WALTHAM, Mass.--(BUSINESS WIRE)--Repligen Corporation (NASDAQ: RGEN) reported today positive top-line results from a Phase 3 study to evaluate the safety and efficacy of RG1068, synthetic human secretin, to improve magnetic resonance imaging (MRI) of the pancreas in patients with pancreatic disease using endoscopy (ERCP) as a diagnostic reference. The study’s co-primary endpoints were achievement of a statistically significant improvement in sensitivity of detection of abnormalities with a loss in specificity of less than 7.5% by two of the three central radiologists reading the MRI images. In this study, all three radiologists achieved a clinically and statistically significant improvement in sensitivity (all radiologists p<0.0001) with minimal loss in specificity (all radiologists <7.5%). In addition, the RG1068-MRI images showed highly significant improvements on image quality (all radiologists p<0.0001), and confidence in the diagnostic findings (all radiologists p<0.0001), when compared to MRI alone. There were no serious adverse events (SAEs) or unscheduled days of hospitalization associated with the RG1068-MRI procedures compared to 55 SAEs and 236 days of unscheduled hospitalization associated with the ERCP procedures.
“This study demonstrated that RG1068 improves the identification of patients with pancreatic disease and allows the radiologist to render a more highly confident diagnosis to the gastroenterologist,” stated Dr. Stuart Sherman, M.D., Professor of Medicine and Radiology at Indiana University School of Medicine and Clinical Director of Gastroenterology and Hepatology and the principal investigator of this study. “RG1068 has the potential to significantly increase a physician’s ability to confidently triage their patients, to improve planning for indicated ERCP and pancreatic surgery and will be an important aid in avoiding unnecessary ERCP procedures which are potentially harmful for patients.”
“We are pleased with the strongly positive results of this study which support the use of RG1068-MRI as a safe, non-invasive approach to the identification of pancreatic abnormalities,” stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. “We intend to request a meeting with the FDA to review these results and to discuss our plan to file a New Drug Application.”
This was a multi-center, baseline controlled, single dose study in which 258 patients enrolled at 29 clinical sites within the U.S. and Canada received an MRI of the pancreas with and without RG1068, and separately an ERCP. The MRI images were randomized and independently reviewed by three radiologists for evaluation of the presence or absence of 10 pre-specified pancreatic duct abnormalities, image quality, visualization of the main pancreatic duct and confidence in diagnostic findings. 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 diagnostic reference for determination of structural abnormalities.
The analysis of the Phase 3 radiographic images was a “re-read” which was agreed to by the Food and Drug Administration and European Medicines Agency based on the determination that the original analysis was flawed and therefore inconclusive due to deficiencies in performance by the contract research organization overseeing the original analysis. A new contract research organization and three new radiologists were hired to conduct the re-read.
RG1068, a synthetic version of human secretin, stimulates the secretion of pancreatic fluid into the pancreatic ducts, thereby filling the ducts with water, which improves the ability to visualize pancreatic abnormalities. Identification of pancreatic duct abnormalities is important in the identification of cause and potential treatment options for diseases such as pancreatitis. 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 approximately 300,000 MRI procedures conducted in the U.S. and Europe each year that could directly benefit from the addition of RG1068.
Conference call This Afternoon at 4:30 p.m. EDT
Repligen’s President and Chief Executive Officer, Walter C. Herlihy, Ph.D., and Dr Stuart Sherman, M.D., the principal investigator of the Phase 3 study, will host a conference call and webcast today, Monday March 14, 2011 at 4:30 p.m. EDT, to discuss the Phase 3 trial results. This call is being webcast with slides by Thomson/CCBN and can be accessed via a link on the Investor Relations section of Repligen’s website at www.repligen.com. You may also listen to the audio portion of the broadcast by calling (800) 299-9086 for domestic calls and (617) 786-2903 for international calls. Participants must provide the following passcode: 58319328. The webcast of the conference call can be accessed via Repligen’s website www.repligen.com. The conference call will include a Q&A session, if you would like to participate in the Q&A session, please listen to the live broadcast through the telephone. The slides accompanying the conference call can be viewed via the webcast link on Repligen’s website. A replay of the call will be available on Repligen’s website promptly following the event.
About Repligen Corporation
Repligen Corporation is a biopharmaceutical company focused on building an integrated company by developing and marketing innovative drugs that deliver the benefits of protein therapies in the fields of neurology and gastroenterology. We have a core competency in the development and manufacturing of biologics products, which is the basis for our bioprocessing business and we have out-licensed certain biologics intellectual property, which provide ongoing sources of revenue. Repligen’s corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453. Additional information may be requested at 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. Investors are cautioned that statements herein which are not strictly historical statements, including, without limitation, expressed or implied statements regarding the regulatory approval of RG1068 and any related clinical development plans, our funding of the regulatory advancement and commercialization of RG1068, the commercial potential of RG1068, if approved, the possibility of seeking a collaboration partner for RG1068, our future financial performance and position, management’s strategy, plans and objectives for future operations, 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: the success of our future clinical trials; our ability to obtain required regulatory approvals; the success of current and future collaborative relationships; our ability to raise additional capital to continue our drug development programs; our ability to obtain, maintain and protect intellectual property rights for our products and product candidates; our ability to successfully develop and commercialize products, including the market acceptance of our products; 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. 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 publication of this press release, except as required by law.