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Looking to Restart Operations, Marina Regains Compliance with SEC Reporting Rules

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NEW YORK (GenomeWeb) – Marina Biotech this week took another step toward reestablishing itself as a player in the nucleic acids drug sector, regaining compliance with US Securities and Exchange Commission financial reporting requirements with the submission of the requisite information for 2012, 2013, and the first two quarters of 2014.

The news gave a shot in the arm to Marina, driving its stock up more than 25 percent to around $0.80 a share on the OTC markets.

Marina President and CEO Michael French — currently the firm's sole employee — also provided a pipeline update, telling Gene Silencing News that a Phase I trial of RNAi-based familial adenomatous polyposis (FAP) drug CEQ508 is set to resume as soon as the first quarter of 2015.

Meanwhile, Marina remains on the lookout for potential industry collaborators, not only for CEQ508, but for the company's suite of therapeutic and drug-delivery technologies, he said.

"With the [upcoming] acquisition of Santaris [by Roche], there really isn't any other player with a bridged nucleic acid that can partner with companies beginning to focus on non-coding RNAs and other mechanisms of action outside of RNAi," French said.

This, along with a willingness to move the company to suit the needs of a big pharma partner, puts Marina in a good position to land a deal, he added.

In mid-2012, Marina was forced to halt essentially all of its operations amid a cash shortfall. As a result, the company had to end a Phase I trial of CEQ508, which uses orally delivered attenuated Escherichia coli to transcribe therapeutic shRNAs, after dosing patients in two of three cohorts.

Earlier this year, however, the firm was able to sell $6 million in convertible preferred stock, giving it funds to operate into May 2015 and the ability to restart its research and development efforts, albeit through contract research and academic collaborations.

Marina has also shifted its focus away from the large indications that it previously pursued, such as cancer and hypercholesterolemia, and toward rare diseases — a trend seen among many of the key players in RNA therapeutics in response to the pharmaceutical industry's growing interest in niche diseases.

CEQ508 remains the company's flagship candidate, and French said that Marina is in the process of conducting the necessary steps to restarting the halted Phase I dose-escalation study. This includes analyzing data from the second cohort and potentially reanalyzing data from the first, as well as preparing and filing the required documentation with the US Food and Drug Administration.

Should everything go according to plan, dosing in the third cohort could begin within the next six months, he added. Ultimately, Marina hopes to commercialize the drug itself in the US and partner it in other markets.

Meanwhile, Marina is laying the groundwork for its nascent myotonic dystrophy program. The exact therapeutic modality used for this effort has not been selected, French said, but it is likely to involve either steric blockers, messenger RNA translation inhibitors, or exon skippers that have been modified with the company's conformationally restricted nucleotide technology.

All three types of molecules will be tested in an animal model and "whichever wins is the lead," French said. In vivo proof-of-concept studies are expected to be completed by the end of this year, with a lead candidate selected in early 2015.

Following behind the myotonic dystrophy program is one in Duchenne's muscular dystrophy (DMD), likely utilizing an exon-skipping approach. French did not specify when the company hopes to have generated proof-of-concept data in this indication, but noted that Marina is closely watching efforts by the RNA medicines companies Prosensa, which has a DMD drug in Phase III testing, and Sarepta Therapeutics, which has a drug for the disease in Phase II development.

Despite the cash infusion earlier this year, Marina remains cautious about its expenditures, however, and remains interested in a tie-up with another company should it be in its best interests, French noted.

"The company has seen death up close and is not interested in seeing that again," he said.

As it stands now, the company's cash on hand is expected to last until May 2015, enough time to restart, and potentially complete, the CEQ508 Phase I trial, French added.

Q2

For the three-month period ended June 30, Marina had cash on hand totaling $4.3 million, and recorded zero revenues.

Research and development spending was $50,000, all of which was related to R&D consulting costs associated with resumption of the CEQ508 program.

General and administrative expenses were $600,000, and were largely linked to expenses stemming from efforts to regain SEC reporting compliance and financial audits.

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