Drugs / Mitapivat
Mitapivat
also known as
AG-348 · AG-348 HEMISULFATE SESQUIHYDRATE · AG-348 sulfate hydrate · AGI-1480 · AGX-0841 · Mitapivat hemisulfate sesquihydrate · mitapivat sulfate · Pkr-in-1 · +1 more
Small molecule targets PKLR via activation
Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed beta-thalassemia and related diseases 1 milestone | |||||
| Filed | EU (EMA) | PYRUKYND | beta-thalassemia and related diseases | — | “A marketing application for mitapivat in thalassemia is currently under review by the European Commission .” agios.com ↗ “A marketing application for PYRUKYND in thalassemia is currently under review by the European Commission .” agios.com ↗ |
| Filed pyruvate kinase deficiency of red cells 2 milestones | |||||
| Filed | EU (EMA) | — | pyruvate kinase deficiency of red cells | 2021-06-28 | “submitted a Marketing Authorisation Application (MAA) for mitapivat to the European Medicines Agency (EMA)” agios.com ↗ |
| Filed | US (FDA) | — | pyruvate kinase deficiency of red cells | 2021-06-21 | “submitted a New Drug Application (NDA) for mitapivat to the U.S. Food and Drug Administration (FDA)” agios.com ↗ |
| Filed sickle cell S-Lepore disease 1 milestone | |||||
| Filed | Other | — | sickle cell S-Lepore disease | 2026-07-30 | “Agios also filed for regulatory approval of mitapivat for sickle cell disease in Saudi Arabia .” agios.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| Label expansion | US (FDA) | PYRUKYND | — | 2025-12-23 | fda.gov ↗ |
| Approved | EU (EMA) | Pyrukynd | — | 2022-11-09 | europa.eu ↗ |
| Approved | US (FDA) | PYRUKYND | — | 2022-02-17 | fda.gov ↗ |
Trials 29
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT04000165 | Jul 2019 → Jun 2021 | sickle cell disease | National Institutes of Health Clinical Center (CC) | Completed | No outcome recorded |
| Phase 110 trials | ||||||
| Phase 1 | NCT06648824 | Oct → Dec 2024 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT05610657 | Jan → Jul 2023 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT04696393 | Jan → Mar 2021 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT04565678 | Sep → Dec 2020 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT04472832 | Jun → Nov 2020 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT03991312 | Jun → Aug 2019 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT03703505 | May → Jun 2018 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT03250598 | Aug → Oct 2017 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT02149966 | May → Nov 2014 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT02108106 | Mar → Aug 2014 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT04610866 | Dec 2020 → Feb 2028 expected | hemolytic anemia, sickle cell disease | National Heart, Lung, and Blood Institute (NHLBI) | Active not recruiting | No outcome recorded |
| Phase 25 trials · 1 met primary | ||||||
| Phase 2 | NCT06286046 | Mar 2026 → May 2027 | sickle cell disease | Agios Pharmaceuticals, Inc. | Withdrawn | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT07055243 | Jun 2025 → Jun 2026 overdue | anemia | University Health Network, Toronto | Recruiting | No outcome recorded |
| Phase 2 | NCT05935202 | Dec 2023 → Jul 2025 overdue | erythrocyte disorder | EuroBloodNet Association | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT03692052 | Mar 2019 → Aug 2020 overdue | thalassemia | Agios Pharmaceuticals, Inc. | Active not recruiting | Met primary |
| Phase 2 | NCT02476916 | Jun 2015 → May 2017 | pyruvate kinase deficiency of red cells | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT05031780 | Feb 2022 → Oct 2025 overdue | sickle cell disease | Agios Pharmaceuticals, Inc. | Active not recruiting | No outcome recorded |
| Phase 310 trials · 5 met primary · 1 missed | ||||||
