A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Subjects With α- or β-Non-Transfusion-Dependent Thalassemia

The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.

Start Date01 Sep 2026

Sponsor / Collaborator

Agios Pharmaceuticals, Inc.

NCT07506863

/ Not yet recruitingPhase 3

The primary objective of this study is to compare the effect of mitapivat versus placebo on transfusion burden in pediatric participants with α- or β-transfusion-dependent thalassemia.

Start Date01 Sep 2026

Sponsor / Collaborator

Agios Pharmaceuticals, Inc.

NCT06286046

/ WithdrawnPhase 2

A Phase 2, Open-label, Multicenter Study of Mitapivat in Subjects With Sickle Cell Disease and Nephropathy (RESIST)

The primary purpose of this study is to evaluate the effect of mitapivat on albumin creatinine ratio (ACR) response in participants with sickle cell disease (SCD) and nephropathy.

Start Date01 Mar 2026

Sponsor / Collaborator

Agios Pharmaceuticals, Inc.

100 Clinical Results associated with Mitapivat

100 Translational Medicine associated with Mitapivat

100 Patents (Medical) associated with Mitapivat

Literatures (Medical) associated with Mitapivat

03 Feb 2026·CIRCULATION

S-Nitrosylation of Pyruvate Kinase Isoform 2 Drives Cardiac Fibrosis by Promoting Mitochondrial Fission

Article

Author: Han, Yi ; Wang, Yu ; Zhang, Yuxiao ; Yu, Bo ; Chen, Xin ; Luo, Shanshan ; Zhou, Miao ; Huang, Zhengrong ; Xie, Liping ; Hu, Lulu ; Sun, Shixiu ; Lv, Mengqi ; Kong, Xiangqing ; Ye, Danyu ; Wang, Xiaoqian ; Ji, Yong ; Zhang, Zhiren ; Zhang, Yan ; Zhong, Ke ; Sun, Changhao

BACKGROUND::

Cardiac fibrosis is a major determinant of adverse clinical outcomes of many heart diseases; currently, therapeutic strategy directly targeting fibroblasts is lacking. Nitric oxide–mediated nitrosative stress is associated with cardiac injury, and excessive nitric oxide can trigger S-nitrosylation (SNO) to specific cysteine thiol. This study aims to investigate the role of SNO in cardiac fibrosis and to identify potential therapeutic target.

METHODS::

SNO proteomic analysis was performed in cardiac tissue isolated from both mice subjected to transverse aortic constriction and spontaneous hypertensive rats. Elevated SNO of pyruvate kinase M2 (PKM2) was identified in cardiac fibroblasts, which was merely detected in cardiomyocytes. Cardiac fibroblast–specific PKM2 knockout mice and mice transfected with wild-type or SNO-resistant PKM2 mutant were used to determine the involvement of SNO of PKM2 (SNO-PKM2) in cardiac fibrosis. Unbiased proteomics and coimmunoprecipitation combined with mass spectrometry analysis were conducted to explore effectors mediating SNO-PKM2–induced activation of cardiac fibroblasts. A recently approved drug for rare blood disorder, mitapivat, was shown to dose-dependently relieve cardiac fibrosis.

RESULTS::

SNO of PKM2 at cysteine 49 and 326 increased in the heart tissue of patients with heart failure, heart tissue of murine cardiac fibrosis models, and cardiac fibroblasts stimulated with angiotensin II. SNO-PKM2 reduced pyruvate kinase activity and tetramerization of PKM2, and cardiac fibroblast–specific PKM2 knockout aggravated cardiac fibrosis, whereas cardiac fibroblast–specific PKM2 knockout mice transfected with SNO-resistant mutant rather than wild-type PKM2 had cardiac function. Mechanistically, SNO-PKM2 drove excessive mitochondrial fission and mitochondrial dysfunction through interfering with its interaction with actin regulatory protein gelsolin. TEPP-46, a pharmacological PKM2 activator, alleviated mitochondrial fission and cardiac fibrosis. Moreover, the US Food and Drug Administration–approved drug mitapivat showed preventive and therapeutical effects on cardiac fibrosis through activating PKM2.

