A phase 3 trial evaluating an investigational therapy for the treatment of pantothenate kinase-associated neurodegeneration (PKAN) did not meet its primary or secondary end points, according to Retrophin, Inc.

The FORT study was investigating the safety and efficacy of fosmetpantotenate, a phosphopantothenate replacement therapy, in patients with PKAN, a rare, genetic, and life-threatening neurologic disorder. Study patients (N=82) were randomized to receive fosmetpantotenate or placebo 3 times daily for 24 weeks; the primary end point of the study was change from baseline in the PKAN-ADL scale (specific activities of daily living measure) through 24 weeks. 

Results showed that while the treatment was found to be generally safe and well-tolerated, a significant change from baseline was not observed when compared with placebo. Data from the trial will be further analyzed and presented at an upcoming scientific congress, according to the Company. The study also included an open-label extension phase in which patients who completed the 24-week treatment period could continue to receive fosmetpantotenate.

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“We are very disappointed in the topline results from the FORT Study, particularly because we have seen the devastating impact of PKAN on patients and their families, and a significant unmet need remains with no approved treatment option,” said Eric Dube, PhD, chief executive officer of Retrophin. “We will work closely with the investigators to further analyze the results of the study and share them with the PKAN community to contribute to the growing knowledge of this rare disorder.”

Worldwide, PKAN, which is caused by a mutation in the PANK2 gene, affects approximately 5000 people. Symptoms of the disorder include dystonia, rigidity, dysphagia, twisting and writhing, and visual impairment.

For more information visit retrophin.com.