Milan Set to Witness Potential Blood Disorder Breakthroughs as Agios Unveils Key Data

Agios Pharmaceuticals will present significant new data on its pioneering pyruvate kinase (PK) activators, mitapivat and tebapivat, at the 30th EHA Congress in Milan.
The findings offer renewed hope for patients suffering from severe rare blood disorders, including sickle cell disease, thalassemia, and PK deficiency, many of whom have limited treatment options.
This major showcase will highlight Agios's leadership in cellular metabolism and its commitment to delivering transformative therapies for devastating conditions.

The hematology world watches with anticipation as Agios Pharmaceuticals prepares to take center stage at the 30th European Hematology Association (EHA) Congress in Milan. From June 12-15, 2025, this trailblazer in cellular metabolism will unleash a compelling wave of new data from its groundbreaking pyruvate kinase (PK) activation portfolio, potentially heralding a new dawn for those battling debilitating rare blood disorders^{6, 8}.

Spearheading this scientific advance are mitapivat (PYRUKYND®) and tebapivat, PK activators at the cutting edge of research. "The clinical results and scientific insights being presented at EHA add to the robust body of efficacy and safety data demonstrating the promise of PK activation," stated Dr. Sarah Gheuens, Agios's Chief Medical Officer. The focus is on delivering "meaningful results" for critical conditions like sickle cell disease, thalassemia, PK deficiency, and myelodysplastic syndromes—diseases that often leave patients with few, if any, lifelines^{1, 5}.

With 14 presentations poised to illuminate the path forward, including crucial updates on mitapivat's journey towards potentially aiding thalassemia patients (with a U.S. FDA decision expected by September 7, 2025)^{5, 7}, and tebapivat's promising developments in sickle cell disease⁴, the stakes are high. While the rigorous path of innovation includes navigating challenges, such as the ACTIVATE-KidsT study outcome⁴ and important PYRUKYND safety considerations, the buzz in Milan signals a potentially pivotal moment. A new chapter in the fight against these relentless diseases may be on the horizon.

Milan Set to Witness Potential Blood Disorder Breakthroughs as Agios Unveils Key Data

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