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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

AGIO
  • 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

  • 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

  • Agios will host ASH 2026 virtual investor event on Monday, December 14, at 1:00 p.m. EST (12:00 p.m. CST)

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’s rare hematology portfolio will be featured in oral and poster presentations during the 68th American Society of Hematology (ASH) Annual Meeting and Exposition (ASH 2026) in New Orleans, Louisiana, December 12-15, 2026.

"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," 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 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 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 2026.

ASH 2026 Accepted Abstracts

TitleNumberDate/TimePresenterAcceptance
Sickle Cell Disease
Long-term hemoglobin

response from the Phase

3 RISE UP trial of

mitapivat in patients with

sickle cell disease		336Saturday,

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		741Monday, 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		1158Saturday,

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		1149Saturday,

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		4434Sunday, 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		4712Monday, 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		6194Monday, 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		4686Monday, 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		4668Monday, 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/AN/AKenneth 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		9Saturday,

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		4655Monday, 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		3772Sunday, 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		3183Sunday, 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 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 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.

About Agios: Fueled by Connections to Transform Rare Diseases™

At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X.

Available Information about Agios

To achieve broad dissemination, Agios may disclose information to the public through a variety of disclosure channels including press releases, SEC filings, and public conference calls and webcasts. Some of the information distributed through these disclosure channels may be considered material information. Investors and others should note that Agios plans to use its website (www.agios.com) as a distribution channel to announce and give notice of Agios’ upcoming events and presentations (including, but not limited to, presentations at medical or healthcare conferences). Such information, which may be deemed material, will be available on the Investors section of the company’s website under the "Events & Presentations" tab. In addition, you may sign up to automatically receive email alerts about Agios’ upcoming events and presentations ("Calendar Alerts") by visiting the "Email Alerts" option under the "IR Resources" tab of the Investors section of the company’s website and submitting your email address.

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of mitapivat and AG-236; Agios’ plans regarding future data presentations; and the potential benefits of Agios’ strategic plans and focus. The words "anticipate," "expect," "goal," "hope," "milestone," "plan," "potential," "possible," "strategy," "will," "vision," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios’ ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236 and cevidoplenib, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.

Contacts:

Investor Contact

Morgan Sanford, Vice President, Investor Relations

Agios Pharmaceuticals

morgan.sanford@agios.com

Media Contact

Eamonn Nolan, Senior Director, Corporate Communications

Agios Pharmaceuticals

eamonn.nolan@agios.com



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