Can This Neurodegeneration Study Find a Way Forward?

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Retrophin Inc. (NASDAQ: RTRX) shares hit a multiyear low after the firm announced late-stage results from its study in patients with pantothenate kinase-associated neurodegeneration (PKAN). Specifically, this is the Phase 3 Fort study evaluating fosmetpantotenate versus a placebo.

Unfortunately, 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.

It is unclear what the future is for the study, but Retrophin intends to further analyze the data. The firm also will be working with study investigators to determine the appropriate next steps for the Fort study, including the ongoing open-label extension of the study.

Retrophin remains focused on progressing its two pivotal Phase 3 programs evaluating sparsentan for the treatment of focal segmental glomerulosclerosis and IgA nephropathy, and continuing the advancement of its commercial portfolio.

Eric Dube, Ph.D., CEO of Retrophin, commented:

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. We would like to thank the patients, their caregivers, study investigators and our employees, whose dedication made this study possible. 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.

Shares of Retrophin traded down about 25% on Thursday, at $13.02 in a 52-week range of $11.84 to $33.00. The consensus price target is $40.71.


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