Retrophin announced results from the Phase 3 FORT study evaluating the safety and efficacy of fosmetpantotenate compared to placebo in patients with pantothenate kinase-associated neurodegeneration (PKAN). The study did not meet its primary endpoint and did not demonstrate a difference between treatment groups. The study also did not meet its secondary endpoint. Fosmetpantotenate was observed to be generally safe and well-tolerated.

FORT  was an international, randomized, double-blind, placebo-controlled trial designed to assess the safety and efficacy of fosmetpantotenate in 84 patients with PKAN. Patients received either three times-daily dosing of fosmetpantotenate or placebo using a 1:1 randomization over 24 weeks. The primary endpoint in the study was the change from baseline in the PKAN-ADL scale through 24 weeks of treatment. 

Retrophin will continue to analyze the data and present finding at an upcoming scientific congress, in addition to working with study investigators to determine the appropriate next steps for the FORT Study, including the ongoing open-label extension of the study.

About pantothenate kinase-associated neurodegeneration (PKAN)

PKAN is a rare, genetic, and life-threatening neurological disorder characterized by a host of progressively debilitating symptoms that typically begin in early childhood. People suffering from PKAN may experience movement disorders such as dystonia (sustained muscle contraction leading to abnormal posture), rigidity, dysphagia (problems swallowing), and twisting and writhing, as well as visual impairment. PKAN is estimated to affect up to 5,000 people worldwide.

PKAN is caused by a mutation in the PANK2 gene, which encodes a critical protein that phosphorylates vitamin B5 (pantothenate), generating phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of coenzyme A (CoA), which is essential in biochemical reactions impacting energy metabolism, membrane integrity, signaling and other critical processes.

About fosmetpantotenate

At the root of PKAN are mutations in the PANK2 gene, which cause a reduction in the activity of the PanK2 enzyme. In the normal biochemical process, the PanK2 enzyme converts pantothenate (PA, vitamin B5) to phosphopantothenate (PPA), and is required for the production of coenzyme A (CoA). CoA is essential for many critical biochemical reactions in the body, including energy metabolism, membrane integrity and cell signaling. Fosmetpantotenate is a small molecule replacement therapy designed to pass the blood-brain barrier and be converted to PPA. This approach is to attempt to restore CoA levels with fosmetpantotenate.

Source: Retrophin

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