Repligen Initiates Phase 1 Clinical Trial Of RG2833 In Patients With Friedreich’s Ataxia

Repligen Corporation (NASDAQ:RGEN) announced today that it has enrolled its first patient in a Phase 1 clinical trial of RG2833 in adult patients with Friedreich’s ataxia (FA). 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. RG2833 is an orally bioavailable, class 1 histone deacetylase inhibitor (HDACi) specifically designed to increase frataxin production in patients with FA. This study is being conducted in Turin, Italy and is the first clinical trial of a drug that targets the core genetic defect in FA.

The Phase 1 trial is a single ascending dose, crossover study in up to 20 adult FA patients. It is designed to evaluate the pharmacokinetic and safety profile of RG2833. Importantly, this study will also evaluate the pharmacodynamic response of RG2833 on various cellular and molecular biomarkers, including frataxin mRNA and frataxin protein.

“This Phase 1 trial in patients will generate valuable information on the safety and pharmacology of RG2833,” said Walter C. Herlihy, Ph.D., President and Chief Executive Officer of Repligen. “In addition, this study has the potential to provide early evidence of clinical activity for RG2833 in the treatment of Friedreich’s ataxia.”

“RG2833 is an attractive drug candidate, given its oral bioavailability and potential to target and activate the defective gene responsible for Friedreich’s ataxia,” said lead investigator Luca Durelli, M.D., Chief of Neurology at San Luigi Gonzaga University Hospital in Turin, Italy and lead investigator for the Phase 1 trial. “The effects of FA are devastating for our young patients and their families. 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.”

Friedreich's ataxia is caused by a single gene defect that results in inadequate production of the frataxin protein. Low levels of frataxin impair the function of nerves coordinating muscle movements in the arms and legs and the nerve tissue in the spinal cord and can lead to a life-shortening cardiomyopathy. RG2833 is a Class 1 HDAC inhibitor that has been designed to upregulate the frataxin gene and has been shown in preclinical studies in animal models and patients’ cells to increase production of this key protein. These results indicate that RG2833 may increase frataxin production in patients and has the potential to be an important treatment for Friedreich’s ataxia.

“Friedreich’s ataxia disease biology provides evidence that a small increase in expression of the defective gene could potentially slow disease progression,” said James R. Rusche, Ph.D., Senior Vice President, Research and Development at Repligen. “RG2833 is the first compound that targets activation of this defective gene. If our unique approach of using small molecules for protein replacement is successful, it 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.”

RG2833 is a new chemical entity that is the subject of a composition of matter patent and will remain in force until 2029 prior to any patent term extensions. Portions of this clinical trial are supported by a grant from the Italy based patient advocacy group GoFAR. Repligen’s additional research efforts in FA have been partially funded with grants from the Friedreich’s Ataxia Research Alliance (FARA), GoFAR, the Muscular Dystrophy Association, the European Friedreich’s Ataxia Consortium for Translations Studies (EFACTS) and the National Ataxia Foundation (NAF). RG2833 has been developed in collaboration with scientists from The Scripps Research Institute and a broad international network of scientific thought leaders. Repligen is also evaluating other HDAC inhibitors in animal models of Huntington’s disease and cognition.

Orphan Drug Designations

Repligen has previously received U.S. Orphan Drug and European Orphan Medicinal Product designations for RG2833 for the treatment of Friedreich’s ataxia. Both orphan programs provide incentives for the research, development and marketing of products intended to diagnose, prevent or treat rare conditions and/or serious or debilitating diseases with unmet medical needs. Orphan designation grants the sponsor exclusive marketing rights for seven years in the U.S. and ten years in the EU following regulatory approval of the designated product.

In the U.S., rare diseases are defined as those affecting fewer than 200,000 Americans. In the EU, rare diseases are considered those that affect no more than five per ten thousand persons in the community. The Company does not have active clinical trials of RG2833 in the U.S. at this time.

