Raptor Submits Investigational New Drug Application For RP103

(RTTNews) - Raptor Pharmaceutical Corp. (RPTP), Tuesday said it submitted an investigational new drug application or IND to the U.S. Food and Drug Administration or FDA, for the clinical development of RP103 as a treatment for Leigh syndrome and other mitochondrial disorders. Currently, RP103 is marketed in the U.S. as PROCYSBI delayed-release capsules for the management of nephropathic cystinosis in adults and children 6 years and older. The clinical plan includes a Phase 2b study in 32 patients, up to a maximum of 64 patients.

The RP103-MITO-001 trial will evaluate the safety, tolerability and efficacy of RP103, and is expected to begin in the 2014 first quarter at four clinical sites in the U.S. The primary endpoint of the study will be the change from baseline in the Newcastle Pediatric Mitochondrial Disease Scale, while the secondary endpoints will include observations of myopathy, dystonia, seizures, motor development, dyskinesia, quality of life, and activities of daily living. Interim results from the initial 24 patients are expected by the end of 2014. Additionally, RP103 is believed to increase mitochondrial glutathione which acts as a scavenging agent of reactive oxygen species.