| Phase 3 | NCT07517133 | Sep 2026 → Mar 2029 expected | alpha thalassemia spectrum, beta thalassemia | Agios Pharmaceuticals, Inc. | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT07506863 | Sep 2026 → Jun 2029 expected | alpha thalassemia spectrum, beta thalassemia | Agios Pharmaceuticals, Inc. | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT07656415 | Aug 2026 → Aug 2029 expected | sickle cell disease | Agios Pharmaceuticals, Inc. | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT05144256 | Jun 2022 → May 2024 overdue | pyruvate kinase deficiency of red cells | Agios Pharmaceuticals, Inc. | Active not recruiting | Missed primary |
| Phase 3 | NCT05175105 | Jun 2022 → Dec 2024 overdue | pyruvate kinase deficiency of red cells | Agios Pharmaceuticals, Inc. | Active not recruiting | Met primary |
| Phase 3 | NCT04770753 | Dec 2021 → Nov 2023 overdue | alpha thalassemia spectrum, beta-thalassemia intermedia | Agios Pharmaceuticals, Inc. | Active not recruiting | Met primary |
| Phase 3 | NCT04770779 | Nov 2021 → Apr 2024 overdue | alpha thalassemia spectrum, beta thalassemia | Agios Pharmaceuticals, Inc. | Active not recruiting | Met primary |
| Phase 3 | NCT03853798 | Mar 2019 → Jul 2024 | pyruvate kinase deficiency of red cells | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 3 | NCT03548220 | Aug 2018 → Oct 2020 | pyruvate kinase deficiency of red cells | Agios Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 3 | NCT03559699 | Jun 2018 → Nov 2020 | pyruvate kinase deficiency of red cells | Agios Pharmaceuticals, Inc. | Completed | Met primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT05777993 | Jul 2023 → Aug 2029 expected | pyruvate kinase deficiency of red cells | Agios Pharmaceuticals, Inc. | Enrolling by invitation | No outcome recorded |
| Comparator or background therapy1 trial | ||||||
| Phase 1 | NCT03397329 Comparator | Oct → Nov 2017 | — | Agios Pharmaceuticals, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 67
| Date | Issuer | Release |
|---|---|---|
| 2026-07-07 | Agios Pharmaceuticals, Inc. | Regulatory U.S. FDA Grants Priority Review to Agios’ sNDA for Mitapivat in Sickle Cell Disease agios.com ↗ |
| 2026-06-13 | Agios Pharmaceuticals, Inc. | Results Agios Showcases RISE UP Phase 3 Results at EHA 2026 Plenary Session Reinforcing Strong Anti-Hemolytic Profile of Mitapivat in Sickle Cell Disease agios.com ↗ |
| 2026-05-22 | Agios Pharmaceuticals, Inc. | Regulatory Agios’ PYRUKYND® (mitapivat) Approved in the European Union for Adults with Thalassemia agios.com ↗
With this decision, PYRUKYND becomes the only medicine approved in all European Union (EU) member states for this broad patient population. |
| 2026-05-12 | Agios Pharmaceuticals, Inc. | Regulatory Agios Submits sNDA to FDA for U.S. Accelerated Approval of Mitapivat in Sickle Cell Disease agios.com ↗
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced the submission of its supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for the U.S. accelerated approval of mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease. |
| 2026-03-02 | Agios Pharmaceuticals, Inc. | Regulatory Agios’ PYRUKYND® (mitapivat) Approved for Adults with Thalassemia in the United Arab Emirates agios.com ↗
With this approval, PYRUKYND becomes the only medicine approved in the UAE for this patient population. |
| 2025-12-23 | Agios Pharmaceuticals, Inc. | Regulatory U.S. FDA Approves Agios’ AQVESME™ (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia agios.com ↗
With this approved indication, AQVESME becomes the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. |
| 2025-11-19 | Agios Pharmaceuticals, Inc. | Results Agios Announces Topline Results from RISE UP Phase 3 Trial of Mitapivat in Sickle Cell Disease agios.com ↗
Trial met primary endpoint of hemoglobin response and key secondary endpoints of change from baseline in hemoglobin concentration and indirect bilirubin |
