The Food and Drug Administration (FDA) has granted Orphan Drug designation to berubicin (CNS Pharmaceuticals) for the treatment of malignant gliomas.

Berubicin is an anthracycline that induces DNA damage in targeted cancer cells by interfering with the action of topoisomerase II. The designation is supported by data from a phase 1 trial conducted by Reata Pharmaceuticals in 2016. Findings from the study showed 44% of patients treated with berubicin (n=11 out of 25) experienced a statistically significant improvement in progression free survival, including 1 patient with a durable complete response who remains cancer-free as of February 20, 2020.

“Glioblastoma currently has a dismal survival rate of only 14.6 months from its diagnosis,” said  Dr Sandra Silberman, Chief Medical Officer of CNS. “We believe berubicin, which based on limited clinical data appears to be the first anthracycline to cross over the blood brain barrier in adults, provides a potentially novel therapy for the treatment of malignant gliomas.”

The Company plans to initiate a phase 2 trial of berubicin for the treatment of glioblastoma mutliforme later this year.

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The FDA’s Orphan Drug designation is granted to treatments for rare diseases that affect ≤200,000 individuals. 

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