Australian company receives FDA orphan drug designation for product to treat malignant melanomas

May 3 2004

Progen Industries Limited, an Australian anti-cancer drug development company, announced today that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for their lead anti-angiogenesis product PI-88 for treatment of malignant melanoma.

Orphan drug designation will provide PI-88 seven years market exclusivity when approved for this disease indication.

The U.S. Orphan Drug Act is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 individuals.

Additional criteria include the ability of the product to address unmet medical need where no approved treatment option exists or, the provision of significant benefit over other available treatments.

Under the Act, upon marketing authorization and during the period of market exclusivity, the FDA does not accept or approve other applications to market the same medicinal product for the same therapeutic indication.

In addition to potential market exclusivity, orphan drug status provides up to 50% in U.S. tax credits, grant funding for research and development and reduced filing fees, expedited review as well as advice on the conduct of clinical trials.

PI-88, is one of a new class of multi-targeted cancer therapeutics inhibiting both angiogenesis or tumor promoting factors such as Vascular Endothelial Growth Factor (VEGF), Fibroblast Growth Factors 1 and 2 (FGF-1 and -2) and heparanase, an enzyme implicated in tumor metastasis (tumor spread).

PI-88 is undergoing a Phase II clinical program in metastatic melanoma as a single agent therapy; advanced non-small cell lung cancer (NSCLC) in combination with chemotherapy (Taxotere(R)); as adjuvant treatment in post- operative primary liver cancer (imminent); and as a single agent therapy in multiple myeloma (completed).

"We believe the strength of PI-88 resides in its ability to inhibit both angiogenesis and metastasis simultaneously and we have seen evidence that this potential might be realized across a variety of cancers," stated Robert Don PhD, Progen's VP of Research and Development. "The FDA's orphan drug designation further strengthens our program for PI-88 by offering important accelerated clinical development and commercialization benefits.