Fosmetpantotenate

Fosmetpantotenate was an investigational therapy being developed for the treatment of pantothenate kinase-associated neurodegeneration (PKAN), a devastating neurological disease. On August 22, 2019, we announced that the Phase 3 FORT Study evaluating the safety and efficacy of fosmetpantotenate compared to placebo in patients with pantothenate kinase-associated neurodegeneration (PKAN) did not meet its primary endpoint and did not demonstrate a difference between treatment groups. The study also did not meet its secondary endpoint. Please see the press release for more information.

We are grateful to the patients and caregivers, advocacy partners, researchers, and team members for their extraordinary work and collaboration over the years. 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.

PATIENT NEED

PKAN is a rare genetic condition estimated to affect up to 5,000 people worldwide. In many cases, the first symptoms of PKAN can be seen in early childhood. PKAN is the most common type of neurodegeneration with brain iron accumulation (NBIA). Symptoms include dystonia (sustained muscle contractions causing repetitive movements), muscular rigidity, cognitive impairment, and difficulty speaking. No FDA-approved treatment options exist for PKAN. Learn more about PKAN .