On November 4, 2026 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, reported that new clinical data across the company’s rare hematology portfolio will be featured in oral and poster presentations during the 68th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (ASH 2026) in New Orleans, Louisiana, December 12-15, 2026.
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"As Agios continues to expand its impact across rare hematologic diseases, ASH (Free ASH Whitepaper) 2026 represents a key opportunity to highlight the strength and differentiation of our portfolio," said Sarah Gheuens, M.D., Ph.D., 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. 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. 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 (Free ASH Whitepaper) 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."
Select Presentations at ASH (Free ASH Whitepaper) 2026
Mitapivat, oral pyruvate kinase (PK) activator
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 ≥1.0 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.
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 m²) at baseline, mitapivat-treated patients (n=92) showed a 14.7 mL/min/1.73 m² decrease in eGFR at Week 52, compared with a 4.9 mL/min/1.73 m² 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.
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/m² among hemoglobin responders in the mitapivat arm (n=40), whereas it increased by 5.8 g/m² among non-responders (n=45) and by 4.6 g/m² in the placebo group (n=27). Hemoglobin responders also achieved a greater decrease from baseline in LAVI at Week 52 (4.7 mL/m²; n=35) compared with non-responders (1.7 mL/m²; n=41) and the placebo group (0.2 mL/m²; 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.
AG-236, small interfering RNA (siRNA) targeting TMPRSS6
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 – a key regulator of iron homeostasis – 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, 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.
In total, 14 abstracts led by Agios and external collaborators have been accepted for presentation and publication at ASH (Free ASH Whitepaper) 2026.
ASH 2026 Accepted Abstracts
Title Number Date/Time Presenter Acceptance
Sickle Cell Disease
Long-term hemoglobin
response from the Phase
3 RISE UP trial of
mitapivat in patients with
sickle cell disease 336 Saturday,
December 12, 2026,
5:15 – 5:30 p.m. CST Kevin Kuo, M.D.,
University of Toronto Oral
Mitapivat is associated
with early and sustained
improvements in eGFR
among patients with
sickle cell disease: A post
hoc analysis of the Phase
3 RISE UP trial 741 Monday, December
14, 2026, 11:00 –
11:15 a.m. CST Kenneth Ataga, M.D.,
Center for Sickle Cell
Disease at University of
Tennessee Health
Science Center Oral
Mitapivat is associated
with favorable cardiac
remodeling in
hemoglobin responders
with sickle cell disease: A
post hoc analysis of the
Phase 3 RISE UP trial 1158 Saturday,
December 12, 2026,
5:30 – 7:30 p.m. CST David Tofovic, M.D.,
University of Illinois
Hospital Poster
Mitapivat rapidly restores
red blood cell membrane
integrity: A window for
pre-emptive intervention
in sickle cell vaso-
occlusive crisis 1149 Saturday,
December 12, 2026,
5:30 – 7:30 p.m. CST Swee Lay Thein, MBBS,
DSc, FRCP, FRCPath,
MRCP, MRCPath,
National Heart, Lung &
Blood Institute Poster
Ongoing burden of sickle
cell disease among
individuals treated with
newly approved
disease-modifying
therapies: A real-world
claims analysis using
United States Medicaid
data 4434 Sunday, December
13, 2026, 6:00 – 8:00
p.m. CST Nirmish Shah, M.D., Duke
University Adult
Comprehensive Sickle
Cell Center Poster
Longitudinal trends in
renal biomarkers of US
patients with sickle cell
nephropathy 4712 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Santosh Saraf, M.D.,
University of Illinois
Hospital Poster
Impact of patient-
reported pain crises on
the lives of adults with
sickle cell disease in the
United States: Results
from the real-world RAISE
study 6194 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Sherif Badawy, M.D.,
Northwestern University
Feinberg School of
Medicine Poster
Trial in progress: Study
design of the
randomized, double-
blind, Phase 3 REIGNITE
trial to evaluate the effect
of mitapivat versus
placebo on transfusion
burden in patients with
sickle cell disease 4686 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Julia Xu, M.D., MScGH,
University of Pittsburgh
Medical Center Poster
Mitapivat promotes
terminal erythroid
maturation in patient-
derived sickle cell
disease erythroid
cultures 4668 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Dr. Wassim El Nemer,
Ph.D.,
French National Blood
Service (EFS)
Poster
Relationship between
markers of hemolysis and
clinical outcomes in
sickle cell disease: A
systematic literature
review and meta-analysis N/A N/A Kenneth Ataga, M.D.,
Center for Sickle Cell
Disease at University of
Tennessee Health
Science Center
Publication
Thalassemia
Long-term changes in
iron overload and
markers of iron regulation
and erythropoiesis with
mitapivat in patients with
α– or β–non–transfusion-
dependent thalassemia:
Results from the Phase
3 ENERGIZE open-label
extension 9 Saturday,
December 12, 2026,
10:00 – 10:15 a.m.
