On September 23, 2019 Auransa Inc., an artificial intelligence (AI)-driven biotechnology company developing precision medicines in areas of significant unmet medical need, reported that new data on AU-409, the company’s novel lead drug candidate being developed for the treatment of hepatocellular carcinoma (HCC), were presented at the 13th Annual Conference of the International Liver Cancer Association (ILCA) in Chicago (Press release, Auransa, SEP 23, 2019, View Source [SID1234635629]). The study results demonstrated promising antitumor activity for AU-409 in a preclinical orthotopic liver cancer model as evidenced by statistically significant reductions in tumor burden. Additionally, the anticancer activity of AU-409 was also demonstrated in culture using cells derived from patients’ tumors. The data were highlighted in an oral presentation at the 2019 ILCA conference by Auransa’s Chief Scientific Officer, Andrew Protter, Ph.D.
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AU-409 is Auransa’s novel lead drug candidate and was generated leveraging the company’s proprietary AI-driven SMarTR Engine. The SMarTR Engine tackles disease heterogeneity and leverages gene expression profiles to predict responder patient populations, as well as compounds that might be effective against those patient populations. Auransa is currently conducting investigational new drug (IND)-enabling studies of AU-409 and expects to initiate first-in-human Phase 1 trials in the first half of 2020.
As part of their work, researchers sought to evaluate the anticancer activity of AU-409 in cell culture. Data demonstrated that AU-409 reduced the viability of HCC cells from a number of patient-derived xenografts, as well as standard HCC stable cell lines.
Additionally, researchers conducted in vivo studies to measure the antitumor activity of AU-409 in an orthotopic mouse model of HCC (Hep3B2.1-7-Luc cells that exhibit bioluminescence measured as total flux, a marker of tumor burden). Following tumor cell implantation into the liver, mice were treated orally with AU-409 (10 mg/kg or 20 mg/kg) or vehicle for four weeks. At Day 28, treatment with AU-409 was associated with a dose-dependent, statistically significant decrease in tumor burden, as compared to vehicle. Tumor burden was reduced by 63% compared to vehicle in the AU-409 10 mg/kg treatment arm (p < 0.001) and 76% compared to vehicle in the AU-409 20 mg/kg treatment arm (p < 0.001).
Treatment with AU-409 in the orthotopic mouse model was well tolerated. There were no clinical observations associated with the treatment. Compared to vehicle, AU-409 treatment was associated with a statistically significant increase in body weight, as well as a decrease in liver enzymes that are indicative of liver toxicity.
"These preclinical data support the development of AU-409 for HCC, as predicted by Auransa’s proprietary AI-driven discovery and development technology platform. The combination of robust anticancer effects in cell culture and in vivo along with evidence of favorable tolerability supports advancing AU-409 into clinical development," said Dr. Protter. "Based on these results, we are working to complete IND-enabling studies for the program with the goal of entering the clinic during the first half of 2020."
"We are pleased by this collection of preclinical data for AU-409 as it offers an early yet important validation of the extent to which Auransa’s SMarTR Engine can accelerate drug discovery and development," stated Pek Lum, Ph.D., chief executive officer of Auransa. "AU-409 is an example of what we believe is possible when we utilize our novel proprietary AI platform to address the drug discovery and development challenges. By interrogating gene expression profiles, we believe we can predict responder patient populations and the potential treatments that may be effective for those patients. We look forward to the ongoing advancement of the AU-409 program, while continuing to build out our broader product pipeline through novel insights generated by the SMarTR Engine."