Retrophin has started dosing patients in a Phase III Fosmetpantotenate Replacement Therapy (FORT) clinical trial of RE-024 to treat patients with pantothenate kinase-associated neurodegeneration (PKAN).
RE-024 is an investigational, small molecule replacement therapy designed to restore the levels of a naturally occurring molecule called Coenzyme A (CoA) that is reduced in PKAN condition.
The product candidate is expected to pass through the blood-brain barrier and then convert to phosphopantothenic acid (PPA), which is an essential step for the CoA biosynthesis.
The international, randomised, double-blind, placebo-controlled, registrational Phase III trial will assess the safety and efficacy of RE-024 in up to 82 subjects aged six to 65 years.
Retrophin research and development senior vice-president and head Bill Rote said: "Individuals with PKAN face a life-threatening neurological disorder, for which there is no approved treatment options, and current therapeutic strategies are limited to symptom management.
"The dosing of the first patient in the Phase III FORT Study is an important milestone for the RE-024 programme, as it continues our progress towards developing a treatment with the potential to make a meaningful difference for the PKAN community."
The FORT trial’s primary endpoint is change in score from baseline through 24 weeks as measured by the Pantothenate Kinase-Associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale.
PKAN-ADL is a PKAN-specific, patient-reported outcome scale for the measure of motor abilities to function in daily lives of PKAN patients.
A previous Phase I trial demonstrated that RE-024 is safe and well-tolerated in healthy subjects.