Maintained by Sreepriya Prasannan

MSc Digital Transformation of Life Sciences (Innopharma Education / Griffith College); MSc & BSc Botany HSE Spark Ignite 2026 — Top 14 finalist

Compiled from ClinicalTrials.gov and CTIS registry records · Last verified 26 Sep 2026 · Editorial standards

At a glance

Agios Pharmaceuticals is the lead sponsor of 17 current clinical trials in our USA, UK, Ireland, China and EU (CTIS) registers. 4 are recruiting patients now. The most common phase is Phase 3. The drugs named most often in its trials are Mitapivat and Tebapivat. The most-studied condition is Sickle Cell Disease.

ClinicalTrials.gov trials by status

Recruiting427%
Active, closed1067%
By invitation17%

Trials by phase

Phase 3747%
Phase 2320%
Phase 1213%
Observational17%
Phase 2/317%
Phase 417%

Clinical trials sponsored by Agios Pharmaceuticals

15 on ClinicalTrials.gov

Study	Phase	Status	Start	Registers
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) NCT07506863 · Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia Drug: Mitapivat	Phase 3	Recruiting	2026-09	USA
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) NCT07656415 · ClinicalTrials.gov ↗ · Sickle Cell Disease Drug: Mitapivat	Phase 3	Recruiting	2026-07	USA
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria NCT07241234 · ClinicalTrials.gov ↗ · Phenylketonuria	Phase 1	Recruiting	2026-04	USA
A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia NCT07517133 · ClinicalTrials.gov ↗ · Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia Drug: Mitapivat	Phase 3	Recruiting	2026-09	USA
A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study NCT05777993 · ClinicalTrials.gov ↗ · Pyruvate Kinase Deficiency, Anemia, Hemolytic, Sickle Cell Disease Drug: Mitapivat	Phase 4	By invitation	2023-07	UK
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 Participants NCT07075640 · Healthy Participants	Phase 1	Active, closed	2025-07	USA
Pyruvate Kinase Deficiency Global Longitudinal Registry NCT03481738 · Pyruvate Kinase Deficiency	Observational	Active, closed	2018-04	Ireland, UK, USA
A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With Sickle Cell Disease (SCD) NCT06924970 · Sickle Cell Disease Drug: Tebapivat	Phase 2	Active, closed	2025-05	Ireland, UK, USA
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT) NCT04770779 · ClinicalTrials.gov ↗ · Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia Drug: Mitapivat	Phase 3	Active, closed	2021-11	UK, USA
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT) NCT04770753 · ClinicalTrials.gov ↗ · Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia Drug: Mitapivat	Phase 3	Active, closed	2021-12	UK, USA, China
A Study of Tebapivat (AG-946) in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS) NCT05490446 · ClinicalTrials.gov ↗ · Myelodysplastic Syndromes Drug: Tebapivat	Phase 2	Active, closed	2022-11	UK, USA
A Study Evaluating the Efficacy and Safety of Mitapivat (AG-348) in Participants With Sickle Cell Disease (RISE UP) NCT05031780 · ClinicalTrials.gov ↗ · Sickle Cell Disease Drug: Mitapivat	Phase 2/3	Active, closed	2022-02	UK, USA
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 Period NCT05144256 · ClinicalTrials.gov ↗ · Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia Drug: Mitapivat	Phase 3	Active, closed	2022-06	UK, USA
A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia NCT03692052 · ClinicalTrials.gov ↗ · Thalassemia Drug: Mitapivat	Phase 2	Active, closed	2019-03	UK, USA
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 Period NCT05175105 · ClinicalTrials.gov ↗ · Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia Drug: Mitapivat	Phase 3	Active, closed	2022-06	USA

Registers list studies that are recruiting, about to open, active but closed to enrolment, or enrolling by invitation. The UK and USA registers cover industry-sponsored studies only. Titles and statuses are copied from the registry as published.

Agios Pharmaceuticals in the EU trials register (CTIS)

2 trials

Authorised or ongoing trials in the EU Clinical Trials Information System with Agios Pharmaceuticals as sponsor. Some are the same study as a ClinicalTrials.gov record above; the two registries share no common ID. Trials with a site in Ireland are listed first.

