31st European Hematology Association Congress
(EHA 2026)
Fueled by Connections to Transform Rare Diseases™
Agios EHA 2026 Data Room
Agios is presenting new data on mitapivat, the company’s oral pyruvate kinase (PK) activator, at EHA 2026.
Select presentations and publications at EHA 2026 will include:
- An oral presentation during the Plenary Abstracts Session on detailed efficacy and safety data from the global 52-week, randomized, double-blind, placebo-controlled RISE UP Phase 3 trial of mitapivat in patients aged 16 years or older with sickle cell disease. Results demonstrate that treatment with mitapivat significantly improved hemoglobin response (a ≥1.0 g/dL increase from baseline in average hemoglobin from Week 24 through Week 52) and reduced markers of hemolysis (red blood cell destruction). Additional analyses, including measures of clinical benefit and patient-reported outcomes that were not previously disclosed in the company’s November 2025 topline report, will also be presented. The RISE UP abstract was one of only six selected for this distinguished plenary session from thousands of submissions.
- An oral presentation on long-term data from the ENERGIZE Phase 3 trial of mitapivat in adult patients with non-transfusion-dependent alpha- or beta-thalassemia. Of the 192 patients who received at least one dose of mitapivat or placebo in the double-blind period of ENERGIZE, 95% (n=182) opted to transition into the corresponding open-label extension (OLE) period, during which all patients receive mitapivat. The results show the durability of mitapivat treatment, with clinically meaningful hemoglobin improvements sustained for up to 127 weeks across the double-blind and OLE periods. During the OLE, nearly half (n=30/61) of non-responders who switched from placebo achieved a hemoglobin improvement (a ≥1 g/dL increase from baseline in average hemoglobin during any two consecutive OLE study visits), supporting a consistent treatment effect with mitapivat. Additionally, about one-third (n=23/68) of non-responders who received mitapivat during the double-blind period achieved a hemoglobin improvement during the OLE, suggesting a potential benefit of continued long-term treatment.
- A poster presentation highlighting a subset of patients with higher baseline hemoglobin levels (≥9.5 g/dL) in ENERGIZE. Among these patients, 38.9% (n=7/18) in the mitapivat arm achieved a hemoglobin response (a ≥1 g/dL increase from baseline in average hemoglobin from Week 12 through Week 24) in the double-blind period, compared with 0% (n=0/11) in the placebo arm. Additionally, patients in the mitapivat arm showed a 5.1-point improvement in patient-reported fatigue scores compared with 0.8 points among those in the placebo arm, as measured by the least squares mean change from baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue from Week 12 to Week 24.
- An oral presentation on the collaborator-led SATISFY Phase 2 trial of mitapivat in 24 patients with one of three rare hemolytic anemias: hereditary spherocytosis, hereditary xerocytosis, or congenital dyserythropoietic anemia type II. Treatment resulted in sustained hemoglobin improvements over 56 weeks. Notably, patients who achieved a hemoglobin response (a ≥1 g/dL increase from baseline in average hemoglobin during at least two consecutive study visits) also showed significant decreases in liver iron content, an important marker of downstream complications in hemolytic anemias.
Events
June 13, 2026, 9:00 a.m. EDT (3:00 p.m. CEST)
EHA 2026 Investor Conference Call and Webcast
Agenda
- Leadership in Rare Hematology: Agios’ Brian Goff will present an overview of the company’s rare hematology programs.
- Key Data at EHA 2026: Agios’ Dr. Sarah Gheuens will highlight the 10 abstracts accepted for presentation and publication at EHA 2026.
- KOL Fireside Chat: Our featured clinical experts will discuss key takeaways from the RISE UP clinical program and the sickle cell disease treatment landscape.
- Unlocking Growth: Agios’ Tsveta Milanova will share the growth opportunity in thalassemia and sickle cell disease.
- Live Q&A: Agios leadership and featured clinical experts will answer questions.
