Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q4 2025 Earnings Presentation
26 slides from Agios Pharmaceuticals's earnings presentation, Feb 12, 2026
Slide 1: Q4 and FY 2025 Financial Results and Business Highlights
Q4 and FY 2025 Financial Results and Business Highlights Conference call for investors and analysts 12 February 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: Q4 and FY 2025 earnings call agenda
Q4 and FY 2025 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 Gheue
Slide 4: CEO Opening Remarks
CEO Opening Remarks Brian Goff, Chief Executive Officer 4
Slide 5: 2026 strategic priorities – driving long-term value creation
2026 strategic priorities – driving long-term value creation Execute high-impact 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 En
Slide 6: 2026 catalysts unlock hematology leadership and expand pipeline potential
2026 catalysts unlock hematology leadership and expand pipeline potential Estimated global market size in 2030* H1 2026 H2 2026 Thalassemia $1B+ AQVESME (mitapivat) U.S. launch underway in thalassemia Mitapivat Tebapivat Sickle Cell Disease $3B+ Pre-sNDA meeting Q1 Phase 2 topline data Tebapivat Low
Slide 7: Q4 2025 – continued portfolio and pipeline delivery
Q4 2025 – continued portfolio and pipeline delivery1 Commercial execution Pipeline advancement Corporate development Phase 3 RISE UP sickle cell disease topline data Pre-sNDA engagement Q1 Anticipate 2026 OpEx to be flat compared to 2025 Q4 2025 Net Revenues AQVESME FDA approval ― $20M, +86% vs prio
Slide 8: Financial Results
Financial Results Cecilia Jones, Chief Financial Officer 8
Slide 9: Q4 and FY 2025 Financial Results
Q4 and FY 2025 Financial Results Statement of Operations ($M) Q4 2025 Q4 2024 FY 2025 FY 2024 PYRUKYND Net Revenue $20.0 $10.7 $54.0 $36.5 US Net Revenue $16.0 - $49.2 $36.4 Ex-US Net Revenue $4.0 - $4.8 $0.1 Cost of Sales $1.9 $1.3 $6.3 $4.2 Research & Development Expense $88.1 $82.8 $339.5 $301.3
Slide 10: Financial discipline to deliver long-term sustainability
Financial discipline to deliver long-term sustainability Anticipate Operating Expenses in 2026 to be flat compared to 2025 with potential for greater efficiencies beyond 20261 Maximize launch Gated investment for Operating model of AQVESME in Sickle Cell Disease refinement thalassemia Clear path to
Slide 11: Commercial Highlights
Commercial Highlights Tsveta Milanova, Chief Commercial Officer 11
Slide 12: Q4 2025 – robust performance driven by strong commercial execution
Q4 2025 – robust performance driven by strong commercial execution PYRUKYND Net Revenues – Quarterly ($M) Key Q4 2025 dynamics • $16.0M U.S. Net Revenues $20.0 ― Increased patient demand due to $4.0 continued promotional focus in PK $12.5 $12.9 deficiency $10.7 $8.7 ― Additional ordering week in Q4
Slide 13: AQVESME – encouraging initial Thalassemia demand
AQVESME – encouraging initial Thalassemia demand 44 prescriptions1 Strong early adoption with a Initial demand driven by through 30 January 2026 wide range of prescribers, transfusion-dependent since FDA approval driven primarily by patients and highly engaged (23 Dec 2025) community physicians NTD
Slide 14: AQVESME – strong early market reception
AQVESME – strong early market reception Physicians Patients Compelling AQVESME profile; confidence in Strong engagement with MyAgios patient support ability to address critical care gap Potential to reduce transfusions and improve REMS viewed as manageable and not a barrier fatigue outweighs additio
Slide 15: AQVESME – prescription-to-treatment initiation
AQVESME – prescription-to-treatment initiation Upon completion of REMS requirements and prior authorization, pharmacy ships AQVESME to patient REMS Patient REMS Pharmacy REMS Physician enrollment certification enrollment and o Baseline liver test Agios uses single certification o Enrollment form Mon
Slide 16: Thalassemia unlocks growth inflection opportunity
Thalassemia unlocks growth inflection opportunity United States Saudi Arabia • FDA approval 23 December 2025 • SFDA approval announced 04 August 2025 • Prescription fulfillment from late January, • Currently PYRUKYND access granted on a following final REMS implementation per patient basis • $425k W
Slide 17: R&D Highlights
R&D Highlights Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of R&D 17
Slide 18: Continued pipeline momentum
Continued pipeline momentum REGULATORY PRECLINICAL EARLY-STAGE LATE-STAGE APPROVAL SUBMISSION PYRUKYND Approved U.S., EU, UK Pyruvate Kinase Mitapivat Deficiency Phase 3 completed1 ACTIVATE – Kids/KidsT First-in-class AQVESME (U.S.) PK activator PYRUKYND (Saudi Arabia) NEW Approved U.S. Dec. 2025; a
Slide 19: Tebapivat – more potent PKa in Phase development
Tebapivat – more potent PKa in Phase 2 development Phase 2 Lower Risk-MDS Phase 2 Sickle Cell Disease Potential to gain key insights into tebapivat role in LR-MDS Potential for enhanced Hemoglobin response • Phase 2a showed clinical PoC but lower exposure in • Phase 1 – mean increase of 1.2g/dL (2mg
Slide 20: CEO Closing Remarks
CEO Closing Remarks Brian Goff, Chief Executive Officer 20
Slide 21: 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 (TMPRSS6) 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 topli
Slide 22: Potential to unlock significant long-term shareholder value
Potential to unlock significant long-term shareholder value >$10B total estimated global market size for current pipeline indications by 2030* ~$54M Net Revenues in FY 2025 with PYRUKYND alone Driving leadership in Capital-efficient path to Thalassemia launch underway rare hematology profitability *
Slide 23: Q&A session
Q&A session FR agios | 23
Slide 24: Appendix
Appendix 24
Slide 25: Appendix – tebapivat Phase 2 Lower Risk-MDS trial
Appendix – tebapivat Phase 2 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 independence5 • Hb <10.0 g/dL during the core period 10mg QD • Up to 2 prior thera
Slide 26: 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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