BioSante Pharmaceuticals, Inc. today announced positive GVAX AML Vaccine results and BioSante's receipt of Orphan Drug designation from the FDA's Office of Orphan Products Development for GVAX AML Vaccine in the treatment of acute myeloid leukemia (AML). This Orphan Drug designation is the second GVAX cancer vaccine from BioSante to secure FDA orphan drug designation.

In a paper published in the peer-reviewed journal Blood, clinical investigators, led by investigators at Johns Hopkins University, reported on the results of a Phase II study of GVAX AML (immunotherapy) accompanied by immunotherapy-primed lymphocytes after autologous stem cell transplantation in hematologic malignancies. Fifty-four subjects were enrolled, with 28 (52 receiving a pre-transplantation GVAX AML dose. A total of 46 (85%) subjects achieved complete remission during the treatment period. For all patients who achieved complete remission, the 3-year relapse-free survival (RFS) rate was 47.4% compared to 61.8% in the GVAX-treated group. While the overall survival rate in all subjects was 57.4%, it was 73.4% in the GVAX-treated group. The authors of the paper conclude, ".immunotherapy in combination with primed lymphocytes and autologous stem cell transplantation shows encouraging signals of potential activity in acute myeloid leukemia."

"The GVAX AML results reported in the medical journal Blood are very exiting. Those patients treated with GVAX had both a relapse-free survival and overall survival rate improvement of approximately 30% compared with the non-GVAX-treated subjects. The GVAX AML results reported today are in addition to the results recently reported for GVAX CML (Chronic Myeloid Leukemia)," said Stephen M. Simes, BioSante's president & CEO. "These encouraging efficacy data along with our second GVAX orphan drug designation should attract increased attention to our portfolio of GVAX cancer vaccines." The paper on GVAX AML results appeared in the peer-reviewed medical journal of the American Society of Hematology, Blood (2009; 114:1736-1745).

The Orphan Drug designation of GVAX Acute Myeloid Leukemia Vaccine for the treatment of acute myeloid leukemia is BioSante's second GVAX regulatory submission and response from the FDA since acquiring this portfolio of cancer vaccines last October. On March 15, BioSante reported receiving orphan drug designation for its GVAX Pancreas Vaccine to treat pancreatic cancer.

"It is our intention to find ways to continue the development of our GVAX cancer vaccines using the benefits conferred by gaining Orphan Drug designation. Further, these orphan drug designations alert the FDA that we are dedicated to bringing better cancer therapy to patients in need," Simes continued.

Currently, clinical trials of GVAX cancer vaccines against many different cancer types are being conducted, including leukemia, pancreatic cancer and breast cancer. It is estimated that approximately 12,810 individuals in the U.S. will be diagnosed with AML in 2010 and approximately 9,000 deaths will occur. AML is quite resistant to currently available treatments, and approximately 76% of these patients will die of their disease. The 3-year overall survival rate currently is about 20%.

The Orphan Drug Act (ODA) provides for granting special status to a product to treat a rare disease or condition upon request of a sponsor. The combination of the product to treat the rare disease or condition must meet certain criteria. This status is referred to as orphan designation. Orphan designation qualifies the sponsor of the product for a tax credit and seven years of marketing exclusivity of the ODA. A marketing application for a prescription drug product that has been designated as a drug for a rare disease or condition is not subject to a prescription drug user fee unless the application includes an indication for other than a rare disease or condition. The disease or condition for which the drug is intended affects fewer than 200,000 people in the United States or, if the drug is a vaccine, diagnostic drug, or preventive drug, the persons to whom the drug will be administered in the United States are fewer than 200,000 per year.