CytRx's Aldoxorubicin Shrinks Tumors, Prolongs Survival in Model of Human Brain Cancer

CytRx Corporation

, a biopharmaceutical research and development company specializing in oncology, today announced that aldoxorubicin, its more potent version of the widely used chemotherapeutic agent doxorubicin, demonstrated statistically significant efficacy (p<.0001) in the treatment of rapidly growing human brain (glioblastoma) cancer in the brains of animals. Complete results from this favorable confirmatory trial, which was conducted in collaboration with Louisiana State University (LSU) School of Medicine, will be presented at the European Society for Medical Oncology being held September 29-October 1 2013 in Amsterdam. “We are surprised and excited about the effectiveness demonstrated by aldoxorubicin in this particularly difficult-to-treat cancer,” said Om Prakash, Ph.D., the study's principal investigator and Research Professor of Medicine, Stanley S. Scott Cancer Center, Louisiana State University Health Sciences Center, New Orleans. “It has been well documented that doxorubicin, although active against glioblastoma cancer cells in tissue culture, does not cross the blood-brain barrier, the body's natural defense system protecting the brain, to effectively treat patients with brain tumors. In fact, in our study doxorubicin was no more effective than saline in suppressing glioblastoma tumor growth. We have shown that aldoxorubicin uptake is confined only to the tumor in the brain and does not enter normal brain tissue. Thus, we would expect toxicity in the central nervous system to be negligible. Our conclusion from this trial is that aldoxorubicin has the potential to safely shrink glioblastoma tumors which could dramatically prolong the average survival time in patients. We initially had observed a similar effect of aldoxorubicin on glioblastoma in a preliminary study, and are quite pleased to have confirmed the result in a larger, well-controlled study that included native doxorubicin.” Dr. Prakash's main focus of his research efforts in the last few years has been to understand the pathogenesis and treatment of glioblastoma multiforme, the most malignant and the most deadly type of brain tumor. He is the corresponding author on several poster presentations in national/international meetings. More recently, he is the first author on a publication, Gliomas and Seizures in the Medical Hypothesis Journal (Prakash et al. 2012; 79:622). “This trial produced remarkable results in a deadly cancer that virtually always returns regardless of whether treated with surgery, radiation, chemotherapy or a combination of methods,” said CytRx President and CEO Steven A. Kriegsman. “Animals treated with aldoxorubicin survived on average more than twice as long as those treated with saline or doxorubicin. “Aldoxorubicin could provide an exciting new approach in how we attack brain tumors. These outstanding results support our plan to initiate a Phase 2b clinical trial with aldoxorubicin in patients with relapsed glioblastoma. We remain on track with expanding our aldoxorubicin clinical development activities and expect our progress to accelerate in the coming months and year,” he added. If the data from the company's planned Phase 2b clinical trial for glioblastoma are positive, it plans to file for breakthrough therapy designation with the U.S. Food and Drug Administration, which could expedite marketing approval for aldoxorubicin. Aldoxorubicin has shown to be superior to doxorubicin in seven different tumor types and animal models of cancer, including ovarian, lung, breast and pancreatic cancer, as well as multiple myeloma, and has demonstrated activity in human trials for the treatment of soft tissue sarcomas and other cancers. Aldoxorubicin is the first drug candidate CytRx is developing based on a novel linker technology that has proven ability to allow attachment of multiple chemotherapeutic agents and is designed to provide both greater anti-cancer activity and to mitigate the toxicity that limits these agents' use.