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.

NCT07055243

/ RecruitingPhase 2IIT

Safety and Efficacy of Mitapivat Sulfate in Adult Patients With Erythrocyte Membranopathies - Canada (SATISFY-CA)

This is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII, a rare sub type of anemia. Nine patients from Princess Margaret who are diagnosed with CDAII will be enrolled to the study. Patients will be in the trial for 57 weeks treatment weeks and a safety follow up week after 30 days from last dose. First 8 weeks will be dose escalating period followed by 48 weeks of fixed dose period. 57th week will be dose tapering week. Data collected from Princes Margaret will be incorporated to the main study conducted in EU for analysis. Overall, approximately 25 patients are expected to be enrolled: Approximately 16 patients at sites in the EU and approximately 9 patients in Canada.

Start Date26 Jun 2025

Sponsor / Collaborator

University Health Network

[+1]

CTIS2024-513528-41-02

/ Not yet recruitingPhase 3IIT

The Effect of Mitapivat on Cerebral Perfusion and Oxygen Metabolism in Sickle Cell Disease

Start Date01 Apr 2025

Sponsor / Collaborator-

100 Clinical Results associated with Mitapivat

100 Translational Medicine associated with Mitapivat

100 Patents (Medical) associated with Mitapivat

Literatures (Medical) associated with Mitapivat

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 ; Luo, Shanshan ; Cheng, Lei

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 ; Hyvönen, Marko ; Rutberg, Mikael ; Nilsson, Oscar ; Liljenberg, Sara ; Håversen, Liliana ; 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.

13 Oct 2025·ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Potent Fluorescent Probe for Target‐Engagement Studies of Allosteric Pyruvate Kinase Modulators

Article

Author: Brear, Paul ; Bogucka, Agnieszka ; Gunnarsson, Anders ; Hyvönen, Marko ; Rutberg, Mikael ; Nilsson, Oscar ; Håversen, Liliana ; Valaka, Anna P. ; Grøtli, Morten ; Köteles, István

Abstract:

Pyruvate kinases (PKs) are highly allosterically regulated enzymes that play a central role in cellular metabolism and are increasingly recognized as valuable therapeutic targets in cancer, metabolic diseases, and diabetes. Despite their biological and clinical significance, methods to directly assess allosteric ligand engagement of PK isoforms remain limited. Here, we report the development of LumiPK, a novel, environment‐sensitive fluorescent tracer designed to monitor allosteric binding to the liver isoform of pyruvate kinase (PKL). LumiPK integrates an environment‐sensitive 4‐sulfamonyl‐7‐aminobenzoxadiazole fluorophore into a potent allosteric modulator scaffold. It emerged as the lead compound from a small ligand series, showing high affinity for PKL (KD = 37 ± 5 nM) in recombinant assays; the most potent fluorescent PK reporter reported to date. A NanoBRET assay using a PKL‐Nluc fusion (PKLNluc) enabled intracellular monitoring of unlabeled ligand engagement. LumiPK maintained high potency (EC50 = 18.4 nM) in cellular experiments. Competitive NanoBRET and fluorescence titration assays confirmed binding of known PKL activators (mitapivat, TEPP‐46, DASA‐58) in both cellular and recombinant settings, with KD values remaining consistent across these methods. LumiPK thus provides a robust tool for probing PKL allosteric modulation and fills a key gap in target engagement technologies for PKL.

144

News (Medical) associated with Mitapivat

12 Feb 2026

·www.biospace.com

Agios Reports Fourth Quarter and Full Year 2025 Financial Results and Provides Business Update

