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Clinical trials
catalyst calendar across sponsors →Studies where AGIO is the lead sponsor, as filed with ClinicalTrials.gov. Completion dates are the sponsor's own projected windows and are revised as a study runs. Active studies first, then completed and stopped — newest readout first within each.
46 interventional · 4 observational · 1 expanded access · 7 with posted results
Held by 13 tracked-famous managers (D. E. Shaw & Co., Inc., SUSQUEHANNA INTERNATIONAL GROUP, LLP, CITADEL ADVISORS LLC, AQR CAPITAL MANAGEMENT LLC, Qube Research & Technologies Ltd, +8 more) · FINRA short interest 5.1 days to cover (settled 2026-07-31) · government filings 180d: 1 congress / 0 executive
| Study | Phase | Status | Interventions | Conditions | Enrollment | Primary completion | Readout in | Updated |
|---|---|---|---|---|---|---|---|---|
| A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat StudyNCT05777993Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat studyOpen-label · Treatment | Phase 4 | Enrolling by invitation | Drug: Mitapivat | Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease, Thalassemia | 59 | Aug 2029 | ≤ 1,108 d | 2026-08-12 |
| A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)NCT07656415Percentage of Subjects who are Transfusion Free From Week 4 Through Week 52Randomized · Double-masked · Treatment | Phase 3 | Recruiting | Drug: Mitapivat Matched Placebo, Mitapivat | Sickle Cell Disease | 159 | Aug 2029 | ≤ 1,108 d | 2026-08-04 |
| A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)NCT07506863Percentage of Participants Who Achieved Transfusion Reduction Response (TRR) Through Week 48Randomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Drug: Mitapivat Matched Placebo, Mitapivat | Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | 54 | Jun 2029 | ≤ 1,046 d | 2026-08-03 |
| A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent ThalassemiaNCT07517133Percentage of Participants Who Achieved a Hemoglobin (Hb) ResponseRandomized · Quadruple-masked · Treatment | Phase 3 | Recruiting | Drug: Mitapivat, Placebo Matching Mitapivat | Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia | 45 | Mar 2029 | ≤ 955 d | 2026-08-03 |
| A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With PhenylketonuriaNCT07241234Number of Subjects with Adverse Events (AEs) and Serious Adverse Events (SAEs) by Type, Severity, and Relationship to the Study DrugNon-randomized · Open-label · Treatment | Phase 1 | Recruiting | Drug: AG-181 | Phenylketonuria | 20 | 2028-01-17 | in 516 d | 2026-07-30 |
| Pyruvate Kinase Deficiency Global Longitudinal RegistryNCT03481738observationalClinical Course of PK Deficiency | — | Active, not recruiting | — | Pyruvate Kinase Deficiency | 500 | 2027-05-31 | in 285 d | 2026-07-30 |
| A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD)NCT06924970Percentage of Participants With Hb ResponseRandomized · Quadruple-masked · Treatment | Phase 2 | Active, not recruiting | Drug: Tebapivat, Tebapivat Matched Placebo | Sickle Cell Disease | 59 | May 2026 | — | 2026-05-29 |
| A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)NCT05490446Phase 2a: Proportion of Participants With Hemoglobin (Hb) ResponseNon-randomized · Open-label · Treatment | Phase 2 | Active, not recruiting | Drug: Tebapivat | Myelodysplastic Syndromes | 87 | 2026-03-17actual | — | 2026-07-30 |
| A Study to Determine the Safety and Tolerability of AG-236 and How it is Absorbed, Broken Down, and Eliminated From the Body in Healthy ParticipantsNCT07075640Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) by Type, Severity, and Relationship to Study DrugRandomized · Double-masked · Treatment | Phase 1 | Active, not recruiting | Drug: AG-236, Placebo | Healthy Participants | 24 | 2026-02-11actual | — | 2026-07-30 |
