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Thursday, August 22, 2019

Retrophin med flunks late-stage study in rare neurodegenerative disorder

A Phase 3 clinical trial, FORT, evaluating Retrophin’s (NASDAQ:RTRXfosmetpantotenate in patients with pantothenate kinase-associated neurodegeneration (PKAN) failed to demonstrate a treatment effect versus placebo.
On a positive note, the phosphopantothenic acid prodrug was safe and well-tolerated.
PKAN in an inherited neurodegenerative disorder characterized by progressive difficulty with movement, typically starting in childhood.
Management will host a conference call this morning at 8:30 am ET to discuss the data.
Shares are down 25% premarket.

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