19 December 2019 - - US-based drugmaker Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) has exercised its option for exclusive rights to two additional targets within the complement pathway for the discovery and development of GalXC RNAi molecules, US-based Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA) said.

This expands the companies' existing research collaboration and license agreement to now encompass four targets within the complement pathway.

In connection with the option exercise, Alexion will pay Dicerna a total of USD 20m, or USD 10m in option exercise fees per additional new target.

The collaboration between Alexion and Dicerna, which was initiated in October 2018, is focused on the discovery and development of subcutaneously delivered GalXC RNAi molecules directed to a total of four complement pathway targets for the treatment of complement-mediated diseases.

Dicerna is leading the joint discovery and research efforts through the preclinical stage, and Alexion will lead development efforts beginning with Phase 1 studies.

The agreement provides Alexion with exclusive worldwide licenses and commercial rights to the GalXC RNAi molecules developed in the collaboration in exchange for development- and approval-related milestones, sales milestones and mid-single to low-double-digit royalties on future product sales.

The complement system is a part of the immune system that enhances, or complements, the ability of antibodies to clear small organisms or damaged cells from an organism, promote inflammation and attack the pathogen's cell membrane.

While the complement system is essential to maintaining health, it can also become over-activated, contributing to the development of numerous devastating diseases.

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.

Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases through the discovery, development and commercialization of life-changing therapies.

As a leader in complement biology and inhibition for more than 20 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor antibody-positive generalised myasthenia gravis and neuromyelitis optica spectrum disorder (NMOSD).

Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia and lysosomal acid lipase deficiency (LAL-D).

In addition, the company is developing several mid-to-late-stage therapies, including a second complement inhibitor, a copper-binding agent for Wilson disease and an anti-neonatal Fc receptor antibody for rare Immunoglobulin G -mediated diseases as well as several early-stage therapies, including one for light chain amyloidosis and a second anti-FcRn therapy.

Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology.

Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries.

Dicerna Pharmaceuticals, Inc., 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 broad range of diseases, focusing on target genes where connections between gene and disease are well understood and documented.

Dicerna intends to discover, develop and commercialize novel therapies either on its own or in collaboration with pharmaceutical partners. Dicerna has strategic collaborations with Novo Nordisk A/S (NYSE: NVO), Roche (OTC: RHHBY), Eli Lilly and Company (NYSE: LLY), Alexion Pharmaceuticals, Inc. and Boehringer Ingelheim International GmbH.