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

Mitapivat data include longer-term hemoglobin responses, while initial AG-236 findings support a potential six-month dosing interval.

Rhea-AI Summary

Agios Pharmaceuticals (AGIO) will present new mitapivat and AG-236 clinical data at ASH, taking place December 12–15, 2026. In the RISE UP sickle cell disease extension, 62.7% of 118 patients continuing mitapivat achieved a hemoglobin improvement; treatment extended up to 115 weeks. Post hoc analyses, conducted after the trial, showed reduced kidney hyperfiltration and favorable cardiac changes among hemoglobin responders.

In 24 healthy volunteers, AG-236 reduced circulating iron availability, hemoglobin and hematocrit through Month 6, with no serious adverse events or dose-limiting toxicities. Agios said the findings support potential every-six-month dosing in polycythemia vera; Phase 2 initiation is expected in the second half of 2026. The FDA extended mitapivat’s sickle cell disease application review goal by three months to February 1, 2027. Overall, 14 abstracts were accepted, including three oral presentations.

12 points · 0 major

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0 major · 4 points

Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate pointMitapivat extension: 62.7% of 118 continuing patients achieved hemoglobin improvement, with treatment up to 115 weeks.
• Moderate point. Forward-looking: it has not happened yet and may not happen.AG-236 Phase 2/3 development: Agios expects Phase 2 initiation in the second half of 2026.
• Minor pointPrior mitapivat non-responders: 39.7% of 63 achieved hemoglobin improvement during continued treatment in the extension.
• Minor pointPlacebo-to-mitapivat crossover: 51.8% of 56 patients achieved hemoglobin improvement during the extension.
• Minor pointMitapivat extension safety profile was consistent with the double-blind period.

7 minor points

• Minor pointKidney hyperfiltration subgroup: Week 52 filtration-rate decreases were 14.7 versus 4.9 mL/min/1.73 m² with mitapivat versus placebo.
• Minor pointCardiac muscle-mass index at Week 52: −5.9 g/m² in mitapivat responders versus +5.8 in non-responders and +4.6 with placebo.
• Minor pointLeft atrial volume index at Week 52: decreases of 4.7 mL/m² in responders versus 1.7 in non-responders and 0.2 with placebo.
• Minor pointAG-236 Phase 1 showed increased hepcidin, an iron-regulating hormone, and reduced circulating iron availability through Month 6.
• Minor pointAG-236 reduced hemoglobin and hematocrit, measures of red blood cells, through Month 6.
• Minor pointAG-236 had no serious adverse events or dose-limiting toxicities across 1.0, 3.0 and 9.0 mg/kg doses.
• Minor point. Forward-looking: it has not happened yet and may not happen.AG-236 findings support potential every-six-month dosing in polycythemia vera, Agios said.

Negative

• Moderate pointMitapivat FDA review goal was extended three months, from November 1, 2026, to February 1, 2027.
• Minor pointMitapivat kidney and cardiac findings came from post hoc analyses, rather than prespecified analyses.
• Minor pointLong-term hemoglobin findings came from an open-label extension in which all patients received mitapivat.
• Minor pointAG-236 initial clinical findings were limited to 24 healthy volunteers, not patients with polycythemia vera.

News Explained

The 62.7% hemoglobin-improvement rate among 118 patients was reported from RISE UP’s open-label extension, where all patients received mitapivat, rather than from its earlier double-blind period.

