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- 14 abstracts accepted at ASH 2026, including three oral presentations featuring new data across mitapivat clinical programs<\/li>\n<\/ul>\n
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- New analyses from RISE UP Phase 3 trial of mitapivat in sickle cell disease show durable hemoglobin improvements for up to two years, as well as potential kidney and cardiac health benefits<\/li>\n<\/ul>\n
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- First AG-236 clinical data from Phase 1 trial in healthy volunteers support every-six-month dosing in polycythemia vera, supporting a potential best-in-class profile <\/li>\n<\/ul>\n
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- Agios will host ASH 2026 virtual investor event on Monday, December 14, at 1:00 p.m. EST (12:00 p.m. CST)<\/li>\n<\/ul>\n
CAMBRIDGE, Mass., Nov. 04, 2026 (GLOBE NEWSWIRE) — Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that new clinical data across the company\u2019s rare hematology portfolio will be featured in oral and poster presentations during the 68th<\/sup> American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2026) in New Orleans, Louisiana, December 12-15, 2026.<\/p>\n
\u201cAs Agios continues to expand its impact across rare hematologic diseases, ASH 2026 represents a key opportunity to highlight the strength and differentiation of our portfolio,\u201d said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. \u201cIn sickle cell disease, new clinical data from the RISE UP Phase 3 trial show that mitapivat can deliver durable hemoglobin improvements for up to two years. RISE UP analyses also show the potential for mitapivat to improve measures of kidney function and cardiac remodeling in this population, which may reflect its unique dual activation of two pyruvate kinase isoforms \u2013 PKR expressed in red blood cells and PKM2 expressed in organ tissues. We will also present the first data from our AG-236 clinical program showing sustained, on-target activity that supports an every-six-month dosing schedule in polycythemia vera, potentially offering patients a best-in-class profile. Together, our ASH 2026 data reinforce the potential for these medicines to transform the treatment of rare hematologic diseases with significant patient needs, and we look forward to sharing them with the hematology community at the congress next month.\u201d<\/p>\n
Select Presentations at ASH 2026 <\/strong>
Mitapivat, oral pyruvate kinase (PK) activator<\/em><\/strong><\/p>\n- \n
- An oral presentation describing hemoglobin improvements with long-term mitapivat treatment (up to 115 weeks) among patients with sickle cell disease in the open-label extension (OLE) period of the RISE UP Phase 3 trial, during which all patients receive mitapivat. Of the 176 patients who completed the double-blind period of RISE UP, a total of 174 (118 from the mitapivat arm and 56 from the placebo arm) enrolled in the OLE period. Among all 118 patients continuing on mitapivat, 62.7% (n=74) achieved a hemoglobin improvement (defined as \u22651.0 g\/dL increase from baseline in average hemoglobin during any two consecutive study visits) during the OLE period. Notably, of the 63 non-responders during the double-blind period, 39.7% (n=25) achieved a hemoglobin improvement with continued mitapivat treatment during the OLE period. Additionally, of the 56 patients who crossed over from placebo to mitapivat, 51.8% (n=29) achieved a hemoglobin improvement during the OLE period. The safety profile in the OLE period was consistent with that observed during the double-blind period. These results continue to support the potential of mitapivat as a durable, disease-modifying treatment for patients with sickle cell disease.<\/li>\n<\/ul>\n
