Bennu acquires potential cystinosis treatment

NOVATO, Calif. Raptor Pharmaceuticals has announced that its clinical development division, Bennu Pharmaceuticals has acquired a clinical product candidate, EC Cysteamine. The company received the exclusive worldwide license to the candidate by way of its acquisition by merger of Encode Pharmaceuticals.

The drug is prescribed for the management of the genetic disorder known as nephropathic cystinosis, a lysosomal storage disease characterized by the poor transport of cystine out of lysosomes. If left untreated, this disorder is associated with a high morbidity rate, including renal failure. EC Cysteamine has received orphan drug designation from the Food and Drug Administration’s Office of Orphan Products Development for the treatment of cystinosis.

“EC Cysteamine will add value, and balance our clinical portfolio with an advanced clinical program focused on a safe and potentially more effective second generation, proprietary treatment for the management of cystinosis,” stated Dr. Christopher M. Starr, Raptor’s chief executive officer. “We plan to continue evaluating EC Cysteamine in other disorders for which it has already shown promise in the clinic.”