PYRUKYND ® (mitapivat) worldwide net revenues of $20.0 million in fourth quarter and $54.0 million for full year AQVESME™ (mitapivat) for thalassemia now available in U.S. following FDA approval Company will have pre-sNDA meeting with FDA for mitapivat in sickle cell disease in first quarter of 2026 Phase 2 tebapivat trial in sickle cell disease fully enrolled; topline results expected in second half of 2026 $1.2 billion dollars in cash, cash equivalents, and marketable securities as of December 31, 2025 CAMBRIDGE, Mass., Feb. 12, 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 fourth quarter and year ended December 31, 2025. “2025 was another year of continued execution across our portfolio, highlighted by the historic U.S. approval of AQVESME – the only medicine approved to treat anemia in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The U.S. launch is off to a strong start, with AQVESME now available and earning an enthusiastic response from the thalassemia community,” said Brian Goff, Chief Executive Officer, Agios. “In 2026, we are focused on driving a high-impact U.S. launch of AQVESME, expanding our PK activation franchise into additional high-value indications such as sickle cell disease and lower-risk myelodysplastic syndromes, and advancing our promising early-stage pipeline to further diversify across hematologic and other rare diseases. With disciplined capital allocation and strong operational execution, we are very well positioned at this critical inflection point to deliver a transformative medicine for the thalassemia community and advance our clinical programs as we work toward our goal of becoming a sustainable rare disease company.” Fourth Quarter 2025 and Recent Corporate Highlights Mitapivat Commercial Performance and Update PYRUKYND ® (mitapivat 5 mg, 20 mg, 50 mg) $16.0 million in U.S. net revenue in the fourth quarter of 2025, driven by continued commercial focus in pyruvate kinase (PK) deficiency ahead of the U.S. commercial launch of AQVESME™ (mitapivat) in thalassemia, an additional ordering week in the fourth quarter, and favorable gross-to-net adjustments. This represents an increase of 49 percent from $10.7 million in the fourth quarter of 2024 and a 24 percent increase from $12.9 million in the third quarter of 2025. $4.0 million in ex-U.S. net revenue in the fourth quarter of 2025, driven by inventory stocking ahead of demand pull-through in Europe for PK deficiency, as patients transition onto commercial supply. AQVESME™ (mitapivat 100 mg) In December 2025, the U.S. Food and Drug Administration (FDA) approved AQVESME as the only medicine for the treatment of anemia in adults with alpha- or beta-thalassemia, regardless of transfusion burden. AQVESME is now available in the U.S. following the implementation of its Risk Evaluation and Mitigation Strategy (REMS) program in late January 2026. R&D Highlights Mitapivat Sickle Cell Disease – Topline results from the RISE UP Phase 3 trial of mitapivat in sickle cell disease were reported in November 2025. Agios will have a pre-supplemental New Drug Application (sNDA) meeting with the FDA in the first quarter of 2026 and intends to submit a U.S. marketing application for mitapivat in sickle cell disease following that engagement. The company will provide an update on its regulatory filing strategy following receipt of the meeting minutes. Tebapivat Sickle Cell Disease – Agios has completed enrollment for the Phase 2 trial of tebapivat in sickle cell disease, and expects to report topline results in the second half of 2026. This double-blind, randomized, placebo-controlled trial is evaluating three tebapivat doses (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. Fourth Quarter 2025 Financial Results For the quarter ended December 31, 2025, net loss was $108.0 million dollars, compared to net loss of $96.5 million dollars for the quarter ended December 31, 2024. Net product revenue from U.S. sales of PYRUKYND for the fourth quarter of 2025 was $16.0 million, compared to $10.7 million for the fourth quarter of 2024. Net product revenue from ex-U.S. sales of PYRUKYND for the fourth quarter of 2025 was $4.0 million. Cost of sales for the fourth quarter of 2025 was $1.9 million. Research and Development (R&D) Expenses were $88.1 million for the fourth quarter of 2025, compared to $82.8 million for the fourth quarter of 2024, associated with the advancement of the company’s early-stage clinical programs. Selling, General and Administrative (SG&A) Expenses were $51.6 million for the fourth quarter of 2025, which were flat compared to $51.7 million for the fourth quarter of 2024. Cash, cash equivalents and marketable securities were $1.2 billion as of December 31, 2025, compared to $1.5 billion as of December 31, 2024. 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. Conference Call Information Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s fourth quarter and full year 2025 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 . 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) December 31, 2025 December 31, 2024 Cash, cash equivalents, and marketable securities $ 1,164,438 $ 1,532,031 Accounts receivable, net 10,577 4,109 Inventory 32,920 27,616 Total assets 1,297,225 1,663,199 Stockholders' equity 1,193,114 1,540,956 Consolidated Statements of Operations Data (in thousands, except share and per share data) (Unaudited) Years Ended Dec 31, 2025 2024 2023 Revenues: Product revenue, net $ 54,028 $ 36,498 $ 26,823 Total revenue 54,028 36,498 26,823 Operating expenses: Cost of sales $ 6,345 $ 4,165 $ 2,881 Research and development 339,535 301,286 295,526 Selling, general and administrative 180,280 156,784 119,903 Total operating expenses 526,160 462,235 418,310 Loss from operations (472,132 ) (425,737 ) (391,487 ) Gain on sale of contingent payments — 889,136 — Milestone payment from gain on sale of oncology business — 200,000 — Interest income, net 56,379 48,083 33,344 Other income, net 1,956 6,487 6,055 Net (loss) income before taxes (413,797 ) 717,969 (352,088 ) Income tax (benefit) expense (1,016 ) 44,244 — Net (loss) income $ (412,781 ) $ 673,725 $ (352,088 ) Net (loss) income per share - basic $ (7.12 ) $ 11.86 $ (6.33 ) Net (loss) income per share - diluted $ (7.12 ) $ 11.64 $ (6.33 ) Weighted-average number of common shares used in computing net (loss) income per share – basic 57,972,004 56,807,415 55,651,487 Weighted-average number of common shares used in computing net (loss) income per share – diluted 57,972,004 57,889,255 55,651,487 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 3Financial Statement

