FORTRESS BIOTECH, INC. Virtual Poster Title: Copper Histidinate Treatment for Menkes Disease (Kinky Hair Syndrome)
Presentation Date:
Session: (VH1410) Section on Advances in Therapeutics and Technology Program
Authors:
'There is a significant unmet need for an approved treatment for patients with Menkes disease. These positive data demonstrate the potential of CUTX-101 to be an effective therapy for patients with this devastating disease. We look forward to working with the
In two completed open-label, single-arm, single-site studies, 129 patients with Menkes disease were treated with CUTX-101 (1450 mcg CUTX-101, equivalent to 250 mcg elemental copper) administered subcutaneously twice daily until 12 months of age, and once daily thereafter, for a total duration of up to three years. Sixty-six patients born after 1999 and with severe loss-of-function ATP7A mutations from these two studies were combined and categorized into an Early Treatment cohort (CuHis-ET; treatment initiated within 4 weeks of birth, corrected for prematurity, n=31) and a Late Treatment cohort (CuHis-LT; treatment initiated after 4 weeks of birth, n=35). A historical control cohort of 18 Menkes disease patients who had not been treated with CUTX-101 were enrolled (including 18 in historical control-early treatment cohort (HC-ET); 17 of whom were also included in historical control-late treatment (HC-LT)). Efficacy of CUTX-101 was assessed by comparing CuHis-ET to untreated HC-ET, and CuHis-LT to untreated HC-LT, using OS as the primary and secondary efficacy endpoints, respectively.
The primary efficacy endpoint comparing CuHis-ET to HC-ET and the secondary efficacy endpoint comparing CuHis-LT to HC-LT were both met. Overall, a 79% reduction in risk of death was observed in CuHis-ET patients compared with HC-ET patients and median OS was 177.1 and 16.1 months, respectively with a hazard ratio (HR) of (95% CI) = 0.208 (0.094, 0.463) p
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