Study	Phase	Status	Countries
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 (ENERGIZEKids) 2025-524706-15-00 · Hemic and Lymphatic Diseases Drug: Mitapivat	Phase 3	Authorised	Italy, Greece
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-Transfusion-Dependent Thalassemia (ENERGIZEKids-T) 2025-524707-71-00 · Hemic and Lymphatic Diseases Drug: Mitapivat	Phase 3	Authorised	Italy, Greece

Drugs in Agios Pharmaceuticals trials

2 compounds

Drugs and biologicals named as interventions in these trials, with the number of this sponsor's trials that name each one. Placebos and generic comparators are excluded.

Mitapivat12 Tebapivat2

Conditions in Agios Pharmaceuticals trials

13 conditions

Sickle Cell Disease4Pyruvate Kinase Deficiency2Transfusion-dependent Alpha-Thalassemia2Transfusion-dependent Beta-Thalassemia2Non-Transfusion-dependent Alpha-Thalassemia2Non-Transfusion-dependent Beta-Thalassemia2Pediatric Pyruvate Kinase Deficiency2Pediatric Hemolytic Anemia2Thalassemia2Myelodysplastic Syndromes1Anemia, Hemolytic1Healthy Participants1Phenylketonuria1

By trial register

Register	Trials	Recruiting
Clinical trials in USA	14	4
Clinical trials in UK	9	0
Clinical trials in Ireland	2	0
Clinical trials in China	1	0
EU register (CTIS)	2	—

A trial with sites in more than one country appears in each of those registers.

Other sponsors in Sickle Cell Disease

Sponsor	Trials
Forma Therapeutics	3
Beam Therapeutics	2
Genetix Biotherapeutics	2
Editas Medicine	2
CSL Behring	1

Sponsors with the most current trials registered for this condition.

Questions about Agios Pharmaceuticals trials

How many clinical trials does Agios Pharmaceuticals sponsor?

Agios Pharmaceuticals is the lead sponsor of 15 current trials on ClinicalTrials.gov in our USA, UK, Ireland and China registers, and 2 authorised or ongoing trials in the EU register (CTIS), as of the registry sync on 26 September 2026. Completed and withdrawn studies are not counted.

Are Agios Pharmaceuticals trials recruiting?

Yes. 4 of its 15 ClinicalTrials.gov trials are recruiting. Eligibility and site contacts are on each trial's record. Only the study team can confirm whether a patient is eligible.

Which drugs is Agios Pharmaceuticals testing?

Its current trials name 2 drugs and biologicals as interventions. The most frequent are Mitapivat (12) and Tebapivat (2). The number in brackets is how many of its trials name each drug.

Does Agios Pharmaceuticals have clinical trials in Ireland?

Yes. 2 trials in our Ireland register (ClinicalTrials.gov). Some may be the same study registered in both. See the Irish clinical trials register for sites and contacts.

Where does this information come from?

Every trial, status, phase and condition on this page is copied from public ClinicalTrials.gov and EU CTIS records by a daily sync, grouped by the sponsor name each registry records. It says nothing about the organisation beyond its registered trials, and it is not medical advice.

How this page is built

Everything on this page is copied from public registry records — it is not written from memory, and nothing here describes the organisation beyond its registered trials. Each trial links to its own record so you can check it yourself.

ClinicalTrials.gov (U.S. National Library of Medicine) ↗ Trial IDs, titles, lead sponsor, phase, overall status, start date, conditions and intervention names
EU Clinical Trials Information System (CTIS) ↗ EU trial numbers, titles, sponsor, phase, status, therapeutic area and member-state countries

This page is built automatically: a daily sync job reads the registry records behind our trial registers and groups every current trial by its lead sponsor name, ignoring legal-form suffixes such as Inc. or Ltd. A small, hand-checked list joins the same organisation's different registry names (for example its EU legal entity). There is no AI drafting and no per-sponsor editorial pass. The “last verified” date above is the timestamp of that sync, not a review date. Our general editorial approach is described in our editorial standards and corrections policy. The the registries data on this page was last retrieved on 26 Sep 2026.

Please verify before you rely on this. This page is general information for life-science and pharmaceutical professionals. It is not medical advice, and it has not been reviewed by a clinician — our editorial team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC) and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not automatically interchangeable, and approvals, brand names and indications differ between the US, the EU/Ireland (EMA/HPRA) and other regions. Spotted an error? Tell us — we correct promptly and log it.

Agios Pharmaceuticals clinical trials