CONCLUSIONS::

SNO-PKM2 specifically increases in cardiac fibroblasts and activated cardiac fibroblasts by inducing excessive mitochondrial fission through a gelsolin-dependent manner. Mitapivat is a potential therapeutic option for attenuating cardiac fibrosis.

01 Feb 2026·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Discovery of 8-quinolinesulfonamide phenylimidazole-based PKM2 agonists for the prevention and delay of aortic dissection

Article

Author: Zhong, Ke ; Xu, Xu ; Zhang, Chi ; Yang, Yanyan ; Li, Junda ; Xu, Shengtao ; Xu, Jinyi ; Chen, Kailin ; Wang, Tao ; He, Chen ; Cheng, Lei ; Luo, Shanshan

Thoracic aortic aneurysm and dissection (TAAD) is a life-threatening cardiovascular condition with a poor prognosis and high mortality rate. Currently, no specific pharmacological therapies are available for TAAD prevention. Clinical management depends mainly on antihypertensive drugs to reduce blood pressure and the risk of aortic rupture, supplemented by surgical intervention when necessary. In this study, guided by structural analysis of known PKM2 agonists and retention of key pharmacophoric features, we initially identified a promising lead compound A2 via fragment-based virtual screening. Through subsequent structure-activity relationship (SAR) optimization, we designed and synthesized 77 novel derivatives, among which, compound D16 emerged as a highly effective PKM2 agonist, demonstrating potent agonist activity (AC₅₀ = 77 nM, Emax = 216 %). D16 significantly suppressed the phenotypic switching of smooth muscle cells without apparent cytotoxicity. Furthermore, in a β-aminopropionitrile (BAPN)-induced TAAD mouse model, D16 treatment effectively prevented aortic dissection and resulted in reduced mortality compared with the approved drug Mitapivat. These results identify D16 as a promising and safe candidate for the prevention of TAAD, supporting its further investigation.

01 Nov 2025·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Fluorescent binding assay for allosteric ligands of liver pyruvate kinase

Article

Author: Bogucka, Agnieszka ; Grøtli, Morten ; Rutberg, Mikael ; Hyvönen, Marko ; Nilsson, Oscar ; Håversen, Liliana ; Liljenberg, Sara ; Köteles, István

Environment-sensitive fluorescent probes are indispensable tools for studying biological systems and advancing drug discovery. This study reports the development of 4-sulfamoyl-7-aminobenzoxadiazole (SBD)-based fluorescent probes for the allosteric site of the liver isoform of pyruvate kinase (PKL). By integrating SBD moieties into known activator scaffolds, such as mitapivat and diarylsulfonamide (DASA) ligands, probes for indicator displacement assays were designed to quantify ligand interactions in the allosteric site. Compound 4a displayed dose-dependent fluorescence enhancement in response to PKL binding and was used in a competitive binding assay with unlabelled ligands: mitapivat, TEPP-46, DASA-58 and reported activator 21. Structure-activity relationship (SAR) analysis revealed key structural features influencing activity and fluorescence sensitivity. The probes report selectively on the allosteric site ligands as the binding was not affected by natural ligands, such as ADP, fructose-1,6-bisphosphate (FBP), phosphoenolpyruvate (PEP), and phenylalanine. These findings provide a practical framework for detecting allosteric ligand engagement in PKL and expand the repertoire of molecular tools for advancing PKL-targeted therapies.

