SMARTICLES(R) Formulated PNT2258 Achieved Meaningful Therapeutic Outcomes
Marina Biotech, Inc. (OTCQB: MRNA), a leading nucleic acid-based drug discovery and development company focused on rare diseases, today reported that its licensee, ProNAi Therapeutics, reported that a number of patients with relapsed or refractory Non-Hodgkin's Lymphoma treated with its first DNAi®-based therapeutic, PNT2258, achieved meaningful therapeutic outcomes and continue to exhibit durable clinical responses. PNT2258 is one of two nucleic acid-based therapeutics in clinical development which are formulated with Marina Biotech's proprietary SMARTICLES® delivery technology.
The interim results from the ongoing pilot Phase II trial of PNT2258, a DNAi-based therapeutic targeting BCL2, were reported in a poster entitled: "The BCL2 Targeted Deoxyribonucleic Acid Inhibitor (DNAi) PNT2258 Is Active in Patients with Relapsed or Refractory Non-Hodgkin's Lymphoma" at the 56th Annual Meeting of the American Society of Hematology (ASH), being held in San Francisco. The investigators for the study concluded that:
"ProNAi Therapeutics' progress in developing this novel BCL2 DNAi therapeutic is exiting," stated J. Michael French, president and Chief Executive Officer of Marina Biotech. "We look forward to the continued success of the ProNAi team and the advancement of PNT2258 in the treatment of relapsed or refractory Non-Hodgkin's Lymphoma. I believe the continued emergence of SMARTICLES as the most versatile delivery technology in the nucleic acid therapeutics sector will lead to additional licensing opportunities going forward."
About PNT2258 and DNAi PNT2258 is the first clinical stage DNAi-based therapeutic, and is designed to exquisitely target the regulatory region of the BCL2 gene, modulating transcription and effecting BCL2 function. PNT2258 is a SMARTICLES formulation of PNT100, a 24-base phosphodiester DNA sequence. After intravenous injection, PNT2258 achieves systemic distribution, where PNT100 enters into nuclei to target the non-coding, non-transcribed regulatory region of the BCL2 gene upstream of the promoter start site resulting in an anti-proliferative and apoptotic effect.
ProNAi's proprietary DNAi® (DNA interference) therapeutic platform delivers rationally-designed DNA oligonucleotides into cells in order to target sequences residing within 5'-non-coding regions of the genome. These regulatory regions are susceptible to DNAi hybridization during transcription, cell cycling and gene up-regulation. The specific hybridization of the DNAi oligonucleotides with the targeted complementary strand of genomic DNA modulates transcription, affecting the function of the target gene.
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.