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news.Dicerna Pharmaceuticals, a leading developer of RNA interference (RNAi) therapeutics, announced a licensing agreement for Dicerna to use Tekmira’s proprietary lipid nanoparticle (LNP) technology for delivery of DCR-PH1, Dicerna’s investigational product candidate for primary hyperoxaluria type 1 (PH1), a rare, inherited liver disorder that often results in kidney failure, and for which there are no approved therapies.
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This announcement follows the successful testing of DCR-PH1 in combination with Tekmira’s LNP technology in animal models, including mice and non-human primates.
Under the agreement, Dicerna will pay Tekmira $2.5 million upfront, as well as $22 million in potential development milestones, and a mid-single-digit royalty on future PH1 sales.
Tekmira’s LNP system has shown in other human clinical studies to provide potent, safe and effective RNA delivery to hepatocytes (liver cells). Licensing Tekmira’s LNP will streamline the development path for DCR-PH1 and allows Dicerna to focus its LNP efforts on its oncology pipeline.
"Dicerna is focused on realizing the full clinical potential of our proprietary pipeline of highly targeted RNAi therapies by applying proven technologies," said Douglas Fambrough, Ph.D., Chief Executive Officer of Dicerna.
"By drawing on Tekmira’s extensive and deep experience with lipid nanoparticle delivery to the liver, the agreement will streamline the development path for DCR-PH1. We look forward to initiating Phase 1 trials of DCR-PH1 in 2015, aiming to fill a high unmet medical need for patients with PH1."
"This new agreement validates our leadership position in RNAi delivery and underscores the significant value we can bring to partners who leverage our LNP technology," said Dr. Mark J. Murray, President and CEO of Tekmira.
"Our LNP technology is enabling the most advanced applications of RNAi therapeutics in the clinic. We are excited to be working with Dicerna in advancing a needed, investigational therapeutic for the treatment of PH1."