Repligen Corporation has enrolled the first patient with Friedreich's ataxia (FA) in its Phase 1 clinical trial of RG2833, an orally bioavailable, class 1 histone deacetylase inhibitor (HDACi).
The Phase 1 trial is a single ascending dose crossover study intended to investigate the pharmacokinetic and safety profile of the RG2833 drug in up to 20 adult FA patients.
RG2833 has received US Orphan Drug and European Orphan Medicinal Product designations for the treatment of Friedreich's ataxia.
Friedreich's ataxia is an inherited neurodegenerative disease caused by a single gene defect that results in inadequate production of the frataxin protein, thereby impairing the function of nerves coordinating muscle movements in the arms and legs and the nerve tissue in the spinal cord.
Symptoms of FA range from loss of strength and coordination in the arms and legs, vision impairment, hearing loss and slurred speech to severe disability and incapacitation requiring wheelchair use.
Repligen president and chief executive officer Walter Herlihy said the Phase 1 trial in patients will generate valuable information on the safety and pharmacology of RG2833.
"In addition, this study has the potential to provide early evidence of clinical activity for RG2833 in the treatment of Friedreich's ataxia," Herlihy added.
Repligen Research and Development senior vice president James Rusche said: "RG2833 is the first compound that targets activation of this defective gene. If our unique approach of using small molecules for protein replacement is successful, it has the potential to significantly improve outcomes for patients with FA."
The company's FA programme is being funding by non-profit organisations including GoFAR, Friedreich's Ataxia Research Alliance (FARA), the Muscular Dystrophy Association (MDA), the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS) and the National Ataxia Foundation (NAF).
Repligen applies its expertise in biologic product development to SecreFlo, a synthetic hormone for the diagnosis of a variety of pancreatic diseases.