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- An oral presentation evaluating changes in kidney filtration rate, as measured by estimated glomerular filtration rate (eGFR), during the double-blind period of the RISE UP Phase 3 trial of mitapivat in sickle cell disease. In this condition, chronic hemolysis and anemia can cause the kidney to filter blood at an abnormally high rate (indicated by elevated eGFR), and this hyperfiltration is associated with long-term kidney damage. In a post hoc analysis, patients who received mitapivat had an early and sustained reduction in eGFR: among 132 patients with hyperfiltration (eGFR >135 mL\/min\/1.73 m\u00b2) at baseline, mitapivat-treated patients (n=92) showed a 14.7 mL\/min\/1.73 m\u00b2 decrease in eGFR at Week 52, compared with a 4.9 mL\/min\/1.73 m\u00b2 decrease with placebo (n=40). This reduction was observed regardless of whether patients achieved a hemoglobin response; taken together, these data suggest that the renal effects of mitapivat may be driven by both PKR- and PKM2-mediated mechanisms.<\/li>\n<\/ul>\n
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- A poster presentation showing favorable cardiac remodeling among patients with sickle cell disease who achieved a hemoglobin response with mitapivat during the double-blind period of the RISE UP Phase 3 trial. This effect was evaluated by left ventricular mass index (LVMI) and left atrial volume index (LAVI), two measures of cardiac size and structure that can be abnormally elevated in sickle cell disease due to chronic hemolysis and anemia. This post hoc analysis found that, at Week 52, LVMI decreased from baseline by 5.9 g\/m\u00b2 among hemoglobin responders in the mitapivat arm (n=40), whereas it increased by 5.8 g\/m\u00b2 among non-responders (n=45) and by 4.6 g\/m\u00b2 in the placebo group (n=27). Hemoglobin responders also achieved a greater decrease from baseline in LAVI at Week 52 (4.7 mL\/m\u00b2; n=35) compared with non-responders (1.7 mL\/m\u00b2; n=41) and the placebo group (0.2 mL\/m\u00b2; n=27). These data indicate that the anti-hemolytic effect of mitapivat may be a key driver of these improvements in cardiac remodeling, which, alongside preclinical evidence showing that PKM2 activation can attenuate cardiac fibrosis, suggests a potential dual cardiac benefit of mitapivat.<\/li>\n<\/ul>\n
AG-236, small interfering RNA (siRNA) targeting TMPRSS6<\/em><\/strong><\/p>\n
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- A poster presentation highlighting first-in-human data from a Phase 1 single ascending dose study of AG-236 in 24 healthy volunteers. The study showed that AG-236 produced sustained increases in hepcidin \u2013 a key regulator of iron homeostasis \u2013 and decreases in circulating iron availability, including reductions in serum iron and transferrin saturation through Month 6 post-dose. Biomarkers of erythropoiesis (red cell production), hemoglobin and hematocrit, were also reduced through Month 6. AG-236 was well tolerated across all dose levels (1.0, 3.0, and 9.0 mg\/kg), with no serious adverse events and no dose-limiting toxicities. In July 2026<\/u><\/a>, Agios announced plans to advance AG-236 into a Phase 2\/3 development program in polycythemia vera, with initiation of the Phase 2 portion expected in the second half of 2026.<\/li>\n<\/ul>\n
In total, 14 abstracts led by Agios and external collaborators have been accepted for presentation and publication at ASH 2026.<\/p>\n
ASH 2026 Accepted Abstracts<\/strong><\/p>\n
- A poster presentation highlighting first-in-human data from a Phase 1 single ascending dose study of AG-236 in 24 healthy volunteers. The study showed that AG-236 produced sustained increases in hepcidin \u2013 a key regulator of iron homeostasis \u2013 and decreases in circulating iron availability, including reductions in serum iron and transferrin saturation through Month 6 post-dose. Biomarkers of erythropoiesis (red cell production), hemoglobin and hematocrit, were also reduced through Month 6. AG-236 was well tolerated across all dose levels (1.0, 3.0, and 9.0 mg\/kg), with no serious adverse events and no dose-limiting toxicities. In July 2026<\/u><\/a>, Agios announced plans to advance AG-236 into a Phase 2\/3 development program in polycythemia vera, with initiation of the Phase 2 portion expected in the second half of 2026.<\/li>\n<\/ul>\n
