Repligen said that the study is a double-blind, single ascending dose, Phase 1 study in healthy volunteers to evaluate the pharmacokinetic and safety profile of RG2833 in up to 40 subjects. The study is also expected to evaluate the pharmacodynamic response of various biomarkers in peripheral blood to RG2833.
RG2833 is a new chemical entity which is the subject of a composition of matter patent application which if allowed, will remain in force until 2029 prior to any patent term extensions. Repligen is also evaluating HDAC-3 inhibitors in animal models of Huntington’s disease and cognition.
Walter Herlihy, president and CEO of Repligen, said: “We are very pleased to file this IND with the FDA. RG2833 is an orally bioavailable compound that targets activation of the defective gene responsible for Friedreich’s ataxia. If this therapeutic approach is successful, it has the potential to change progression of the disease and significantly impact patients’ lives.”