Slide 12: Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview
Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Landscape is an ultra-rare, acquired, life-threatening disease caused by Current approved therapies by MOA: uncontrolled complement-mediated intravascular hemolysis • C5 inhibitors – Ultomiris (rav
Slide 13: PNH Phase 3 ACCESS-1 registrational study
PNH Phase 3 ACCESS-1 registrational study Two cohort study evaluating cemdisiran + pozelimab in patients with PNH who are naïve to, or have not recently received, complement inhibitor therapy Screening 26-week Open-label Treatment Period (OLTP) Cohort A Up to 6 weeks Primary Endpoint: Percent change
Slide 14: PNH Phase 3 ACCESS-1 registrational study
PNH Phase 3 ACCESS-1 registrational study Data from Cohort A validated the novel combination approach while demonstrating potentially best-in-class disease control Screening 26-week Open-label Treatment Period (OLTP) Cohort A Up to 6 weeks Primary Endpoint: Percent change in LDH Pozelimab 400 mg SC
Slide 15: PNH Phase 3 cohort A: complete, rapid, uninterrupted and durable inhibition
ASH PNH Phase 3 cohort A: cemdisiran + pozelimab enabled complete, 2024 rapid, uninterrupted and durable inhibition of terminal complement Data presented at ASH 2024; safety profile of the combination was generally consistent with approved C5 inhibitors Primary Endpoint: % change in lactate Cemdisir
Slide 16: PNH Phase 3 ACCESS-1 registrational study
PNH Phase 3 ACCESS-1 registrational study Enrollment in cohort B completed, registration-enabling data expected in late Q4 2026 Screening 26-week Open-label Treatment Period (OLTP) Up to 6 weeks Pozelimab 400 mg SC + Cemdisiran 200 mg SC, Q4W Cohort A: (n=25) PNH 108-week Supportive R extension stud
Slide 17: Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview
Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment Landscape is an ultra-rare, acquired, life-threatening disease caused by Current approved therapies by MOA: uncontrolled complement-mediated intravascular hemolysis • C5 inhibitors – Ultomiris (rav
Slide 18: Regeneron Roundtable – C5 Complement
REGENERON ROUNDTABLE – C5 COMPLEMENT C5 Program Overview and Strategy Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Geographic Atrophy (GA) Overview Generalized Myasthenia Gravis (gMG) Overview Commercial Outlook and Vision Conclusion and Q&A 18
Slide 19: Geographic Atrophy (GA) Overview
Geographic Atrophy (GA) Overview Geographic Atrophy (GA) is an advanced, irreversible stage Treatment Landscape of dry age-related macular degeneration (dAMD) that leads to Current approved therapies: progressive vision loss and central blind spots • C3 inhibitor – Syfovre (pegcetacoplan), IVT Q4W/Q
Slide 20: Phase 3 GA exploratory cohort fully enrolled
Phase 3 GA exploratory cohort fully enrolled Initial data from exploratory cohort A expected in Q4 2026, enrollment in cohort B underway SIENNA Study Design Phase 3, multi-center, double-masked, placebo-controlled, global study in participants (N=975) with GA secondary to AMD Cohort A Cohort A: Supp
Slide 21: Differentiated systemic approach in treating a leading cause of blinding eye disease
Differentiated systemic approach in treating a leading cause of blinding eye disease Significant opportunity in a large, high-unmet-need indication where disease-modifying treatments remain limited despite approved therapies Current Geographic Atrophy Landscape Regeneron Opportunity (cemdisiran ± po
Slide 22: Regeneron Roundtable – C5 Complement
REGENERON ROUNDTABLE – C5 COMPLEMENT C5 Program Overview and Strategy Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Geographic Atrophy (GA) Overview Generalized Myasthenia Gravis (gMG) Overview Commercial Outlook and Vision Conclusion and Q&A 22
Slide 23: Generalized Myasthenia Gravis (gMG) overview
Generalized Myasthenia Gravis (gMG) overview Generalized Myasthenia Gravis (gMG) Na+ Activation of complement cascade is a rare and chronic autoimmune disease in ACh Autoantibody which abnormal antibodies activate the complement system (including C5), disrupting ACh Axon neuromuscular signaling and
Slide 24: Phase 3 NIMBLE trial in gMG
Phase 3 NIMBLE trial in gMG Trial evaluated adults with symptomatic gMG who have antibodies to the acetylcholine receptor (anti-AChR) and may be receiving standard of care immunosuppressants based on the investigator’s discretion 24-week DBTP Placebo-Controlled Double-Blind Extension Treatment Perio
Slide 25: Cemdisiran monotherapy met primary and all key secondary endpoints in NIMBLE
Annual AAN Cemdisiran monotherapy dosed every 3 months met the Meeting primary & all key secondary endpoints in Phase 3 NIMBLE trial Robust efficacy achieved without waning of efficacy between dosing intervals, underscoring cemdisiran’s potential best-in-class profile MG-ADL (Myasthenia Gravis Activ
Slide 26: Cemdisiran was generally well tolerated
Cemdisiran was generally well tolerated; no serious infections AAN Annual Meeting in the cemdisiran arm and no meningococcal infections in any group Potential best-in-class efficacy achieved with partial complement inhibition, enabling potentially differentiated safety profile Placebo Cemdisiran Com