About Friedreich’s Ataxia

Friedreich’s ataxia is an inherited neurodegenerative disease caused by a single gene defect that results in inadequate production of the protein frataxin. Symptoms of FA typically emerge between the ages of five and 15 and include loss of strength and coordination in the arms and legs, vision impairment, hearing loss and slurred speech. Symptoms often progress to severe disability and incapacitation requiring wheelchair use. Survival for individuals with FA is generally 15 to 20 years after the first appearance of symptoms. Patients generally die in early adulthood from the associated cardiovascular, neuromuscular and/or diabetic complications. There are approximately 15,000 individuals worldwide living with Friedreich’s ataxia, however there are no available treatments today.

About our External Collaborators

Repligen’s FA program continues to be supported by strong relationships with and funding from non-profit organizations including GoFAR, Friedreich’s Ataxia Research Alliance (FARA), the Muscular Dystrophy Association (MDA), the European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS) and the National Ataxia Foundation (NAF).

GoFAR - Friedreich’s Ataxia Research is an Italian, non-profit organization dedicated to facilitating and promoting scientific research for the treatment of Friedreich’s ataxia. GoFAR has provided a $500,000 grant to support the operations of this clinical study and provides additional logistics support for participating families. GoFAR has also collaborated with Repligen by coordinating patients and families to participate in non-treatment research that has been important for developing clinical biomarkers of drug treatment. For more information, go to

The Friedreich’s Ataxia Research Alliance (FARA) is a national, public, non-profit, tax-exempt organization dedicated to the pursuit of scientific research leading to treatments and a cure for Friedreich’s ataxia. FARA’s mission is to slow, stop, and reverse the damage caused by this disorder. For more information, go to FARA has also participated in scientific collaborations by organizing patient participation in research studies associated with drug development.

The Muscular Dystrophy Association (MDA) is the nonprofit health agency dedicated to curing muscular dystrophy, ALS, FA and related diseases by funding worldwide research. The Association also provides comprehensive health care and support services, advocacy and education. For the latest research news and information about diseases in MDA’s program, visit and follow MDA on Facebook ( and Twitter (@MDAnews).

The European Friedreich’s Ataxia Consortium for Translational Studies (EFACTS) is a European Community funded collaboration of 15 centers supported through the fp7 initiative to coordinate basic research and develop a clinical network for the advancement of treatments for FA. Repligen is the only non-EU member.

The National Ataxia Foundation (NAF) is dedicated to improving the lives of persons affected by ataxia through support, education, and research. NAF is a membership supported, nonprofit organization established in 1957 to help persons with ataxia and their families. The Foundation's primary purpose is to support promising ataxia research and to provide vital programs and services for ataxia families. For more information go to

About Repligen Corporation

Repligen Corporation is a leading supplier of critical biologic products used to manufacture biologic drugs. Repligen also applies its expertise in biologic product development to SecreFlo™, a synthetic hormone being developed as a novel imaging agent for the diagnosis of a variety of pancreatic diseases. In addition, the Company has two central nervous system (CNS) rare disease programs in Phase 1 clinical trials. Repligen’s corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453. Additional information may be requested at

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. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, express or implied statements regarding future financial performance and position, the Food and Drug Administration approval of our NDA for SecreFlo, plans and objectives for future operations, plans and objectives for product development, plans and objectives for regulatory approval, product development, our market share and product sales and other statements identified by words like “believe,” “expect,” “may,” “will,” “should,” “seek,” or “could” and similar expressions, 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: our ability to develop and commercialize products and the market acceptance of our products; reduced demand for our products that adversely impacts our future revenues, cash flows, results of operations and financial condition; the ability to obtain, and the timing and receipt of, FDA approval for our NDA; our ability to obtain other required regulatory approvals; the success of current and future collaborative or supply relationships; our ability to compete with larger, better financed bioprocessing, pharmaceutical and biotechnology companies; the success of our clinical trials; new approaches to the treatment of our targeted diseases; our compliance with all Food and Drug Administration and EMEA 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 capabilities; our volatile stock price; and other risks detailed in Repligen’s annual report on Form 10-K on file with the Securities and Exchange Commission and the other reports that Repligen periodically files with the Securities and Exchange Commission. Actual results may differ materially from those Repligen contemplated by these forward-looking statements. These forward looking statements reflect management’s current views and Repligen does not undertake to update any of these forward-looking statements to reflect a change in its views or events or circumstances that occur after the date hereof except as required by law.

Copyright Business Wire 2010

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