| 2025-11-03 | Agios Pharmaceuticals, Inc. | Results Agios to Present New Mitapivat Data in Rare Blood Disorders at 67th ASH Annual Meeting and Exposition agios.com ↗ |
| 2025-10-17 | Agios Pharmaceuticals, Inc. | Regulatory Agios’ PYRUKYND® (mitapivat) Receives Positive CHMP Opinion for Adults with Thalassemia agios.com ↗ |
| 2025-09-04 | Agios Pharmaceuticals, Inc. | Regulatory Agios Provides Update on U.S. PDUFA Goal Date for PYRUKYND® (mitapivat) in Thalassemia agios.com ↗ |
| 2025-08-04 | Agios Pharmaceuticals, Inc. | Regulatory Agios’ PYRUKYND® (mitapivat) Approved for Adults with Thalassemia in Saudi Arabia agios.com ↗
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the Saudi Food and Drug Authority (SFDA) has approved PYRUKYND ® (mitapivat) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. |
| 2025-05-14 | Agios Pharmaceuticals, Inc. | Results Agios to Highlight Pyruvate Kinase Activation Portfolio with New Data in Rare Blood Disorders at 30th EHA Congress agios.com ↗
The study showed a clinically meaningful reduction in transfusion burden with mitapivat, with a higher proportion of patients achieving the primary endpoint of transfusion reduction response compared to placebo, though the prespecified statistical criterion was not met. |
| 2025-02-13 | Agios Pharmaceuticals, Inc. | Results Agios’ Phase 3 ACTIVATE-Kids Study of Mitapivat in Children with Pyruvate Kinase (PK) Deficiency Not Regularly Transfused Met Primary Endpoint agios.com ↗ |
| 2025-01-13 | Agios Pharmaceuticals, Inc. | Regulatory Agios Announces Key 2025 Milestones for Innovative Rare Disease Portfolio agios.com ↗
FDA Accepted Agios’ Supplemental New Drug Application for PYRUKYND ® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia; PDUFA Goal Date is September 7, 2025 |
| 2025-01-08 | Agios Pharmaceuticals, Inc. | Regulatory FDA Accepts Agios’ Supplemental New Drug Application for PYRUKYND® (mitapivat) in Adult Patients with Non-Transfusion-Dependent and Transfusion-Dependent Alpha- or Beta-Thalassemia agios.com ↗ |
| 2024-12-18 | Agios Pharmaceuticals, Inc. | Regulatory European Commission Adopts Positive Decision for Orphan Medicinal Product Designation of Agios’ Mitapivat in Sickle Cell Disease agios.com ↗ |
| 2024-12-08 | Agios Pharmaceuticals, Inc. | Results Agios Presents Positive Results from Phase 3 ENERGIZE-T Study of Mitapivat at ASH 2024 and Provides Regulatory Update on Mitapivat agios.com ↗ |
| 2024-08-01 | Agios Pharmaceuticals, Inc. | Results Agios Reports Business Highlights and Second Quarter 2024 Financial Results agios.com ↗
This morning, we were pleased to report topline data in the Phase 3 ACTIVATE-KidsT study of mitapivat, which is the first study to report safety and efficacy data in children with PK deficiency. |
| 2024-08-01 | Agios Pharmaceuticals, Inc. | Results Agios Announces Results from Phase 3 ACTIVATE-KidsT Study of Mitapivat in Children with PK Deficiency Who Are Regularly Transfused agios.com ↗ |
| 2024-06-15 | Agios Pharmaceuticals, Inc. | Results Agios Presents Positive Results from Phase 3 ENERGIZE Study of Mitapivat in Non-Transfusion-Dependent Thalassemia in Plenary Session at the European Hematology Association 2024 Hybrid Congress agios.com ↗
The ENERGIZE study achieved its primary endpoint, with mitapivat demonstrating a statistically significant increase in hemoglobin response rate compared to placebo. |
| 2024-06-03 | Agios Pharmaceuticals, Inc. | Results Agios Announces Phase 3 ENERGIZE-T Study of Mitapivat Met Primary Endpoint and All Key Secondary Endpoints in Adults with Transfusion-Dependent Alpha- or Beta-Thalassemia agios.com ↗ |
| 2024-01-03 | Agios Pharmaceuticals, Inc. | Results Agios Announces Phase 3 ENERGIZE Study of Mitapivat Met Primary Endpoint and Both Key Secondary Endpoints in Adults with Non-Transfusion-Dependent Alpha- or Beta-Thalassemia agios.com ↗
