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news.Repligen has enrolled its first patient in a Phase 1 clinical trial of RG2833 in adult patients with Friedreich's ataxia (FA).
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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.
Orally bioavailable, class 1 histone deacetylase inhibitor (HDACi) RG2833 is specifically designed to increase frataxin production in patients with FA.
Being conducted in Turin, Italy, the study is the first clinical trial of a drug that targets the core genetic defect in FA.
The Phase 1 trial includes single ascending dose, crossover study in up to 20 adult FA patients, to evaluate the pharmacokinetic and safety profile of RG2833.
The pharmacodynamic response of RG2833 on various cellular and molecular biomarkers, including frataxin mRNA and frataxin protein, will also be evaluated in this study.
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 said.
San Luigi Gonzaga University Hospital in Turin, Italy, chief of Neurology and the Phase 1 trial’s lead investigator said RG2833 is an attractive drug candidate, given its oral bioavailability and potential to target and activate the defective gene responsible for Friedreich’s ataxia.
"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," Durelli added.
Repligen Research and Development senior vice president R Rusche said if the company’s approach of using small molecules for protein replacement is successful, RG2833 has the potential to significantly improve outcomes for patients with FA.
"We are hopeful that the Phase 1 trial will elucidate the role for HDAC inhibition in FA, and inform future efficacy studies," Rusche added.