Fortress-Backed Cyprium's Formulated Copper Histidinate Shows Efficacy In Rare Pediatric Disease

Fortress Biotech Inc's FBIO partner company, Cyprium Therapeutics Inc announced results from an efficacy and safety analysis of data integrated from two CUTX-101 studies in 129 Menkes disease patients.
In both pre-specified primary and secondary efficacy analyses, treatment with CUTX-101 demonstrated a significantly greater median overall survival (OS) than untreated historical control patients.
The data will be presented at the 2021 American Academy of Pediatrics National Conference & Exhibition.
CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate (CuHis).
In the early treatment cohort, a 79% reduction in risk of death was observed in CuHis-ET patients compared with historical control HC-ET patients. The median OS was 177.1 and 16.1 months, respectively, with a hazard ratio (HR).
A 75% reduction in the risk of death was also observed in CuHis-LT (late treatment) patients compared with HC-LT subjects, and median OS was 62.4 and 17.6 months, respectively.
CUTX-101 was shown to be well tolerated. In CuHis-ET and CuHis-LT cohorts, the most common treatment-emergent adverse events were pneumonia (30.3%), seizures (21.2%), dehydration (18.2%), failure to thrive (16.7%), and respiratory distress (15.2%).
No patient discontinued due to an adverse event considered related to treatment.
Menkes disease is a rare X-linked recessive pediatric disease caused by gene mutations of copper transporter ATP7A.
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