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism and pioneering therapies for rare diseases, today announced that the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary endpoint of hemoglobin response. |
| 2023-12-09 | Agios Pharmaceuticals, Inc. | Results Agios Presents Positive Results from Phase 2 Portion of the RISE UP Pivotal Study in Sickle Cell Disease at 65th ASH Annual Meeting and Exposition agios.com ↗
During the Phase 2 double-blind, placebo-controlled study, treatment with mitapivat demonstrated statistically significant improvement in hemoglobin response across both mitapivat dose levels (50 mg and 100 mg BID), compared to placebo. |
| 2023-11-02 | Agios Pharmaceuticals, Inc. | Results Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Rare Blood Disorders at 65th ASH Annual Meeting and Exposition agios.com ↗
Both Mitapivat Dose Arms Achieved Statistically Significant Hemoglobin Response in Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease, Compared to the Placebo Arm; |
| 2023-08-03 | Agios Pharmaceuticals, Inc. | Results Agios Reports Business Highlights and Second Quarter 2023 Financial Results agios.com ↗ |
| 2023-06-26 | Agios Pharmaceuticals, Inc. | Results Agios Announces Positive Results from Phase 2 Portion of RISE UP Pivotal Study in Sickle Cell Disease with Both Mitapivat Dose Arms Achieving Statistically Significant Hemoglobin Response agios.com ↗
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that the Phase 2 portion of the global RISE UP study of mitapivat in sickle cell disease met its primary endpoint of hemoglobin response for patients in both the 50 mg and 100 mg twice daily (BID) mitapivat arms. |
| 2022-12-11 | Agios Pharmaceuticals, Inc. | Results Agios Presents Updated PYRUKYND® (mitapivat) Long-term Extension Data Demonstrating Sustained Clinical Benefits in Adults with Pyruvate Kinase (PK) Deficiency at 64th ASH Annual Meeting and Exposition agios.com ↗
Long-term extension data (abstract # 2328) show that previously reported effects of PYRUKYND ® on hemoglobin and transfusion burden were maintained over time. |
| 2022-12-10 | Agios Pharmaceuticals, Inc. | Results Agios Presents Updated PYRUKYND® (mitapivat) Data Highlighting Long-term Safety Profile and Durable Improvement in Hemoglobin and Markers of Hemolysis and Ineffective Erythropoiesis in Non-transfusion-dependent α- and β-Thalassemia at 64th ASH Annual Meet agios.com ↗
Consistent with previously reported data , durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients. |
| 2022-11-10 | Agios Pharmaceuticals, Inc. | Regulatory PYRUKYND® (mitapivat) Approved in the EU for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients agios.com ↗ |
| 2022-11-03 | Agios Pharmaceuticals, Inc. | Results Agios Pharmaceuticals to Present Broad Set of Clinical and Translational Data in Chronic Hemolytic Anemias at 64th ASH Annual Meeting and Exposition agios.com ↗
Long-term Data for PYRUKYND ® (mitapivat) in Adults with Pyruvate Kinase (PK) Deficiency and Adults with Thalassemia Demonstrate Sustained Improvement in Hemoglobin, Markers of Hemolysis and Other Disease Impacts |
| 2022-09-16 | Agios Pharmaceuticals, Inc. | Regulatory Agios Receives Positive CHMP Opinion for PYRUKYND® (mitapivat) for the Treatment of Pyruvate Kinase (PK) Deficiency in Adult Patients agios.com ↗ |
| 2022-08-18 | Agios Pharmaceuticals, Inc. | Results Agios Announces Publication of Phase 3 ACTIVATE-T Data in The Lancet Haematology Demonstrating Benefits of PYRUKYND® (mitapivat) for Adults with Pyruvate Kinase Deficiency agios.com ↗ |
| 2022-08-11 | Agios Pharmaceuticals, Inc. | Results Agios Announces Publication of Phase 2 Data in The Lancet Demonstrating Safety and Efficacy of PYRUKYND® (mitapivat) in Non-transfusion-dependent α- and β-Thalassemia agios.com ↗ |
