Retrophin, Inc., a biotechnology company focused on the discovery and development of orphan drugs for the treatment of rare and life-threatening diseases, has announced positive results from a series of in vitro and in vivo experiments with RE-024. RE-024 rescued the phenotype of pantothenate kinase associated neurodegeneration (PKAN) in vitro and in vivo, demonstrating successful replacement therapy proof-of-concept.

“We are delighted with the outcome of the experiments conducted by St. Jude Children’s Research Hospital, which assessed RE-024 in a broad array of customized assays in PKAN,” said Martin Shkreli, founder and chief executive officer of Retrophin. “The promising results we’ve seen to date are a testament to our chemistry team’s design of RE-024. On the basis of these results, we are accelerating the timeline for filing of an IND for RE-024.”

PKAN is an inherited, progressive and fatal neurodegenerative disease. Symptoms of PKAN vary but often include ataxia, dystonia, and a general failure to thrive. Onset usually occurs before 10 years of age and typically results in premature death. While the exact incidence of PKAN is uncertain, it is estimated to affect one to three per million people worldwide. There is currently no FDA approved treatment for PKAN.