Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — J.P. Morgan Healthcare Conference 2026
33 slides from Agios Pharmaceuticals's conference presentation, Jan 14, 2026
Slide 1: J.P. Morgan Healthcare Conference 2026
J.P. Morgan Healthcare Conference 2026 Agios Pharmaceuticals Brian Goff, Chief Executive Officer 14 January 2026
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation and various remarks we make during this presentation contain 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 AQVESMETM
Slide 3: Agios – positioned for a growth inflection, advancing pipeline with path to profitability
Agios – positioned for a growth inflection, advancing pipeline with path to profitability Potential to unlock PK activator franchise as Clear path to profitability additional value with standard of care in multiple with existing commercial robust early and mid-stage hemolytic anemias portfolio pipel
Slide 4: Pipeline foundation in hematology, maturing early-stage pipeline offers potential for diversification
Pipeline foundation in hematology, maturing early-stage pipeline offers potential for diversification REGULATORY PRECLINICAL EARLY-STAGE LATE-STAGE APPROVAL SUBMISSION Approved U.S., EU, UK Pyruvate Kinase Deficiency Mitapivat Phase 3 completed1 ACTIVATE – Kids/KidsT First-in-class NTD and TD α- and
Slide 5: Potential to unlock significant growth with current pipeline
Potential to unlock significant growth with current pipeline >$10B total estimated global market size for current pipeline indications by 2030* ~$35M Net Revenues in 2025 as of Q3 in ultra-rare indication PK Deficiency Scalable rare disease commercial model Dedicated field teams with Capital efficie
Slide 6: 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 7: Execute high impact launch for AQVESME in thalassemia
Execute high impact launch for AQVESME in thalassemia 7
Slide 8: AQVESME (mitapivat) – now approved in U.S. with broad thalassemia label
AQVESME (mitapivat) – now approved in U.S. with broad thalassemia label A pyruvate kinase activator indicated for the treatment of anemia in adults with alpha- or beta-thalassemia Only FDA approved medicine for anemia in both non-transfusion dependent and transfusion-dependent alpha- or beta-thalass
Slide 9: Delivering a series of “firsts” in thalassemia
– delivering a series of “firsts” in thalassemia First medicine to address α- or β-thalassemia, regardless of transfusion burden First oral medicine First medicine to show quality of life improvements in NTDT patients1 First medicine to show durable reduction in transfusion burden in TDT patients2 1
Slide 10: Ready to execute a high-impact commercial launch in the U.S.
– ready to execute a high-impact commercial launch in the U.S. 4,000 $425k addressable patients at annual U.S. WAC for leading patient launch thalassemia support services Potential to deliver $1B global peak-year-sales across PKD and thalassemia indications 10
Slide 11: Potential to expand PK activation franchise into Sickle Cell Disease and LR-MDS
Potential to expand PK activation franchise into Sickle Cell Disease and LR-MDS 11
Slide 12: PK activation proven mechanism in hemolytic anemias
PK activation proven mechanism in hemolytic anemias Hemolytic anemias – reduced RBC Activating PK in glycolytic pathway improves lifespan drives pathophysiology health, energy and lifespan of RBCs Healthy Red Blood Cell – 120 days RBC lifespan Increase ATP Decrease 2,3-DPG to match energy reversibly
Slide 13: Mitapivat has proven the transformative potential of PK activation in hemolytic anemias
Mitapivat has proven the transformative potential of PK activation in hemolytic anemias 6 positive well designed, randomized trials across 3 hemolytic anemias PK deficiency Thalassemia Sickle Cell Disease ACTIVATE ENERGIZE Phase 2 RISE UP ACTIVATE-T ENERGIZE-T Phase 3 RISE UP1 Statistically signific
Slide 14: Sickle Cell Disease – significant need for novel treatments
Sickle Cell Disease – significant need for novel treatments 2023 withdrawn from EU due to Meaningful opportunity with lack failed trial of disease-modifying innovation Crizanlizumab Approved 2019 Gene therapies Hydroxyurea • ~100,000 diagnosed adult and Approved 1998 Approved 2023 pediatric patients
Slide 15: Mitapivat – potential to expand in Sickle Cell Disease
Mitapivat – potential to expand in Sickle Cell Disease Strong anti-hemolytic profile Clinically meaningful Favorable in total trial population with benefits in Hb-responders safety profile SCPC trend • 40.6% of patients achieved • 1.6 g/dL mean increase in Hemoglobin • No similar pattern of HCI as o
Slide 16: Tebapivat – more potent PK activator
