AUSTIN, Texas -- (BUSINESS WIRE) -- Mirna Therapeutics, Inc., a biotechnology company pioneering microRNA (miRNA) replacement therapies for cancer, announced that new preclinical data from its lead product candidate will be presented this week at the Keystone conference on Non-coding RNAs in Development and Cancer in Vancouver, Canada, January 20th – 25th.
New data will be presented on the pharmacology, pharmacokinetics and toxicology of MRX34, a liposomal formulation containing a mimic of the miR-34 tumor suppressor. miR-34 represses the expression of more than 20 oncogenes and inhibits processes required for cancer cell viability, cancer stemness, metastasis, and chemoresistance. The company has previously demonstrated that intravenous administration of the miR-34 mimic can inhibit the growth of pre-existing B-cell lymphoma, liver, lung and prostate cancer (Craig et al., 2012; Wiggins et al., 2010, Trang et al., 2011, Liu et al., 2011).
MRX34 was well tolerated in rodents and non-human primates during IND-enabling studies and did not induce RNAi-mediated toll-like receptor activation or immune response at anticipated therapeutic dose levels. The pharmacokinetic profiles suggest a satisfactory residence time in blood and will be used to determine the recommended human starting dose and treatment regimen in the clinic.
“These data continue to support the safety profile and therapeutic potential of MRX34. We are on track to file an Investigational New Drug application (IND) for MRX34 with the US Food and Drug Administration and initiate clinical testing of this miRNA replacement therapy approach in the first half of this year in patients diagnosed with primary liver cancer or advanced solid cancers with liver involvement,” said Paul Lammers, M.D., President and Chief Executive Officer of Mirna Therapeutics.
This project was funded in part by a Cancer Prevention and Research Institute of Texas (CPRIT) Commercialization grant.
MicroRNAs (miRNAs) are approximately 20-25 nucleotides long and affect gene expression by interacting with messenger RNAs. Unlike siRNAs, miRNAs are encoded in the human genome and are used as natural regulators of global gene expression. More than 1,500 miRNAs are encoded in the human genome and comprise approximately 2% of all mammalian genes. Since each miRNA appears to regulate the expression of tens to hundreds of different genes, miRNAs can function as “master-switches,” efficiently regulating and coordinating multiple cellular pathways and processes. By coordinating the expression of multiple genes, miRNAs are responsible for guiding proper embryonic development, immunity, inflammation, as well as cellular growth and proliferation. Misregulation of miRNAs appears to play a fundamental role in the occurrence, growth and dissemination of many cancers, and replacement of down regulated miRNAs in tumor cells results in a positive therapeutic response.
About Mirna Therapeutics
Mirna Therapeutics, Inc. (Mirna) is a biotechnology company focused on the development and commercialization of miRNA therapeutics. The Company has a foundational intellectual property portfolio on the therapeutic use of miRNAs developed by its own scientists as well as in-licensed from other institutions. Mirna’s IP portfolio contains >300 miRNAs with applications in oncology and other diseases. Oncology-directed miRNAs include those that are key tumor suppressors in cancer, such as miR-34 and let-7 that have been shown to block tumor growth in a number of different pre-clinical animal studies. The Company, founded in 2007 and located in Austin, Texas, has received significant funding from private investors as well as the State of Texas, both through the State’s Emerging Technology Fund and from the Cancer Prevention and Research Institute of Texas (CPRIT). For more information, visit www.mirnarx.com.