Agreements

Date: 2017-07-27

Type of information: Termination of an agreement

Compound: lipid nanoparticle (LNP) technology

Company: Arbutus Biopharma (Canada) Alexion Pharmaceuticals (USA - CT)

Therapeutic area: Infectious diseases

Type agreement: licensing

Action mechanism:

RNAi. Lipid Nanoparticle (LNP) technology is a RNAi delivery technology. It allows RNAi drugs to be encapsulated in tiny particles made of lipids. These tiny particles – LNP – travel through the bloodstream to target tissues. They are designed to stay in the circulation long enough to accumulate at disease sites, such as the liver or cancerous tumors. Through a process called endocytosis, cells take up the LNPs which allows them to migrate into the cell. The LNPs then undergo an interaction within the cell and the RNAi trigger molecules are released, mediating RNAi.

Disease: chronic hepatitis B

Details:

• On March 16, 2017, Arbutus Biopharma announced it has licensed to Alexion Pharmaceuticals its proprietary lipid nanoparticle (LNP) technology for exclusive use in one of Alexion's rare disease programs. This transaction enables Alexion to address delivery for therapeutic application of messenger RNA and rapidly enter clinical development with its mRNA product candidate. In addition, Arbutus will conduct technology development and provide manufacturing and regulatory support for the rapid advancement of Alexion's mRNA product candidate.

Financial terms:

Under the terms of the license agreement, Alexion will pay Arbutus $7.5 million upfront, and payments of up to $75 million for achievement of development, regulatory, and commercial milestones, as well as single digit royalties.

Latest news:

• On July 27, 2017, Alexion Pharmaceuticals announced that the company is redefining its R&D strategy to create greater efficiency and focus on its expertise in complement biology and core therapeutic areas of hematology, nephrology, neurology, and metabolic disorders. To optimize and align R&D investments and development efforts with the Company's redefined strategy, Alexion is de-prioritizing the ALXN1101 (cPMP replacement therapy) and ALXN6000 (samalizumab) clinical development programs and will seek to out-license these assets. Alexion is also discontinuing its preclinical programs with mRNA therapies as well as other preclinical programs that are outside of the complement franchise, and is therefore terminating its partnerships with Moderna Therapeutics, Blueprint Medicines and Arbutus Biopharma.