| 2022-06-10 | Agios Pharmaceuticals, Inc. | Results Agios Presents New Clinical Data Supporting the Benefits of PYRUKYND® (mitapivat) Treatment in Adults with PK Deficiency at European Hematology Association Annual Congress agios.com ↗
Data demonstrate that treatment with PYRUKYND ® was associated with normalization of hemoglobin levels. |
| 2022-04-14 | Agios Pharmaceuticals, Inc. | Results Agios Announces Publication of Phase 3 ACTIVATE Study in New England Journal of Medicine Demonstrating Benefits of PYRUKYND® (mitapivat) for Adults with Pyruvate Kinase Deficiency agios.com ↗ |
| 2022-02-17 | Agios Pharmaceuticals, Inc. | Regulatory Agios Announces FDA Approval of PYRUKYND® (mitapivat) as First Disease-Modifying Therapy for Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency agios.com ↗ |
| 2021-12-13 | Agios Pharmaceuticals, Inc. | Results Agios Presents Mitapivat Long-term Extension Data Demonstrating Durability of Hemoglobin Response and Transfusion Burden Reduction in Adults with Pyruvate Kinase (PK) Deficiency at 63rd ASH Annual Meeting and Exposition agios.com ↗
Long-term extension data (abstract #848) demonstrate that previously reported effects of mitapivat on hemoglobin and transfusion burden were maintained over time. |
| 2021-12-13 | Agios Pharmaceuticals, Inc. | Results Agios Presents Mitapivat Data Highlighting Long-term Safety Profile and Durable Improvement in Hemoglobin and Markers of Hemolysis in Non-transfusion-dependent α- and β-Thalassemia at 63rd ASH Annual Meeting and Exposition agios.com ↗
Consistent with previously reported data , durable improvements in hemoglobin concentration and markers of hemolysis and ineffective erythropoiesis, were observed for up to 72 weeks of treatment in both α- and β-thalassemia patients. |
| 2021-12-13 | Agios Pharmaceuticals, Inc. | Results Agios Announces Data from Investigator-led Studies of Mitapivat in Adults with Sickle Cell Disease Demonstrating Improvements in Anemia, Hemolysis and Sickling Parameters at 63rd ASH Annual Meeting and Exposition agios.com ↗
The data demonstrate the potential of mitapivat to provide clinically meaningful outcomes for patients, including improvements in anemia, hemolysis and red blood cell sickling. |
| 2021-11-04 | Agios Pharmaceuticals, Inc. | Results Agios to Present Broad Set of Clinical and Translational Data in Chronic Hemolytic Anemias at 63rd ASH Annual Meeting and Exposition agios.com ↗
Extension Data for Mitapivat in PK Deficiency and Thalassemia Demonstrate Long-term Safety Profile and Durable Improvement in Hemoglobin and Markers of Hemolysis |
| 2021-08-17 | Agios Pharmaceuticals, Inc. | Regulatory Agios Announces FDA Acceptance and Priority Review of New Drug Application for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s New Drug Application (NDA) for mitapivat for the treatment of adults with pyruvate kinase (PK) deficiency. |
| 2021-06-28 | Agios Pharmaceuticals, Inc. | Regulatory Agios Submits Marketing Authorisation Application to European Medicines Agency for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency agios.com ↗
This submission follows the company’s recent New Drug Application (NDA) submission of mitapivat to the U.S. Food and Drug Administration (FDA) for treatment of adults with PK deficiency in the U.S. |
| 2021-06-21 | Agios Pharmaceuticals, Inc. | Regulatory Agios Submits New Drug Application to FDA for Mitapivat for Treatment of Adults with Pyruvate Kinase Deficiency agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism developing and delivering innovative treatments for genetically defined diseases, today announced that it has submitted a New Drug Application (NDA) for mitapivat to the U.S. Food and Drug Administration (FDA) for the treatment of adults with pyruvate kinase (PK) deficiency. |