29 Jan 2026

·endpoints.news

Summit says FDA will decide to approve its cancer drug by November

The leader in the next frontier of immuno-oncology officially has a date with the FDA. Summit Therapeutics announced Thursday morning that US regulators have accepted its submission package for the experimental drug ivonescimab, a bispecific antibody targeting PD-1 and VEGF, to treat a specific form of lung cancer. FDA is expected to make a decision on whether to approve the drug by Nov. 14. Thursday’s announcement marks the first FDA review for a new class of drugs that is expected to advance I-O efforts just as the best-selling PD-(L)1 inhibitors approach their patent cliffs. It also comes after a bumpy 2025 for Summit and as a field of significant competitors emerged, including Bristol Myers Squibb, Pfizer, Merck and AbbVie. Ivonescimab’s review will likely be closely scrutinized as the broader pharma industry aims to see how receptive the FDA is to these bispecifics. Notably, regulators already granted Summit permission for an unusual trial design: The study was originally conducted only in China, but FDA allowed Summit to enroll Western patients several months after all Chinese participants had started in the study. The “sequential nature” of the trial caused a large divergence in follow-up times between patients in and outside China, execs have previously said. Co-CEO Bob Duggan has also emphasized that the permission for sequential enrollment was unexpected. Despite this scrutiny, the market for this specific lung cancer population — second-line treatment for patients with EGFR-mutated non-squamous non-small cell lung cancer — is not expected to be large. Analysts have estimated peak sales at about $500 million per year, though there is greater potential in first-line lung cancer and other types of tumors . In a press release, Summit said it expects the review to proceed normally, with all aspects being completed “subject to major deficiencies not being identified.” That includes ivonescimab’s potential label, according to Summit. Labeling discussions caused the FDA to miss another company’s deadline last month, when Agios said regulators wouldn’t meet a Dec. 7 PDUFA date for its blood disorder drug Pyrukynd. Agios’ review was previously delayed due to safety concerns. Another FDA review last June for KalVista was delayed due to agency resource constraints and, as Endpoints News later reported , an attempt by Commissioner Marty Makary to reject the drug. KalVista’s drug, called Ekterly, was eventually approved in July.

