Cyprium Therapeutics, with the support of its licensing partner Sentynl, a wholly-owned subsidiary of Cadila Healthcare (Zydus), announced positive results from an efficacy and safety analysis of data integrated from two completed pivotal studies in patients with Menkes disease treated with CUTX-101, copper histidinate (CuHis). In both pre-specified primary and secondary efficacy analyses, treatment with CUTX101 demonstrated a significantly greater median overall survival (OS) compared to untreated historical control patients.

"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 U.S. Food and Drug Administration ("FDA") to begin our rolling submission of a new drug application ("NDA") for CUTX-101 in the fourth quarter of this year," said Lung S. Yam, M.D., Ph.D., President and Chief Executive Officer of Cyprium.

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 controllate 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<0.0001. A 75% reduction in the risk of death was also observed in CuHis-LT patients compared with HC-LT subjects and median OS was 62.4 and 17.6 months, respectively; HR (95% CI) = 0.253 (0.119, 0.537); p<0.0001.

Stephen G. Kaler, M.D., M.P.H., a physician-scientist in the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children's Hospital, is Principal Investigator of the clinical studies and is also a professor of Pediatrics and Genetics at The Ohio State University College of Medicine.

Cyprium has partnered with Sentynl to bring CUTX-101 to market. Cyprium will retain development responsibility of CUTX-101 through approval of the NDA by the FDA, and Sentynl will be responsible for commercialisation of CUTX-101 as well as progressing newborn screening activities.