| 2021-06-11 | Agios Pharmaceuticals, Inc. | Results Agios Presents Positive Results from Phase 2 Study of Mitapivat in Non-transfusion-dependent α- and β-Thalassemia at the European Hematology Association Virtual Congress agios.com ↗ |
| 2021-06-11 | Agios Pharmaceuticals, Inc. | Results Agios Announces Updated Data from ACTIVATE and ACTIVATE-T Phase 3 Studies of Mitapivat in Pyruvate Kinase (PK) Deficiency at the European Hematology Association Virtual Congress agios.com ↗
Consistent with previously announced topline data, the ACTIVATE and ACTIVATE-T studies met primary and secondary endpoints, including PRO outcomes that address symptom burden and quality-of-life impact of PK deficiency in adults. |
| 2021-01-26 | Agios Pharmaceuticals, Inc. | Results Agios Announces Phase 3 ACTIVATE-T Trial of Mitapivat Achieved Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and genetically defined diseases, today announced that the global, open-label Phase 3 ACTIVATE-T trial of mitapivat in regularly transfused adults with PK deficiency demonstrated a statistically significant and clinically meaningful reduction in transfusion burden. |
| 2020-12-07 | Agios Pharmaceuticals, Inc. | Results Agios Announces Updated Data from Phase 1 Study of Mitapivat, First-in-Class PKR Activator, in Sickle Cell Disease agios.com ↗
As of the October 6, 2020 data cut-off, six of 11 efficacy evaluable patients (55%) achieved a hemoglobin increase of ≥1.0 g/dL from baseline. |
| 2020-12-01 | Agios Pharmaceuticals, Inc. | Results Agios Announces the Phase 3 ACTIVATE Trial of Mitapivat Achieved Its Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the global Phase 3 ACTIVATE trial of mitapivat in adults with pyruvate kinase (PK) deficiency who do not receive regular transfusions met its primary endpoint. |
| 2020-11-12 | Agios Pharmaceuticals, Inc. | Regulatory Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Sickle Cell Disease agios.com ↗
CAMBRIDGE, Mass. , Nov. 12, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s first-in-class pyruvate kinase R (PKR) activator mitapivat for the treatment of patients with sickle cell disease. |
| 2020-06-12 | Agios Pharmaceuticals, Inc. | Results Agios First-in Class PKR Activator Mitapivat Demonstrates Sustained Hemoglobin Responses in Non-transfusion-dependent α- and β-Thalassemia in Phase 2 Study agios.com ↗ |
| 2020-06-12 | Agios Pharmaceuticals, Inc. | Results Agios Announces Clinical Proof-of-Concept Has Been Established in Phase 1 Study of Mitapivat, First-in Class PKR Activator, in Sickle Cell Disease agios.com ↗
today announced that clinical proof-of-concept has been established based on a preliminary analysis in the Phase 1 trial of mitapivat (AG-348) in patients with sickle cell disease. |
| 2020-06-08 | Agios Pharmaceuticals, Inc. | Regulatory Agios Announces FDA Orphan Drug Designation Granted to Mitapivat for Treatment of Thalassemia agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s first-in-class pyruvate kinase-R (PKR) activator mitapivat for the treatment of patients with thalassemia. |
| 2020-05-14 | Agios Pharmaceuticals, Inc. | Results Agios to Present Updated Clinical Proof-of-Concept Data from the Phase 2 Study of Mitapivat in Thalassemia in Oral Presentation at the European Hematology Association Annual Congress agios.com ↗
Proof of concept for the oral pyruvate kinase activator mitapivat in adults with non-transfusion-dependent thalassemia: Interim results from an ongoing, phase 2, open-label, multicenter study |
| 2020-03-30 | Agios Pharmaceuticals, Inc. | Regulatory Agios Receives Positive Opinion on Orphan Drug Designation from the European Medicines Agency for Mitapivat in Pyruvate Kinase Deficiency agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued a positive opinion on the company’s application for orphan drug designation for its investigational medicine mitapivat as a potential treatment for pyruvate kinase (PK) deficiency, a rare, debilitating, hemolytic anemia. |
