Mirna Therapeutics Presents Updates on Development Program for Lead Therapeutic Candidate
- Category: DNA RNA and Cells
- Published on Tuesday, 22 May 2012 16:16
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Formulated mimic of tumor suppressor miR-34 causes tumor regression in mouse models of liver cancer
AUSTIN, TX, USA I May 22, 2012 I Mirna Therapeutics, Inc., a biotechnology company pioneering microRNA replacement therapies for cancer, announced that new preclinical data from its lead therapeutic program will be presented this week at two different conferences: 2012 TIDES Oligonucleotide and Peptide Research, Technology, and Product Development Conference in Las Vegas, Nevada, and the 2012 European Microsymposium on Small RNAs in Basel, Switzerland.
Scientists from Mirna Therapeutics will discuss recent progress and development plan for the Company’s lead therapeutic candidate, a mimic of the miR-34 tumor suppressor. miR-34 is a key component of the p53 pathway that affects cell cycle progression, apoptosis, and cancer stem cell development via its ability to regulate the expression of more than twenty oncogenes. When delivered systemically, the Company’s mimic of miR-34 inhibits the growth of mature tumors in mouse models of B-cell lymphoma, liver, lung, and prostate cancer.
Mirna Therapeutics has received an exclusive license from Marina Biotech for a SMARTICLES® formulation that is being used to enhance the delivery of the miR-34 mimic to solid and hematological tumors. The SMARTICLES-formulated miR-34 mimic causes complete tumor regression in two different and aggressive mouse models of liver cancer and has proven effective in inhibiting the growth of tumors in mouse models of other cancers. Preliminary toxicity studies have revealed no adverse effects and no immunostimulatory activity at therapeutic dose levels. The Company expects to submit an IND application and initiate a Phase I clinical trial within the next year.
“The exceptional therapeutic activity and favorable safety profile produced by our lead therapeutic candidate has us eager to transition into a clinical program where we can demonstrate proof of concept for the miRNA replacement therapy approach,” said Dr. Paul Lammers, President and CEO of Mirna Therapeutics. “Because miRNAs co-regulate multiple oncogenes and oncogenic pathways, miRNA-based therapies are expected to impact a broader range of tumor cells than other targeted cancer therapies, and therefore yield greater clinical benefits.”
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 the State of Texas, both through the State’s Emerging Technology Fund and from the Cancer Prevention and Research Institute of Texas (CPRIT). Mirna Therapeutics is the recipient of a $10.3 million commercialization award from CPRIT. For more information, visit www.mirnarx.com.
SOURCE: Mirna Therapeutics