Repligen, Pharmacology data and biomarker activity from our Phase 1 trial in Friedreich's ataxia (FA) patients of our product candidate, RG2833,

Repligen, Pharmacology data and biomarker activity from Phase 1 trial in Friedreich's ataxia (FA) patients of the product candidate, RG2833.. Reuters, Thu Aug 1, 2013

We analyzed the pharmacology data and biomarker activity from our Phase 1 trial in Friedreich's ataxia (FA) patients of our product candidate, RG2833, a compound that interferes with the function of the histone deacetylase (HDAC) class of enzymes. The trial results show that RG2833 was well tolerated and able to improve the expression of frataxin - a disease biomarker – in a dose-dependent manner. While there were no adverse events reported, the generation of potentially toxic metabolites was observed. Overall, the Phase 1 results support the use of an oral HDAC inhibitor in the treatment of FA, however we believe that other compounds in our HDAC inhibitor portfolio may be more suitable than RG2833 for clinical development. Consistent with our focus on bioprocessing, we are seeking non-profit and biopharmaceutical development partners to support future development.