Biotech firm Roquefort Therapeutics PLC (LSE:ROQ, OTCQB:ROQAF) has reported significant progress in its quest to treat Midkine-expressing cancers.
The programme uses anti-cancer RNA oligonucleotides, small DNA-like molecules used in gene therapy, and has shown over 90% efficacy in laboratory tests on human liver and neuroblastoma cancer cells.
The development work is carried out in partnership with the University of Sydney's Faculty of Medicine and Health, and the University of New South Wales's Immune Oncology Laboratory.
The team at the University of Sydney, under Professor Mark Molloy, detected a new peptide, or short chain of amino acids, related to the truncated, non-functional Midkine protein from cancer cells.
This study confirms the creation of a non-functional Midkine protein by the company's anti-sense oligonucleotides.
Further research by the team at the University of New South Wales has shown promising results in laboratory tests for hepatocellular carcinoma (HCC), a common form of liver cancer.
Using a unique combination of Roquefort's oligonucleotides, they achieved significant reductions in full-length Midkine and created a new non-functional Midkine. HCC accounts for about 90% of liver cancers and is the fourth leading cause of cancer deaths worldwide.
Roquefort now plans to advance its anti-cancer RNA oligonucleotide program into animal studies, which are expected to be completed by the end of 2023.
"Liver cancer is growing with incidences forecast to increase to one million new patients per year by 2025," said Roquefort's chief executive, Ajan Reginald.
"It remains an area of high unmet medical need with a five-year survival of only 21% because the existing medicines have limited effect.
"Midkine is associated with progression, metastasis and resistance and we feel this is a cancer in which we should focus our portfolio of Midkine targeting medicines including antibodies, mRNA and the anti-sense oligonucleotides.
"These oligonucleotides attack a different Midkine region versus the antibodies and mRNA, and this diversity of targeting may be helpful in developing mono or combination therapies."