Promising top-line results for CUTX-101 in Menkes disease

Shares of Fortress Biotech (Nasdaq: FBIO), formerly Coronado Biosciences, were up more than 6% at $3.51 in early trading today, after it announced that its Cyprium Therapeutics subsidiary reported positive top-line clinical efficacy results for CUTX-101, a potential treatment for Menkes disease, a rare X-linked recessive pediatric disorder caused by gene mutations of copper transporter ATP7A.

The study demonstrated statistically-significant improvement in overall survival for Menkes disease subjects who received early treatment (ET) with CUTX-101, compared to an untreated historical control (HC) cohort, with a nearly 80% reduction in the risk of death (Hazard Ratio = 0.21, p<0.0001). Median survival for the ET cohort was 14.8 years (177.1 months) compared to 1.3 years (15.9 months) for the untreated HC cohort.

“These positive top-line clinical efficacy data highlight the potential of CUTX-101 as an effective therapy for Menkes disease patients. With no currently approved US Food and Drug Administration (FDA) treatments, Menkes disease is a serious condition with a significant unmet medical need. We look forward to presenting these top-line data to the FDA at our upcoming pre-new drug application (pre-NDA) meeting and will begin our rolling submission of a New Drug Application (NDA) for CUTX-101 in the fourth quarter of this year,” said Dr Lung Yam, president and chief executive of Cyprium.