| 2019-12-09 | Agios Pharmaceuticals, Inc. | Results Agios Presents Updated Data for Mitapivat from Extension Phase of the DRIVE PK Study in Patients with Pyruvate Kinase Deficiency agios.com ↗
Agios Pharmaceuticals, Inc. (NASDAQ: AGIO), a leader in the field of cellular metabolism to treat cancer and rare genetic diseases, today reported new data from the extension phase of the DRIVE PK Phase 2 study of mitapivat (AG-348) in adults with pyruvate kinase (PK) deficiency at the 2019 American Society of Hematology (ASH) Annual Meeting. |
| 2019-12-08 | Agios Pharmaceuticals, Inc. | Results Agios Establishes Proof-of-Concept for Mitapivat in Non-transfusion-dependent Thalassemia Based on Preliminary Phase 2 Results agios.com ↗ |
| 2019-11-06 | Agios Pharmaceuticals, Inc. | Results Agios to Present Broad Set of Clinical and Translational Data for Oncology and Rare Genetic Disease Programs at the 2019 ASH Annual Meeting agios.com ↗
Updated DRIVE PK Data for Mitapivat in PK Deficiency Patients Show Robust, Sustained Hemoglobin Responses in Extension Phase for More Than Three Years; New Data Characterize Comorbidities and Complications Associated with PK Deficiency |
| 2019-09-04 | Agios Pharmaceuticals, Inc. | Results Agios Announces Publication of Data for Mitapivat from Core and Extension Phases of the DRIVE PK Study in Patients with Pyruvate Kinase Deficiency in the New England Journal of Medicine agios.com ↗
Data from the study demonstrated rapid, clinically significant increases in hemoglobin in 50 percent of patients, and for patients in the extension phase, the response was sustained for up to 35 months, |
| 2017-12-10 | Agios Pharmaceuticals, Inc. | Results Agios Presents Updated Data from DRIVE PK Study Demonstrating AG-348 is Well-Tolerated and Results in Clinically Relevant, Rapid and Sustained Hemoglobin Increases in Patients with Pyruvate Kinase Deficiency agios.com ↗
Agios Pharmaceuticals, Inc. (Nasdaq: AGIO ) presented updated data today from its wholly owned pyruvate kinase-R (PKR) activator, AG-348, demonstrating its potential as the first disease-modifying treatment for patients with pyruvate kinase (PK) deficiency at the 2017 American Society of Hematology (ASH) Annual Meeting and Exposition. |
| 2017-06-24 | Agios Pharmaceuticals, Inc. | Results Agios Announces Updated Data from Fully Enrolled DRIVE PK Study Demonstrating AG-348’s Potential as the First Disease-modifying Treatment for Patients with Pyruvate Kinase Deficiency agios.com ↗
Agios Pharmaceuticals, Inc. (Nasdaq:AGIO) presented updated data from its wholly owned pyruvate kinase-R (PKR) activator demonstrating the potential for the first disease-modifying treatment for patients with pyruvate kinase (PK) deficiency at the 22nd Congress of the European Hematology Association (EHA). |
| 2016-12-04 | Agios Pharmaceuticals, Inc. | Results Agios Announces New Data from AG-348 and AG-519 Demonstrating Potential for First Disease-modifying Treatment for Patients with PK Deficiency agios.com ↗
AG-348 Is Well-Tolerated and Demonstrates Clinically Relevant, Rapid and Sustained Hemoglobin Increases in 15 of 26 Patients with at Least One Missense Mutation and 15 out of 32 Patients Overall |
| 2016-06-11 | Agios Pharmaceuticals, Inc. | Results AG-348 Achieves Proof-of-Concept in Ongoing Phase 2 DRIVE-PK Study and Demonstrates Rapid and Sustained Hemoglobin Increases in Adults with Pyruvate Kinase Deficiency agios.com ↗
Agios Pharmaceuticals, Inc. (Nasdaq:AGIO) today announced initial data demonstrating that AG-348 achieved proof-of-concept in an ongoing Phase 2 study (DRIVE-PK) of patients with pyruvate kinase (PK) deficiency, a rare, potentially debilitating, congenital anemia. |