Key Figures

Hemoglobin improvement: 62.7% (74/118 patients) Hemoglobin improvement among prior non-responders: 39.7% (25/63 patients) Hemoglobin improvement after placebo crossover: 51.8% (29/56 patients) +5 more

Hemoglobin improvement

62.7% (74/118 patients)

Mitapivat open-label extension

Hemoglobin improvement among prior non-responders

39.7% (25/63 patients)

Continued mitapivat in the open-label extension

Hemoglobin improvement after placebo crossover

51.8% (29/56 patients)

Mitapivat open-label extension

eGFR change

−14.7 vs. −4.9 mL/min/1.73 m²

Week 52; mitapivat vs. placebo among patients with baseline hyperfiltration

LVMI change

−5.9 vs. +5.8 vs. +4.6 g/m²

Week 52; hemoglobin responders vs. non-responders vs. placebo

LAVI decrease

4.7 vs. 1.7 vs. 0.2 mL/m²

Week 52; hemoglobin responders vs. non-responders vs. placebo

AG-236 study size

24 healthy volunteers

Phase 1 single ascending dose study

AG-236 dosing interval

Every six months

Potential polycythemia vera dosing schedule supported by Phase 1 data

Previous Clinical trial Reports

2 past events · Latest: Jun 13

Same Type 2 events

1. Jun 13

   RISE UP Phase 3 results

   24h Move

   +8.0%

   Reported 52-week mitapivat results with significant hemoglobin responses and reduced transfusion burden.

2. Nov 19

   RISE UP topline results

   24h Move

   -50.9%

   RISE UP met its hemoglobin-response endpoint, while the reported 24-hour share reaction was negative.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

lvmi, sirna, pdufa, snda

4 terms

lvmi medical

"evaluated by left ventricular mass index (LVMI)"

Left ventricular mass index (LVMI) measures the weight of the heart’s main pumping chamber (left ventricle) adjusted for a person’s body size, similar to comparing vehicle engine size relative to car weight. It matters to investors because changes in LVMI are used as a clinical sign of heart disease progression or improvement in trials of drugs and devices, and can influence regulatory decisions, market potential and reimbursement outcomes.

sirna medical

"small interfering RNA (siRNA) targeting TMPRSS6"

Small interfering RNA (siRNA) is a short strand of genetic material that binds to and destroys the messenger RNA that carries instructions for making a specific protein, effectively switching that gene off. Investors care because siRNA is a platform for precise medicines: successful trials or approvals can create high-value drugs, while delivery challenges, manufacturing complexity, patent positions and regulatory risk can sharply affect a biotech company's prospects.

pdufa regulatory

"extended the Prescription Drug User Fee Act (PDUFA) goal date"

PDUFA is the Prescription Drug User Fee Act, the U.S. law under which drug companies pay fees that fund the FDA's review of new medicines. In company news the term usually appears as the PDUFA date, the target deadline by which the FDA aims to decide on a drug application; that date tells investors when to expect the approval or rejection decision for the product.

snda regulatory

"accepted its supplemental New Drug Application (sNDA)"

A SNDA (Subordination, Non‑Disturbance and Attornment Agreement) is a legal pact among a property owner’s lender, the owner’s tenants, and sometimes the landlord that sets who keeps lease rights if the property is sold or a mortgage is enforced. Think of it as a rulebook that decides whether a tenant can stay and keep paying rent or must answer to a new owner after a foreclosure. For investors, an SNDA matters because it protects predictable rental income, clarifies who has priority on claims against a property, and therefore affects a property’s value and the security of related loans.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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• 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 68^th 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

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 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

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did Agios report about long-term mitapivat responses in sickle cell disease?

During the RISE UP extension, 62.7% of 118 patients continuing mitapivat achieved a hemoglobin improvement. Among 63 patients who had not responded during the double-blind period, 39.7% improved with continued treatment. Among 56 patients switching from placebo, 51.8% improved.

What did Agios report from the first AG-236 clinical study?

AG-236 produced sustained changes in iron regulation through Month 6 in a Phase 1 study of 24 healthy volunteers. No serious adverse events or dose-limiting toxicities occurred. Agios said the findings support potential every-six-month dosing in polycythemia vera.

How was hemoglobin improvement defined in Agios' RISE UP extension?

Hemoglobin improvement meant a ≥1.0 g/dL increase from baseline in average hemoglobin during any two consecutive study visits. Of 176 patients who completed the double-blind period, 174 entered the extension: 118 from the mitapivat arm and 56 from the placebo arm.