British biotech MiNA Therapeutics’ innovative RNA treatment may enhance liver cancer patients’ response to standard cancer therapies, suggest early results from a Phase I/IIa clinical trial.
When they received the biotech’s small activating RNA (saRNA) treatment, two patients showed complete responses after being given the cancer treatment sorafenib and one showed a partial response to another treatment, lenvatinib. A complete tumor response is the best result you can hope for in cancer therapy and is particularly rare in liver cancer patients.
This is the first trial of MiNA’s lead saRNA therapy in humans. As the research is still at an early stage, the biotech now hopes to collect more evidence for the drug’s potential. For example, modifying the current clinical trial protocol to include a combination study with other therapies.
The company’s lead candidate is a double strand of RNA, which activates a target gene called CEBPA. It is packaged in lipid nanoparticles to help the treatment penetrate the normally hard-to-reach liver cells and control the gene expression in the cell nucleus. Low levels of expression of this gene are linked to liver cancer and other liver diseases. In laboratory studies, increasing expression of CEBPA returned levels of its protein to normal and helped reduce the growth of cancer cells.
The company also hopes to test the same drug candidate in liver cirrhosis patients in the future and is also working on other projects targeting liver disease in partnership with Boehringer Ingelheim.
After a long time in development, RNA-based treatments are beginning to enter the market. Unlike MiNA’s therapy, which is designed to activate gene expression, most RNA therapies in development use RNA interference (RNAi) technology that reduces gene expression.
Recently, the European Commission approved the first ever RNAi drug, Onpattro, developed by Alnylam, as a treatment for polyneuropathy. European companies working on RNAi therapies include Spain-based Sylentis, France-based Sanofi and Silence Therapeutics in the UK.