151

News (Medical) associated with Mitapivat

29 Apr 2026

·www.biospace.com

Agios Reports First Quarter 2026 Financial Results and Provides Business Update

Mitapivat (PYRUKYND ® and AQVESME™) generated worldwide net revenues of $20.7 million in the first quarter of 2026, compared to $8.7 million in the first quarter of 2025 Strong initial U.S. commercial launch of AQVESME in thalassemia, with 242 prescriptions written as of March 31, 2026 Company plans to submit mitapivat sNDA for sickle cell disease in the second quarter of 2026 Pipeline advancing to multiple value-driving inflection points in 2026, including two Phase 2 readouts for next-generation PK activator tebapivat $1.0 billion in cash, cash equivalents, and marketable securities as of March 31, 2026 CAMBRIDGE, Mass., April 29, 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 financial results and updates for the first quarter ended March 31, 2026. “Our first-quarter performance reflects strong execution and significant progress against our 2026 strategic objectives,” said Brian Goff, Chief Executive Officer, Agios. “The solid early momentum of our U.S. commercial launch of AQVESME in thalassemia highlights both the medicine’s clinical value and strong community reception. Additionally, following collaborative engagement with the FDA, we now plan to submit our mitapivat sNDA for sickle cell disease under the U.S. accelerated approval pathway in the second quarter. 2026 marks an important growth inflection point for Agios as we continue to build a sustainable rare disease company that is rooted in hematology and focused on delivering differentiated medicines that create meaningful long-term value for patients and shareholders.” First Quarter 2026 and Recent Corporate Highlights Mitapivat (PYRUKYND ® and AQVESME™) Commercial Performance and Update $18.8 million in U.S. net revenue and $1.9 million in ex - U.S. net revenue in the first quarter of 2026. U.S. net revenue was driven by the U.S. commercial launch of AQVESME™ (mitapivat) in thalassemia in late January 2026. Ex-U.S. net revenue reflected demand for PYRUKYND ® (mitapivat) in thalassemia in Saudi Arabia. 242 AQVESME prescriptions for thalassemia were written by Risk Evaluation and Mitigation Strategy (REMS)-certified U.S. physicians as of March 31, 2026, driven by motivated prescribers and highly engaged patients. R&D Highlights Mitapivat Thalassemia – In March 2026, the Emirates Drug Establishment of the United Arab Emirates (UAE) approved PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. With this approval, PYRUKYND becomes the only medicine approved in the UAE for this broad patient population. Mitapivat is now approved for adults with thalassemia in the U.S., Saudi Arabia, and the UAE. A marketing application for mitapivat in thalassemia is currently under review by the European Commission. Sickle Cell Disease – Agios confirmed plans to pursue U.S. accelerated approval for mitapivat in sickle cell disease, following completion of its pre-supplemental New Drug Application (sNDA) meeting with the U.S. Food and Drug Administration (FDA). The FDA’s accelerated approval pathway expedites the availability of medicines that can fill a medical need for a serious condition, with the requirement of a confirmatory clinical trial to convert to a traditional approval. The company now plans to submit an sNDA for mitapivat in sickle cell disease in the second quarter of 2026. Tebapivat Lower-Risk Myelodysplastic Syndromes (LR-MDS) – Agios expects to report topline results from its Phase 2b trial in the first half of 2026. Based on findings from the Phase 2a trial, the Phase 2b open-label trial is evaluating three higher daily dose levels of tebapivat (10 mg, 15 mg, and 20 mg) over a 24-week period. The primary endpoint is the proportion of participants achieving transfusion independence, defined as being transfusion-free for at least 8 consecutive weeks during the 24-week period. Sickle Cell Disease – Agios expects to report topline results from its Phase 2 trial in the second half of 2026. This double-blind, randomized, placebo-controlled trial is evaluating three daily dose levels of tebapivat (2.5 mg, 5 mg, and 7.5 mg) versus matched placebo over a 12-week period. The primary endpoint is hemoglobin response, defined as a ≥1.0 g/dL increase in average hemoglobin concentration from Week 10 through Week 12 compared with baseline. First Quarter 2026 Financial Results For the quarter ended March 31, 2026, net loss was $99.1 million, compared to net loss of $89.3 million for the quarter ended March 31, 2025. Net product revenue from U.S. sales of mitapivat (PYRUKYND and AQVESME) for the first quarter of 2026 was $18.8 million, compared to $8.7 million for the first quarter of 2025. Net product revenue from ex-U.S. sales of mitapivat (PYRUKYND) for the first quarter of 2026 was $1.9 million. Cost of sales for the first quarter of 2026 was $1.3 million. Research and Development (R&D) Expenses were $81.1 million for the first quarter of 2026, compared to $72.7 million for the first quarter of 2025, due to workforce-related expenses supporting pipeline advancement efforts, as well as increased mitapivat process development expenses. Selling, General and Administrative (SG&A) Expenses were $48.3 million for the first quarter of 2026, compared to $41.5 million for the first quarter of 2025, due to an increase in activities to support the U.S. commercial launch of AQVESME in thalassemia, as well as an increase in stock compensation expense. Cash, cash equivalents and marketable securities were $1.0 billion as of March 31, 2026, compared to $1.2 billion as of December 31, 2025. Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to execute the U.S. commercial launch of AQVESME in thalassemia, prepare for the potential U.S. commercial launch of mitapivat in sickle cell disease, advance the company’s existing clinical programs, and opportunistically expand its pipeline through both internally- and externally-discovered assets. First Quarter 2026 Conference Call Information Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s first quarter 2026 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website ( ) 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 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 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 ( ) 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 PYRUKYND ® (mitapivat), AQVESME™ (mitapivat), tebapivat, AG-236 and AG-181; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including mitapivat, tebapivat, AG-236 and AG-181; Agios’ expectations for the review of marketing applications for mitapivat by regulatory agencies, including the FDA and European Commission; Agios’ strategic vision and goals; 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 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. Consolidated Balance Sheet Data (in thousands) (Unaudited) March 31, 2026 December 31, 2025 Cash, cash equivalents, and marketable securities $ 1,045,492 $ 1,164,438 Accounts receivable, net 16,132 10,577 Inventory 35,087 32,920 Total assets 1,184,990 1,297,225 Stockholders' equity 1,109,115 1,193,114 Consolidated Statements of Operations Data (in thousands, except share and per share data) (Unaudited) Three Months Ended March 31, 2026 2025 Revenues: Product revenue, net $ 20,746 $ 8,726 Total revenue 20,746 8,726 Operating expenses: Cost of sales $ 1,319 $ 1,085 Research and development 81,148 72,743 Selling, general and administrative 48,304 41,527 Total operating expenses 130,771 115,355 Loss from operations (110,025) (106,629) Interest income, net 10,795 16,087 Other income, net 119 1,253 Net loss $ (99,111) $ (89,289) Net loss per share - basic and diluted $ (1.69) $ (1.55) Weighted-average number of common shares used in computing net loss per share – basic and diluted 58,782,241 57,459,195 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