Slide 27: Cemdisiran's differentiated clinical profile offers potential advantages in gMG
Cemdisiran’s differentiated clinical profile offers potential advantages to currently approved advanced therapies for gMG Cemdisiran’s potential clinical differentiation MG-ADL Total Score • Potential first-in-class siRNA treatment for gMG, offering a novel 0 Cemdisiran mechanism of action for this
Slide 28: Cemdisiran's differentiated clinical profile (vs. Ultomiris)
Cemdisiran’s differentiated clinical profile offers potential advantages to currently approved advanced therapies (vs. Ultomiris) MG-ADL Total Score (Cemdisiran) Cemdisiran’s potential clinical differentiation 0 Cemdisiran Placebo • Potential first-in-class siRNA treatment for gMG, offering a novel
Slide 29: Cemdisiran's differentiated clinical profile (vs. Vyvgart)
Cemdisiran’s differentiated clinical profile offers advantages to currently approved advanced therapies (vs. Vyvgart) MG-ADL Total Score (Cemdisiran) Cemdisiran’s potential clinical differentiation 0 Cemdisiran Placebo • Potential first-in-class siRNA treatment for gMG, offering a novel LS mean chan
Slide 30: Cemdisiran's differentiated clinical profile (vs. Vyvgart)
Cemdisiran’s differentiated clinical profile offers advantages to currently approved advanced therapies (vs. Vyvgart) MG-ADL Total Score (Cemdisiran) Cemdisiran’s potential clinical differentiation 0 Cemdisiran Placebo • Potential first-in-class siRNA treatment for gMG, offering a novel LS mean chan
Slide 31: Cemdisiran's differentiated clinical profile (vs. Uplizna)
Cemdisiran’s differentiated clinical profile offers potential advantages to currently approved advanced therapies (vs. Uplizna) MG-ADL Total Score (Cemdisiran) Cemdisiran’s potential clinical differentiation 0 Cemdisiran Placebo • Potential first-in-class siRNA treatment for gMG, offering a novel LS
Slide 32: Regeneron Roundtable – C5 Complement
REGENERON ROUNDTABLE – C5 COMPLEMENT C5 Program Overview and Strategy Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Geographic Atrophy (GA) Overview Generalized Myasthenia Gravis (gMG) Overview Commercial Outlook and Vision Conclusion and Q&A 32
Slide 33: Significant commercial opportunity across multiple complement-mediated diseases
Significant commercial opportunity across multiple complement-mediated diseases Cemdisiran Monotherapy We believe late-stage, differentiated C5 Myasthenia Gravis program with near-term launches across U.S. Launch expected Q4 2026 multiple indications creates meaningful 2025 U.S. Prevalence (patients
Slide 34: U.S. gMG market is growing rapidly, driven by newer agents
U.S. gMG market is growing rapidly, driven by newer agents and increasing rates of advanced treatment use U.S. gMG patients on treatment: U.S. gMG net sales (advanced treatments only)*: % of patients on advanced therapy (Tx) will increase gMG commercial opportunity expected to more than double by 20
Slide 35: Cemdisiran commercial vision in gMG
Cemdisiran commercial vision in gMG Position cemdisiran as a differentiated treatment with compelling efficacy, generally manageable safety, and quarterly subcutaneous dosing Educate Establish Optimize • Position cemdisiran as a first-in- • Target current C5 inhibitor • Transition from HCP-administe
Slide 36: Regeneron Roundtable – C5 Complement
REGENERON ROUNDTABLE – C5 COMPLEMENT C5 Program Overview and Strategy Paroxysmal Nocturnal Hemoglobinuria (PNH) Overview Geographic Atrophy (GA) Overview Generalized Myasthenia Gravis (gMG) Overview Commercial Outlook and Vision Conclusion and Q&A 36
Slide 37: Key Roundtable Takeaways: C5 Program
Key Roundtable Differentiated MoA (siRNA +/- mAb) Takeaways: Late-stage differentiated clinical programs rapidly advancing in 3 distinct indications C5 Program Generalized Myasthenia Gravis (gMG) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Geographic Atrophy (GA) Clinical Validation Emerging data
Slide 38: Question & Answer Session
Question & Answer Session Regeneron Roundtable – C5 Complement Program George Yancopoulos Andres Sirulnik Umesh Chaudhari Soma Gupta Board co-Chair Senior Vice President Vice President Vice President President Clinical Development Global Program Head (C5) Commercial New Products Chief Scientific Off
Slide 39: Alnylam royalty and milestone obligations for C5 program
Alnylam royalty and milestone obligations for C5 program Cemdisiran Monotherapy Cemdisiran + Pozelimab* Combination Royalties Tiered, double-digit royalties Royalties Flat, low double-digit royalty Tiered by net sales (up to 15%) Calendar-year net sales in a calendar year Development Milestones Mode
Slide 40: gMG: competitive landscape
gMG: competitive landscape Maintenance Route of dosing Cyclic or Select Placebo controlled Company Target administration frequency chronic dosing safety risks* response in Ph3 FDA approval Cemdisiran SC Q3M -2.3 MG-ADL (W24) 2026 (estimated) Eculizumab (Soliris) IV Q2W -1.9 MG-ADL (W26) 2017 Infecti
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