Drug ApprovalAccelerated ApprovalPriority Review

14 Jan 2026

·www.fiercepharma.com

JPM26, Day 3: AbbVie touts 'clear line of sight' to continued growth into 2030s

With news continuing to pour in from San Francisco, be sure to check back throughout this week for the latest. The first two days of the J.P. Morgan Healthcare Conference are in the books, and attendees are now pushing through to finish strong. Today, UCB, AbbVie and Legend Biotech are among the companies presenting at the conference. And like the past two days, Fierce reporters and editors are busy on the ground meeting with industry leaders to bring you all the news from the event.For updates from prior days, here are our trackers from day 1 and day 2.And for the biotech side of the business, check out Fierce Biotech's day 1 and day 2 JPM trackers. UPDATE: 2:29 p.m. ETAs Legend Biotech’s J&J-partnered CAR-T cell therapy Carvykti continues its ascent in multiple myeloma, the company is aiming to reach a key financial milestone in 2026, CEO Ying Huang, Ph.D., said at the JPM conference Wednesday.Namely, this is the year that Legend expects to achieve profitability, Huang said, noting that the company already hit positive operating profit for Carvykti in 2025’s third quarter.“Few companies in our sector combine this commercial scale and also next-generation pipeline optionality that Legend has,” he said. “This year, we expect to become a profitable, fully scaled CAR-T leader.”Taking a closer look at the numbers, Carvykti—which has now reached more than 10,000 patients—ginned up roughly $1.7 billion in sales for the 12 months that ended with last year’s third quarter, Legend noted in a conference presentation. Remarking on that performance, Huang noted that Carvykti has enjoyed the “fastest launch among CAR-T therapies to date,” reiterating Legend and J&J’s annual peak sales estimate for the drug of more than $5 billion.As with other CAR-Ts, manufacturing is a crucial component of—and potential limiting factor in—access to Legend’s one-time therapy. On that front, Legend noted that “all” of the facilities in its manufacturing network are now online, covering locations in Belgium and New Jersey. The company currently boasts a median turnaround time of fewer than 30 days to produce the product and get it back to patients, whom it treats at 279 treatment sites globally, 141 of which have been activated in the U.S.As of 2026, Legend says it boasts an annualized capacity for Carvkyti of 10,000 doses. Not content to rest on its laurels, however, Legend aims to boost that yearly capacity to 20,000 doses “in the next three years,” Huang said. UPDATE: 2:20 p.m. ET AbbVie is looking at a “clear line of sight” to continue its growth journey for its key products well into the 2030s, chief financial officer Scott Reents said during a fireside chat Wednesday at the J.P. Morgan Healthcare Conference. The exec pointed to “very strong momentum of the business,” resulting in 8% revenue growth during 2025, with sales in its growth platform specifically climbing 19% versus the prior year.AbbVie’s performance leans heavily on immunology blockbusters Skyrizi and Rinvoq, two “exceptional products” that have still have room to capture additional share in the immunology market over the next decade, chief commercial officer Jeffrey Stewart added. Part of the company's confidence comes from the ten head-to-head trials it's planning against “all major mechanisms” in “most of the major indications,” in theory giving the drugs a chance to show off their strengths, the exec said.At the same time, specific immunology markets such as psoriatic arthritis and inflammatory bowel disease (IBD) are “great markets” that “cascade on top of each other” and expand each year, Stewart added.Much like how AbbVie built up its Skyrizi and Rinvoq as successors to its off-patent Humira, Johnson & Johnson has done the same with Tremfya as biosimilars challenge the New Jersey pharma's Stelara. AbbVie considers Tremfya’s market performance “in line with what we thought,” Stewart said, and hasn’t seen any degradation of its “strong” new patient starts for Skyrizi as a result of the competition.Skyrizi’s largest indication is in psoriasis, where it holds over 60% of the market among first-line patients, according to AbbVie. This is an area in which Tremfya has already been cranking up the competition for several years now.