Marina Biotech Reports Clinical and Preclinical Updates

BOTHELL, WA--(Marketwired - Nov 19, 2015) - Marina Biotech, Inc. ( OTCQB : MRNA ), a leading nucleic acid-based drug discovery and development company focused on rare diseases, today reported that its SMARTICLES ^® licensee Mirna Therapeutics, Inc. ( NASDAQ : MIRN ) of Austin, TX, recently announced the presentation of interim results from its ongoing Phase I clinical trial of MRX34, Mirna's lead therapeutic candidate. Mirna's poster presentation was titled, "Safety, tolerability, and clinical activity of MRX34, the first-in-class liposomal miR-34 mimic, in patients with advanced solid tumors" and presented on November 8, 2015 at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston, MA. The poster reported interim Phase I clinical results for MRX34 from 75 patients with advanced solid tumors.

In addition, SMARTICLES licensee MiNA Therapeutics Limited recently announced the presentation of pre-clinical data on its MTL-CEBPA program in which the compound was shown to improve several markers of liver function in a rat model of liver failure. MiNA's oral presentation was titled, "Systemic administration of novel development candidate, MTL-CEBPA, up-regulates the liver enriched transcription factor C/EBP-α, and reverses CCI4-induced liver failure in vivo " and presented on November 16, 2015 at the 2015 American Association for the Study of Liver Diseases (AASLD) Liver Meeting in San Francisco, CA.

"We are extremely pleased with the clinical and preclinical progress of SMARTICLES-delivered nucleic acid compounds," stated J. Michael French, president and chief executive officer of Marina Biotech. "Over the past 30 days, ProNAi Therapeutics announced the initiation of their Brighton Phase II trial, Mirna Therapeutics announced interim results of their Phase I trial and MiNA Therapeutics announced positive preclinical results which they believe will allow them to advance their program into the clinic in 2016. We look forward to the continued positive progress of these programs and expect to complete additional similar SMARTICLES licenses in the future." 

Mirna Therapeutics' MRX34 is a double-stranded "mimic" of the naturally occurring tumor suppressor microRNA miR-34, encapsulated in the SMARTICLES liposomal delivery formulation. Interim safety, efficacy and biomarker data from the multicenter, open-label Phase 1 clinical trial in solid tumor patients show that MRX34 has a safety profile manageable with standard interventions or tests used by oncologists, and demonstrate the therapeutic potential of miR-34 replacement therapy. Additionally, dose-dependent effects on miR-34 target genes in patients' white blood cells have been observed. As of August 13, 2015, two patients with advanced, metastatic Stage IV cancer have achieved clinical responses after treatment with MRX34: one patient with primary liver cancer (hepatocellular carcinoma, HCC) metastatic to the lung, and one patient with acral melanoma, metastatic to lymph nodes, showed more than 30 percent tumor shrinkage (confirmed partial responses).

MiNA Therapeutics' MTL-CEBPA is a SMARTICLES liposomal formulation of a short activating RNA targeting the CEBPA gene. In the experiments covered by the presentation, MTL-CEBPA was administered systemically for two weeks in male Sprague Dawley rats with CCl4-induced liver failure. In the treated animals, MTL-CEBPA restored CEBPA mRNA levels as compared to normal and negative vehicle controls. The CEBPA gene encodes CCAAT/enhancer binding protein alpha (C/EBP‐α), a transcription factor that plays an important role in normal hepatocyte function and response to injury. The treatment regime further reduced fibrosis and fatty infiltration as well as normalized levels of several important liver function markers, such as serum bilirubin, serum transaminase levels and coagulation time. In addition, treatment also led to an increase in the treated animals' body weight and attenuated hyper-ammonaemia, a marker of impaired liver detoxification.

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, MiNA Therapeutics, 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 .

About Mirna Therapeutics, Inc. Mirna is a clinical-stage biopharmaceutical company developing a broad pipeline of microRNA-based oncology therapeutics and is the first to establish clinical proof-of concept for a microRNA replacement therapy for cancer. Mirna's lead product candidate, MRX34, a mimic of naturally occurring microRNA-34 (miR-34), is currently being studied in a Phase 1 clinical trial in patients with primary liver cancer, advanced solid tumors and hematological malignancies. miR-34 is one of the most widely published microRNAs and is considered a key regulator of multiple oncogenes across key oncogenic pathways, with the capacity to regulate more than 30 different oncogenes and repress the immune checkpoint signaling molecule PD-L1. The potential capacity to simultaneously affect multiple pathways and processes that are critical to cancer cell viability may make mimics of tumor suppressor microRNAs potent anti-cancer agents and less susceptible to drug resistance. Mirna plans to develop MRX34 as a monotherapy and in combination with other therapeutic modalities, such as targeted therapies and immuno-oncology agents. The company was founded in 2007 and is located in Austin, Texas. For more information, visit www.mirnarx.com .

About MiNA Therapeutics Limited Harnessing the innate mechanism of gene activation, MiNA Therapeutics' platform enables the development of new medicines that restore normal function to patients' cells. MiNA is applying its technology and clinical know-how to transform the therapy landscape of severe liver diseases. MiNA expects that its initial product candidate will achieve clinical proof of concept in 2017.

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.