Featured Speakers
- Brian Goff – Chief Executive Officer, Agios
- Sarah Gheuens, M.D., Ph.D. – Chief Medical Officer and Head of R&D, Agios
- Tsveta Milanova – Chief Commercial Officer, Agios
- Alan Anderson, M.D. (KOL Fireside Chat) – Associate Professor of Pediatrics at the University of South Carolina School of Medicine Greenville and Director of the Comprehensive Lifespan Sickle Cell Disease Program at Prisma Health
- Kenneth Ataga, M.D. (KOL Fireside Chat) – Plough Foundation Endowed Chair in Sickle Cell Disease and Director of the Center for Sickle Cell Disease at the University of Tennessee Health Science Center
- Ahmar Zaidi, M.D. (KOL Fireside Chat Moderator) – Senior Medical Director, Clinical Development, Agios, and former Pediatric Hematologist at the Comprehensive Sickle Cell Center at Children’s Hospital of Michigan
News
June 13, 2026
Agios Showcases RISE UP Phase 3 Results at EHA 2026 Plenary Session Reinforcing Strong Anti-Hemolytic Profile of Mitapivat in Sickle Cell Disease
May 12, 2026
Agios to Present New Data at EHA 2026 Reinforcing the Significant Therapeutic Impact of Mitapivat Across Multiple Rare Hemolytic Anemias
Agios- and Collaborator-Led Presentations & Publications
Sickle Cell Disease
Oral Plenary Session:
Efficacy and Safety of Mitapivat in Sickle Cell Disease: Results From the Global, Randomized, Phase 3 RISE UP Trial
Abstract: S102
Presenter: Biree Andemariam, M.D., University of Connecticut Health
Session Date: Saturday, June 13
Session Time: 12:00 – 1:30 p.m. CEST
View presentation >
Poster Presentations:
Metabolic Dysregulation of Energy, Arginine Metabolism and Inflammation Associated With Point of Sickling in Patients With Sickle Cell Anemia
Abstract: PF1264
Presenter: Amira Idrizovic, Ph.D., Vall d’Hebron Research Institute, Barcelona, Spain
Session Date: Friday, June 12
Session Time: 6:45 – 7:45 p.m. CEST
View poster >
Impaired Function of Pyruvate Kinase Is Associated With Hemolysis-Related Inflammation, Ineffective Erythropoiesis, and Endothelial Dysfunction in Adults With Sickle Cell Anemia
Abstract: PF1269
Presenter: Marissa J.M. Traets, M.D., University Medical Center Utrecht, Netherlands
Session Date: Friday, June 12
Session Time: 6:45 – 7:45 p.m. CEST
View poster >
Thalassemia
Oral Presentation:
Long-Term Hemoglobin Improvements in Non-Transfusion-Dependent Alpha- or Beta-Thalassemia: Results From the Open-Label Extension of the Ongoing Phase 3 ENERGIZE Trial
Abstract: S296
Presenter: Hanny Al-Samkari, M.D., Mass General Brigham Cancer Institute
Session Date: Saturday, June 13
Session Time: 5:15 – 6:30 p.m. CEST
View presentation >
Poster Presentation:
Efficacy of Mitapivat in Patients With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia With Baseline Hemoglobin ≥9.5 g/dL: Subgroup Analysis From the Phase 3 ENERGIZE Trial
Abstract: PF1281
Presenter: Khaled M. Musallam, M.D., Ph.D., Burjeel Medical City, Abu Dhabi, United Arab Emirates
Session Date: Friday, June 12
Session Time: 6:45 – 7:45 p.m. CEST
View poster >
Publication Only:
ENERGIZEKIDS: Mitapivat in Pediatric Patients With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia
Abstract: PB4160
Lead Author: Kathryn Dickerson, M.D., University of Texas Southwestern Medical Center
ENERGIZEKIDS-T: Mitapivat in Pediatric Patients With Transfusion-Dependent Alpha- or Beta-Thalassemia
Abstract: PB4162
Lead Author: Janet Kwiatkowski, M.D., MSCE, Children’s Hospital of Philadelphia
Other
Publication Only:
The Patient Experience of Fatigue in Individuals With Sickle Cell Disease, Thalassemia, and Pyruvate Kinase Deficiency
Abstract: PB4417
Lead Author: Biree Andemariam, M.D., University of Connecticut Health
Oral Presentation:
Effects of Mitapivat on Iron Burden and Spleen Size in Erythrocyte Membranopathies and Congenital Dyserythropoietic Anemia Type II: 56-Week Follow Up Results From the SATISFY Study
Abstract: S304
Presenter: Thomas Doeven, M.D., University Medical Center Utrecht, Netherlands
Session Date: Thursday, June 11
Session Time: 4:45 – 6:00 p.m. CEST
View presentation >
Poster Presentation:
Diamond-Blackfan Anemia Syndrome Erythroid Progenitors Display Abnormal Metabolomic Profile
Abstract: PS1781
Presenter: Veronica Riccardi, M.D., University of Verona, Italy
Session Date: Saturday, June 13
Session Time: 6:45 – 7:45 p.m. CEST
View poster >