| A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP)NCT05031780Phase 2: Percentage of Participants With Hemoglobin (Hb) ResponseRandomized · Quadruple-masked · Treatment | Phase 2/3 | Active, not recruiting | Drug: Mitapivat · Other: Mitapivat-matching placebo, Mitapivat-matching placebo | Sickle Cell Disease | 286 | 2025-10-30actual | — | 2026-06-23 |
| A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension PeriodNCT05175105Percentage of Participants Achieving a Hemoglobin (Hb) ResponseRandomized · Quadruple-masked · Treatment | Phase 3 | Active, not recruiting | Drug: Mitapivat, Mitapivat-matching placebo | Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia | 30 | 2024-12-13actual | — | 2026-07-30 |
| A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension PeriodNCT05144256Percentage of Participants Achieving Transfusion Reduction Response (TRR)Randomized · Quadruple-masked · Treatment | Phase 3 | Active, not recruiting | Drug: Mitapivat, Mitapivat-matching placebo | Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia | 49 | 2024-05-03actual | — | 2026-07-30 |
| A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)NCT04770779results2025-05-25Double-blind Period: Percentage of Participants Who Achieved Transfusion Reduction Response (TRR)Randomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Active, not recruiting | Drug: Placebo Matching Mitapivat, Mitapivat | Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia | 258 | 2024-04-11actual | — | 2026-07-31 |
| A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)NCT04770753results2025-01-24Double-Blind Period: Percentage of Participants Who Achieved Hemoglobin (Hb) Response From Week 12 Through Week 24 Compared With BaselineRandomized · Quadruple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Active, not recruiting | Drug: Placebo Matching Mitapivat, Mitapivat | Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia | 194 | 2023-11-13actual | — | 2026-07-31 |
| A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent ThalassemiaNCT03692052results2021-11-15Percentage of Participants Achieving a Hemoglobin Response (HR)Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Active, not recruiting | Drug: AG-348 | Thalassemia | 20 | 2020-08-20actual | — | 2026-03-30 |
| A Study of Mitapivat in Participants With Sickle Cell Disease and NephropathyNCT06286046Percentage of Participants With Albumin Creatinine Ratio (ACR) ResponseOpen-label · Treatment | Phase 2 | Withdrawnsponsor's stated reason: Withdrawn prior to study start per sponsor decision. No patients were enrolled. | Drug: Mitapivat | Sickle Cell Disease, Nephropathy | 0 | May 2027 | ≤ 285 d | 2026-04-03 |
| Single and Multiple Ascending Dose Study and Food Effect Study for AG181NCT06286033SAD: Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) by Type, Severity, and Relationship to Study DrugRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: AG-181, Placebo | Healthy Volunteers | 88 | 2025-10-09actual | — | 2025-12-23 |
| A Study to Determine How Tebapivat is Absorbed, Broken Down, and Removed From the Body and the Extent to Which Tebapivat is Made Available in the Body in Healthy ParticipantsNCT06745271Percentage of Radioactive Dose Excreted in Urine Over a Time Interval (feut1-t2) of [14C]-TebapivatOpen-label · Treatment | Phase 1 | Completed | Drug: [14C]-tebapivat, [13C2,15N3]-tebapivat | Healthy Participants | 8 | 2025-02-21actual | — | 2025-03-18 |
| Study of How Mitapivat Affects Midazolam Blood Levels in Healthy ParticipantsNCT06648824Area Under the Plasma Concentration-Time Curve Extrapolated From Time Zero to Infinity (AUC0-infinity) of MidazolamOpen-label · Treatment | Phase 1 | Completed | Drug: Mitapivat, Midazolam | Healthy Participants | 20 | 2024-12-03actual | — | 2025-01-28 |
| Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007NCT03853798results2025-11-18All Cohorts: Number of Participants With at Least One Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, Related TEAEs and TEAEs With Severity Greater Than or Equal to Grade 3Non-randomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Mitapivat | Pyruvate Kinase Deficiency | 90 | 2024-07-03actual | — | 2025-11-18 |
| A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell DiseaseNCT04536792SAD and MAD: Number of Participants Who Experienced at Least One Adverse Event (AE), by SeverityRandomized · Quadruple-masked · Treatment | Phase 1 | Completed | Drug: AG-946, AG-946 Matched Placebo | Healthy Volunteers, Anemia, Sickle Cell | 122 | 2023-12-19actual | — | 2024-04-16 |
| Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008NCT04995315observationalChange Over Time in Cogstate Brief Battery (CBB) Scores | — | Completed | — | Pyruvate Kinase Deficiency | 13 | 2023-09-22actual | — | 2023-10-16 |
| A Pharmacokinetic Study of Mitapivat in Participants With Moderate Hepatic Impairment Compared to Matched Healthy Control Participants With Normal Hepatic FunctionNCT05610657Area Under the Plasma Concentration-Time Curve From Time 0 (Predose) to Extrapolated to Infinity Time (AUC∞) of MitapivatOpen-label · Basic science | Phase 1 | Completed | Drug: Mitapivat | Moderate Hepatic Impairment | 20 | 2023-07-21actual | — | 2023-09-21 |
| Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO)NCT04964323observationalChange from Baseline in Pyruvate Kinase Deficiency Diary (PKDD) Score | — | Terminatedsponsor's stated reason: Lack of enrollment and participant compliance with surveys. | — | Pyruvate Kinase Deficiency, Pyruvate Kinase Deficiency Anemia, Anemia, Anemia, Hemolytic | 15 | 2023-06-30actual | — | 2023-09-07 |
| A Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Healthy Adult ParticipantsNCT04696393Area Under the Plasma Concentration Versus Time Curve (AUC) of Mitapivat From Time 0 to the Last Quantifiable Concentration (AUC0-t)Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: Mitapivat tablet | Healthy Volunteers | 26 | 2021-03-19actual | — | 2021-04-13 |
| A Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy ParticipantsNCT04565678Maximum Observed Concentration (Cmax) of Mitapivat Under Fasted ConditionsRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: Mitapivat tablets, Mitapivat coated granules | Healthy Volunteers | 32 | 2020-12-03actual | — | 2020-12-10 |
| A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Mitapivat (AG-348) in Healthy Adult ParticipantsNCT04472832Area Under the Plasma Concentration Versus Time Curve (AUC) From Time 0 to the Last Quantifiable Concentration (AUC0-T) for Mitapivat Under Fasted and High-Fat Meal ConditionsRandomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: Placebo for Treatment A, Mitapivat 100 mg, Placebo for Treatment B, Mitapivat 300 mg, Placebo for Treatment C | Healthy Volunteers | 32 | 2020-11-23actual | — | 2021-02-18 |
| A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)NCT03559699results2021-12-10Percentage of Participants Achieving a Reduction in Transfusion Burden in Part 2Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: AG-348 | Pyruvate Kinase Deficiency, Anemia, Hemolytic | 27 | 2020-11-12actual | — | 2022-01-04 |
| A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)NCT03548220results2022-05-24Percentage of Participants Achieving a Hemoglobin (Hb) Response (HR)Randomized · Triple-masked · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 3 | Completed | Drug: Placebo, AG-348 | Pyruvate Kinase Deficiency, Anemia, Hemolytic | 80 | 2020-10-09actual | — | 2022-05-24 |
