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Nimbus, Shire Partner on Rare Genetic Disease Drug Discovery

DNA strand (NSF)

(James. J. Caras, National Science Foundation)

Nimbus Discovery, a biotechnology company in Cambridge, Massachusetts will discover and develop new small-molecule drug candidates to treat a set of rare genetic disorders, for the specialty pharmaceutical company Shire plc based in Ireland. Under the deal, Shire will provide Nimbus with periodic milestone payments, and while financial details of the agreement were not disclosed, at least some of the deal is financed through a special venture capital investment.

Nimbus Discovery uses high-throughput computational techniques for simulating chemical and biological processes to screen drug candidates, particularly for small molecule — low molecular weight — therapies for disorders that are difficult to treat.

The agreement with Shire covers candidates to treat lysosomal storage diseases, a set of some 50 rare inherited disorders, mainly affecting children, where the body lacks the enzymes needed to digest and eliminate macromolecules that accumulate and can result in dysfunctions in eyes, connective tissues, and central nervous system.

Under the agreement Nimbus will use its computational research platform to screen potential molecules against lysosomal storage disease targets and provide Shire with a therapy candidate ready for late preclinical testing. Shire, at that point, will have an exclusive option to acquire the program, and then be responsible for subsequent clinical development and commercialization.

Nimbus will be eligible to receive milestone payments at unspecified preclinical, clinical, and commercialization stages. According to the companies, this is the first deal structured under an arrangement between Shire and venture capital company Atlas Ventures, also in Cambridge, to underwrite early stage research for therapies to address rare genetic disorders.

The financing arrangement between Shire and Atlas Ventures, announced in December 2011, aims to combine Shire’s experience in drug development with Atlas’s expertise in company development to generate therapies for rare diseases. Nimbus Discovery was founded in 2009 as a joint venture of Atlas Ventures and the scientific software company Schrödinger Inc.

The company’s seed-stage investors include Microsoft founder Bill Gates. Atlas Ventures also took part in Nimbus’s first venture round after seed stage.

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