Drug ApprovalPhase 2Accelerated ApprovalFinancial Statement

20 Apr 2026

·firstwordpharma.com

Novo's oral sickle cell drug etavopivat hits bullseyes in pivotal trial

Novo Nordisk said Monday a pivotal trial of its oral pyruvate kinase (PK) activator etavopivat met both primary efficacy goals, demonstrating a notable reduction in vaso-occlusive crises (VOCs) and improved haemoglobin (Hb) response against placebo in adults and adolescents with sickle cell disease (SCD). The company intends to commence marketing submissions in the second half."Etavopivat has the potential to be a first and best-in-class therapy and transform the lives of people with sickle cell disease, who currently have limited therapeutic options,” said Martin Holst Lange, chief scientific officer and R&D head at Novo. The Phase III HIBISCUS study enrolled 385 patients aged 12 years and older with SCD, who were randomised to receive etavopivat 400 mg once daily or placebo for 52 weeks, while being permitted to continue standard-of-care treatment throughout.Top-line results showed that on the VOC primary endpoint, etavopivat achieved a 27% reduction in annualised crisis rate compared with placebo, with the median time to first VOC nearly doubling — 38.4 weeks on etavopivat versus 20.9 weeks on placebo. The other co-primary goal of Hb response also favoured the study drug, with 48.7% of etavopivat recipients achieving an Hb increase >1 g/dL at week 24 versus 7.2% in the placebo arm, translating to an adjusted rate difference of 41.2%. Additionally, an exploratory analysis showed etavopivat significantly lowered the risk of blood transfusion.Novo also noted etavopivat was well tolerated, exhibiting a safety profile consistent with its previous studies. Complete data from HIBISCUS will be presented at a medical conference later this year. Etavopivat — gained through Novo's $1.1-billion takeover of Forma Therapeutics in 2022 — also continues to be investigated in the Phase IIIb HIBISCUS2 trial pitting it against standard-of-care or placebo.PK activators are trying to fill a major void in the oral SCD therapies space left by Pfizer's global withdrawal of its Hb polymerisation inhibitor Oxbryta (voxelotor) in 2024 following a number of patient deaths. Etavopivat potentially holds an edge over its rival Pyrukynd (mitapivat) from Agios Pharmaceuticals — which only managed to clear one of two primary endpoints in the pivotal RISE UP trial last year.Etavopivat, alongside the haemophilia therapy Mim8, also underscores Novo’s push beyond diabetes and obesity to build a stronger presence in rare diseases (see — Spotlight On: Novo throws weight behind budding rare disease pipeline).

Phase 3Clinical Result

20 Apr 2026

·www.biospace.com

Novo’s late-stage sickle cell win piles pressure on competitors

iStock, beast01 Novo Nordisk’s etavopivat elicited a 27% drop in vaso-occlusive crises and 48.7% hemoglobin response after 24 weeks, creating “separation amongst PK class candidates,” Truist analysts said on Monday. Novo plans to seek FDA approval in the back half of 2026. Novo Nordisk’s drug candidate for sickle cell disease significantly reduced severe pain crises in a Phase 3 study, opening the path for a regulatory submission later this year—and putting pressure on other drugmakers in this space. The readout “creates separation amongst PK class candidates,” analysts at Truist Securities wrote to investors on Monday. Data from the Phase 3 HIBISCUS study showed a 27% drop in vaso-occlusive crises (VOC) in patients taking etavopivat, as compared with placebo, according to Novo’s Monday morning release . The drug, taken orally once-daily, is a pyruvate kinase-R (PK) activator that works by preventing the sickling of red blood cells. Etavopivat came to Novo in September 2022 when the pharma acquired Forma Therapeutics for $1.1 billion . Novo’s drug also delayed the time to the first VOC episode, with this occurring at 38.4 weeks in etavopivat-treated patients, compared to 20.9 weeks in placebo recipients. Hemoglobin response above 1 g/dL—a measure of how well a patient responded to the treatment—at 24 weeks reached 48.7% in the etavopivat arm versus 7.2% in placebo. Novo will use the HIBISCUS readout to build toward a regulatory submission in the second half of 2026. These data also shine a light on competitor programs. In its Monday note, Truist pointed to Agios Pharmaceuticals, which is working on its own orally available PK activator mitapivat for sickle cell disease (SCD). Phase 3 data released in November 2025 demonstrated a 40.6% hemoglobin response in patients on mitapivat, compared with 2.9% in the placebo group. There have been no direct head-to-head studies assessing mitapivat against etavopivat, and cross-trial comparisons are inconclusive. “We think AGIO could come under pressure,” Truist wrote, “and the competitor update is another threat to AGIO shares.” Still, the analysts noted that they still see “a place for mitapivat in SCD given the high unmet need globally.” Agios is down 15% in premarket trading on Monday. Mitapivat is currently marketed under the brand name Pyrukynd for pyruvate kinase deficiency, for which it won approval in 2022. The drug picked up another approval for anemia in alpha- or beta-thalassemia in December. Last month, Agios announced it was working with the FDA to settle on an accelerated pathway for mitapivat in SCD, with a supplemental drug application planned for “the coming months.” If ultimately approved, Novo’s etavopivat will face off against other medicines in the sickle cell arena, including the gene therapies Casgevy (from Vertex Pharmaceuticals and CRISPR Therapeutics) and Lyfgenia (from bluebird bio). These therapies were approved in December 2023 but have had a hard time getting a foothold on the market. At least for Casgevy, however, things seem to be turning around. Vertex and CRISPR said in February that patient infusions have been on the rise and that sales of the gene therapy could see encouraging growth this year. Gene therapy Vertex, CRISPR Set Lofty Goal for Casgevy Gene Therapy as Patient Starts Ramp Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple in 2026, as patient access to the sickle cell disease and beta thalassemia gene therapy grows. February 13, 2026 · 3 min read · Dan Samorodnitsky Read more

