The University of Southern California (USC) is collaborating with Auransa Inc., on a phase 1 clinical trial to evaluate a new kind of treatment for cancers of the liver and solid tumors with liver dominant disease. The drug, known as AU409, was developed by Auransa, a clinical stage drug development company focused on identifying novel drug candidates for oncology, inflammatory diseases and diseases of the central nervous system. In preclinical trials, AU409, has been shown to work in a unique fashion by limiting the cancer cell’s ability to translate the message from various genes that are essential for the cancer cell’s ability to survive and multiply. In other words, AU409 interrupts the cancer cell’s machinery, preventing it from using DNA code to produce proteins that it needs to grow.

Liver cancer is estimated to be the third most common cause of cancer-related deaths worldwide, and hepatocellular carcinoma (HCC) accounts for about 80% of all liver cancers. Despite major improvements in treatment, patients with advanced HCC continue to have limited median overall survival due to primary or secondary resistance to existing therapies. While chronic hepatitis B and C infections continue to be important risk factors for liver cancer, the rising prevalence of obesity, non-alcoholic steatohepatitis (NASH), and alcohol consumption are becoming the dominant risk factors for liver cancer in the United States as well as the rest of the world.

The trial will involve patients from the USC Norris Comprehensive Cancer Center with advanced primary liver cancers or with advanced solid tumors with liver predominant metastatic disease. Its principal investigator is Dr. Anthony El-Khoueiry, a leading expert in early drug development for solid tumors, particularly gastrointestinal cancers. Dr. El-Khoueiry is the Director of the phase I program and Associate Director for Clinical Research at the USC Norris cancer center and Associate Professor of Clinical Medicine at the Keck School of Medicine of USC.

“We are excited at the potential impact our collaboration with Auransa can have for patients with solid tumors with liver predominant disease, in particular patients with hepatocellular cancer and cholangiocarcinoma. AU409 has a unique mechanism of action that impacts gene expression in the tumor and may represent a novel therapeutic approach for these tumors with high unmet need.” said Dr. El-Khoueiry.

“The collaboration with the USC Norris Comprehensive Cancer Center is an important first step in the clinical development of AU409 which is a new chemical entity with a unique mechanism of action.  We plan to work closely with the team at USC to advance this very differentiated potential new drug for patients with liver cancers, specifically hepatocellular carcinoma (HCC), who may not respond to the currently available drugs.” said Pek Lum, Ph.D., co-founder and chief executive officer of Auransa.

“Dr. El-Khoueiry’s clinical trial experience spanning first-in-human, phase 2 and phase 3 trials, together with his interest in liver cancer, makes him the perfect lead of the AU409 clinical trial.” said Andrew Protter, Ph.D., Chief Scientific Officer of Auransa.

Auransa and USC are also collaborating on a broader approach to bring novel and much needed therapeutics to cancer patients, leveraging both Auransa’s proprietary artificial intelligence (AI) drug discovery platform to identify novel disease mechanisms and capabilities from the Rosalie and Harold Rae Brown Center for Cancer Drug Development at the USC Norris Comprehensive Cancer Center.

“The Brown Center for Cancer Drug Development takes an integrated academic-industry approach to achieve our mission of accelerating promising oncology therapeutics into the clinic through collaborative drug development,” said Caryn Lerman, PhD, director of USC Norris Comprehensive Cancer Center, and associate dean for cancer programs at the Keck School of Medicine at USC.   “Together with Auransa, we are optimistic that we can develop new therapeutics for patients in need and further our mission.”

Disclosure: If this trial is successful, USC has the potential to receive royalty payments under the collaboration agreement with Auransa.

About University of Southern California’s Norris Comprehensive Cancer Center

The USC Norris Comprehensive Cancer Center (USC Norris), located in Los Angeles, is a major regional and national resource for cancer research, treatment, prevention, education, and community engagement. The National Cancer Institute (NCI) has designated the USC Norris Comprehensive Cancer Center as one of the nation’s 52 comprehensive cancer centers, a select group of institutions providing leadership in cancer treatment, research, prevention, and education. USC Norris has held this designation since 1973, at which time it was named as one of the first eight such centers in the country. The Rosalie and Harold Rae Brown Center for Cancer Drug Development (CCDD) at the USC Norris was launched in 2020 with a generous gift from the Rosalie and Harold Rae Brown Foundation.  The CCDD employs an integrated academic-industry-focused management model to engage investigators from different disciplines, collapse gaps in expertise, resources, and funding, and ensure that programs have a high likelihood of progressing to the clinic.