PIPELINE

Pre-clinical

Phase I

Phase II

Phase III

NDA

CUTX-101 (Copper Histidinate) for Menkes Disease

In development by partner, Sentynl Therapeutics; NDA accepted for Priority Review by FDA; PDUFA target action date: 9/30/2025
80%

AAV-ATP7A Gene Therapy for Menkes Disease

Preclinical
17%

= Orphan Drug Designation
= Fast Track Designation
= Rare Pediatric Disease
= Orphan Medicinal Product (Positive Opinion Granted)
= Breakthrough Therapy Designation