Agios to Present Rare Blood Disorder Data at EHA 2024

Agios Pharmaceuticals, Inc., a pioneer in cellular metabolism and PK activation therapies for rare diseases, has announced that it will present data from its ongoing research programs at the European Hematology Association 2024 (EHA2024) Hybrid Congress, scheduled to take place from June 13 to 16, 2024, in Madrid, Spain.

Key highlights include presentations from the Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia. The ENERGIZE study, which focuses on alpha- and beta- non-transfusion-dependent thalassemia, will be featured in a plenary session on June 15 from 14:45 to 15:15 CEST by Dr. Ali T. Taher of the American University of Beirut Medical Center. Furthermore, a poster session on June 14 from 18:00 to 19:00 CEST will shed light on improvements in fatigue and six-minute walk test outcomes in adults with this condition, led by Dr. Kevin H. M. Kuo from the University of Toronto.

Additionally, Agios will present information on the design of the Phase 3 portion of the RISE UP study of mitapivat in sickle cell disease, which is a randomized, double-blind, placebo-controlled trial. Dr. Biree Andemariam from the University of Connecticut Health will lead this presentation on June 14 at 9:00 CEST. Another key presentation will discuss a Phase 2 study of mitapivat in patients with sickle cell disease and nephropathy, led by Dr. Fuad El Rassi of Emory University School of Medicine, also scheduled for June 14 at 9:00 CEST. Furthermore, Dr. La’Ron Browne from St. Jude Children's Research Hospital will present a cost analysis of care expenditures for sickle cell disease patients on hydroxyurea therapy, also on June 14 at 9:00 CEST.

Data on pyruvate kinase deficiency will also be presented, focusing on the characteristics of pediatric patients with iron overload. This poster presentation, scheduled for June 14 from 18:00 to 19:00 CEST, will be led by Dr. Rachael F. Grace from Harvard Medical School.

Agios’ collaborators will present new findings on mitapivat as a potential treatment for other rare hemolytic anemias. An oral presentation on June 16 from 11:30 to 12:45 CEST by Jonathan R.A. de Wilde, a Ph.D. candidate from Utrecht University, will examine ex vivo pyruvate kinase activation in hereditary spherocytosis and xerocytosis, focusing on improved enzyme function and red cell properties.

Agios Pharmaceuticals will also hold a virtual investor event on June 16, 2024, at 10 a.m. Eastern Time (4 p.m. Central European Summer Time) to discuss the key clinical presentations from the EHA meeting. This event will be webcast live and can be accessed via the "Events & Presentations" section of the company's website.

Agios is renowned for its pioneering work in PK activation and remains dedicated to developing transformative therapies for patients with rare diseases. The company currently markets a pyruvate kinase activator for adults with PK deficiency, the first treatment of its kind for this debilitating hemolytic anemia. Building on its expertise in classical hematology and cellular metabolism, Agios is advancing a robust clinical pipeline, including therapies for alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, MDS-associated anemia, and phenylketonuria. The company is also working on a preclinical TMPRSS6 siRNA project as a potential treatment for polycythemia vera.

Agios Pharmaceuticals remains at the forefront of developing innovative treatments for rare hematologic diseases, showcasing its commitment through ongoing research and data presentations at significant medical conferences like EHA2024.