Agios to Highlight Strength and Differentiation of Rare Hematology Portfolio with New Clinical Data for Mitapivat in Sickle Cell Disease and AG-236 in Polycythemia Vera at ASH 2026
Globe Newswire•04/11/2026•09:01 ET
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Key Highlights
- ➤Mitapivat improved hemoglobin in 62.7% of continuing patients through 115 weeks
- ➤Mitapivat reduced eGFR 14.7 versus 4.9 mL/min/1.73 m² with placebo at Week 52
- ➤Mitapivat responders reduced LVMI 5.9 g/m², versus increases for non-responders and placebo
- ➤AG-236 sustained hepcidin increases and reduced iron availability through Month 6
- ➤AG-236 Phase 1 showed no serious adverse events or dose-limiting toxicities across 24 volunteers
Expert Statements
Sarah Gheuens, Chief Medical Officer and Head of R&D, Agios
“As 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”
Sarah Gheuens, Chief Medical Officer and Head of R&D, Agios
“In 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.”
Sarah Gheuens, Chief Medical Officer and Head of R&D, Agios
“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 – PKR expressed in red blood cells and PKM2 expressed in organ tissues.”
Sarah Gheuens, Chief Medical Officer and Head of R&D, Agios
“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.”
Sarah Gheuens, Chief Medical Officer and Head of R&D, Agios
“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.”
* 14 abstracts accepted at ASH 2026, including three oral presentations featuring new data across mitapivat clinical programs
* 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
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