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Retrophin Reports Positive Data from Preclinical Trial of RE-024 for the Treatment of PKAN

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Retrophin, Inc. (OTC: RTRX) today announced that it has received positive survival results from interim preclinical tests for the Company's compound, RE-024, for the treatment of the ultra-orphan disease Pantothenate Kinase-Associated Neurodegeneration (PKAN). RE-024 is a replacement therapy for phosphopantothenate, the substrate that is missing in patients with PKAN. Tests were conducted on mice that were administered a PANK inhibitor to induce a PKAN-like phenotype.

The interim results of this two-week study assessed the effect of RE-024 in mice dosed with RE-024 and Hopantenate (HoPan), a pantothenate kinase inhibitor that simulates PKAN by inhibiting PANK, compared to mice dosed with HoPan alone. Data from the study showed that 92.5% (37/40) of the mice dosed with HoPan and RE-024 survived the experiment, while no (0/14) mice dosed with HoPan alone were alive at the end of the study period (p

PKAN is a rare and life-threatening neurological disorder caused by a mutation in the PANK2 gene, which prevents patients from being able to properly metabolize vitamin B5 (pantothenate) into phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of Coenzyme A (CoA) and iron accumulation in the brain. As a result, patients present with dystonia (sustained muscle contraction leading to abnormal posture), rigidity, dysphagia (problems swallowing), weakness, pigmentary retinopathy (visual impairment), tremors, as well as a number of other symptoms. Onset of PKAN typically occurs prior to the age of 10 and has an estimated prevalence of 5,000-10,000 patients worldwide. Many patients die within 10 years of being diagnosed.

“We are proud of what we have accomplished with this program in a short period of time,” said Martin Shkreli, Founder, President and Chief Executive Officer of Retrophin. “When our team read about the plight of PKAN patients, we moved quickly to develop a series of phosphopantothenate analogs that we believed would rescue the phenotype of patients suffering from this horrific, catastrophic disease. Today I am so proud of our team for its dedication to these patients. We expect that we will be able to start a human study in first-quarter 2014.”

Suzanne Jackowski, PhD, a member of the faculty at St. Jude Children's Research Hospital and a leading expert in Coenzyme A metabolism, commented, "I am encouraged by the results of this most recent experiment with RE-024. The positive preclinical survival data generated by this study serve to further confirm our own proof-of-concept research, conducted earlier this year, pointing to RE-024 as a potential promising new approach to treating this debilitating and life-threatening disease.”

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