Repligen Corporation (NASDAQ:RGEN) announced today that it has enrolled its first patient in a Phase 1 clinical trial of RG2833 in adult patients with Friedreich’s ataxia (FA). FA is an inherited neurodegenerative disease caused by low levels of the protein frataxin which results in symptoms that typically present in childhood and lead to progressive loss of muscle and nerve function, often resulting in loss of life by early adulthood. RG2833 is an orally bioavailable, class 1 histone deacetylase inhibitor (HDACi) specifically designed to increase frataxin production in patients with FA. This study is being conducted in Turin, Italy and is the first clinical trial of a drug that targets the core genetic defect in FA.
The Phase 1 trial is a single ascending dose, crossover study in up to 20 adult FA patients. It is designed to evaluate the pharmacokinetic and safety profile of RG2833. Importantly, this study will also evaluate the pharmacodynamic response of RG2833 on various cellular and molecular biomarkers, including frataxin mRNA and frataxin protein.
“This Phase 1 trial in patients will generate valuable information on the safety and pharmacology of RG2833,” said Walter C. Herlihy, Ph.D., President and Chief Executive Officer of Repligen. “In addition, this study has the potential to provide early evidence of clinical activity for RG2833 in the treatment of Friedreich’s ataxia.”
“RG2833 is an attractive drug candidate, given its oral bioavailability and potential to target and activate the defective gene responsible for Friedreich’s ataxia,” said lead investigator Luca Durelli, M.D., Chief of Neurology at San Luigi Gonzaga University Hospital in Turin, Italy and lead investigator for the Phase 1 trial. “The effects of FA are devastating for our young patients and their families. I am happy to be involved with the study and to help address the critical need for a therapy that has the potential to slow disease progression.”