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RNA Therapy Biotech Earns $44 Million in IPO

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1 October 2015. A biotechnology company developing therapies based on RNA, genetic material that sends signals to cells, raised $43.75 million in its initial public stock offering. Mirna Therapeutics Inc. in Austin, Texas issued 6.25 million shares today at $7.00, and trades on the Nasdaq exchange under the symbol MIRN. Its shares closed today at $7.06.

Mirna Therapeutics develops therapies based on the actions of microRNAs or miRNAs, genetic molecules serving as regulators of the genome. They start out small, but evolve into more complex molecules that interact with another type of RNA — messenger RNA — to repress the expression of genes that respond to various proteins. More than 1,500 miRNA genes control some 30 percent of all human genes, and can regulate the expression of tens to hundreds of genes, involving such functions as embryonic development, immune and related inflammatory responses, and cellular growth and proliferation.

The company’s technology uses a process called miRNA replacement therapy, to introduce material that simulates functioning miRNA, replacing the non-functioning miRNAs that allowed cancer cells to proliferate, and reactivating pathways encouraging a therapeutic response.  Mirna Therapeutics says its miRNA replacement material has the same genetic sequence as naturally occurring miRNA, replacing the depleted miRNA and addressing the same genes as the original. Thus, says the company, the miRNA replacement  is less likely by itself to produce an adverse effect.

Mirna’s lead product, code-named MRX34, is a cancer therapy that the company says simulates a miRNA associated with suppression of tumor cells, and can generate an immune response to kill cancer cells. In MRX34, miRNAs are encased in nanoscale liposomes, cell membrane material, and administered as intravenous infusions.

MRX34 is currently being tested in an early-stage clinical trial, enrolling 200 participants at several sites in the U.S. and Korea. Participants in the trial have primary liver cancer, small-cell lung cancer, and melanoma, as well as several types of blood-related cancers. The study is still underway, testing MRX34 primarily for safety to determine the maximum tolerated dose and recommend a dosage level for a later intermediate-stage trial.

Mirna Therapeutics reported early results from the trial at a meeting of the American Association for Cancer Research in April 2015. At the meeting, the company said MRX34 had “a manageable safety profile” in participants with solid tumors and blood-related cancers. The study team also found regression in cancer-related genes of the miRNA targets, revealed by an analysis of white blood cells from the participants.

At the same meeting, the company reported on preclinical research on lab mice induced with non-small cell lung cancer showing MRX34 represses a checkpoint signaling molecule, that turns off the immune system’s abilities to attack cancer cells. The study also found MRX34 led to an increase in immune system T cells that infiltrate tumors, and a decrease in T cells hampered by the checkpoint signaling molecule.

Mirna Therapeutics was founded in 2007, and received four rounds of venture financing totaling $83 million.

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Hat tip: Fortune/Term Sheet

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