A POTENT ORAL THERAPY FOR CYTOMEGALOVIRUS INFECTION

DESCRIPTION (provided by applicant): Cytomegalovirus infection represents a major health concern in the immunocompromised population, causing a variety of serious and even life threatening disorders. The current standard of care for CMV infection, ganciclovir, suffers from complications of low oral bioavailability, bone marrow toxicity and emerging resistance in the clinic. Although two alternatives to ganciclovir exist, foscarnet and cidofovir, their own issues of severe renal toxicity limit them to use only in situations of ganciclovir failure. A new series of purine nucleoside analogues, the MP analogs, have been shown to be more potent and less toxic than ganciclovir. In addition, the MP analogs also exhibit increased activity against all gancicIovir resistant strains tested to date. Microbiotix, Inc. has exclusive license to the MP analogs and proposes to develop this class of compounds for the treatment of CMV disease. The focus of this Phase I Small Business Innovation Research (SBIR) application is the identification of a lead compound through a thorough investigation of in vitro efficacy and cytotoxicity. The applicants intend to advance this lead compound into pre-Investigational New Drug (IND) studies in the Phase II application.