ZyStor Receives FDA Clearance For ZC-701

ZyStor Therapeutics (ZyStor) has received clearance from the FDA to proceed into a clinical trial for its first drug candidate, ZC-701, an enzyme replacement therapy for the treatment of Pompe disease.

ZC-701 is a recombinant protein containing ZyStor’s proprietary Glycosylation Independent Lysosomal Targeting (GILT) tag fused to GAA, thereby allowing more efficient delivery of GAA to the lysosome of muscle cells.

Reportedly, the result suggested that in preclinical research, ZC-701 was found to be both safe and highly efficacious in well-studied animal models. In animal models, preclinical efficacy was seen at doses much lower than those reported for the currently approved drug for the treatment of Pompe disease. It is anticipated that the first-in-human trial of ZC-701 will be conducted in late-onset Pompe patients.

Jonathan LeBowitz, chief scientific officer of ZyStor, said: “We are pleased and very proud to announce that ZC-701 has been cleared by the FDA for a phase I human safety trial. A small and dedicated team has brought ZC-701 from concept to the clinic and now ZyStor is poised to bring this promising treatment to Pompe patients. We hope that these patients will benefit from the improved targeting of ZC-701 to the tissues in the body that are most affected by Pompe disease.”