Press Release: Aspreva Pharmaceuticals Corporation Says FDA Fast-Tracks CellCept Lupus Drug
Aspreva Pharmaceuticals Corporation Says FDA Fast-Tracks CellCept Lupus Drug VICTORIA, June 6 - Aspreva Pharmaceuticals Corporation today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for CellCept (mycophenolate mofetil, MMF) for the treatment of lupus nephritis. Aspreva is currently evaluating CellCept for the treatment of lupus nephritis in a global phase III study. The FDA's Fast Track designation is designed to expedite the application and review process for products that have the potential to address a serious or life-threatening condition. There has been no new approved treatment for lupus in the United States in over thirty years. In granting Fast Track designation, the FDA noted that type III-V lupus nephritis is a severe inflammation of the kidney associated with systemic lupus erythematosus, and is a life-threatening disease. They continued stating that the partnership between Aspreva and Roche shows a commitment to study clinically important outcomes including death, need of dialysis, or loss of renal function in this serious disease. Finally the FDA noted that CellCept received Fast Track designation on the basis that, given the current development program in lupus nephritis, it may have the potential to address an unmet medical need in patients with this disease. "Lupus nephritis is one of the most serious manifestations of lupus, and if left untreated can result in progressive kidney failure", said Dr. Usman Azam, Aspreva's Executive Vice-President and Chief Medical Officer. "We believe that Fast Track designation from the FDA will be of great assistance in our efforts to bring an evidence-based treatment option to this patient population where there is a clear unmet medical need." Aspreva's lupus nephritis study is one of the largest phase III studies ever conducted in this disease. This two-phase induction to maintenance study was designed as a randomized open-label comparison of MMF with intravenous cyclophosphamide for the first six months (induction phase), followed by a double-blind comparison of MMF to azathioprine for up to three years (maintenance phase). The first patient of this study was treated in July 2005 and patient enrollment was completed at the end of September 2006. Preliminary results from the induction phase of this trial are expected towards the end of June 2007. Re-randomization into maintenance phase is complete and this phase of the study is ongoing. About Lupus Nephritis Systemic lupus erythematosus (SLE), commonly called lupus, is a chronic autoimmune disease that causes the body to attack its own tissues and joints. Lupus nephritis is the most serious manifestation of the disease, which, if left untreated, can lead to kidney failure, requiring dialysis. It is a complicated disease as patients typically fluctuate between periods of intense disease activity when the patient's own immune system is actively attacking and causing damage in their kidney, interspersed with periods of remission. Clinicians estimate that one third to one half of lupus patients have lupus nephritis. There has been no new approved treatment for SLE or lupus nephritis in the United States in over thirty years. Current treatments involve the off-label use of existing cancer drugs such as cyclophosphamide, steroids, and other immunosuppressant drugs such as azathioprine. It is important to note that MMF is not currently approved by the FDA for the treatment of any autoimmune disease. About CellCept CellCept is Roche's leading immunosuppressant or "anti-rejection" drug used in combination with other immunosuppressive drugs (cyclosporine and corticosteroids) for the prevention of rejection in patients receiving heart, kidney and liver transplants. CellCept was first approved for use in combination therapy for the prevention of acute organ rejection in kidney transplantation in 1995 and has since been approved worldwide for prevention of organ rejection in adult kidney, heart and liver transplantation. In some countries, it has also been approved for paediatric kidney transplantation. This therapeutic success represents 11 years of clinical experience and patient benefits, including reduced toxicities and prolonged graft and patient survival. Over the last decade, CellCept has become the world's most widely studied immunosuppressant and research is ongoing both in organ transplantation and related areas, such as autoimmune disease, to help provide clinical benefit to a wider range of patients. In July 2003, Aspreva signed a collaboration agreement with Roche for the exclusive worldwide rights (excluding Japan) to develop and, upon regulatory approval, commercialize CellCept for all autoimmune disease applications. About Aspreva Pharmaceuticals Aspreva is a global pharmaceutical company focused on identifying, developing, and, upon approval, commercializing evidence-based medicines for patients living with less common diseases. Aspreva common stock is traded on the NASDAQ Global Select Market under the trading symbol "ASPV" and on the Toronto Stock Exchange under the trading symbol "ASV". Learn more at www.aspreva.com. This news release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995 and forward-looking information within the meaning of applicable securities laws in Canada (collectively, "forward-looking statements"). The words "anticipates", "believes", "budgets", "could", "estimates", "expects", "forecasts", "intends", "may", "might", "plans", "projects", "schedule", "should", "will", "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this news release include, but are not limited to, statements about: our expectations with respect to the Fast Track designation; our strategy, future operations, clinical trials, prospects and plans of management; the effects of CellCept on patients; our expectations with respect to our existing collaboration agreement with Roche for the development of CellCept in autoimmune indications; and our phase III clinical program underway with CellCept: for the treatment of lupus nephritis. With respect to the forward-looking statements contained in this news release, we have made numerous assumptions regarding, among other things: our ability to predict the effects of CellCept on patients; our ability to continue our phase III clinical program underway with CellCept: for the treatment of lupus nephritis; our ability to protect our intellectual property rights and to not infringe on the intellectual property rights of others; our ability to comply with applicable governmental regulations and standards; and our ability to succeed at establishing a successful commercialization program for any of our potential products. Readers are cautioned that the plans, intentions or expectations disclosed in any forward-looking statements and underlying assumptions may not be achieved and that they should not place undue reliance on any forward-looking statement. Actual results or events could differ materially from the plans, intentions, expectations, and assumptions expressed or implied in any forward-looking statements as a result of numerous risks, uncertainties and other factors, including those relating to: difficulties or delays in the progress, timing and results of clinical trials and studies; difficulties or delays in obtaining regulatory approvals; the FDA may determine that the design and planned analysis of our clinical trials do not adequately address the trial objectives in support of our regulatory submission; future sales of CellCept may be less than expected; our future operating results are uncertain and likely to fluctuate; we may not be able to develop and obtain regulatory approval for CellCept in the treatment of autoimmune indications and any future products in our targeted indications; we may not be able to establish marketing and sales capabilities and the costs of launching CellCept in the treatment of autoimmune indications and any future products for our targeting indications may be greater than anticipated; the risk that we may not sustain our profitability; our ability to attract and retain collaborations relating to the development and commercialization of new indications; competition from other pharmaceutical or biotechnology companies; our ability to raise additional financing required to fund further research and development, clinical studies, and obtain regulatory approvals, on commercially acceptable terms or at all; economic and capital market conditions; our ability to obtain and protect patents and other intellectual property rights; our ability to operate without infringing the intellectual property rights of others; our ability to comply with applicable governmental regulations and standards; currency exchange rates; and our ability to successfully attract and retain skilled and experienced personnel. For a more thorough discussion of the risks associated with Aspreva's business, see the "Risk Factors" section in Aspreva's Quarterly Report on Form 10-Q for the quarter ended March 31, 2007, filed with the U.S. Securities and Exchange Commission at www.sec.gov and with securities regulatory authorities in Canada at www.sedar.com. Although we have attempted to identify important risks, uncertainties and other factors that could cause actual results or events to differ materially from those expressed or implied in the forward-looking statements, there may be other factors that cause actual results or events to differ from those expressed or implied in the forward-looking statements. All forward-looking statements are qualified in their entirety by this cautionary statement and Aspreva undertakes no obligation to revise or update any forward-looking statements as a result of new information, future events or otherwise after the date hereof.