| 2015-06-12 | Agios Pharmaceuticals, Inc. | Results Agios Reports New, Final Data from Phase 1 Multiple Ascending Dose (MAD) Study in Healthy Volunteers for AG-348, an Investigational Medicine for Pyruvate Kinase (PK) Deficiency agios.com ↗ |
| 2015-03-24 | Agios Pharmaceuticals, Inc. | Regulatory Agios Pharmaceuticals Announces Orphan Drug Designation of AG-348 for Treatment of Pyruvate Kinase Deficiency agios.com ↗ |
| 2014-12-08 | Agios Pharmaceuticals, Inc. | Results Agios Reports Positive Phase 1 Data in Healthy Volunteers for AG-348, a First-in-Class Investigational Medicine That Targets the Underlying Cause of Pyruvate Kinase (PK) Deficiency agios.com ↗
These results provide early proof-of-mechanism for AG-348, a novel, first-in-class, oral activator of both wild type (normal) and mutated pyruvate kinase-R (PKR) enzymes. |
| 2014-11-06 | Agios Pharmaceuticals, Inc. | Results Agios Pharmaceuticals to Present Clinical and Preclinical Data at the 2014 American Society of Hematology Annual Meeting agios.com ↗
In addition, the emerging data from the Phase 1 healthy volunteer studies for AG-348 allow us to advance into patients in early 2015 and lend support to the hypothesis that targeting the underlying defect of PK deficiency could potentially improve clinical outcomes. |
| 2013-12-09 | Agios Pharmaceuticals, Inc. | Results Agios Presents Preclinical Data from Lead Programs at American Society of Hematology Annual Meeting agios.com ↗
The results demonstrate that AG-348 potently activates a spectrum of PKR mutant proteins, the isoform of pyruvate kinase that is present in red blood cells, leading to a normalization of metabolic balance in patient-derived blood samples. |
All press releases naming this drug 78 releases
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 33 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Mitapivat | “AG-348 (Mitapivat), an allosteric activator of red blood cell pyruvate kinase, increases enzymatic activity, protein stability, and ATP levels over a broad range of PKLR genotypes.” PMID 31974203 ↗ Jan 2021 |
| Known as | AG-348 | “Investigational drug mitapivat (also known as AG-348, AGI-1480 and AGX-0841) is an orally bioavailable, broad-spectrum allosteric activator of alleles of the RBC-specific form...” NCT04610866 ↗ |
| Known as | AG-348 HEMISULFATE SESQUIHYDRATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4299940 ↗ |
| Known as | AG-348 sulfate hydrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL4299940 ↗ |
| Known as | AGI-1480 | “Investigational drug mitapivat (also known as AG-348, AGI-1480 and AGX-0841) is an orally bioavailable, broad-spectrum allosteric activator of alleles of the RBC-specific form...” NCT04610866 ↗ |
| Known as | AGX-0841 | “Investigational drug mitapivat (also known as AG-348, AGI-1480 and AGX-0841) is an orally bioavailable, broad-spectrum allosteric activator of alleles of the RBC-specific form...” NCT04610866 ↗ |
| Known as | Mitapivat hemisulfate sesquihydrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL4299940 ↗ |
| Known as | mitapivat sulfate | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03991312 ↗ |
| Known as | Pkr-in-1 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4299940 ↗ |
| Known as | Pyrukynd | ChEMBL registry synonym — accepted as the source's own label CHEMBL4299940 ↗ |
| Action | Activate | “AG-348, a first-in-class allosteric PK-R activator” PMID 30091852 ↗ Aug 20181“Pyruvate kinase isozymes R/L activator” CHEMBL4299940 ↗ |
| Modality | Small molecule | “AG-348 / mitapivat is a novel, orally bioavailable, small molecule allosteric activator of PK-R” NCT04000165 ↗ |
| Route | Oral | “AG-348 will be administered as a single oral dose on Day 1.” NCT02108106 ↗ |
| Target | PKLR | “Pyruvate kinase isozymes R/L activator” CHEMBL4299940 ↗ |