Pipeline

Passion for Discovery

Agios has leveraged its core capabilities in cellular metabolism to build a productive research engine that is focused on genetically defined diseases.


CANDIDATE

Drug Discovery
Early Stage Clinical Development
Late Stage Clinical Development
Regulatory Submission

APPROVED
Non-transfusion Dependent Adult PK Deficiency
 
ACTIVATE
Transfusion Dependent Adult PK Deficiency
ACTIVATE-T
Non-transfusion Dependent Adult Thalassemia
ENGERGIZE
Transfusion Dependent Adult Thalassemia
ENERGIZE-T
Adult Sickle Cell Disease
Phase 2/3
Pediatric PK Deficiency
Pediatric Thalassemia
Pediatric Sickle Cell Disease
Other PKR/PKM2
development candidates
PAH
BCAT-II

Clinical Programs

Agios is focused on discovering and developing novel investigational medicines to treat genetically defined diseases through scientific leadership in the field of cellular metabolism. In addition to an active research and discovery pipeline, Agios has a first-in-class investigational therapy in clinical development.

Mitapivat

(PKR Activator)

Mitapivat is an investigational, wholly owned, first-in-class, novel, oral activator of both wild-type (normal) and mutated pyruvate kinase-R (PKR) enzymes.

Mutations in PKR cause deficiencies in red blood cell glycolysis, which lead to a disease known as pyruvate kinase (PK) deficiency. Agios announced positive topline data for mitapivat in adults with PK deficiency who are not regularly transfused and in adults with PK deficiency who are regularly transfused, and expects to file for U.S. and EU regulatory approval in this rare disease.

Additionally, Agios has expanded the clinical application of mitapivat into thalassemia and sickle cell disease where it has the potential to provide therapeutic benefit by activating wild-type PKR. Based on preliminary clinical data, proof-of-concept has been established for mitapivat as a potential treatment for both of these hemolytic anemias.

DISEASE PROGRAM STUDY PHASE STATUS LEARN MORE
Pyruvate kinase deficiency Mitapivat Phase 3 Study (ACTIVATE) to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Completed clinicaltrials.gov
Pyruvate kinase deficiency Mitapivat Phase 3 Study (ACTIVATE-T) Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD) Completed clinicaltrials.gov
Pyruvate kinase deficiency Mitapivat Phase 2 DRIVE PK Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency Active, not recruiting clinicaltrials.gov
Thalassemia Mitapivat A Phase 2, Open-label, Multicenter Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Subjects With Non-transfusion-dependent Thalassemia Active, not recruiting clinicaltrials.gov
Thalassemia (NTD) Mitapivat 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) Not yet recruiting clinicaltrials.gov
Thalassemia (TD) Mitapivat 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) Not yet recruiting clinicaltrials.gov
Pyruvate kinase deficiency Mitapivat Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007 Active, not recruiting clinicaltrials.gov

For additional details about these trials, please visit our Clinical Trials page or visit www.clinicaltrials.gov and search for Agios

AG-946

(PKR Activator)

AG-946 is a next-generation oral activator of both wild-type and mutated PKR enzymes. The Phase 1 study is evaluating AG-946 in healthy volunteers in a single ascending dose cohorts followed by multiple ascending dose cohorts. The goal is to evaluate safety, tolerability, pharmacokinetics and pharmacodynamics of AG-946. In addition, we have included the option to enroll additional multi-ascending dose cohorts in adults with sickle cell disease.

DISEASE PROGRAM STUDY PHASE STATUS LEARN MORE
Sickle Cell Disease Healthy Volunteers AG-946 A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-946 in Healthy Volunteers and in Participants With Sickle Cell Disease Recruiting clinicaltrials.gov

For additional details about these trials, please visit our Clinical Trials page or visit www.clinicaltrials.gov and search for Agios.

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