19 November 2019 - - US-based pharmaceutical company Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA) and Denmark-based Novo Nordisk A/S have inked an agreement to discover and develop novel therapies for the treatment of liver-related cardio-metabolic diseases using Dicerna's proprietary GalXC RNAi platform technology, the companies said.
The collaboration plans to explore more than 30 liver cell targets and may deliver multiple clinical candidates for disorders including chronic liver disease, non-alcoholic steatohepatitis, type 2 diabetes, obesity, and rare diseases.
Dicerna will conduct and fund discovery and preclinical development to clinical candidate selection for each liver cell target, and Novo Nordisk will be responsible for all further development.
The agreement represents a significant investment by Novo Nordisk to secure access to Dicerna's proprietary GalXC RNAi platform, which complements its existing technology base.
The collaboration provides Novo Nordisk with the capability to inhibit hepatocyte targets involved in disease regulation and has the potential to generate a number of clinical development candidates.
The agreement enables each company to co-develop and co-commercialize product candidates discovered under the collaboration.
Novo Nordisk will lead programs targeting cardio-metabolic disorders and other indications with Dicerna having the option to opt into two programmes during clinical development.
Dicerna retains rights to initiate two new orphan liver disease programs for which Novo Nordisk can opt in.
For all co-development programs, the companies will share in the profit/loss of net sales of products consistent with each company's contribution to co-development costs.
Under the terms of the agreement, Dicerna will receive an upfront payment of 175m, a 50m equity investment in Dicerna at a premium and 25m annually during each of the first three years of the collaboration, contingent on Dicerna delivering RNAi molecules for a defined number of targets.
The deal also includes up to USD 357.5m per target in development, regulatory, and commercialisation milestone payments, plus tiered royalties on product sales ranging from the mid-single-digits to mid-teens.
The transaction is subject to the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 and other customary conditions.
Dicerna's proprietary RNA interference technology platform, called GalXC, aims to advance the development of next-generation RNAi-based therapies designed to silence disease-driving genes in the liver and other tissues.
Liver-targeted GalXC-based compounds enable subcutaneous delivery of RNAi therapies that are designed to bind specifically to receptors on liver cells, leading to internalization and access to the RNAi machinery within the cells.
The GalXC approach seeks to optimise the activity of the RNAi pathway so that it operates in the most specific and potent fashion.
Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas, including both liver and non-liver indications.
Dicerna Pharmaceuticals is a biopharmaceutical company using ribonucleic acid interference to develop medicines that silence genes that cause disease.
The company is applying its proprietary GalXC technology to develop potent, selective, and safe RNAi therapies for treatment of rare diseases, chronic liver diseases, cardiovascular diseases, neurodegenerative diseases, pain, and viral infectious disease.
Dicerna aims to treat disease by addressing the underlying causes of illness with capabilities that extend beyond the liver to address a range of diseases, focusing on target genes where connections between gene and disease are well understood and documented.