Repligen Corporation Files Investigational New Drug Application with FDA for First Drug Targeting the Core Genetic Defect of Friedreich's Ataxia

Published: May 13, 2010

WALTHAM, Mass., May 13 /PRNewswire-FirstCall/ -- 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.

About Friedreich's Ataxia

Friedreich's ataxia is characterized by limb ataxia, sensory polyneuropathy, hypertrophic cardiomyopathy and diabetes. At present, there are no effective treatments for Friedreich's ataxia and generally within 15 to 20 years after the first appearance of symptoms in adolescence, affected individuals are confined to a wheelchair. In later stages, patients become completely incapacitated and generally die in early adulthood from the associated heart disease, neuromuscular or diabetic complications. There are approximately 15,000 people worldwide with Friedreich's ataxia.

About Repligen Corporation

Repligen Corporation is a biopharmaceutical company focused on the development of novel therapeutics for neurological disorders. In addition, we are the world's leading supplier of recombinant Protein A, the sales of which partially fund the advancement of our development pipeline while supporting our financial stability. Repligen's corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453. Additional information may be requested from

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding the FDA's acceptance of the above-described IND for the Phase 1 study of RG2833, current or future financial performance and position, including our ability to finance the Phase 1 study of RG2833, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials, including trials utilizing RG2833, and results of such trials, plans and objectives for regulatory approval of our product candidates, including RG2833, litigation and intellectual property matters, including statements regarding the composition of matter patents covering RG2833, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of our clinical trials, the success of current and future collaborative relationships, the market acceptance of our products, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of product revenues and profits, our ability to generate future revenues, our ability to raise additional capital to continue our drug development programs, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited sales and manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in Repligen's filings with the Securities and Exchange Commission. Repligen assumes no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.

SOURCE Repligen Corporation

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