Retrophin's Late-Stage PKAN Drug Fails Against Placebo

Stocks down

Shares of Retrophin have plunged more than 24% in premarket trading after the San Diego-based company announced its Phase III treatment for pantothenate kinase-associated neurodegeneration (PKAN) did not stack up against placebo.

This morning, Retrophin, Inc. said results from the late-stage FORT study evaluating the safety and efficacy of fosmetpantotenate compared to placebo in PKAN patients did not demonstrate a difference between the treatment groups, meaning the trial drug did not distinguish itself. In addition to failing to hit its primary endpoint, Retrophin said it also failed to hit its secondary endpoint. In its announcement, Retrophin did not provide full details of the Phase III failure. The company said it intended to analyze the data and present the information at a later medical conference. Additionally, Retrophin said it will work with study investigators to analyze the appropriate next steps for fosmetpantotenate, including the ongoing open-label extension of the study. In the FORT trial, fosmetpantotenate was observed to be generally safe and well-tolerated, the company added.

As could be expected, the company noted its disappointment in the FORT study. Eric Dube said that much of that disappointment surrounded the fact that throughout the studies, the company has seen the “devastating impact of PKAN on patients and their families.” Dube said there remains a significant unmet need in this patient population, as there is no approved treatment.

PKAN is a rare, genetic neurological disorder caused by a mutation in the PANK2 gene. The disruption of this metabolic pathway ultimately leads to decreased levels of coenzyme A, which is essential in biochemical reactions impacting energy metabolism, membrane integrity, signaling and other critical processes. The disease is characterized by a host of progressively debilitating symptoms that can be life-threatening. People suffering from PKAN may experience movement disorders such as dystonia, a sustained muscle contraction leading to abnormal posture, rigidity, problems swallowing, and twisting and writhing. PKAN is estimated to affect up to 5,000 people worldwide.

Dube said the company will work closely with the study investigators to further analyze the results and share them with the PKAN community to “contribute to the growing knowledge of this rare disorder.”

As the company determines what went wrong with FORT, the company said it remains focused on progressing its two pivotal Phase III programs evaluating sparsentan for the treatment of focal segmental glomerulosclerosis and IgA nephropathy, and continuing the advancement of its commercial portfolio.


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