| A Study of AG-636 in the Treatment of Subjects With Advanced LymphomaNCT03834584The frequency of dose limiting toxicities (DLTs) associated with AG-636 administration during the first cycle (first 28 days) of treatment.Open-label · Treatment | Phase 1 | Terminatedsponsor's stated reason: Terminated due to sponsor decision. | Drug: AG-636 | Lymphoma | 11 | 2020-06-17actual | — | 2020-08-25 |
| A Study to Compare the Relative Bioavailability of Two AG-881 Formulations and Evaluate the Effect of Food and Omeprazole on the Pharmacokinetics of AG-881NCT04128787Area Under the Concentration-Time Curve From Time 0 to the Last Observed Non-Zero Concentration (AUC0-t) of AG-881 Formulation 1 without FoodRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: AG881 Formulation 1, AG881 Formulation 2, Omeprazole | Healthy Volunteers | 36 | 2019-12-18actual | — | 2021-07-21 |
| A Study to Evaluate AG-881 in Healthy Japanese and Non-Asian ParticipantsNCT04145128Area Under the Plasma Concentration Versus Time Curve from Time Zero to the Time of the Last Measurable Concentration (AUC0-last) of AG881Open-label · Treatment | Phase 1 | Completed | Drug: AG881 | Healthy Volunteers | 32 | 2019-12-11actual | — | 2020-04-02 |
| A Study to Evaluate the Effect of AG-881 on the Pharmacokinetics of a Single Dose of Lamotrigine in Healthy AdultsNCT04015687Area under the Concentration-time Curve from Time 0 to Infinity (AUC0-inf) for Lamotrigine Administered with Interacting Drug AG-881Non-randomized · Open-label · Basic science | Phase 1 | Completed | Drug: AG-881, Lamotrigine | Healthy Participants | 22 | 2019-10-09actual | — | 2019-10-14 |
| Pre-Screening Study to Identify MTAP Loss in Advanced Solid Tumors or LymphomaNCT03361358observationalDetermination of MTAP status in tumors | — | Completed | — | Advanced Solid Tumors, Lymphoma | 44 | 2019-09-30actual | — | 2020-03-16 |
| A Safety and Pharmacokinetic Study of AG-881 in Healthy Male Participants Following Administration of a Single Oral Dose of [14C] AG-881 and Concomitant Intravenous Microdose of [13C315N3] AG-881NCT03960502Amount of AG-881 Excreted in Urine (Aeu)Open-label · Basic science | Phase 1 | Completed | Drug: AG-881, [13C315N3]AG-881 | Healthy Male Participants | 5 | 2019-09-07actual | — | 2019-10-23 |
| Study to Evaluate the Effect of Multiple Doses of Itraconazole and Rifampin on the Single-Dose Pharmacokinetics of Mitapivat Sulfate (AG-348) in Healthy Adult ParticipantsNCT03991312Area Under the Concentration-Time Curve, from Time 0 to the Last Measurable Concentration (AUC0-t) of Mitapivat Sulfate with and without ItraconazoleNon-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: itraconazole, rifampin, mitapivat sulfate | Healthy Volunteers | 28 | 2019-08-14actual | — | 2019-10-23 |
| A Study to Evaluate the Absorption, Distribution, Metabolism, Excretion and Absolute Bioavailability of AG-348 in Healthy Male Participants Following Administration of a Single Oral Dose of [14C]AG-348 and Concomitant Single Intravenous Microdose of [13C6]AG-348NCT03703505AG-348 Excreted Through Urine [A(eu)]Open-label · Basic science | Phase 1 | Completed | Drug: AG-348, [13C6]AG-348 | Healthy Volunteers | 8 | 2018-06-01actual | — | 2018-10-12 |
| A Study of AG-120 (Ivosidenib) in Subjects With Mild or Moderate Hepatic Impairment or Normal Hepatic FunctionNCT03282513Maximum observed plasma concentration (Cmax)Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: AG-120 (Ivosidenib) | Hepatic Impairment | 33 | 2018-03-31actual | — | 2018-04-27 |
