Cyprium Therapeutics, Inc. (Cyprium), a Fortress Biotech partner company, is focused on the development of novel therapies for the treatment of Menkes disease and related copper metabolism disorders. In March 2017, Cyprium entered into a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), part of the National Institutes of Health (NIH), to advance the clinical development of CUTX-101 (Copper Histidinate injection) for the treatment of Menkes disease.
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.
In addition, Cyprium and NICHD entered into a worldwide, exclusive license agreement to develop and commercialize adeno-associated virus (AAV)-based gene therapy, called AAV-ATP7A, to deliver working copies of the copper transporter that is defective in Menkes patients, and to be used in combination with CUTX-101.
Both CUTX-101 and AAV-ATP7A gene therapy were granted orphan drug designation by the FDA. The FDA granted Breakthrough Therapy Designation, 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.
Cyprium is a majority‐owned subsidiary of Fortress Biotech (NASDAQ: FBIO) and is based in New York City.