Marina Biotech Strengthens the Intellectual Property Estate for the Transkingdom RNA Interference Nucleic Acid Delivery Techn...
Marina Biotech Strengthens the Intellectual Property Estate for the Transkingdom RNA Interference Nucleic Acid Delivery Technology
Company Expects to Restart Its Phase 1 Human Clinical Trial in FAP Patients in First Half of 2014
BOSTON, MA--(Marketwired - Jan 14, 2014) - Marina Biotech, Inc. (PINKSHEETS: MRNA), a leading oligonucleotide-based drug discovery and development company, announced today that the Canadian Intellectual Property Office (CIPO) has issued a Notice of Allowance in Canada Patent Application 2,591,565, for "Compositions for Bacterial Mediated Gene Silencing and Methods of Use." This patent allowance supports the Company's transkingdom RNAi (tkRNAi) nucleic acid delivery technology. The tkRNAi technology is an integral part of the Company's broad nucleic acid-based drug discovery platform and was utilized in the development of CEQ508 for the potential treatment of Familial Adenomatous Polyposis (FAP). CEQ508 has thus far been orally administered in two cohorts of a four cohort dose-escalating study in a Phase 1 human clinical trial in FAP patients. The Company has received U.S. Food and Drug Administration Orphan Drug Designation for this program.
"We continue to develop Marina Biotech's patent estate with respect to our unique nucleic acid drug discovery platform," stated J. Michael French, President and Chief Executive Officer at Marina Biotech. "Our other chemistry and delivery technologies provide us the means to target coding and non-coding RNA via intravenous, subcutaneous and pulmonary administration. With this particular patent, we strengthen the portfolio in order to develop proprietary and novel nucleic acid-based therapeutics against oncology and inflammatory diseases throughout the gastrointestinal tract. Moreover, the tkRNAi technology permits us to develop orally administered nucleic acid-based therapeutics. With regard to our START-FAP (Safety and Tolerability of An RNAi Therapeutic in Familial Adenomatous Polyposis) clinical trial with CEQ508, we expect to be able to restart the trial in the first half of 2014."
J. Michael French, President and CEO, will present an update on the company at the upcoming Biotech Showcase™ 2014, on Wednesday, January 15, 2014 at 4:15 pm at the Parc 55 Wyndham San Francisco - Union Square at 55 Cyril Magnin Street, San Francisco, California. The webcast URL for both the live webcast and the webcast replay of the presentation is http://www.media-server.com/m/p/qstvqrqe. The webcast replay will be available approximately two hours after the webcast presentation ends and is accessible for 30 days.
About CEQ508
CEQ508 is the first drug candidate in a novel class of therapeutic agents utilizing the transkingdom RNA interference (tkRNAi) platform. CEQ508 comprises attenuated bacteria that are engineered to enter into dysplastic tissue and release a payload of short-hairpin RNA (shRNA), a mediator in the RNAi pathway. The shRNA targets the mRNA of beta-catenin, which is known to be dysregulated in classical FAP. CEQ508 is being developed as an orally administered treatment to reduce the levels of beta-catenin protein in the epithelial cells of the small and large intestine. Under the trial plan, upon enrollment, patients are placed in one of four dose-escalating cohorts. Following completion of the dose escalation phase, the trial plan calls for a stable-dose phase in which additional patients will receive the highest safe dose. CEQ508 will be administered daily in an oral suspension for 28 consecutive days.
About FAP
CEQ508 is being developed for the treatment of Familial Adenomatous Polyposis (FAP), a hereditary condition that occurs in approximately 1:10,000 persons worldwide. FAP is caused by mutations in the Adenomatous Polyposis Coli (APC) gene. As a result of these mutations, epithelial cells lining the intestinal tract have increased levels of the protein beta-catenin, which in turn, results in uncontrolled cell growth. Proliferation of the epithelial cells results in the formation of numerous (hundreds to thousands) non-cancerous growths (polyps) throughout the large intestine. By age 35, 95% of individuals with FAP have developed polyps and most will experience adverse effects including increased risk of bleeding and the potential for anemia. In more severe cases, obstruction of the intestines, abdominal pain, and severe bouts of diarrhea or constipation can occur. FAP patients are also at an increased risk of various cancers, the most concerning of which is a nearly 100% occurrence of colon cancer if measures are not taken to prevent the formation of polyps. For many patients, complete colectomy (surgical removal of the entire large intestine), usually performed in the late teenage years or early twenties, is the only viable option for treatment. However, surgical intervention is not curative as the risk of polyps forming in the remaining portions of the intestinal tract and in the small intestine continues after colectomy.
About Marina Biotech, Inc.
Marina Biotech is an oligonucleotide therapeutics company with the broadest drug discovery platform in the sector providing the ability to develop proprietary single and double-stranded nucleic acid therapeutics including siRNAs, microRNA mimics, antagomirs, and antisense compounds, including messengerRNA therapeutics. The platform was built via a roll-up strategy to discover and develop any number of 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 causing a particular disease. We believe that Marina Biotech is the only company in the sector with this breadth of capability, and as a result the platform has unique strengths as a drug discovery engine for the development of nucleic acid-based therapeutics for rare and orphan diseases. Further, we believe Marina 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 two preclinical programs -- in bladder cancer and 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.
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 and supplement forward-looking statements because of subsequent events.
Marina Biotech, Inc.