| Study of Orally Administered AG-881 in Patients With Advanced Hematologic Malignancies With an IDH1 and/or IDH2 MutationNCT02492737Safety/tolerability; incidence of adverse eventsOpen-label · Treatment | Phase 1 | Completed | Drug: AG881 | Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome, Hematologic Malignancies | 46 | 2018-03-21actual | — | 2019-03-08 |
| Study to Evaluate the Relative Bioavailability and Safety of the AG-348 Tablet and Capsule Formulations in Healthy AdultsNCT03397329CmaxRandomized · Open-label · Other | Phase 1 | Completed | Drug: AG-348 Sequence A, AG-348 Sequence B | Healthy Volunteers | 26 | 2017-11-13actual | — | 2018-01-12 |
| Study to Evaluate the Pharmacokinetics, Safety, and QTc Effect of AG-348 in Healthy Subjects of Japanese Origin and Non-Asian OriginNCT03250598AUCNon-randomized · Open-label · Other | Phase 1 | Completed | Drug: AG-348, AG-348, AG-348 | Healthy, Japanese, Non-Asian | 60 | 2017-10-02actual | — | 2017-10-19 |
| Japanese Bridging Study of Ivosidenib (AG-120) in Healthy SubjectsNCT03071770AUCOpen-label · Treatment | Phase 1 | Completed | Drug: ivosidenib (AG-120) | Healthy | 60 | 2017-05-09actual | — | 2018-01-11 |
| A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) DeficiencyNCT02476916results2020-06-11Percentage of Participants Experiencing at Least One Adverse Event (AEs) in the Core PeriodRandomized · Open-label · TreatmentStudy Protocol ↗ · Statistical Analysis Plan ↗ | Phase 2 | Completed | Drug: AG-348 | Pyruvate Kinase Deficiency | 52 | 2017-05-08actual | — | 2026-05-01 |
| A Safety and Tolerability Study of AG-519 in Healthy SubjectsNCT02630927Incidence of Treatment-Emergent Adverse EventsRandomized · Double-masked · Treatment | Phase 1 | Terminatedsponsor's stated reason: Agios is no longer developing its second pyruvate kinase-R (PKR) activator, AG-519, and withdrew its investigational new drug (IND) application in December 2016 | Drug: AG-519, Placebo | Anemia | 108 | Dec 2016actual | — | 2017-06-29 |
| Drug-Drug Interaction Study of AG120 in Healthy SubjectsNCT02831972AG120 Area Under the Curve [AUC]Non-randomized · Open-label · Treatment | Phase 1 | Completed | Drug: AG120, Itraconazole | Healthy | 22 | Sep 2016actual | — | 2017-06-27 |
| Food Effect Study of AG120 in Healthy SubjectsNCT02579707CmaxRandomized · Open-label · Treatment | Phase 1 | Completed | Drug: AG120 | Healthy | 36 | Feb 2016actual | — | 2016-11-29 |
| Study to Investigate the Absorption, Metabolism, and Excretion of [14C]-AG-120NCT02489513Pharmacokinetics - Total [14C] Whole blood and plasmaOpen-label | Phase 1 | Completed | Drug: [14C]-AG-120 | Healthy Volunteers | 8 | Sep 2015actual | — | 2015-12-14 |
| Food Effect Study of AG-221 in Healthy Male SubjectsNCT02218346Pharmacokinetic (PK) profile of AG-221Randomized · Open-label · Treatment | Phase 1 | Completed | Drug: AG221 | Healthy Volunteers | 30 | Dec 2014actual | — | 2015-12-14 |
| A Phase I Study of AG-348 in Healthy VolunteersNCT02149966Incidence of adverse eventsRandomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: AG-348, Placebo | Healthy Volunteer | 48 | Nov 2014actual | — | 2014-11-10 |
| A Phase I Study of AG-348 in Healthy VolunteersNCT02108106Safety and tolerabilityRandomized · Triple-masked · Treatment | Phase 1 | Completed | Drug: AG-348, Placebo | Healthy Volunteer | 48 | Aug 2014actual | — | 2014-10-22 |
| Ivosidenib Expanded Access Program in Relapsed/Refractory AML With an IDH1 MutationNCT03245424expanded access | — | Approved for marketing | Drug: ivosidenib (AG-120) | Acute Myeloid Leukemia, Relapsed Adult AML, Relapsed Pediatric AML | — | — | — | 2018-07-24 |
Primary completion is the date the sponsor filed with the registry — their own projection, revised whenever they revise it, unless the row is marked actual.
Source: ClinicalTrials.gov, retrieved 2026-08-19