Marina is entitled to $14 million in total fees per gene target plus royalties.

Marina Biotech and ProNAi Therapeutics entered into an exclusive license agreement regarding DNAi-based therapeutics. Marina Biotech’s Smarticles® liposomal delivery technology will be used, and ProNAi will have the option to select any number of gene targets. Marina could receive up to $14 million for each target in total up-front fees as well as clinical and commercialization milestone payments.

ProNAi will have full responsibility for the development and commercialization of any products arising under the agreement. Marina is entitled to royalties on sales. “DNAi are short single-strand unmodified oligonucleotides designed to silence genes by interfering with DNA,” explains Charles L. Bisgaier, Ph.D., president and CEO of ProNAi Therapeutics. “The DNAi silencing approach is differentiated from that of RNAi, antisense, or miRNA in that it targets genomic sequences within the noncoding 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,” Dr. Bisgaier continues. “ProNAi is now positioned to advance additional cancer therapies from its preclinical 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.”

Dr. Bisgaier notes that 22 patients have been dosed with lead candidate PNT2258 in a Phase I trial in advanced solid tumors. In this open-label, single-arm, dose-escalation trial, patients receive PNT2258 as an intravenous infusion once daily for five consecutive days of every 21-day cycle. ProNAi plans to report the results of this Phase I study later this year.

PNT2258 is 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,” Dr. Bisgaier notes.

Marina Biotech’s Smarticles technology has also been licensed to Mirna Therapeutics for the delivery of miRNA mimics. Inked last December, the deal could earn Marina Biotech $63 million in total up-front fees as well as clinical and commercialization milestone payments over and above royalties on sales.

Marina Biotech’s own pipeline comprises a clinical program in familial adenomatous polyposis and two preclinical programs in bladder cancer and malignant ascites. The company also has an exclusive agreement with The Debiopharm Group covering the bladder cancer program. “Besides advancements within our own internal research programs, 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,” remarks J. Michael French, president and CEO of Marina Biotech.

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