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Drug

Mitapivat

Approved medicine
ClassPyruvate kinase isozymes R/L activatorResearchOngoing clinical research

Also known as Aqvesme, Pyrukynd.

RxNorm2594468ChEMBLCHEMBL4299940ATCB06AX04UNII2WTV10SIKH
11
Active clinical trials
Pyruvate kinase PKLR
Primary target
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE)

Clinical trial2026-08-27Results posted · ClinicalTrials.gov

Evaluation of the Safety,Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat

Clinical trial2026-07-07Results expected Q1 2028 · ClinicalTrials.gov

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

Clinical trial2026-06-30Primary completion · ClinicalTrials.gov

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZE-T)

Clinical trial2026-06-23Results posted · ClinicalTrials.gov

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

Clinical trial2026-06-23Results expected Q2 2029 · ClinicalTrials.gov

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

Clinical trial2026-06-23Results expected Q1 2029 · ClinicalTrials.gov

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease

Clinical trial2026-06-23Results expected Q3 2029 · ClinicalTrials.gov

A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Clinical trial2026-06-23Results expected Q3 2029 · ClinicalTrials.gov

An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies

Clinical trial2025-11-18Results posted · ClinicalTrials.gov

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

Clinical trial2025-10-30Primary completion · ClinicalTrials.gov

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period

Clinical trial2024-12-13Primary completion · ClinicalTrials.gov

A Phase 1, Open-label, One-Sequence Crossover Study to Assess the Effect of Mitapivat on the Pharmacokinetics of the CYP3A Substrate (Midazolam) in Healthy Participants

Clinical trial2024-12-03Completed · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Mitapivat
Aliases & brands
AqvesmePyrukynd
RxNorm CUI
2594468
ChEMBL ID
CHEMBL4299940
ATC codes
B06AX04
UNII
2WTV10SIKH
Primary mechanism
Pyruvate kinase isozymes R/L activator

Pharmacology & targets

1 target

Known molecular targets and mechanisms supported by curated pharmacology databases.

Pyruvate kinase PKLR
PKLR · P30613
ACTIVATORPyruvate kinase isozymes R/L activator

Clinical trials

18 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
18
registered trials across all phases
LATEST COMPLETION 2024
11
Active studies
5
Recruiting
10
Late-stage (III+)
6
Completed
1
Discontinued
PHASE DISTRIBUTIONn = 18
Phase 15Phase 1 / 21Phase 22Phase 2 / 31Phase 38Phase 41

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Enrolling by invitationPH IVAgios Pharmaceuticals, Inc.n=62029

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

Not yet recruitingPH IIIAgios Pharmaceuticals, Inc.n=542032

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

Not yet recruitingPH IIIAgios Pharmaceuticals, Inc.n=452032

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease

Not yet recruitingPH IIIAgios Pharmaceuticals, Inc.n=1592030

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

Active, not recruitingPH II/IIIAgios Pharmaceuticals, Inc.n=2862030

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 1, Open-label, One-Sequence Crossover Study to Assess the Effect of Mitapivat on the Pharmacokinetics of the CYP3A Substrate (Midazolam) in Healthy Participants

CompletedPH IAgios Pharmaceuticals, Inc.n=202024

An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies

CompletedPH IIIAgios Pharmaceuticals, Inc.n=902024

A Phase 1, Open-label, Single-dose, Pharmacokinetic Study of Mitapivat in Subjects With Moderate Hepatic Impairment Compared to Matched Healthy Control Subjects With Normal Hepatic Function

CompletedPH IAgios Pharmaceuticals, Inc.n=202023

A Phase 1, Open-Label, Randomized, Two-Period, Crossover Study to Compare the Pharmacokinetics and Safety of Mitapivat 100 mg Tablet Formulation With Mitapivat 2 × 50 mg Tablet Formulation in Healthy Adult Subjects

CompletedPH IAgios Pharmaceuticals, Inc.n=262021

A Phase 1, Open-label, Randomized 4-period Crossover Study to Assess the Relative Bioavailability and Effect of Food on the Coated Granule Formulation of Mitapivat in Healthy Subjects

CompletedPH IAgios Pharmaceuticals, Inc.n=322020

A Phase 1, Randomized, Four-Period, Crossover Food-Effect Study With Mitapivat Sulfate In Healthy Subjects

CompletedPH IAgios Pharmaceuticals, Inc.n=322020
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials