Repligen files IND for Phase 1 study of RG2833 selective HDAC-3 inhibitor for Friedreich's ataxia

Repligen Corporation (Nasdaq: RGEN) announced today that it has filed an Investigational New Drug Application (IND) with the Food and Drug Administration (FDA) for a Phase 1 study of RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor.  This 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.  This study will also evaluate the pharmacodynamic response of various biomarkers in peripheral blood to RG2833.  Pending FDA approval, this study will be the first step in the clinical development of RG2833 as a potential treatment for Friedreich's ataxia.

"We are very pleased to file this IND with the FDA," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation.  "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."

Friedreich's ataxia is an inherited neurodegenerative disease caused by a defect in a single gene that results in inadequate production of the protein frataxin.  Low levels of frataxin lead to degeneration of both the nerves controlling muscle movements in the arms and legs and the nerve tissue in the spinal cord.  Preclinical studies in animal models and patients' cells have shown that RG2833 crosses the blood brain barrier, activates the defective frataxin gene and increases production of the protein frataxin.  These results indicate that RG2833 may increase frataxin production and arrest disease progression in patients with Friedreich's ataxia.

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.  RG2833 has been developed in collaboration with scientists from The Scripps Research Institute and a broad network of international scientific thought leaders.  Repligen's research efforts have been partially funded with grants from the Muscular Dystrophy Association, the Friedreich's Ataxia Research Alliance, GoFAR and the National Ataxia Foundation.

SOURCE Repligen Corporation


  1. Joann Joann United States says:

    WOW! To Repligen! This spells real hope for families affected by the progressive illness, Friedreich's Ataxia. Keep up the good work and Thank-You!

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