FDA Grants Orphan Drug Designation for Novel Akt Inhibitor Vqd-002 In Multiple Myeloma

VioQuest Pharmaceuticals announced that the U.S. FDA has granted orphan drug designation to the company's product candidate VQD-002 (triciribine phosphate monohydrate or TCN-P) for the treatment of multiple myeloma. VQD-002 is a small molecule anticancer compound that inhibits protein kinase B (PKB or AKT), a key component of a signaling pathway known to promote cancer cell growth and survival as well as resistance to chemotherapy and radiotherapy. VioQuest Pharmaceuticals has reported preliminary Phase I results from ongoing studies in both solid tumors and hematological malignancies and the company plans to advance VQD-002 into Phase II development in 2008.

"Activation of the Akt pathway is increasingly being recognized as a critical factor promoting cancer cell survival and proliferation in multiple myeloma and many other cancers," commented Michael D. Becker, president and chief executive officer of VioQuest Pharmaceuticals. "By de-activating this pathway, VQD-002 has the potential to be an effective treatment for many cancers including multiple myeloma. Orphan drug designation significantly strengthens our opportunity to advance this promising product candidate to late stage clinical development and commercialization."

Orphan drug designation entitles VioQuest Pharmaceuticals to seven years of marketing exclusivity for VQD-002 upon regulatory approval, as well as the opportunity to apply for grant funding from the U.S. government to defray costs of clinical trial expenses, tax credits for clinical research expenses and potential waiver of the FDA's application user fee. Orphan status is granted by the FDA to promote the development of new drug therapies for the treatment of diseases that affect fewer than 200,000 individuals in the United States.

Preclinical studies in myeloma cell lines have demonstrated the synergy of VQD-002 alone and in combination with a range of therapeutic agents. These results support the initiation of clinical stage research. VioQuest Pharmaceuticals is exploring a range of clinical research study opportunities in collaboration with Oncotherapeutics, Inc. in California.

"Activation of the Akt pathway is a critical event in malignant transformation and survival, and its importance in multiple myeloma continues to emerge," commented James Berenson, M.D., president of Oncotherapeutics. "Inhibition of Akt has already been shown to cause cell death, reduce angiogenesis and reverse drug resistance in preclinical studies across various cancers. With its unique mechanism of action, coupled with the benign safety profile evidenced from ongoing Phase I studies, we are hopeful that VQD-002 will find a place in our cancer treatment armamentarium."

About VQD-002

VQD-002 (triciribine phosphate monohydrate, or TCN-P), a tricyclic nucleoside that inhibits the phosphorylation of Akt, has demonstrated anti-tumor activity against a wide spectrum of cancers in preclinical and clinical studies. Amplification, overexpression, or activation of Akt, also named protein kinase B, have been detected in a number of human malignancies, including prostate, breast, ovarian, colorectal, pancreatic, and hematologic cancers. Activation of Akt is associated with cell survival, malignant transformation, tumor invasiveness, and chemo-resistance, while inhibition of Akt activity has been shown to cause cell death. These attributes make Akt an attractive target for cancer therapy.