Clinical Trials

Date: 2015-04-30

Type of information: Clinical trial autorization

phase: 1

Announcement: clinical trial authorisation

Company: Retrophin (USA - CA)

Product: RE-024 (fosmetpantotenate)

Action mechanism:

RE-024 is a small molecule discovered by Retrophin that is being developed as a replacement therapy for phosphopantothenate. Preclinical studies in PANK-deficient animal models and cell lines have demonstrated the ability of RE-024 to restore Coenzyme A levels. PKAN is caused by a mutation in the PANK2 gene, which encodes a critical protein that metabolizes vitamin B5 (pantothenate) to phosphopantothenate. The disruption of this metabolic pathway ultimately leads to decreased levels of CoA, an important substrate for many functions such as mitochondrial energy metabolism. Clinical manifestations of PKAN include developmental delay in children, dystonia sometimes causing intractable pain, parkinsonism with Parkinson’s-like freezing and bradykinesia, choreoathetosis, status dystonicus, dysarthria, spasticity, rigidity, and dysphagia often leading to feeding tube placement.

Disease: pantothenate kinase-associated neurodegeneration (PKAN)

Therapeutic area: Rare diseases - Neurodegenerative diseases - Genetic diseases

Country:

Trial details:

The Phase 1 trial is a randomized, double-blind, placebo-controlled single ascending dose study of orally administered RE-024 in healthy volunteer subjects. The primary objective of the study is to assess safety and tolerability of single oral doses of RE-024. As a secondary objective, pharmacokinetics of RE-024 will be assessed at multiple time points. Pending study completion, safety and pharmacokinetic data will be submitted for publication.

Latest news:

• On April 30, 2015, Retrophin announced that the FDA has reviewed the Investigational New Drug (IND) application for RE-024, a novel phosphopantothenate replacement therapy, and has granted clearance to begin clinical studies. The company is initiating a Phase 1 trial in healthy adult subjects to evaluate safety, tolerability, and pharmacokinetics, with enrollment expected to begin in the coming weeks. RE-024 is being developed for the treatment of pantothenate kinase-associated neurodegeneration (PKAN), a rare and lethal autosomal recessive neurodegenerative disorder believed to affect approximately one to three persons per million worldwide, with no approved treatment currently available.