A note about our Phase 3 clinical trial for PKAN
Last year we announced that the FORT Study, a Phase 3 clinical trial evaluating the safety and effectiveness of fosmetpantotenate for the treatment of pantothenate kinase-associated neurodegeneration (PKAN), did not meet either the primary or the secondary endpoint, and fosmetpantotenate did not show a benefit when compared to placebo. Please see the press release for more information.
After seeing the initial results, our medical and clinical teams worked to further analyze the data to understand if there were any age groups, differences in stage of PKAN progression, or signs that extending treatment for a longer period could produce a different outcome. Unfortunately, there was no evidence to suggest that fosmetpantotenate was having a positive impact in any of these additional analyses. Given these results, we made the difficult decision to discontinue the open-label extension of the FORT Study, as well as compassionate use protocols.
We are grateful to the patients, their caregivers, study investigators and our employees, whose dedication made this study possible. The patient community enabled us to genuinely recognize the significant unmet need this community faces with no approved treatment option.
We are finalizing plans to donate the full fosmetpantotenate program information and data to the PKAN community with the hope that this research may further the field’s understanding of the disease and contribute to others’ work in developing potential treatments.