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Orphan Drug designation for Tocosol Paclitaxel in bladder cancer indication

BOTHELL, WASH., January 27, 2005 -- Sonus Pharmaceuticals announced that the FDA has granted an Orphan Drug designation to its lead oncology candidate, Tocosol Paclitaxel, for the treatment of non-superficial urothelial cancer. The most common form of urothelial cancer is bladder cancer.

The Orphan Drug designation adds to the Fast-track designation awarded in 2003 by the FDA Division of Oncology Drug Products for the development of Tocosol Paclitaxel for the treatment of metastatic or locally advanced, inoperable transitional cell carcinoma (TCC) of the urothelium. Tocosol Paclitaxel is a novel formulation of paclitaxel, a widely prescribed anti-cancer drug for the treatment of solid tumors.
In parallel with developing Tocosol Paclitaxel for the treatment of bladder cancer, Sonus is pursuing initial market entry through a 505(b)(2) New Drug Application in an indication for which paclitaxel-based products have already been approved, e.g. breast, ovarian or lung cancers. The Company is currently in discussions with the FDA to finalize the pivotal Phase 3 protocol for Tocosol Paclitaxel, which Sonus believes will be initiated in 2005.

The Company is conducting Phase 2b bladder cancer studies in Cleveland, Philadelphia, Baltimore and Seattle, and will soon be opening sites in Spain and the U.K. In addition to the bladder cancer program, Sonus is also conducting a Phase 2b study of Tocosol Paclitaxel in patients with metastatic breast cancer.

Sonus has completed patient enrollment in Phase 2a studies of Tocosol Paclitaxel in bladder, ovarian and lung cancer patients, and these trials have generated encouraging data on safety and anti-tumor activity. The Company reported results of the Phase 2a studies in three presentations at the annual meeting of the American Society of Clinical Oncology (ASCO) in 2004. Tocosol Paclitaxel offers the convenience of a ready-to-use formulation, which does not require reconstitution, dilution or pharmacy preparation as is required with the marketed paclitaxel products. Additionally, Tocosol Paclitaxel is administered to patients in a short 15-minute infusion, compared to the three-hour infusion required with Taxol, the innovator paclitaxel product.

The FDA's Fast Track program is intended to facilitate the development and review of new drugs intended to treat serious or life-threatening conditions, and for which the product has the potential to address an unmet medical need.

Source: Sonus Pharmaceuticals

Posted: January 2005