The US Food and Drug Administration (FDA) has reviewed Retrophin's investigational new drug (IND) application for RE-024, a new phosphopantothenate replacement therapy, and granted clearance to begin a Phase I clinical trial in healthy adult volunteers.
The trial is designed to evaluate safety, tolerability and pharmacokinetics of orally administered RE-024 to treat pantothenate kinase-associated neurodegeneration (PKAN).
Patient enrolment in the randomised, double-blind, placebo-controlled single ascending dose Phase I trial is expected to be initiated in the coming weeks.
PKAN is a rare and lethal autosomal recessive neurodegenerative disorder which is believed to affect about one to three persons per million worldwide and it currently has no approved treatment available.
Retrophin Research & Development executive vice-president and head Dr Alvin Shih said: "We are excited to see RE-024 move forward into the clinic and get one step closer to reaching patients who are suffering from this rare and debilitating disease.
"With the continued support of the scientific and patient communities, our research and development team will continue to rapidly advance RE-024 through clinical development with the hope of providing these patients with a much needed treatment option."
The trial's primary objective is to evaluate the safety and tolerability of single oral doses of RE-024 and as a secondary objective, pharmacokinetics of RE-024 will be assessed at multiple time points.
Following completion of the trial, safety and pharmacokinetic data will be submitted for publication.
The preclinical trials in PANK-deficient animal models and cell lines have showed the ability of RE-024 to restore Coenzyme A (CoA) levels.
The approved products of the company include Chenodal, Cholbam, and Thiola, and its pipeline comprises of compounds for several catastrophic diseases, including focal segmental glomerulosclerosis (FSGS), pantothenate kinase-associated neurodegeneration (PKAN), infantile spasms, nephrotic syndrome and others.