Novo Nordisk Becomes Dicerna’s Fifth Big Pharma Partnership

After four pharma partners, now Novo Nordisk has shown faith in Dicerna’s proprietary GalXC™ platform. This collaboration aims to develop novel therapies for the treatment o

RNA therapeutics is a budding field as compared to small molecules or protein biologics. For decades, several optimizations have been carried out to improve the stability, pharmacokinetics, and cellular uptake of RNA drugs. However, getting the drug into the cytosol, past the cell’s lipid bilayer has proved to be a major challenge in the field. Over the years, lipid nanoparticle (LNP) carriers became a predominant strategy for RNA drug delivery but toxicity issues have made drug developers look for alternatives.

At present, Alnylam pharmaceutical’s Onpattro is the only FDA-approved RNAi drug. A big breakthrough came when the chemical conjugation of RNA drugs with trivalent N-acetylgalactosamine (GalNAc) was developed. GalNAc conjugated RNA drugs can be targeted to the liver with better accuracy with the help of Asialoglycoprotein (ASGPR) receptors that are highly expressed on the surface of hepatocytes.

Dicerna’s Most Sought After GalXC™ RNAi Platform

Cambridge-based Dicerna Pharmaceuticals uses its GalXC™ RNAi platform technology to advance next-generation RNAi therapies. GalXC-based therapies utilize the Dicer enzyme which initiates RNA interference mechanism to silence disease-causing genes in the liver. The improved delivery mechanism and cellular uptake of this system have progressed several RNA based therapeutics to late-stage clinical trials including Dicerna’s.

The platform has also helped the company sign lucrative partnerships with various pharmaceutical giants. In 2017, it entered a collaboration with Boehringer Ingelheim to work on liver diseases. Last year it struck two major partnerships; one with Alexion pharmaceuticals for the preclinical development of candidates treating complement-mediated diseases, another with Eli Lilly to develop therapies for pain and neurodegeneration. Last month, it signed the biggest deal with Roche for the development of investigational Hepatitis B therapy, DCR-HBVS.

Deal with Novo Nordisk, the Fifth Big Pharma Partnership for Dicerna

This morning, Dicerna announced its new collaboration with Novo Nordisk to discover and develop novel therapies for liver-related cardio-metabolic diseases using its GalXC™ technology. This collaboration aims to explore more than 30 liver cell targets and develop clinical candidates for a multitude of disorders including chronic liver disease, non-alcoholic steatohepatitis (NASH), type 2 diabetes, obesity, and rare diseases. The news sent Dicerna’s stocks soaring since morning.

According to the agreement, Dicerna will receive an upfront payment of $175 million and $50 million equity investment in Dicerna at a premium. In addition to that, it will receive an annual payment of $25 million each for the first three years on the condition that it delivers RNAi molecules for a defined number of targets. Lastly, it will receive up to $357.5 million per target in milestone payments, plus tiered royalties on product sales ranging from the mid-single-digits to mid-teens.

Douglas M. Fambrough, Ph.D., president, and chief executive officer of Dicerna expressed his excitement over the collaboration. “We are excited to collaborate with Novo Nordisk on this broad research and development effort that extends the reach of our GalXC platform to a wide range of liver cell targets and maximizes our opportunities in serious liver diseases. Our efforts will benefit from Novo Nordisk’s expertise in cardio-metabolic diseases and years of experience developing and commercializing innovative therapies worldwide, which will help us advance novel RNAi treatments for underserved patient populations.”

“Through this important collaboration with Dicerna, we gain access to an innovative technology and deep expertise in RNA interference,” said Marcus Schindler, senior vice president of Global Drug Discovery in Novo Nordisk. “Dicerna is the ideal partner to discover and develop molecules for targets that may yield multiple potential treatments across disease areas such as diabetes, obesity, cardiovascular, and NASH. We will work closely together to unlock the true potential of treating a range of diseases using RNAi therapies, for the benefit of patients.”

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Author

Rajaneesh K. Gopinath