Marina Biotech, Inc. Receives Decision To Grant Japanese Patent For Its SMARTICLES Nucleic Acid Delivery Technology

BOSTON, MA--(Marketwired - June 05, 2014) - Marina Biotech, Inc. (PINKSHEETS: MRNA), a leading nucleic acid-based drug discovery and development company focused on rare diseases, announced today that a decision to grant a patent has been issued for the Company’s SMARTICLES® delivery technology in Japan (Ser. No. 2008-530426). The claims of this National Stage Application cover delivery technology based on Marina’s proprietary SMARTICLES amphoteric liposomes and directly support the clinical programs of the company’s licensees ProNAi Therapeutics, Inc. and Mirna Therapeutics, Inc. The granted claims cover amphoteric liposomes that are suitable for tailoring delivery systems for the release of therapeutic cargos, including DNA oligonucleotides, antisense and/or decoy entities, ribozymes, DNAzymes or aptamers, as well as proteins, peptides, and RNA-based drugs such as siRNA and miRNA.

“The decision to grant from the Japan Patent Office further extends the scope of our intellectual property covering our SMARTICLES technology, while the positive clinical delivery experiences of both ProNAi and Mirna provide significant scientific and clinical validation of SMARTICLES,” stated J. Michael French, President and CEO of Marina Biotech. “We believe that SMARTICLES, which is broadly covered by the company’s patent estate, is a leading means for the successful delivery of nucleic acid compounds. Further, we believe it is the only delivery technology in development that has delivered both a single- and a double-stranded oligonucleotide in a clinical setting. We believe that the breadth and flexibility of the SMARTICLES platform provides significant competitive advantages and differentiation as we move forward with our rare disease clinical pipeline.

The claims of this National Stage Application cover delivery technology based on SMARTICLES amphoteric liposomes, which are composed of phosphatidylcholine (PC) and phosphatidylethanolamine (PE), along with chargeable amphiphiles. The chargeable amphiphiles include at least one pH sensitive anionic lipid and at least one pH sensitive cationic lipid. These amphoteric liposomes impart a unique in vivo serum stability to the delivery formulation. Serum stability is enhanced by synergistically balancing the overall charge of the amphoteric liposomes using the pH sensitive anionic lipids and pH sensitive cationic lipids. The stable amphoteric liposomes can have a particle size of from 50 to 500 nm, and represent a leading technology for delivering therapeutics in the fields of oligonucleotide, decoy and antisense drugs, as well as RNA-based drugs. A significant aspect of the granted intellectual property is the breadth of the covered technology. For example, the ratio of phosphatidylethanolamine to phosphatidylcholine can be varied over a very wide range from 0.5 to 8. Further, the compositional balance between the PC/PE components and the chargeable amphiphiles can be varied over a wide range from 5% to 95%. These features give SMARTICLES technology an extraordinary flexibility in selection and development of lead formulations for clinical trials.

About SMARTICLES Clinical Experience.
To date, SMARTICLES-delivered nucleic acid drug candidates have demonstrated: delivery to tumor in Phase 1 and 2 clinical trials; statistically significant, dose-dependent, and specific knockdown of a gene target in a Phase 1 clinical trial; single agent anti-tumor activity in patients with recurrent or refractory non-Hodgkin’s lymphoma (NHL) in a Phase 2 clinical trial; and anti-tumor efficacy with both single- and double-stranded oligonucleotides in rodent models. These achievements represents the combined preclinical and clinical experiences (a total of approximately 50 patients) of, licensees ProNAi Therapeutics, Inc., Plymouth, MI and Mirna Therapeutics, Inc., Austin, TX. ProNAi Therapeutics’ clinical compound, PNT2258, is a first-in-class, 24-base, single-stranded, chemically-unmodified DNA oligonucleotide drug targeting BCL2. Mirna Therapeutics’ clinical compound, MRX34, is a double-stranded microRNA “mimic” of the naturally occurring tumor suppressor miR-34, which inhibits cell cycle progression and induces cancer cell death.

About Marina Biotech, Inc.
Marina Biotech is an oligonucleotide therapeutics company with broad drug discovery technologies providing the ability to develop proprietary single and double-stranded nucleic acid therapeutics including siRNAs, microRNA mimics, antagomirs, and antisense compounds, including messengerRNA therapeutics. These technologies were built via a roll-up strategy to discover and develop different types of nucleic acid therapeutics in order to modulate (up or down) a specific protein(s) which is either being produced too much or too little thereby causing a particular disease. We believe that the Marina Biotech technologies have unique strengths as a drug discovery engine for the development of nucleic acid-based therapeutics for rare and orphan diseases. Further, we believe Marina Biotech is the only company in the sector that has a delivery technology in human clinical trials with differentiated classes of payloads, through licensees ProNAi Therapeutics and Mirna Therapeutics, delivering single-stranded and double-stranded nucleic acid payloads, respectively. Our novel chemistries and other delivery technologies have been validated through license agreements with Roche, Novartis, Monsanto, and Tekmira. The Marina Biotech pipeline currently includes a clinical program in Familial Adenomatous Polyposis (a precancerous syndrome) and a preclinical program in myotonic dystrophy. Marina Biotech’s goal is to improve human health through the development of RNAi- and oligonucleotide-based compounds and drug delivery technologies that together provide superior therapeutic options for patients. Additional information about Marina Biotech is available at www.marinabio.com.

Marina Biotech Forward-Looking Statements
Statements made in this news release may be forward-looking statements within the meaning of Federal Securities laws that are subject to certain risks and uncertainties and involve factors that may cause actual results to differ materially from those projected or suggested. Factors that could cause actual results to differ materially from those in forward-looking statements include, but are not limited to: (i) the ability of Marina Biotech to obtain additional funding; (ii) the ability of Marina Biotech to attract and/or maintain manufacturing, research, development and commercialization partners; (iii) the ability of Marina Biotech and/or a partner to successfully complete product research and development, including preclinical and clinical studies and commercialization; (iv) the ability of Marina Biotech and/or a partner to obtain required governmental approvals; and (v) the ability of Marina Biotech and/or a partner to develop and commercialize products prior to, and that can compete favorably with those of, competitors. Additional factors that could cause actual results to differ materially from those projected or suggested in any forward-looking statements are contained in Marina Biotech’s most recent filings with the Securities and Exchange Commission. Marina Biotech assumes no obligation to update or supplement forward-looking statements because of subsequent events.