Aspreva Pharmaceuticals Corp (VICTORIA, British Columbia), an emerging pharmaceutical company focused on identifying, developing, and commercializing new indications for approved drugs and late-stage drug candidates for patients with less common diseases, and Roche (BASEL, Switzerland) released preliminary results from a phase III clinical trial comparing CellCept (oral mycophenolate mofetil, MMF) with intravenous cyclophosphamide (IVC), the current standard of care in patients suffering from lupus nephritis.

Although response rates were similar in both arms, 56.2% in the MMF arm and 53% in the IVC arm, the trial did not meet its primary objective of demonstrating that MMF was superior to IVC in inducing treatment response in patients after 24 weeks of induction therapy. The trial comprised 185 patients in the MMF arm and 185 in the IVC arm. Aspreva is conducting additional analyses to determine the potential for regulatory submission, and the company plans to present the final results at an appropriate scientific forum in the future.

In general, preliminary analysis indicates that the adverse events (AEs) experienced by patients in both arms of the study are consistent with those observed in lupus nephritis patients receiving immunosuppressive therapy. Overall incidence of AEs was comparable in both treatment arms.

Clinicians estimate that one third to one half of lupus patients have lupus nephritis. Current treatments involve the off-label use of existing cancer drugs such as cyclophosphamide, steroids, and other immunosuppressant drugs such as azathioprine.

CellCept is Roche's leading immunosuppressant and is used in combination with other immunosuppressive drugs (cyclosporine and corticosteroids) to prevent organ rejection in patients receiving heart, kidney, and liver transplants. 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. CellCept has not been approved by the US FDA for the treatment of any autoimmune disease.

—A. Techman