Repligen Announces FDA and EMA Approval of Re-analysis of Images from Phase 3 Trial of RG1068 for Pancreatic Imaging

WALTHAM, Mass., May 27 /PRNewswire-FirstCall/ -- Repligen Corporation (Nasdaq: RGEN) announced today that the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have approved the Company's proposal to re-analyze the images from our Phase 3 study to establish the utility of RG1068, synthetic human secretin, in improving magnetic resonance imaging (MRI) of the pancreas (Phase 3 re-read).  The FDA and EMA have agreed to the Phase 3 re-read based on the numerous deficiencies with the analysis of the radiographic images by the contract research organization hired to oversee analysis of the Phase 3 data.  A successful re-read of the Phase 3 data may support registration of RG1068 for MRI imaging of the pancreas.  The goal of the Phase 3 study is to evaluate the sensitivity and specificity of RG1068 in combination with MRI to improve the detection of structural abnormalities of the pancreatic ducts relative to MRI alone.  Detailed visual assessment of the pancreatic ducts is important in the assessment, diagnosis and treatment of diseases such as acute and chronic pancreatitis.

"We are very pleased with the response of the FDA and EMA to our re-read proposal," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen.  "Based on the positive feedback we received from both regulatory agencies, we believe that a successful re-read may provide the basis for registration, and we have no plans to initiate additional clinical studies of RG1068 in this indication.  Pending finalization of the protocol, we anticipate completing the Phase 3 re-read by the end of the year."

Based on our discussions, we do not anticipate any changes in the primary endpoints or significant modifications to the statistical analysis plan for the study.  The EMA has requested that we add an additional secondary endpoint to document that RG1068 in combination with MRI imaging reduces the use of endoscopic retrograde cholangiopancreatography (ERCP).  ERCP is an invasive procedure used to diagnose and treat diseases of the pancreas and gallbladder.  ERCP is associated with significant morbidity, which has generated interest in the development of safer non-invasive tests to diagnose gastrointestinal disorders.  We believe that there is sufficient evidence to document that the use of RG1068 in combination with a non-invasive procedure such as MRI leads to the avoidance of unnecessary ERCP procedures.

In December, we reported the top-line results from our Phase 3, multi-center, baseline-controlled, single dose study in which 258 patients with a history of pancreatitis received an MRI of the pancreas with and without RG1068, and independently an ERCP.  The MRI images were randomized and independently reviewed by three radiologists for evaluation of pancreatic duct abnormalities, image quality, number of duct segments visualized and confidence in diagnosis.  The study's co-primary endpoints were an improvement in sensitivity of detection of structural abnormalities of the pancreatic ducts using RG1068 in combination with MRI compared to MRI alone with minimal loss in specificity (<7.5%).  The study design used ERCP as the standard for determination of structural abnormalities.  The predetermined criteria for a successful study were achievement of a statistically significant improvement in sensitivity with minimal loss in specificity from two of the three central radiologists reading the MRI images.  In this study, one radiologist achieved a statistically significant improvement in sensitivity with RG1068 (p<0.0001) while a second radiologist showed a trend but did not achieve statistical significance (p=0.088).  There was minimal loss in specificity for all radiologists.  The RG1068-MRI images showed highly statistically significant improvements on other endpoints for all radiologists including improvements in image quality (p<0.0001), ability to see all three segments of the pancreatic duct (p<0.0001), and physician confidence in their ability to identify pancreatic duct abnormalities (p<0.0001), when compared to MRI alone.  There were no serious adverse events (SAEs) associated with the RG1068-MRI procedures compared to 55 SAEs associated with the ERCP procedures.  The most commonly reported SAE following ERCP was acute pancreatitis requiring hospitalization.

The FDA has granted RG1068 Orphan Drug status and Fast Track Designation, a process designed to facilitate the development and expedite the review of drugs that treat serious diseases and fill an unmet medical need based on the need for safer non-invasive tests to diagnose pancreatic disorders.  There are more than 300,000 MRI procedures conducted in the U.S. and Europe each year that could directly benefit from the addition of RG1068.

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 www.repligen.com.

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 current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance such as the anticipated growth in the monoclonal antibody market and our other target markets and projected growth in product sales, 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 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, the success of our clinical trials, 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