“Tremfya is a good competitor. It’s a strong drug,” Stewart said. But the exec is clear that “it’s not a zero-sum game” and that it's not exactly as simple as “one over the other.”Combination therapies will be a key aspect to Skyrizi and Rinvoq’s growth journeys, with CEO Robert Michael citing a combo as the way to clear the efficacy bar that the drugs on their own have already established.UPDATE: 1:50 p.m. ET Agios is projecting (PDF) $1 billion-plus in peak sales potential for its two mitapivat treatments. Aqvesme, an oral medicine which was approved by the FDA three weeks ago, is for the treatment of anemia in adults with alpha- or beta-thalassemia, while Pyrukynd is on the market for the rare, genetic blood disorder pyruvate kinase deficiency (PKD).The separate brand names for what is essentially the same agent is to distinguish Aqvesme and its Risk Evaluation and Mitigation Strategy (REMS) program for liver monitoring in thalassemia from Pyrukynd, which has no REMS requirement in PKD.“This is in so many ways an historical moment for the thalassemia community that has waited for this for decades,” Agios CEO Brian Goff said Wednesday at the J.P. Morgan Healthcare Conference. “We’re really proud of this profile. It puts us in a great position to make Aqvesme the standard of care in thalassemia.”UPDATE: 1:45 p.m. ETWuXi Biologics has been busy in recent months, and heading into 2026 the contract research, development, and manufacturing organization (CRDMO) has no plans to slow down, CEO Chris Chen, Ph.D., said during his company’s presentation at the J.P. Morgan Healthcare Conference Wednesday.All told, WuXi Biologics lassoed 209 integrated projects across its CRDMO business in 2025, compared to 150 in 2024, Chen said, noting that roughly two-thirds of those contracts cover bi- or multispecifics or antibody-drug conjugates (ADCs). Half of those projects are also based out of the U.S., Chen added.All told, WuXi Biologics is now engaged with 74 late-stage and 25 commercial programs, according to a company presentation (PDF) prepared for the event.Chen noted that WuXi Biologics’ manufacturing revenue might look slightly lower than its peers because many of the commercial projects it’s partnered on are just now launching, stressing that sales streams from those collaborations will continue to increase. “In the meantime, we keep adding more programs into the funnel,” he said, referring to WuXi Bio’s continued uptake of new projects.The CEO also took time to reflect on WuXi Bio’s growing manufacturing footprint. In the U.S. where, the company boasts facilities for drug substance, drug product and process development, WuXi Biologics and its conjugation spinoff WuXi XDC have raised their total investment plan to about $1 billion, according to the CEO.The company is also setting up an end-to-end biologics hub in Singapore that's expected to start generating revenue this year, Chen said.Elsewhere, WuXi Bio is putting down roots in a sixth country with the memorandum of understanding it signed with the Qatar Free Zones Authority back in December, Chen pointed out. UPDATE: 10:20 a.m. ETAstraZeneca may have had its share of doubters when the company in 2024 shared the goal to generate $80 billion in annual sales by 2030. But as the company's pipeline and launch cadence comes into clearer focus, it's winning over nonbelievers.At JPM on Tuesday, AZ CFO Aradhana Sarin stressed that the goal is “very much within reach,” particularly if AZ is able to continue its winning streak in the clinic. StoryUPDATE: 10:15 a.m. ETDuring his debut appearance at JPM, Novo Nordisk's new CEO Maziar Mike Doustdar didn’t hesitate to acknowledge the company’s “difficult 2025.” The plan for 2026, he said, is to focus on commercial execution in the "consumer"-style obesity market, advance Novo's pipeline in select disease areas and balance financial discipline while “investing where the investments are needed.”A big focus for Novo this year will be direct-to-patient sales programs in the United States as the company continues to face fierce obesity competition from Eli Lilly—and as new players make moves to enter the space. Story

