12:00 AM
May 20, 2013
 |  BioCentury  |  Strategy

Let a thousand ideas bloom

How Shire is breaking the mold to fuel external discovery in rare diseases

Shire plc has long been a growth through acquisition company, but the pace of its external discovery is picking up steam and the company now has over 20 ongoing projects to show for it.

Traditional research collaborations are target- or MOA-driven, with the pharma or biotech supporting academic institutions that have a program focused on a particular disease-related gene or a novel disease pathway.

But Shire's research collaborations are not restricted to a given pathway, target or disease. Instead, the specialty biopharma is leaving those decisions to its academic partners, offering a set amount of funding and allowing the researchers to propose a range of programs that address rare diseases.

Like many pharmas and large biotechs that have venture groups, Shire also invests as part of a syndicate of VCs in early stage companies with a target or platform that may address an area of interest for the company.

But Shire also has tweaked this model. In October 2011, it partnered with Atlas Ventures to co-invest in newcos where Shire has the exclusive option to certain programs.

The first deal under the alliance was announced on May 8 - an option deal with Atlas portfolio company Nimbus Discovery LLC - and more are in the offing, Philip Vickers, SVP of R&D, told BioCentury.

While none of Shire's external R&D programs has transitioned to the company's pipeline, Vickers said some could within three years.

And despite recent changes announced by new CEO Flemming Ornskov to focus more on internal R&D, Shire expects its external efforts in rare disease to continue at a swift pace (see "R&D Rewind," A8).

Foundation model

Shire has at least 12 early stage partnerships or collaborations. While some are typical platform deals, the company has joined a growing list of other big companies that are trying less traditional formats (see "Shire's Rare Deals," A6).

For example, Shire's research collaboration model operates more like a foundation, where the company offers a given amount of money to experts in the field, along with broad search criteria, and then evaluates the proposals.

"We ask what are the patient needs, and then try to think creatively about how we can access world-class science and align our interests with those parties doing the work," Vickers said.

In the past year, Shire has formed two different partnerships that illustrate the approach.

In October, Shire announced a collaboration with Fondazione Telethon's Telethon Institute of Genetics and Medicine (TIGEM). Shire will provide $22 million over five years to fund research on 13 undisclosed rare disease indications. Telethon will retain rights to IP developed under the deal, and Shire will have an option to license the programs.

TIGEM has three research programs - cell biology of genetic diseases, systems biology and functional genomics, and...

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