Auransa Inc. and the University of Southern California (USC) are collaborating on a Phase I clinical trial to evaluate AU409, a new kind of treatment for cancers of the liver and solid tumors with liver dominant disease. In preclinical studies, the compound showed anticancer activity against hepatocellular carcinoma (HCC).
“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 Anthony El-Khoueiry, principal investigator for the trial and associate director for Clinical Research at the USC Norris Comprehensive Cancer Center.
The company reports that in preclinical trials, AU409 has been shown to limit liver cancer cells’ ability to translate the message from various genes that are essential for them to survive and multiply. In other words, it prevents the cells from producing proteins they need to grow.
AU409 is the first of Auransa’s drug candidates to enter clinical trial stage. The company uses a proprietary AI Platform (SMarTR Engine). Auransa says it has built a “drug-discovery engine that bypasses the need for a drug target as a starting point. By analyzing diverse molecular data obtained from public data sources, the SMarTR engine can predict drug candidates for particular patient populations.”
The company has about a dozen compounds in its pipeline, three of which are in preclinical development. The majority of these are anti-cancer drug candidates. If the trial of AU409 is successful, USC has the potential to receive royalty payments under the collaboration agreement.
AU409’s anti-cancer activity is distinct from FDA approved tyrosine kinase inhibitors (such as sorafenib or regorafenib) currently used for hepatocellular carcinoma, the company reports. The compound is a novel small molecule that has been shown to modulate transcription of certain genes thereby altering the gene expression profile of liver cancer cells.
More than 800,000 people are diagnosed with liver cancer each year, according to the American Cancer Society, and this disease causes an estimated 700,000 deaths worldwide. Over 700 drug trials are ongoing to test liver cancer treatments, according to ClinicalTrials.gov.
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.
“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.
Auransa and USC reportedly are also collaborating on “a broader approach to bring novel therapeutics to cancer patients.”