Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q1 2026 Earnings Presentation
25 slides from Agios Pharmaceuticals's earnings presentation, Apr 29, 2026
Slide 1: Q1 2026 Financial Results and Business Highlights
Q1 2026 Financial Results and Business Highlights Conference call for investors and analysts 29 April 2026
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation and various remarks we make during this presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND
Slide 3: Q1 2026 earnings call agenda
Q1 2026 earnings call agenda 1 Introduction Morgan Sanford, VP Investor Relations 2 CEO Opening Remarks Brian Goff, Chief Executive Officer 3 Financial Results Cecilia Jones, Chief Financial Officer 4 Commercial Highlights Tsveta Milanova, Chief Commercial Officer 5 R&D Highlights Sarah Gheuens, MD,
Slide 4: CEO Opening Remarks
CEO Opening Remarks Brian Goff, Chief Executive Officer 4
Slide 5: Continued focus on delivering on 2026 strategic priorities
Continued focus on delivering on 2026 strategic priorities Execute high-impact U.S. launch for AQVESME (mitapivat) in thalassemia Potential to expand PK activation franchise into sickle cell disease and LR-MDS Unlock future value in hematology and other rare disease by advancing early-stage pipeline
Slide 6: Q1 2026 – early progress toward growth inflection
Q1 2026 – early progress toward growth inflection Strong commercial execution Progress against 2026 strategic priorities • Q1 2026 Net Revenues $20.7M, $8.7M in Q1 2025 Strong initial AQVESME thalassemia launch progress – 242 prescriptions1 • 2026 OpEx expected to be approximately flat compared to p
Slide 7: Agios – positioned for near-term growth inflection and sustained longer-term value creation
Agios – positioned for near-term growth inflection and sustained longer-term value creation Maximizing PKa franchise Differentiated next-gen PKa Pipeline diversification to • AQVESME launch execution • Tebapivat – upcoming catalysts drive sustained growth • Mitapivat sNDA filing in sickle ― LR-MDS P
Slide 8: Financial Results
Financial Results Cecilia Jones, Chief Financial Officer 8
Slide 9: Q1 2026 Financial Results
Q1 2026 Financial Results Statement of Operations ($M) Q1 2026 Q1 2025 Mitapivat Net Revenue $20.7 $8.7 US Net Revenue $18.8 $8.7 Ex-US Net Revenue $1.9 - Cost of Sales $1.3 $1.1 Research & Development Expense $81.1 $72.7 Selling, General & Administrative Expense $48.3 $41.5 Net (Loss) Income ($99.1
Slide 10: Disciplined capital allocation to drive sustained long-term value creation
Disciplined capital allocation to drive sustained long-term value creation Operating expense Strategic pipeline Focused investment to management aligned diversification through maximize AQVESME U.S. with driving sustained disciplined internal and launch in thalassemia long-term growth external innov
Slide 11: Commercial Highlights
Commercial Highlights Tsveta Milanova, Chief Commercial Officer 11
Slide 12: Q1 2026 – robust performance driven by strong commercial execution
Q1 2026 – robust performance driven by strong commercial execution Mitapivat Net Revenues – Quarterly ($M) Key Q1 2026 dynamics $20.7 $20.0 $1.9 $4.0 $18.8 • $18.8M U.S. Net Revenues $12.9 $16.0 ― Strong initial AQVESME U.S. launch $12.5 execution in thalassemia $8.7 • $1.9M ex-U.S. Net Revenues ― D
Slide 13: AQVESME – early U.S. launch reflects high-quality demand
AQVESME – early U.S. launch reflects high-quality demand • Strong early adoption among highly-engaged patients, exiting Q1 with growing NTDT patients • Broadening community-based prescriber uptake • Shorter-than-expected time to treatment, reflecting patient engagement and readiness 242 prescription
Slide 14: AQVESME – early U.S. launch unlocking path to sustained adoption
AQVESME – early U.S. launch unlocking path to sustained adoption AQVESME profile addresses unmet need in Priorities in coming quarters to drive broad thalassemia patient population sustained AQVESME adoption Expand prescriber breadth across • Compelling clinical profile and broad indication communit
Slide 15: R&D Highlights
R&D Highlights Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of R&D 15
Slide 16: Continued pipeline momentum
Continued pipeline momentum REGULATORY PRECLINICAL EARLY-STAGE LATE-STAGE APPROVAL SUBMISSION Approved U.S., EU, UK PYRUKYND® PK Deficiency Mitapivat Phase 3 completed1 ACTIVATE-Kids/KidsT First-in-class AQVESME (U.S.) Approved U.S., KSA, UAE; filing under review in EU2 PK activator PYRUKYND® (KSA,
Slide 17: Mitapivat advancing toward U.S. accelerated approval in sickle cell disease
Mitapivat advancing toward U.S. accelerated approval in sickle cell disease U.S. accelerated approval pathway regulatory process1 • Pre-sNDA meeting completed • Confirmatory trial designed to demonstrate clinical benefit, Pre-sNDA sNDA sNDA regulatory balancing operationally feasible meeting submiss
Slide 18: Tebapivat – next-generation PK activator – distinct design supporting potential for differentiated clinical profile
Tebapivat – next-generation PK activator – distinct design supporting potential for differentiated clinical profile Next-generation PK activator Early data supports potential differentiation vs first-generation PKa Sickle Cell Disease Lower Risk-MDS Structurally differentiated, next-generation PK ac
Slide 19: CEO Closing Remarks
CEO Closing Remarks Brian Goff, Chief Executive Officer 19
Slide 20: Strong catalyst flow across pipeline in 2026
Strong catalyst flow across pipeline in 2026 Sickle Cell Disease Lower-Risk MDS Polycythemia Vera H1 2026 Mitapivat tebapivat AG-236 (TMPRSS6i) Pre-sNDA meeting Q1 Phase 2b topline data Phase 1 HV topline data Sickle Cell Disease Phenylketonuria H2 2026 tebapivat AG-181 (PAH stabilizer) Phase 2 topl
Slide 21: Agios – pipeline advancement driving leadership in rare hematology and other rare diseases
Agios – pipeline advancement driving leadership in rare hematology and other rare diseases Foundation in rare hematology Other rare Sickle Cell Polycythemia PK deficiency Thalassemia LR-MDS PKU Disease Vera AQVESME/ Mitapivat/ PYRUKYND Tebapivat AG-236 AG-181 PYRUKYND1 Tebapivat TMPRSS6i Mechanism o
Slide 22: Q&A session
Q&A session FR agios | 22
Slide 23: Appendix
Appendix 23
Slide 24: Appendix – tebapivat Phase 2b Lower Risk-MDS trial
Appendix – tebapivat Phase 2b Lower Risk-MDS trial Tebapivat ı Phase 2b trial Key Inclusion Criteria Primary Endpoint for LR-MDS1 • Lower-risk MDS3 • Proportion of participants with • Transfusion dependent4 transfusion independence for at • Hb <10.0 g/dL least 8 consecutive weeks5 10mg QD • Up to 2
Slide 25: Appendix – tebapivat Phase 2 Sickle Cell Disease trial
Appendix – tebapivat Phase 2 Sickle Cell Disease trial Tebapivat ı Phase 2 trial for Key Inclusion Criteria Primary Endpoint Sickle Cell Disease1 • Hb ≥5.5 - ≤10.5 g/dL • Hb response3 at baseline, and • HU use permitted, if dose has from Weeks 10 - 12 been stable for at least 90 days before randomiz
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