AG-348

Phase 2Small molecule

AAgios Pharmaceuticals, Inc.

AG-348 is one of 71 small molecule programs targeting Sickle Cell Disease. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Sickle Cell DiseaseActive, not recruiting

ThalassemiaActive, not recruiting

Sickle Cell DiseasePhase 1

Active, not recruiting

ThalassemiaPhase 2

Active, not recruiting

Competition

Competitive Landscape

272 competitors in Sickle Cell Disease

#DrugCompanyPhaseTrials

Adakveo

Novartis

Phase 3

7 2

Exjade

Novartis

Phase 3

4 3

Brilinta

AstraZeneca

Phase 3

3 4

Campath

GGenzyme, a Sanofi Company

Phase 3

12 5

Ritalin

Novartis

Phase 3

2 6

Cysteine Hydrochloride

Novartis

Phase 3

2 7

Eliquis

Bristol-Myers Squibb

Phase 3

1 8

Oxbryta

GGlobal Blood Therapeutics

Phase 3

5 9

Aqvesme

AAgios Pharmaceuticals, Inc.

Phase 3

4 10

Effient

CCosette Pharmaceuticals, Inc.

Phase 3

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2030

Phase 2Active, not recruiting

Thalassemia

NCT03692052

Clinical

Trials by Indication[1]

Adult2 trials

Study to Evaluate the Relative Bioavailability and Safety of the AG-348 Tablet and Capsule Formulations in Healthy Adults

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT03397329·26 enrolled

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

Completed·AAgios Pharmaceuticals, Inc.·Phase 2·NCT02476916·52 enrolled

Pyruvate Kinase Deficiency2 trials

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Completed·AAgios Pharmaceuticals, Inc.·Phase 3·NCT03559699·27 enrolled

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Completed·AAgios Pharmaceuticals, Inc.·Phase 3·NCT03548220·80 enrolled

Sickle Cell Disease1 trial

A Dose-Finding Study of AG-348 in Sickle Cell Disease

Completed·NNational Institutes of Health Clinical Center (CC)·Phase 1·NCT04000165·17 enrolled

Tics1 trial

Study to Evaluate the Pharmacokinetics, Safety, and QTc Effect of AG-348 in Healthy Subjects of Japanese Origin and Non-Asian Origin

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT03250598·60 enrolled

Intravenous1 trial

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-348

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT03703505·8 enrolled

2 more indications

Thalassemia1 trial

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

Active, not recruiting·AAgios Pharmaceuticals, Inc.·Phase 2·NCT03692052·20 enrolled

Unspecified2 trials

A Phase I Study of AG-348 in Healthy Volunteers

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT02108106·48 enrolled

A Phase I Study of AG-348 in Healthy Volunteers

Completed·AAgios Pharmaceuticals, Inc.·Phase 1·NCT02149966·48 enrolled

Sources

References

[1]
ClinicalTrials.gov — 10 trial records for AG-348. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — AG-348 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for AG-348. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Agios Pharmaceuticals, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: AAgios Pharmaceuticals, Inc.

AG-348

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q3 2030

Trials by Indication[1]

Adult2 trials

Pyruvate Kinase Deficiency2 trials

Sickle Cell Disease1 trial

Tics1 trial

Intravenous1 trial

Thalassemia1 trial

Unspecified2 trials

References

Drug Info

Quick Facts

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