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Mirna Series B Sees Slight Delay as miRNA Rx Field Grows

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By Doug Macron

While Mirna Therapeutics had expected to close a $25 million Series B financing round by the end of September, the company missed its goal after encountering an overall slowdown in venture capital investment activity during the summer, the company's top official said this week.

However, "interest has peaked again, [and] several VC firms have closed new funds so they have money to invest," Mirna President and CEO Paul Lammers told Gene Silencing News in an e-mail.

"We have done a lot of meetings, [and] there is definitely interest in our approach to miRNA-based therapeutics," he added. Unlike companies developing drugs that inhibit the small, non-coding RNAs, Mirna is focused on miRNA mimics.

Although its pipeline includes eight candidates, the company's lead projects include miR-Rx34, a mimic of mir-34 for treating prostate and other solid tumors; and miR-Rx02, a mimic of an undisclosed miRNA for solid tumors including non-small cell lung cancer. The company expects that both programs could yield investigational new drug applications next year.

In June, Mirna received $10.3 million from the state of Texas to advance these compounds. At the time, Lammers told Gene Silencing News that the company was also nearing the completion of the Series B, which would provide enough money to fund operations into 2014 (GSN 6/24/2010).

Although it ran into delays, Mirna is now expected to close the Series B by the end of the fourth quarter, he said.

"Perhaps it is a sign of the times that closing a round just takes longer than it used to," Lammers added.

And to add to the difficulty in raising funds, besides the state of the economy, which has undoubtedly impacted companies' efforts to secure financing, there has been an increase in the number of companies operating in the miRNA space in recent years, many of which are on the lookout for their share of available investment dollars.

Mirrx Therapeutics, for instance, this year obtained an undisclosed amount of seed financing from three Danish investment firms to fund its so-called Blockmir technology (GSN 2/4/2010).

A spinout of Stealth Biotech, Mirrx is focused on developing Blockmirs as therapeutic miRNA antagonists. Unlike other miRNA-inhibition approached, Blockmirs bind to specific miRNA binding sites in target RNAs to thereby prevent the small, non-coding RNAs from binding to the same site, according to the company. This allows the compounds to regulate one specific mRNA, rather than all of a particular miRNA's targets.

With the seed money in hand, Mirrx expects to be able to complete its planned in vivo proof-of-concept work and continue operations into 2012.

Meanwhile, in April, startup Mirina closed a Series A-1 financing round (GSN 4/29/2010). While the size of that round is also undisclosed, David McElligott, Mirina’s vice president of research and development, told Gene Silencing News that it would be sufficient to support the company's operations for 12 to 15 months.

Included in Mirina's plans for that time is the selection and validation of drug candidates, based on the firm's minor groove binder technology, for late-stage preclinical testing.

MGBs are peptide antibiotics that non-covalently bind to the minor grooves of nucleic acid duplexes, and had originally been developed as cancer therapeutics, according to developer Nanogen. Although they did not prove effective for this application, they were later shown to stabilize DNA duplexes and potentially boost the target selectivity and potency of miRNA antagonists.

Nanogen later sold the rights to MGBs for RNA-based therapeutics to investment firm Accelerator, which passed them along to spinout Mirina.

And in July, an official from Miragen Therapeutics disclosed to Gene Silencing News that the company is preparing for a Series B financing (GSN 7/15/2010). While President and CEO Bill Marshall declined to specify how much is expected to be raised in the round, he said that it would be sufficient to advance one of the firm's two lead programs to the IND stage next year.

Those two efforts center around inhibitors of miR-15 as a treatment for post-myocardial infarction remodeling, and miR-208 for chronic heart failure. At the time, Marshall said it wasn't clear which one would be advanced into clinical studies first, and that Miragen would prioritize the candidates based on the results of animal studies.

One miRNA drugs company that does not appear to be looking for private investments is Regulus Therapeutics. Established as a joint venture between Isis Pharmaceuticals and Alnylam Pharmaceuticals in 2007, the company was initially funded with $10 million from Alnylam (GSN 9/13/2007).

Since that time, Regulus has had quite a bit of success on the partnering front, inking a pair of deals with GlaxoSmithKline and, in June, an alliance with Sanofi-Aventis that included a $25 million upfront fee and a promise of a $10 million equity investment down the road (GSN 6/24/2010).

And it seems that Regulus may be considering going public in the not-too-distant future. As reported by Gene Silencing News, the company is currently seeking a financial-reporting manager whose duties will include preparing "internal management reporting and external SEC reporting documents" (GSN 9/23/2010).

Specifically, the manager will create Sarbanes-Oxley compliance procedures "to ensure that Regulus is fully compliant and ready to become a publicly traded organization," the job listing states.