About Menkes Disease
Menkes disease is a rare X-linked pediatric disease caused by gene mutations of the copper transporter ATP7A. The condition is characterized by sparse, depigmented, kinky hair; failure to thrive; low/poor muscle tone (hypotonia); connective tissue disorders; and severe neurological symptoms such as seizures. If untreated, mortality for Menkes disease is high, with many patients dying before the age of 3. Currently, there is no FDA-approved treatment for Menkes disease and related disorders in copper metabolism.
To learn more about Menkes disease, please visit aboutmenkes.com.
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 National Institute of Child Health and Human Development (NICHD), early treatment of Menkes patients with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival.
Cyprium Therapeutics, Inc., with support from Sentynl, is developing CUTX-101 to restore copper homeostasis and maintain copper levels in patients with Menkes disease and related disorders.
In October 2021, Cyprium announced positive results from a safety and efficacy analysis of data integrated from two completed pivotal studies in patients with Menkes disease treated with CUTX-101. These data were presented as a virtual poster at the 2021 American Academy of Pediatrics National Conference & Exhibition.
In December 2021, Cyprium initiated a rolling submission of a New Drug Application to the U.S. Food and Drug Administration (FDA) for CUTX-101 for the treatment of Menkes disease.
CUTX-101 has been granted Orphan Drug Designation by the FDA. FDA previously granted Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations 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.
Learn more about Menkes disease at the National Institutes of Health (NIH) and National Organization for Rare Disorders (NORD).