Tebapivat – more potent PK activator Binds longer and stronger Reduced potential for Improved metabolic to PKR and PKM2 enzymes Drug-drug interaction1 stability – longer half-life that regulate RBC energy and once-daily dosing Potential best-in-class PKa – opportunity to deepen Hb response and expan
Slide 17: Tebapivat – potential to show leading clinical profile in sickle cell disease
Tebapivat – potential to show leading clinical profile in sickle cell disease Potential to become leading Phase 1 tebapivat data demonstrates strong proof of concept in SCD PK activator in SCD • Enhanced PK/PD profile drives Mean change from baseline increased potency in Hb across dosing cohorts (2m
Slide 18: Tebapivat – novel mechanism and oral dosing with potential for durable response
Tebapivat – novel mechanism and oral dosing with potential for durable response Phase 2a showed clinical PoC Ongoing open-label Phase 2b designed to test higher doses in for tebapivat in LR-MDS heterogeneous patient population reflective of real world • 40% of low transfusion burden 2L+ cohort achie
Slide 19: Drive future growth with diversified rare disease pipeline
Drive future growth with diversified rare disease pipeline 19
Slide 20: Expanding in hematology – AG-236 for Polycythemia Vera
Expanding in hematology – AG-236 for Polycythemia Vera 100K diagnosed in U.S. AG-236 – GalNAc siRNA-based TMPRSS6 inhibitor targeting iron homeostasis regulation 35% phlebotomy dependent Currently approved treatments Q6M dosing – GalNAc siRNA has well No titration, single consist of phlebotomy, HU,
Slide 21: Diversification beyond hematology – AG-181 for PKU
Diversification beyond hematology – AG-181 for PKU Significant opportunity to AG-181 novel AG-181 unique attributes transform treatment of PKU mechanism of action Oral medicine targeting • PKU caused by dysfunctional classical Phenylketonuria Phe Tyr PAH enzyme Potential fast onset and PAH • ~15-20k
Slide 22: Financial discipline to achieve growth and long-term sustainability
Financial discipline to achieve growth and long-term sustainability 22
Slide 23: 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 24: Agios – Transforming for tomorrow
Agios – Transforming for tomorrow 24
Slide 25: 2025 in review – continued track record for execution
2025 in review – continued track record for execution EARLY MID-YEAR LATE Pediatric PK Deficiency Sickle Cell Disease Thalassemia PYRUKYND tebapivat AQVESMETM (mitapivat) FDA approval Phase 3 readout ACTIVATE-Kids Initiate enrollment in Phase 2 trial Polycythemia Vera Sickle Cell Disease AG-236 mita
Slide 26: 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 27: 2026 catalysts reinforce hemolytic anemia leadership and rare disease pipeline potential
2026 catalysts reinforce hemolytic anemia leadership and rare disease pipeline potential 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 teb
Slide 28: Appendix
Appendix 28
Slide 29: Appendix – AQVESME is first-and-only PK activator indicated for use in adult thalassemia patients
Appendix – AQVESME is first-and-only PK activator indicated for use in adult thalassemia patients AQVESME is indicated for the Indication statement treatment of anemia in adults with alpha- or beta-thalassemia Pyruvate kinase activator that allosterically Mechanism of action binds to the pyruvate ki
Slide 30: Appendix – AQVESME REMS for liver monitoring
Appendix – AQVESME REMS for liver monitoring Warnings and Precautions Label Language During the double-blind period, 2 of 301 patients (0.66%) with thalassemia treated with AQVESME experienced adverse reactions REMS Monitoring suggestive of hepatocellular injury. Three additional patients experience
Slide 31: Appendix – AQVESME availability in late January
Appendix – AQVESME availability in late January 6,000 diagnosed adult thalassemia patients in U.S. Addressable launch population | 4,000 patients Remaining 2,000 diagnosed adult thalassemia patients Higher frequency of visits, transfusion dependent and/or symptomatic Younger Older patient Co-morbid
Slide 32: Appendix – tebapivat Phase 2 Sickle Cell Disease trial
Appendix – tebapivat Phase 2 Sickle Cell Disease trial Tebapivat Phase 2 trial for Sickle Cell Disease1 Key Inclusion Criteria Primary Endpoint • 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 randomizat
Slide 33: Appendix – tebapivat Phase 2 Lower Risk-MDS trial
Appendix – tebapivat Phase 2 Lower Risk-MDS trial Tebapivat Phase 2b trial for Key Inclusion Criteria Primary Endpoint LR-MDS1 • Lower-risk MDS3 • Proportion of participants with • Transfusion dependent4 transfusion independence5 10mg QD • Hb <10.0 g/dL during the core period Extension • Up to 2 pri
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