J. Michael French
President and Chief Executive Officer
(425) 892-4322
admin@marinabio.com
Marina Biotech Strengthens the Intellectual Property Estate for the Transkingdom RNA Interference Nucleic Acid Delivery Technology
Company Expects to Restart Its Phase 1 Human Clinical Trial in FAP Patients in First Half of 2014
BOSTON, MA--(Marketwired - Jan 14, 2014) - Marina Biotech, Inc. (PINKSHEETS: MRNA), a leading oligonucleotide-based drug discovery and development company, announced today that the Canadian Intellectual Property Office (CIPO) has issued a Notice of Allowance in Canada Patent Application 2,591,565, for "Compositions for Bacterial Mediated Gene Silencing and Methods of Use." This patent allowance supports the Company's transkingdom RNAi (tkRNAi) nucleic acid delivery technology. The tkRNAi technology is an integral part of the Company's broad nucleic acid-based drug discovery platform and was utilized in the development of CEQ508 for the potential treatment of Familial Adenomatous Polyposis (FAP). CEQ508 has thus far been orally administered in two cohorts of a four cohort dose-escalating study in a Phase 1 human clinical trial in FAP patients. The Company has received U.S. Food and Drug Administration Orphan Drug Designation for this program.
"We continue to develop Marina Biotech's patent estate with respect to our unique nucleic acid drug discovery platform," stated J. Michael French, President and Chief Executive Officer at Marina Biotech. "Our other chemistry and delivery technologies provide us the means to target coding and non-coding RNA via intravenous, subcutaneous and pulmonary administration. With this particular patent, we strengthen the portfolio in order to develop proprietary and novel nucleic acid-based therapeutics against oncology and inflammatory diseases throughout the gastrointestinal tract. Moreover, the tkRNAi technology permits us to develop orally administered nucleic acid-based therapeutics. With regard to our START-FAP (Safety and Tolerability of An RNAi Therapeutic in Familial Adenomatous Polyposis) clinical trial with CEQ508, we expect to be able to restart the trial in the first half of 2014."
J. Michael French, President and CEO, will present an update on the company at the upcoming Biotech Showcase™ 2014, on Wednesday, January 15, 2014 at 4:15 pm at the Parc 55 Wyndham San Francisco - Union Square at 55 Cyril Magnin Street, San Francisco, California. The webcast URL for both the live webcast and the webcast replay of the presentation is http://www.media-server.com/m/p/qstvqrqe. The webcast replay will be available approximately two hours after the webcast presentation ends and is accessible for 30 days.
About CEQ508
CEQ508 is the first drug candidate in a novel class of therapeutic agents utilizing the transkingdom RNA interference (tkRNAi) platform. CEQ508 comprises attenuated bacteria that are engineered to enter into dysplastic tissue and release a payload of short-hairpin RNA (shRNA), a mediator in the RNAi pathway. The shRNA targets the mRNA of beta-catenin, which is known to be dysregulated in classical FAP. CEQ508 is being developed as an orally administered treatment to reduce the levels of beta-catenin protein in the epithelial cells of the small and large intestine. Under the trial plan, upon enrollment, patients are placed in one of four dose-escalating cohorts. Following completion of the dose escalation phase, the trial plan calls for a stable-dose phase in which additional patients will receive the highest safe dose. CEQ508 will be administered daily in an oral suspension for 28 consecutive days.
About FAP
CEQ508 is being developed for the treatment of Familial Adenomatous Polyposis (FAP), a hereditary condition that occurs in approximately 1:10,000 persons worldwide. FAP is caused by mutations in the Adenomatous Polyposis Coli (APC) gene. As a result of these mutations, epithelial cells lining the intestinal tract have increased levels of the protein beta-catenin, which in turn, results in uncontrolled cell growth. Proliferation of the epithelial cells results in the formation of numerous (hundreds to thousands) non-cancerous growths (polyps) throughout the large intestine. By age 35, 95% of individuals with FAP have developed polyps and most will experience adverse effects including increased risk of bleeding and the potential for anemia. In more severe cases, obstruction of the intestines, abdominal pain, and severe bouts of diarrhea or constipation can occur. FAP patients are also at an increased risk of various cancers, the most concerning of which is a nearly 100% occurrence of colon cancer if measures are not taken to prevent the formation of polyps. For many patients, complete colectomy (surgical removal of the entire large intestine), usually performed in the late teenage years or early twenties, is the only viable option for treatment. However, surgical intervention is not curative as the risk of polyps forming in the remaining portions of the intestinal tract and in the small intestine continues after colectomy.
About Marina Biotech, Inc.
Marina Biotech is an oligonucleotide therapeutics company with the broadest drug discovery platform in the sector providing the ability to develop proprietary single and double-stranded nucleic acid therapeutics including siRNAs, microRNA mimics, antagomirs, and antisense compounds, including messengerRNA therapeutics. The platform was built via a roll-up strategy to discover and develop any number of 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 causing a particular disease. We believe that Marina Biotech is the only company in the sector with this breadth of capability, and as a result the platform has unique strengths as a drug discovery engine for the development of nucleic acid-based therapeutics for rare and orphan diseases. Further, we believe Marina 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 two preclinical programs -- in bladder cancer and 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.
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 and supplement forward-looking statements because of subsequent events.
Marina Biotech, Inc.
J. Michael French
President and Chief Executive Officer
(425) 892-4322
admin@marinabio.com
