AnorMED Inc has filed an Investigational New Drug Application (IND) with the U.S. Federal Drug Agency for approval to begin its clinical program for AMD-070, a novel therapeutic approach for the treatment of HIV infection. AMD-070 is a new type of anti-HIV agent that targets the CXCR4 cell receptor and prevents HIV from entering and infecting healthy cells. The filing is the first step in initiating a clinical program to assess the safety and efficacy of AMD-070 in healthy volunteers and HIV patients. AnorMED plans to start this clinical program in 2003 and is also exploring potential partnering opportunities for use of AMD-070 in combination with other HIV therapeutics.

"AMD-070 is a CXCR4 drug candidate in a new class of anti-HIV agents, called HIV entry inhibitors. The filing of an IND is a significant milestone for AnorMED. It represents the culmination of several years of very focused science and marks the transition from the laboratory into the clinic," said Dr. Michael Abrams, President and CEO of AnorMED Inc. He added, "We are excited about AMD-070 and its potential as part of a new paradigm in the treatment of HIV patients. Unlike many existing HIV drugs that target the virus after it has infected a healthy cell, HIV entry inhibitors such as AMD-070, block the virus from entering the cell and beginning its replication process".

CXCR4 is a chemokine receptor. Chemokine receptors expressed on the surface of immune cells are known to play a critical role in virus infection and transmission. CXCR4 and another chemokine receptor CCR5, are involved in HIV infection. In order to enter and infect cells, HIV must bind to either the CXCR4 or CCR5 chemokine receptor. Different strains of HIV prefer one receptor or the other, or may use either receptor to infect cells. An infected individual may harbor different levels of both CXCR4 and CCR5-using strains.

Both CXCR4 and CCR5 inhibitors belong to the new investigational class of antiretrovirals known as HIV entry inhibitors. This new class includes various experimental compounds designed to block cell surface receptors, such as CCR5 or CXCR4, as well as other novel compounds that block HIV fusion with the cell surface. AMD-070 targets CXCR4-using HIV strains.

Several classes of CXCR4 inhibitor molecules have emerged from AnorMED's preclinical program. From these, AMD-070 was selected to be the lead candidate in July 2002. AMD-070 has suitable pharmacokinetics for oral dosing and is synergistic with all other approved anti-HIV inhibitors. In addition, in vitro studies of AMD-070 have shown it to be a potent and specific CXCR4 antagonist which strongly inhibits viral infection against both CXCR4 using virus as well as HIV strains that use either CXCR4 or CCR5 (referred to as the dual tropic strain of HIV). AMD-070 is specific for the CXCR4 receptor and does not interact with any one chemokine receptor other than CXCR4, as evaluated in an in vitro analyses. This data was recently presented at the 11th Retroviral and Opportunistic Infections conference held in February 2003.