Dicerna Pharmaceuticals will license Tekmira’s lipid nanoparticle (LNP) technology for delivery of DCR-PH1, Dicerna’s investigational drug for the rare liver disorder primary hyperoxaluria type 1 (PH1), Dicerna said today. The deal could generate up to $24.5 million, plus royalties, for Tekmira.
The licensing deal follows the successful testing of DCR-PH1 in combination with Tekmira's LNP technology in animal models, including mice and non-human primates.
LNP was shown to provide potent, safe, and effective RNA delivery to hepatocytes in human clinical studies. By licensing Tekmira’s LNP, Dicerna aims to streamline the development path for DCR-PH1 while focusing its LNP efforts on its oncology pipeline.
DCR-PH1 is designed to target and destroy the messenger RNA (mRNA) produced by HAO1, a gene implicated in the pathogenesis of PH1. HAO1 encodes glycolate oxidase, a protein involved in producing oxalate. Reducing oxalate is intended to prevent complications of PH1.
In preclinical studies, DCR-PH1 was shown to induce potent and long-term inhibition of HAO1 and significantly reduce levels of urinary oxalate, while demonstrating long-term efficacy and tolerability in animal models of PH1.
“We look forward to initiating Phase I trials of DCR-PH1 in 2015, aiming to fill a high unmet medical need for patients with PH1,” Dicerna CEO Douglas Fambrough, Ph.D., said in a statement.
If it succeeds in clinical trials and wins approval, DCR-PH1 would be the first therapy on the market indicated for PH1.
Dicerna agreed to pay Tekmira $2.5 million upfront, as well as $22 million in payments tied to development milestones, and a mid-single-digit royalty on future PH1 sales.
Tekmira’s LNP technology (formerly called stable nucleic acid-lipid particles, or SNALP) encapsulates RNAi triggers with high efficiency in uniform lipid nanoparticles that are effective in delivering therapeutic compounds to disease sites. LNP formulations comprise several lipid components that can be adjusted to suit the specific application.