Slide 1: Q2 2026 Financial Results and Business Highlights
Q2 2026 Financial Results and Business Highlights Conference call for investors and analysts 30 July 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: Q2 2026 earnings call agenda
Q2 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: Agios – advancing a diversified rare disease portfolio with multiple growth drivers
Agios – advancing a diversified rare disease portfolio with multiple growth drivers Immune Thalassemia Sickle Cell Disease Polycythemia Vera Phenylketonuria Thrombocytopenia First medicine approved Strong anti-hemolytic Next-gen SYK inhibitor, TMPRSS6i designed for PAH enzyme stabilizer, for broad t
Slide 6: Q2 2026 – commercial growth, pipeline expansion and financial discipline
Q2 2026 – commercial growth, pipeline expansion and financial discipline Commercial launch Near-term growth Strategic pipeline Strong capital momentum catalyst diversification position • $44.7M worldwide net • Mitapivat sNDA accepted • In-licensed cevidoplenib, • ~$965B in cash, cash revenue, includ
Slide 7: Financial Results
Financial Results Cecilia Jones, Chief Financial Officer 7
Slide 8: Q2 2026 Financial Results
Q2 2026 Financial Results Statement of Operations ($M) Q2 2026 Q2 2025 Mitapivat Net Revenue $44.7 $12.5 U.S. Net Revenue $40.9 $12.2 Ex-U.S. Net Revenue $3.8 $0.3 Cost of Sales $3.0 $1.7 Research & Development Expense $100.8 $91.9 Selling, General & Administrative Expense $51.5 $45.9 Net (Loss) Inc
Slide 9: Commercial Highlights
Commercial Highlights Tsveta Milanova, Chief Commercial Officer 9
Slide 10: Q2 2026 performance reflects strong launch execution and expanding demand
Q2 2026 performance reflects strong launch execution and expanding AQVESME/PYRUKYND (mitapivat) demand Mitapivat Total Net Revenues – Quarterly ($M) $44.7 Key Q2 2026 dynamics $3.8 • $40.9M U.S. Net Revenues ― AQVESME launch execution in thalassemia, driven by increased demand $20.7 $20.0 $40.9 ― ~$
Slide 11: AQVESME – Q2 execution reinforces confidence in thalassemia launch trajectory
AQVESME – Q2 execution reinforces confidence in thalassemia launch trajectory Time to treatment initiation Patient mix reflects growing starting to approach expected proportion of NTDT patients 10-12 week average Coverage policies Broad and expanding 75% established for prescriber base 442 cumulativ
Slide 12: AQVESME – thalassemia launch entering next phase in H2
AQVESME – thalassemia launch entering next phase in H2 Early launch wave Broadening patient adoption Maturing launch H1 2026 H2 2026 2027+ Q1 benefitted from PDUFA delay, Adoption expanding into broader Durable base of patients on higher launch anticipation NTDT population therapy Highly motivated p
Slide 13: Focused launch planning for mitapivat in sickle cell disease
Focused launch planning supports potential first-in-class opportunity for mitapivat in sickle cell disease Compelling initial launch target within a Key launch preparation ahead of broader market opportunity potential U.S. approval Potential to expand over time • Target prescribers – mapped and prio
Slide 14: R&D Highlights
R&D Highlights Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of R&D 14
Slide 15: Focused pipeline advancing multiple opportunities for long-term growth
Focused pipeline advancing multiple opportunities for long-term growth First patient dosed in REIGNITE Phase 3 confirmatory trial for mitapivat in sickle cell disease 1. Defined as completion of double-blind randomized portion of the trial. PK = pyruvate kinase; EU = European Union; UK = United King
Slide 16: Cevidoplenib in-licensing adds next-generation SYK inhibitor for ITP
Cevidoplenib in-licensing adds next-generation SYK inhibitor designed for durable platelet control in ITP First-generation SYKi Cevidoplenib Cevidoplenib – next-generation, highly- selective SYK inhibitor Inconsistent target Sustained target Efficacy inhibition associated with inhibition to support
Slide 17: Strong presence at the 2026 EHA Congress
Strong presence at the 2026 EHA Congress with data across thalassemia and sickle cell disease 10 abstracts accepted — RISE UP Phase 3 selected for EHA plenary session RISE UP Phase 3 trial in sickle cell disease1 – new SATISFY Phase 2 trial – 56-week follow-up results2 PRO and transfusion burden dat
Slide 18: Advancing AG-236 into Phase 2/3 program in polycythemia vera
Advancing AG-236 into operationally seamless Phase 2/3 program in polycythemia vera on positive Phase 1 HV data Addressing broad target population Includes patients requiring frequent phlebotomy and those on stable cytoreductive therapy Efficient registrational strategy Operationally seamless Phase
Slide 19: CEO Closing Remarks
CEO Closing Remarks Brian Goff, Chief Executive Officer 19
Slide 20: Continued execution positions Agios for near- and long-term value drivers
Continued execution positions Agios for multiple near- and long-term value drivers Execute high-impact U.S. launch for AQVESME 442 cumulative prescriptions1 for AQVESME (mitapivat) in thalassemia +200 in Q2 2026 Potential to expand PK activation into Mitapivat sNDA accepted with Priority Review sick
Slide 21: 2026 catalyst progress sharpens focus on highest-value opportunities
2026 catalyst progress sharpens focus on highest-value opportunities 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 Clinical activity observed did not meet Support advancement to Pha
Slide 22: Pipeline advancement driving leadership in rare hematology and other rare diseases
Agios – pipeline advancement driving leadership in rare hematology and other rare diseases Diversified across rare hematology Beyond hematology Sickle Cell PK Deficiency Thalassemia ITP Polycythemia Vera PKU Disease PYRUKYND AQVESME Mitapivat Cevidoplenib AG-236 AG-181 Mechanism of action PK activat
Slide 23: Q&A session
Q&A session FR agios | 23
Slide 24: Appendix
Appendix 24
Slide 25: Appendix – REIGNITE mitapivat confirmatory trial in sickle cell disease
Appendix – REIGNITE mitapivat confirmatory trial in sickle cell disease Double-blind period (52 weeks) Confirmatory trial design based on RISE UP Phase 3 trial Mitapivat 100 mg BID Open-label • 98% power for primary endpoint and ≥90% power for extension key secondary endpoints N=159 (52 weeks) 2:1 •
Slide 26: Appendix – Cevidoplenib Phase 2 secondary endpoints
Appendix – Cevidoplenib Phase 2 secondary endpoints show clinically meaningful platelet responses aligned with registrational standards Cevidoplenib Placebo 2-sided (400 mg BID) N=12 p-value N=22 Primary endpoint limited assessment of benefit PLT ≥30,000/μL and AVG PLT • Does not measure durability