Santaris Pharma to discover and develop new RNA-based medicines for treating rare genetic disorders

Santaris Pharma A/S and Shire plc to Engage in a Multi-Year Research Program Utilizing Santaris Pharma's Proprietary Locked Nucleic Acid (LNA) Drug Platform to Identify and Select Drug Candidates Against Certain Targets for the Treatment of Rare Genetic Disorders

Santaris Pharma A/S to Receive Significant Upfront Payments, Milestone Payments and Royalties for Providing Access to its LNA Technology, Exclusivity for Three Pre-Defined Targets and Funding for Discovery

Partnership for the Treatment of Rare Genetic Disorders Further Demonstrates Versatility of the LNA Drug Platform to Develop RNA-Based Medicines for a Range of Disease Areas Including Metabolic Disorders, Cancer, Infectious and Inflammatory Diseases

Santaris Pharma A/S, a privately-held biopharmaceutical company focused on developing RNA-based drugs targeted to disease-related mRNAs and microRNAs, today announced a multi-year worldwide strategic alliance with Shire plc to discover and develop new RNA-based medicines to treat rare genetic disorders.

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 US$6.5 million covering technology access, exclusivity for three pre-defined targets and initial discovery funding, and an additional early stage payment of US$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.

"We are delighted that the increasing interest in the LNA Drug Platform and our Drug Discovery Engine so quickly has materialized in our fourth strategic alliance. The alliance with Shire demonstrates the broad utility of Santaris Pharma's proprietary LNA Drug Platform for developing treatments for an extensive range of diseases, now also including rare genetic disorders," said S0ren Tulstrup, President and CEO of Santaris Pharma A/S. "This fourth partnership follows only seven months after the announcement of our new strategic alliance with Wyeth Pharmaceuticals and further demonstrates our leading position in the rapidly evolving RNA-based therapeutics field."

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 US$72 million for each of the potential five drug candidates and customary royalties on the worldwide sales of commercialized products arising from the alliance.

"We are excited to be collaborating with Santaris Pharma A/S, a leader in the field of RNA-based therapeutics. Its LNA technology and experienced management team are a good fit for Shire," said Dr. Whaijen Soo, Senior Vice President, Research and Development, Shire Human Genetic Therapies. "We look forward to working closely with the Santaris Pharma A/S team and expanding our pipeline by discovering and developing new RNA-based medicines to treat rare genetic disorders."

Santaris Pharma A/S' worldwide strategic alliance with Shire to discover and develop new medicines targeting rare genetic disorders adds to the growing list of mRNA and microRNA drug discovery and development partnerships that include Wyeth (delivery of lead candidates against up to ten mRNA and microRNA targets), GlaxoSmithKline (four viral disease drug candidates) and Enzon Pharmaceuticals (eight cancer targets successfully delivered).

About Locked Nucleic Acid (LNA) Drug Platform

The Locked Nucleic Acid (LNA) Drug Platform developed by Santaris Pharma A/S creates synthetic chemical versions called LNAs of the normal nucleic acid building blocks of ribonucleic acids (RNA). These LNAs improve the drug-like qualities of resulting therapeutics, called oligonucleotides, by boosting resistance to metabolism, increasing half-life and improving tissue uptake. LNA-based therapeutics demonstrate improved binding affinity to their target RNA, which increases potency many-fold over other nucleotide therapeutics. The greater potency of LNA in binding complementary RNA sequences also allows for the use of significantly shorter LNA oligonucleotide drugs which can be more effective than previous antisense or siRNA drugs.