A Phase I Study of AG-348 in Healthy Volunteers

Phase 1 Completed 48 enrolled Agios Pharmaceuticals, Inc.

RandomizedParallel-groupTriple-blindTreatment

Summary

This is a dose escalation study that will be conducted in healthy volunteers. Multiple cohorts of volunteers will receive ascending (increasingly higher) oral doses of AG-348 daily for 14 days to evaluate the safety and tolerability of multiple oral doses of AG-348, the pharmacokinetics (PK) of multiple doses of AG-348, and the pharmacodynamic profile of AG-348 with specific biomarkers assessed in the blood.

Timeline

Start: 2014-05
Primary completion: 2014-11
Completion: 2014-11

Publications

Yang H, Merica E, Chen Y, Cohen M, Goldwater R, Kosinski PA, Kung C, Yuan ZJ, Silverman L, Goldwasser M, Silver BA, Agresta S, Barbier AJ. Phase 1 Single- and Multiple-Ascending-Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG-348, a First-in-Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers. Clin Pharmacol Drug Dev. 2019 Feb;8(2):246-259. doi: 10.1002/cpdd.604. Epub 2018 Aug 9.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Mitapivat	Small molecule	15 mg	Oral
Subject	Mitapivat	Small molecule	120 mg	Oral
Subject	Mitapivat	Small molecule	700 mg	Oral

Indications

No indication recorded.