100 Deals associated with Mitapivat

External Link

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB16236

R&D Status

Approved

10 top approved records.

to view more data

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
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.

to view more data

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
Hemoglobin SC Disease	Phase 3	United States	Agios Pharmaceuticals, Inc.	11 Feb 2022
Hemoglobin SC Disease	Phase 3	Belgium	Agios Pharmaceuticals, Inc.	11 Feb 2022
Hemoglobin SC Disease	Phase 3	Brazil	Agios Pharmaceuticals, Inc.	11 Feb 2022
Hemoglobin SC Disease	Phase 3	Canada	Agios Pharmaceuticals, Inc.	11 Feb 2022
Hemoglobin SC Disease	Phase 3	France	Agios Pharmaceuticals, Inc.	11 Feb 2022
Hemoglobin SC Disease	Phase 3	Germany	Agios Pharmaceuticals, Inc.	11 Feb 2022
Hemoglobin SC Disease	Phase 3	Israel	Agios Pharmaceuticals, Inc.	11 Feb 2022
Hemoglobin SC Disease	Phase 3	Italy	Agios Pharmaceuticals, Inc.	11 Feb 2022

Clinical Result

Indication

Phase

Evaluation

View All Results

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	llxwjkdbox(ntlcgeqrbe) = vaxqgcghbx spiqyolqlp (aoxzuwjaei, 16.892) View more	Positive	06 Dec 2025
NCT05031780 (RISE UP, Company_Website) Manual	Phase 3	Anemia, Sickle Cell	-	Mitapivat	xndqotktbh(ygardxfght) = gglijixvdj htlafkaiyj (nhgdgrqwrg ) Met View more	Positive	19 Nov 2025
				Placebo	xndqotktbh(ygardxfght) = oqjkdiuqru htlafkaiyj (nhgdgrqwrg ) Met View more
NCT05935202 (SATISFY, EHA2025)	Phase 2	Anemia, Dyserythropoietic, Congenital	24	Mitapivat 100mg twice daily	kkzuyxnhbb(qrndccjoio) = TEAEs were generally mild and consistent with mitapivat’s known safety profile, most frequently headache (50%), upper respiratory infection (46%), and insomnia (38%) weyqtuozkz (xapgozjspb ) View more	Positive	13 Jun 2025
NCT04770779 (ENERGIZE-T, CTgov)	Phase 3	Transfusion-dependent Thalassemia \| Transfusion-dependent Beta Thalassemia	258	Mitapivat (Mitapivat)	yjujhtsrdx = pdkxqmhfdl bnbczrmurt (hyjewrgahy, uurccefrob - yypoxmmdaq) View more	-	25 May 2025
				Placebo Matching Mitapivat+Mitapivat (Placebo)	yjujhtsrdx = uufezlqlss bnbczrmurt (hyjewrgahy, yvwtigfxlu - ispedocniv) View more
NCT05144256 (ACTIVATE-KidsT, EHA2025)	Phase 3	Pyruvate Kinase Deficiency	49	Mitapivat	xdekpxnifw(nkwlbgzwep) = qglzveiwxt zwimrjmkih (opktzzndub ) View more	Positive	14 May 2025
				Placebo	xdekpxnifw(nkwlbgzwep) = kwtbenmjfd zwimrjmkih (opktzzndub )
ESTIMATE study (EHA2025)	Phase 2	Anemia, Sickle Cell HbSS \| HbS/β0 \| HbS/β+	10	Mitapivat 100mg BID	jghqmwesbz(dvfiffzlkf) = xzyhsdlynl vtmwxtmjpk (solhosscud ) View more	Positive	14 May 2025
				Placebo	oqidbakugi(xqbmegzvjc) = eguhdpcbok gflpifpvcg (kffibagchy )
NCT05175105 (ACTIVATE-Kids, Company_Website) Manual	Phase 3	Pyruvate Kinase Deficiency	30	Mitapivat twice-daily	lkuyetwfwj(bgdzttjita) = qgtbfjqpao zyckepqszr (avtytzyxqr ) Met	Positive	13 Feb 2025
				Matched Placebo	lkuyetwfwj(bgdzttjita) = kwiocbdeeg zyckepqszr (avtytzyxqr ) Met
NCT04770753 (ENERGIZE, CTgov)	Phase 3	Beta-Thalassemia \| Non-transfusion dependent thalassaemia \| Alpha-Thalassemia	194	Mitapivat (Mitapivat)	sxhlskqiws = wlvmnxxykg oypyrudhav (ghrbsakizp, txbgjlleow - vnfqvtqulb) View more	-	24 Jan 2025
				Placebo Matching Mitapivat+Mitapivat (Placebo)	sxhlskqiws = dfsycintiu oypyrudhav (ghrbsakizp, kurrhtedwt - zztipeivtw) View more
NCT05935202 (Satisfy, ASH2024) Manual	Phase 2	Anemia, Dyserythropoietic, Congenital	24	Mitapivat 50 mg BID	vftdeymgwm(wkdndbgtrh) = One non-treatment related SAE (grade 3) occurred vtalwvyumn (kkcaeotuzz ) View more	Positive	09 Dec 2024
NCT04770779 (ENERGIZE-T, ASH2024) Manual	Phase 3	Transfusion-dependent Thalassemia	258	Mitapivat 100 mg	nnwjrynqsn(tielfxalec) = kyoqchnbvx iwgbhbcddi (wrjznwrtmj ) View more	Positive	08 Dec 2024
				Placebo	nnwjrynqsn(tielfxalec) = eerhyrwxft iwgbhbcddi (wrjznwrtmj ) View more

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