MZE001 / Maze Therap, Sanofi, Shionogi - LARVOL DELTA

First-in-Human Evaluation of Safety, Pharmacokinetics and Muscle Glycogen Lowering of a Novel Glycogen Synthase 1 Inhibitor for the Treatment of Pompe Disease. (PubMed, Clin Pharmacol Ther) - "A muscle biopsy sub-study demonstrated that 10 days of MZE001 (480 mg BID) dosing safely and substantially lowered muscle glycogen stores in healthy adults. This correlated with the PBMC exposure response and supports the use of PBMC glycogen reduction as a surrogate for muscle response, and MZE001 potential for development as the first oral substrate reduction therapy for patients with Pompe disease."

Journal • P1 data • PK/PD data • Metabolic Disorders • Pompe Disease • Respiratory Diseases

A Randomized Phase 1 of of MZE001 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=121 | Completed | Sponsor: Maze Therapeutics | Recruiting ➔ Completed

Trial completion

A Randomized Phase 1 of of MZE001 in Healthy Volunteers (clinicaltrials.gov) - P1 | N=104 | Recruiting | Sponsor: Maze Therapeutics | N=80 ➔ 104

Enrollment change