Basingstoke, England-based drugmaker Shire has moved to strengthen its human genetics business by forming a research collaboration with Danish biopharmaceutical group Santaris Pharma to develop new therapies for rare diseases.

The partnership plans to utilise Santaris’ proprietary Locked Nucleic Acid (LNA) Drug Platform in the discovery of drug targets for certain rare genetic disorders, from which Shire will then cherry pick its favourites to take under its wing to develop further and commercialise those that successfully complete a clinical programme.

According to the group, LNA technology offers the advantage of speedy validated target to proof of concept turnaround, which not only accelerates the development process but also makes it cheaper, factors that Shire will be hoping can help to sharpen the competitive edge of its HGT unit.

Shire HGT also has a successful platform producing human cell derived enzyme replacement therapies for a wide spectrum of rare human genetic disorders, but as its president Sylvie Grégoire explains: “With this novel platform technology Shire has the potential to move into a wider range of genetic orphan diseases”, and she confirmed that the alliance will focus on the development of “treatments for people with a variety of rare life-altering and life-threatening conditions that to date, could not be effectively addressed by ERT”.

Cash terms
On the financial side of the deal, Santaris stands to receive initial early-stage payments of $6.5 million in return for access to its technology, exclusivity for three pre-defined targets and funding for the initial discovery process over the two-year duration of the deal (which can be extended by a further two on Shire’s say so, as well as a potential further $13.5 million if certain initial studies are completed.

In addition, the Danish group will get funding for all additional discovery activities performed under the contract, payments on Shire’s nomination of up to two additional targets, and development, regulatory and sales related milestone payments and royalties of up to $72 million for each of the potential five drug candidates arising from the alliance.

Santaris is evidently delighted with its latest partnership, which, according to Søren Tulstrup, the firm’s president and chief executive, demonstrates the “broad utility” of its LNA Drug Platform “for developing treatments for an extensive range of diseases, now also including rare genetic disorders”.