- A poster presentation showing favorable cardiac remodeling among patients with sickle cell disease who achieved a hemoglobin response with mitapivat during the double-blind period of the RISE UP Phase 3 trial. This effect was evaluated by left ventricular mass index (LVMI) and left atrial volume index (LAVI), two measures of cardiac size and structure that can be abnormally elevated in sickle cell disease due to chronic hemolysis and anemia. This post hoc analysis found that, at Week 52, LVMI decreased from baseline by 5.9 g\/m\u00b2 among hemoglobin responders in the mitapivat arm (n=40), whereas it increased by 5.8 g\/m\u00b2 among non-responders (n=45) and by 4.6 g\/m\u00b2 in the placebo group (n=27). Hemoglobin responders also achieved a greater decrease from baseline in LAVI at Week 52 (4.7 mL\/m\u00b2; n=35) compared with non-responders (1.7 mL\/m\u00b2; n=41) and the placebo group (0.2 mL\/m\u00b2; n=27). These data indicate that the anti-hemolytic effect of mitapivat may be a key driver of these improvements in cardiac remodeling, which, alongside preclinical evidence showing that PKM2 activation can attenuate cardiac fibrosis, suggests a potential dual cardiac benefit of mitapivat.<\/li>\n<\/ul>\n
- An oral presentation evaluating changes in kidney filtration rate, as measured by estimated glomerular filtration rate (eGFR), during the double-blind period of the RISE UP Phase 3 trial of mitapivat in sickle cell disease. In this condition, chronic hemolysis and anemia can cause the kidney to filter blood at an abnormally high rate (indicated by elevated eGFR), and this hyperfiltration is associated with long-term kidney damage. In a post hoc analysis, patients who received mitapivat had an early and sustained reduction in eGFR: among 132 patients with hyperfiltration (eGFR >135 mL\/min\/1.73 m\u00b2) at baseline, mitapivat-treated patients (n=92) showed a 14.7 mL\/min\/1.73 m\u00b2 decrease in eGFR at Week 52, compared with a 4.9 mL\/min\/1.73 m\u00b2 decrease with placebo (n=40). This reduction was observed regardless of whether patients achieved a hemoglobin response; taken together, these data suggest that the renal effects of mitapivat may be driven by both PKR- and PKM2-mediated mechanisms.<\/li>\n<\/ul>\n
- An oral presentation describing hemoglobin improvements with long-term mitapivat treatment (up to 115 weeks) among patients with sickle cell disease in the open-label extension (OLE) period of the RISE UP Phase 3 trial, during which all patients receive mitapivat. Of the 176 patients who completed the double-blind period of RISE UP, a total of 174 (118 from the mitapivat arm and 56 from the placebo arm) enrolled in the OLE period. Among all 118 patients continuing on mitapivat, 62.7% (n=74) achieved a hemoglobin improvement (defined as \u22651.0 g\/dL increase from baseline in average hemoglobin during any two consecutive study visits) during the OLE period. Notably, of the 63 non-responders during the double-blind period, 39.7% (n=25) achieved a hemoglobin improvement with continued mitapivat treatment during the OLE period. Additionally, of the 56 patients who crossed over from placebo to mitapivat, 51.8% (n=29) achieved a hemoglobin improvement during the OLE period. The safety profile in the OLE period was consistent with that observed during the double-blind period. These results continue to support the potential of mitapivat as a durable, disease-modifying treatment for patients with sickle cell disease.<\/li>\n<\/ul>\n
- Agios will host ASH 2026 virtual investor event on Monday, December 14, at 1:00 p.m. EST (12:00 p.m. CST)<\/li>\n<\/ul>\n
- First AG-236 clinical data from Phase 1 trial in healthy volunteers support every-six-month dosing in polycythemia vera, supporting a potential best-in-class profile <\/li>\n<\/ul>\n
- New analyses from RISE UP Phase 3 trial of mitapivat in sickle cell disease show durable hemoglobin improvements for up to two years, as well as potential kidney and cardiac health benefits<\/li>\n<\/ul>\n