CUTX-101 (Copper Histidinate)
CUTX-101 (Copper Histidinate) is in development to restore copper homeostasis and maintain copper levels in patients with Menkes disease and related disorders. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under current Good Manufacturing Practice (cGMP) that is intended to improve tolerability due to its physiological pH, and to bypass the oral absorption of copper, which is impaired in patients with Menkes disease. In the Phase 1/2 clinical study conducted at NICHD, early treatment of Menkes patients with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A Phase 3 study of CUTX-101 is ongoing at NICHD.
Cyprium is completing a natural history study to collect data on Menkes disease patients who have not been treated with copper supplements. Data from this natural history study will serve as a historical control to demonstrate the efficacy and safety of CUTX-101. CUTX-101 has been granted orphan drug designation by the FDA. FDA granted Fast Track Designation and Rare Pediatric Disease Designation to CUTX-101. In July 2020, European Medicines Agency (EMA) Committee for Orphan Medicinal Products issued a positive opinion on the Orphan Medicinal Product Designation for CUTX-101.