LONDON—MiNA Therapeutics, which works in the area of RNA activation therapeutics, announced recently top-line results from the Phase Ib dose escalation and cohort expansion study, OUTREACH, of lead candidate MTL-CEBPA in combination with sorafenib standard of care in patients with advanced hepatocellular carcinoma (HCC or liver cancer).
The study met its primary endpoints of safety and tolerability for MTL CEBPA administered either concomitantly or sequentially with sorafenib. In addition, five patients experienced objective tumour responses, including two complete responses during the combination treatment.
“We are delighted to have confirmed objective tumor responses in a Phase Ib study in advanced liver cancer patients who are poorly served by existing treatments,” commented Robert Habib, CEO of MiNA Therapeutics. “Combined with previous positive results, these data suggest that by reducing immune suppression in the tumor microenvironment, MTL CEBPA may increase the effectiveness of sorafenib standard of care.”
Treatment was associated with a reduction in both the number of immature immune suppressor cells as well as genetic markers of immune suppression in patient samples. These biomarker data validate the mechanism of action of MTL CEBPA in reducing immune suppression, which has been identified as a resistance mechanism of solid tumors to cancer treatment, including sorafenib.
These encouraging Phase Ib data add to the previously published positive Phase I results in which four out of five patients experienced a durable, objective response to off-study sorafenib treatment after discontinuation of MTL CEBPA, the company noted, adding that as a single-agent treatment, sorafenib is associated with a very low objective response rate.