Apr 18, 2002
PR Newswire - USA
Repligen Corporation announced today that it has received Medicines Control Agency (MCA) approval to conduct a clinical trial in the United Kingdom to assess the safety and efficacy of CTLA4-Ig in patients with refractory auto-immune thrombocytopenic purpura (ITP). A total of 12 patients with ITP will be treated in a Phase 1/2 open label, dose escalating clinical trial. In 2001, we completed a Phase 1 clinical trial of intravenously administered CTLA4-Ig in 12 normal adults who received escalating doses of CTLA4-Ig in which no serious adverse events or toxicities were observed.
"We are pleased to initiate this trial which will serve to evaluate the in vivo activity of CTLA4-Ig in an auto-immune disease patient population," said Walter C. Herlihy, President and CEO of Repligen. "If successful, we believe that CTLA4-Ig may additionally be useful in treating other auto-immune diseases such as multiple sclerosis and rheumatoid arthritis."
ITP is an auto-immune disease in which the body makes antibodies that target a patient's own blood platelets resulting in premature platelet destruction and an impaired ability to form blood clots. The clinical consequences of ITP are largely dependent on platelet count and range from bruising, mucosal bleeding and nosebleeds to intracranial hemorrhage, which can be fatal. There are approximately 25,000 people in the United States with refractory ITP. CTLA4-Ig is being evaluated in this patient population for its ability to interrupt the body's attack on the platelets. Clinical outcome will be monitored by change in platelet number following treatment.
Repligen also announced today that The United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance on a patent application entitled "CTLA4-Cgamma4 Fusion Proteins." A Notice of Allowance is the final step in the patent prosecution process, which indicates that the USPTO will issue the patent. The patent, which will remain in force until 2016, covers the specific composition of the Company's CTLA4-Ig product form. A CTLA4- Cgamma4 fusion protein is a genetically engineered form of CTLA4 which is comprised of natural sequence CTLA4 fused to a portion of an immunoglobulin (Ig) to make a soluble form of CTLA4. The molecule covered by the patent has additionally been engineered to eliminate those portions of the Ig molecule that have the potential to induce certain undesirable natural biological mechanisms, including cell lysis or cellular toxicity which may cause side effects in a patient.
Repligen and the University of Michigan (the University) previously filed a complaint against Bristol-Myers Squibb in the United States District Court for the Eastern District of Michigan seeking correction of inventorship on certain CTLA4 related patents issued to Bristol-Myers Squibb. The suit seeks to add an inventor whose rights are assigned to the University of Michigan who was engaged in collaboration with Bristol-Myers Squibb involving, among other things, CTLA4. A correction of inventorship would result in the University being designated as a co-assignee on any corrected patent. Repligen is the exclusive licensee of the University of Michigan's CTLA4 patent rights. Both Repligen and Bristol-Myers Squibb have filed motions for summary judgement. Repligen's motion requests correction of inventorship on certain Bristol-Myers Squibb patents and Bristol-Myers Squibb's motion requests dismissal of the complaint. A hearing on these motions is scheduled for the end of May during which the judge will question both parties' counsel concerning their summary motions in which each has substantiated their position for judgement based on evidence collected during the investigation of the complaint. This hearing is part of the customary legal process, following which the judge is expected to make a decision on the motions and/or on the scheduling of the trial.
About CTLA4-Ig for Auto-Immune Disease
CTLA4 is a T cell regulatory protein which is one of the immune system's natural "off switches." We have developed a soluble form of CTLA4 (CTLA-Ig) which has immunosuppressive activity for use in organ transplantation and auto-immune diseases. CTLA4-Ig has the potential to inactivate only those cells that are initializing an unwanted immune response without compromising the body's ability to fight off infections. A form of CTLA4-Ig has been shown to be active in several clinical and non-clinical studies and was recently reported by Bristol-Myers Squibb to have activity in a Phase 2 clinical trial in 214 patients with refractory rheumatoid arthritis.
About Repligen Corporation
Repligen Corporation develops new drugs for debilitating pediatric disorders including autism, immune and metabolic disorders. Repligen has exclusive worldwide rights to market SecreFlo(TM), which was recently approved by the FDA for pancreatic assessment and manufactures and markets a set of patented products based on Protein A, which are used by the pharmaceutical industry to produce therapeutic antibodies. Its corporate headquarters are located at 117 Fourth Avenue, Needham MA, 02494. Additional information may be requested from http://www.repligen.com/.
This release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 21E of the Securities Exchange Act of 1934. 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, management's strategy, plans and objectives for future operations, clinical trials and results and product plans and performance, 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 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, 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.
Contact: Walter C. Herlihy, Ph.D., President and Chief Executive Officer of Repligen Corporation, +1-781-449-9560, ext. 2000; or Jonathan Fassberg (investors) of The Trout Group, +1-212-477-9007, ext. 16
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