Phase 3AcquisitionDrug ApprovalClinical ResultGene Therapy

100 Deals associated with Mitapivat

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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Alpha-Thalassemia	Saudi Arabia	Agios Pharmaceuticals, Inc.	04 Aug 2025
Beta-Thalassemia	Saudi Arabia	Agios Pharmaceuticals, Inc.	04 Aug 2025
Thalassemia	Saudi Arabia	Agios Pharmaceuticals, Inc.	04 Aug 2025
Anemia, Hemolytic	United States	Agios Pharmaceuticals, Inc.	17 Feb 2022
Pyruvate Kinase Deficiency	United States	Agios Pharmaceuticals, Inc.	17 Feb 2022

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Non-transfusion dependent thalassaemia	NDA/BLA	United States	Agios Pharmaceuticals, Inc.	08 Jan 2025
Transfusion-dependent Thalassemia	NDA/BLA	United States	Agios Pharmaceuticals, Inc.	08 Jan 2025
Anemia, Sickle Cell	Phase 3	United States	Agios Pharmaceuticals, Inc.	11 Feb 2022
Anemia, Sickle Cell	Phase 3	Belgium	Agios Pharmaceuticals, Inc.	11 Feb 2022
Anemia, Sickle Cell	Phase 3	Brazil	Agios Pharmaceuticals, Inc.	11 Feb 2022
Anemia, Sickle Cell	Phase 3	Canada	Agios Pharmaceuticals, Inc.	11 Feb 2022
Anemia, Sickle Cell	Phase 3	France	Agios Pharmaceuticals, Inc.	11 Feb 2022
Anemia, Sickle Cell	Phase 3	Germany	Agios Pharmaceuticals, Inc.	11 Feb 2022
Anemia, Sickle Cell	Phase 3	Israel	Agios Pharmaceuticals, Inc.	11 Feb 2022
Anemia, Sickle Cell	Phase 3	Italy	Agios Pharmaceuticals, Inc.	11 Feb 2022

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04770779 (ENERGIZE-T, ASH2025)	Phase 3	Transfusion-dependent Thalassemia \| Transfusion-dependent Beta Thalassemia	-	Mitapivat 100 mg	xtiucdmqun(mjduxourvy) = fmbwptkbls omdjipkjzs (ckuinpkxvt, 16.892) View more	Positive	06 Dec 2025
NCT05031780 (RISE UP, Company_Website) Manual	Phase 3	Anemia, Sickle Cell	-	Mitapivat	pdetcmwhsc(etnzoejafq) = cfgndmtzpy fxtmvwnnha (srqavtdhxt ) Met View more	Positive	19 Nov 2025
				Placebo	pdetcmwhsc(etnzoejafq) = bhpefvieqc fxtmvwnnha (srqavtdhxt ) Met View more
NCT04610866 \| NCT04000165 \| NCT05031780 (Pubmed)	Phase 1/2	Anemia, Sickle Cell	15	Mitapivat 50 mg BID escalated to 100 mg BID after 4 weeks	eryaxacces(ttejyblxuw) = duqbyxwlom itzzhbouyj (wmrzpriybv ) View more	Positive	01 Sep 2025
NCT05935202 (SATISFY, EHA2025)	Phase 2	Anemia, Dyserythropoietic, Congenital	24	Mitapivat 100mg twice daily	hebozdnhlj(jxayozuvxr) = TEAEs were generally mild and consistent with mitapivat’s known safety profile, most frequently headache (50%), upper respiratory infection (46%), and insomnia (38%) ynpnhweghb (vjfxnceksl ) View more	Positive	13 Jun 2025
NCT04770779 (ENERGIZE-T, CTgov)	Phase 3	Transfusion-dependent Thalassemia \| Transfusion-dependent Beta Thalassemia	258	Mitapivat (Mitapivat)	mjwzrdoxxk = ecxdrqfnoy efnsxpvapw (knzdbsqvji, xzpxgxwuck - npsnmfjfog) View more	-	25 May 2025
				Placebo Matching Mitapivat+Mitapivat (Placebo)	mjwzrdoxxk = kqdmmvmadt efnsxpvapw (knzdbsqvji, pdhvxadmdk - jekwzdbujl) View more
NCT05144256 (ACTIVATE-KidsT, EHA2025)	Phase 3	Pyruvate Kinase Deficiency	49	Mitapivat	abaoxgfbwm(ewqcaacxlh) = qeactnxpwa nfoafmhgkz (qzngemxrek ) View more	Positive	14 May 2025
				Placebo	abaoxgfbwm(ewqcaacxlh) = vlmpntzctj nfoafmhgkz (qzngemxrek )
ESTIMATE study (EHA2025)	Phase 2	Anemia, Sickle Cell HbSS \| HbS/β0 \| HbS/β+	10	Mitapivat 100mg BID	muxagvbcqs(lcgcximmhy) = muugfecrgd rqbqpgxvxu (igvwlfpiei ) View more	Positive	14 May 2025
				Placebo	lkutmpvupc(hguucqbsdw) = visrzlkghp fotzqpnxkg (ffsrptxzmw )
NCT05175105 (ACTIVATE-Kids, Company_Website) Manual	Phase 3	Pyruvate Kinase Deficiency	30	Mitapivat twice-daily	izdtsslplm(yhjobchdqw) = fepihoekjy ueymojfbhk (kxrlcuekzt ) Met	Positive	13 Feb 2025
				Matched Placebo	izdtsslplm(yhjobchdqw) = vmscvkabep ueymojfbhk (kxrlcuekzt ) Met
NCT04770753 (ENERGIZE, CTgov)	Phase 3	Beta-Thalassemia \| Non-transfusion dependent thalassaemia \| Alpha-Thalassemia	194	Mitapivat (Mitapivat)	cqkyefinma = mtqnmbhavi avrmlhpwvs (vmiskijzmr, lvxrcfjehi - cqjadgbrvv) View more	-	24 Jan 2025
				Placebo Matching Mitapivat+Mitapivat (Placebo)	cqkyefinma = nhtxkpcxct avrmlhpwvs (vmiskijzmr, jxwgepgwtq - cqvejtbmua) View more
NCT05935202 (Satisfy, ASH2024) Manual	Phase 2	Anemia, Dyserythropoietic, Congenital	24	Mitapivat 50 mg BID	dxjlexdrts(hlrxcdwrii) = One non-treatment related SAE (grade 3) occurred lmftdrcltw (yxsfnztzqp ) View more	Positive	09 Dec 2024

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Core Patent

Boost your research with our Core Patent data.

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Clinical Trial

Identify the latest clinical trials across global registries.

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Approval

Accelerate your research with the latest regulatory approval information.

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Regulation

Understand key drug designations in just a few clicks with Synapse.

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Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

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Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis