on June 22, 2020 twoXAR Pharmaceuticals, a drug discovery and development company focused on bringing first-in-class small molecules to market, reported that preclinical data for its investigational treatment TXR-311 with a novel MOA demonstrated significant efficacy and good tolerability in in vivo studies for the potential treatment of hepatocellular carcinoma (HCC), with results comparable to sorafenib, today’s standard-of-care (Press release, TwoXAR, JUN 22, 2020, View Source [SID1234561361]). The data was presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Virtual Annual Meeting II. The full poster and accompanying audio of the data is available at www.twoxar.com.
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"We are pleased that our TRX-311 in vivo data was selected for presentation at AACR (Free AACR Whitepaper). This study is another demonstration that our approach to drug discovery is effective in identifying novel molecules that have high likelihood of showing positive safety and efficacy signals in preclinical studies," stated Andrew A. Radin, co-founder and CEO of twoXAR. "We are thrilled with the progress of our HCC clinical program thus far and look forward to advancing additional drug candidates in disease areas with similar unmet needs."
HCC is a complex and heterogenous tumor and the most common type of primary liver cancer. It is difficult to diagnose and treat, with poor survival. It is most commonly associated with underlying chronic liver disease, including hepatitis and alcoholic and non–alcoholic steatohepatitis (NASH).
"While we have some beneficial treatments for hepatocellular carcinoma, this tumor type is a growing problem worldwide and more options are needed," said Dr. Ghassan K. Abou-Alfa, MD, medical oncologist at Memorial Sloan Kettering. "These early results are interesting and exciting because they showed safety and efficacy comparability against sorafenib but with a completely novel mechanism of action."
In validation studies, TRX-311 demonstrated greater activity and selectivity in killing HCC tumor cells than standard of care, sorafenib, and significantly inhibited growth in two HCC patient-derived xenograft (PDX) tumor models. TRX-311 also demonstrated in vivo efficacy comparable to sorafenib with good tolerability.
As part of a larger oncology drug discovery program, twoXAR identified 10 novel MOAs each for HCC and pancreatic ductal adenocarcinoma (PDAC) through its AI-driven drug discovery approach that builds an in-silico disease model using multiple sets of biological, clinical and chemical data. TRX-311 showed the most promising results and was chosen to proceed into lead optimization studies for HCC. TRX-311 also showed in vitro activity at low µm concentrations in PDAC cancer cells.
About Hepatocellular Carcinoma
Hepatocellular carcinoma (HCC) occurs most often in people with chronic liver diseases, such as cirrhosis caused by hepatitis B or hepatitis C infection. HCC is a multifactorial disorder and does not cause symptoms in the early stage, which often leads to a delay in diagnosis. The exact cause of HCC is not fully understood.