Retrophin Announces Topline Results from Phase 3 FORT Study

Retrophin (NASDAQ:RTRX) has announced that the Phase 3 FORT study evaluating the safety and efficacy of fosmetpantotenate compared to a placebo in patients with pantothenate kinase-associated neurodegeneration (PKAN) didn’t meet its primary endpoints and did not show any differences between treatment groups.

As quoted in the press release:

“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,” said Eric Dube, Ph.D., 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.”

Data from the FORT Study will be further analyzed and are expected to be presented at an upcoming scientific congress, and the Company 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.

Consistent with its mission of identifying, developing and delivering life-changing therapies to people living with rare disease, Retrophin remains focused on progressing its two pivotal Phase 3 programs evaluating sparsentan for the treatment of focal segmental glomerulosclerosis (FSGS) and IgA nephropathy, and continuing the advancement of its commercial portfolio.

Click here to read the full press release.