Shire strikes discovery deal in rare diseases with Bill Gates-backed Nimbus

Shire ($SHPG) has nailed down another deal to fuel its growing rare disease unit. The pharma group has tapped the Cambridge, MA-based startup Nimbus Discovery, which will use its computational approach to find small-molecule drugs for rare genetic diseases known as lysosomal storage disorders.

The deal marks the first announcement of a corporate partnership for Nimbus, which was founded in 2009 by Atlas Venture and the chemical software company Schrödinger, and gained an early seed investment from Microsoft ($MSFT) Chairman Bill Gates. It's also a vote of confidence for the startup's use of computational screening for drug-like compounds against disease targets that have been unreachable with traditional approaches to discovery.

The companies declined to reveal any financial details of the pact, yet Nimbus's Jonathan Montagu tells FierceBiotech that the deal offers "significant" perks, including funding of all discovery research and potential milestones if Shire takes an option to acquire the program. 

Shire seeks small molecules from Nimbus against an undisclosed target associated with lysosomal storage disorders (LSDs), which include 50 or so inherited diseases where missing enzymes cause buildups of certain complex carbohyrdate in the lysosomes of cells and lead to organ damage. Shire's Lexington, MA-based Human Genetic Therapies unit, Sanofi's ($SNY) Genzyme and others provide pricy enzyme-replacement therapies for LSDs, which include rare diseases such as Fabry, Gaucher and Hunter syndrome. Oral small molecules could simplify treatment for patients with the diseases, which often strike them during childhood.

Genzyme, which markets the enzyme therapy Cerezyme for Gaucher, is in late-stage development of a new small-molecule drug for that disease called eliglustat tartrate. If approved, the treatment would grab market share and offer Sanofi/Genzyme an edge in the lucrative business. Yet Nimbus believes that its discovery platform could yield compounds that give Shire something unique.

"The cool thing about this collaboration is that, whereas traditional enzyme-replacement therapy for lysosomal storage disorders really just treats one specific disease," Montagu, Nimbus's vice president of business development and operations, said, "this approach with a small molecule could potentially treat multiple lysosomal storage disorders with the same molecule."

Though Montagu declined to provide details, he said that Nimbus would organize the work in LSDs with the same structure as other discovery efforts at the company. The startup has formed a series a LLCs for specific programs, giving Nimbus and its investors the opportunity to cash out on the acquisitions of multiple LLC units. Under this structure, Nimbus is in preclinical development of a lead candidate for targeting a metabolic enzyme known as acetyl-CoA carboxylase for metabolic diseases and cancers. 

Shire's pact with Nimbus is its latest in a string of deals this year in rare diseases. In March, the Ireland-based drugmaker revealed the acquisition of Uppsala, Sweden-based Premacure, which is in mid-stage development of a protein replacement therapy for a cause of blindness in premature infants called retinopathy of prematurity. And earlier this year the company scooped up venture-backed Lotus Tissue Repair in Cambridge, MA to acquire its replacement therapy for Dystrophic Epidermolysis Bullosa.

In December 2011, Shire formed an alliance with Altas Venture, Nimbus's founding investor, to hunt for investment options in rare diseases.

- here's Nimbus' release

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