CUTX-101 (Copper Histidinate)
CUTX-101 (Copper Histidinate) is in development to supplement 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 and NICHD are currently conducting 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 may 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. Through the Cooperative Research and Development Agreement (CRADA) between Cyprium and NICHD, Cyprium is authorized to file NDA for Copper Histidinate for the treatment of Menkes disease using data generated at NICHD. FDA recently granted Fast Track Designation to CUTX-101.