rare genetic disorders and genetic diseases

Santaris Pharma A/S has a multi-year worldwide strategic alliance with Shire plc to discover and develop new RNA-based medicines to treat rare genetic disorders. It is estimated that there are between 5,000 and 8,000 known rare diseases affecting 250 million people in the world.

The alliance will use Santaris Pharma A/S’ proprietary Locked Nucleic Acid (LNA) Drug Platform to identify and select drug candidates against certain targets.  Shire will be responsible for the selection of appropriate drug candidates, as well as further development and commercialization of products arising from the alliance.

Under the terms of the agreement Santaris Pharma A/S will receive initial early stage payments of $6.5 million covering technology access, exclusivity for three pre-defined targets and initial discovery funding, and an additional early stage payment of $13.5 million upon successful completion of certain initial studies. The initial term of the research collaboration is two years; Shire has an option to extend for up to two additional years. 

In addition to these initial payments Santaris Pharma A/S will receive funding for all additional discovery activities to be performed under the contract, payments on Shire’s nomination of up to two additional targets, and will be eligible to receive development, regulatory and sales related milestone payments of up to $72 million for each of the potential five drug candidates and customary royalties on the worldwide sales of commercialized products arising from the alliance.