Marina Biotech Inc., a leading nucleic acid-based drug discovery and development company, and ProNAi Therapeutics Inc., a privately-held biotechnology company pioneering DNA interference (DNAi) therapies for cancer, have entered into an exclusive license agreement regarding the development and commercialization of DNAi-based therapeutics utilizing Marina Biotech's novel Smarticles liposomal delivery technology.

ProNAi will have full responsibility for the development and commercialization of any products arising under the agreement.

Under terms of the agreement, Marina Biotech could receive up to $14 million for each gene target in total upfront, clinical and commercialization milestone payments, as well as royalties on sales, with ProNAi having the option to select any number of additional gene targets. For example, if ProNAi licenses five products over time under this agreement, Marina Biotech could receive up to $70 million in total milestones, plus royalties. Further terms of the agreement were not disclosed.

“We are pleased that twenty-two patients have been dosed with PNT2258 in our phase I clinical trial in advanced solid tumor patients to evaluate safety and tolerability, maximum tolerated dose, pharmacokinetics and pharmacodynamics. PNT2258 is our first DNAi oligonucleotide targeted against the anti-apoptotic bcl-2 oncogene and encapsulated in Marina's Smarticles technology. This novel delivery technology offers protection for the DNAi oligonucleotide during systemic administration with good circulation times and extrahepatic tumor exposure. DNAi are short single-strand unmodified oligonucleotides designed to silence genes by interfering with DNA. The DNAi silencing approach is differentiated from that of RNAi, anti-sense or miRNA in that it targets genomic sequences within the non-coding region of DNA disrupting transcription. The progress and delivery validation in the clinic this past year on the novel DNAi-SMARTICLES formulation gives us confidence to bring forward more first-in-class drug candidates alone or with partners. ProNAi is now positioned to advance additional cancer therapies from its pre-clinical leads targeting other oncogenes such as c-myc and k-ras while also exploring other disease targets in areas such as inflammation and genetics diseases,” said Charles L Bisgaier, PhD, president and CEO of ProNAi Therapeutics.

“We are extremely pleased to have entered into a relationship with a company like ProNAi that is developing a first-in-class nucleic acid therapeutic,” stated J Michael French, president and CEO of Marina Biotech. “In addition, we are excited to see the continued advancement of oligonucleotide-based therapeutics using our Smarticles technology. Besides advancements within our own internal research programmes, we have now been able to establish two license agreements broadening the application of the Smarticles technology to the systemic administration of both single- and double-stranded oligonucleotide therapeutics. We look forward to the rapid advancement of ProNAi Therapeutics' clinical pipeline and the opportunity to bring novel therapeutics to patients in need.”

ProNAi is conducting an open-label, single arm, phase I dose-escalation study of PNT2258 in patients with advanced solid tumours for which no standard therapy exists at START in San Antonio, Texas. PNT2258 is ProNAi's first drug candidate from the DNAi drug platform. Patients receive PNT2258 as an intravenous infusion once daily for 5 consecutive days (Days 1-5) of every 21-day cycle (3 weeks). ProNAi plans to report the results of this phase I study at oncology conferences later this year and initiate the next phase I/II safety and efficacy studies in select cancer patients based upon the safety and dose findings from this phase I study.

Marina Biotech is focused on the development and commercialization of oligonucleotide-based therapeutics utilizing multiple mechanisms of action including RNA interference (RNAi) and messenger RNA translational blocking.