Austin, TX's microRNA replacement specialist Mirna Therapeutics last week announced its up to $81 million Nasdaq IPO plans on the same day that the Nasdaq Biotechnology Index plummeted 23%, though it ended the day down 4%. But the biotech hopes the financial backing of big players like Baxalta ($BXLT), Celgene ($CELG) and Pfizer ($PFE), plus nifty drug delivery tech can help it ride out the biotech storm.
The company is developing a tumor suppressor or known as MRX34 that inhibits multiple oncogene pathways to fight cancer while (hopefully) limiting the development of drug resistance. The candidate is in currently in a Phase I dose escalation study to determine its maximum tolerated dose against a variety of cancers, including primary liver cancer, lymphoma and melanoma.
The microRNA is delivered using the Smarticles platform and formulation, licensed from Marina Biotech, which describes its tech as "amphoteric liposomes composed of unique combinations of lipids having anionic and cationic groups that work together to enable cell uptake, to provide serum stability, and to provide pH-triggered endosomal escape."
MicroRNAs are inhibited in cancerous patients by antagonists that bind to the molecules, which prevents them from repressing the expression of multiple proteins. MRX34 contains double-stranded microRNA replicas to replace the inhibited, naturally occurring microRNA.
Mirna says that miR-34, the naturally occurring microRNA that its candidate aims to mimic, regulates 30 oncogenes and several checkpoint molecules, which must be overcome in order to unleash a full immune response against cancer.