CST Hanny Al-Samkari, M.D.,
Mass General Brigham
Cancer Institute Oral
Clinical and patient-
centered implications of
reduced transfusion
burden with mitapivat in
transfusion-dependent
thalassemia: A post hoc
analysis of ENERGIZE-T 4655 Monday, December
14, 2026, 6:00 – 8:00
p.m. CST Sujit Sheth, Weill Cornell
Medicine Poster
Polycythemia Vera
Safety and tolerability of
AG-236, an
investigational TMPRSS6-
targeting small interfering
RNA for polycythemia
vera, in healthy
participants 3772 Sunday, December
13, 2026, 6:00 – 8:00
p.m. CST
Jonathan Meiman, M.D.,
University of Wisconsin-
Madison Poster
Other Conditions
Distinct metabolic
signatures differentiate
Rps19 and Rpl5
haploinsufficiency in
Diamond-Blackfan
Anemia syndromes 3183 Sunday, December
13, 2026, 6:00 – 8:00
p.m. CST Jonathan de Wilde, M.D.,
University Medical Center
Utrecht, Netherlands
Poster
Please refer to the ASH (Free ASH Whitepaper) 2026 website for full session details and data presentation listings and visit the Agios booth (#929) onsite.
ASH 2026 Investor Event
Agios will host an ASH (Free ASH Whitepaper) 2026 virtual investor event on Monday, December 14, at 1:00 p.m. EST (12:00 p.m. CST). The live webcast will be accessible on the Investors section of the company’s website (www.agios.com) under the "Events & Presentations" tab. A replay of the webcast will be available on the company’s website approximately two hours after the event.
About Mitapivat in Sickle Cell Disease
Sickle cell disease is a rare, inherited blood disorder in which abnormal hemoglobin causes red blood cells to become rigid and sickle-shaped, leading to chronic hemolysis (red blood cell destruction) that contributes to severe anemia and progressive, life-threatening complications, including damage to the lungs, kidneys, and heart. Mitapivat is an oral medicine designed to enhance red blood cell energy by activating the pyruvate kinase (PK) enzyme, which can increase levels of adenosine triphosphate (ATP) and lower levels of 2,3-diphosphoglycerate (2,3-DPG). Mitapivat has a unique dual mechanism of action that activates two isoforms of the PK enzyme – PKR expressed in red blood cells and PKM2 expressed in organ tissues – and has shown the potential to address both chronic hemolysis and pathways involved with long-term organ damage.
In July 2026, Agios announced that the U.S. Food and Drug Administration (FDA) accepted its supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease with a Priority Review. In October 2026, Agios announced the FDA extended the Prescription Drug User Fee Act (PDUFA) goal date for this sNDA by three months, from November 1, 2026, to February 1, 2027. The mitapivat sNDA is based on data from the global, randomized, double-blind, placebo-controlled RISE UP Phase 2 and Phase 3 trials (NCT05031780) in patients aged 16 years or older with sickle cell disease.
About AG-236 in Polycythemia Vera
Polycythemia vera (PV) is a rare blood cancer characterized by the overproduction of blood cells driven by an underlying genetic mutation in Janus kinase 2 (JAK2). Patients with PV typically have reduced levels of hepcidin, a critical hormone that regulates iron in the body. AG-236 is an investigational, small interfering RNA (siRNA) designed to silence the expression of the transmembrane serine protease 6 (TMPRSS6) gene, which normally downregulates hepcidin. By silencing TMPRSS6, AG-236 is designed to upregulate hepcidin, thereby restricting iron availability and controlling the overproduction of red blood cells associated with PV.
Based on promising results in a Phase 1 trial in healthy volunteers, Agios is advancing AG-236 into a Phase 2/3 development program (NCT07859423) in polycythemia vera, with initiation of the Phase 2 portion expected in the second half of 2026.
AG-236 is licensed from Alnylam Pharmaceuticals, Inc.
(Press release, Agios Pharmaceuticals, NOV 4, 2026, View Source [SID1234674242])