Nimbus Discovery, Shire to Co-Develop Oral Rare Disease Drugs

5/8/13Follow @benthefidler

Nimbus Discovery splashed onto the scene in 2011 with a big-name investor group that included Bill Gates and a plan to discover drugs with the help of computer software. On Tuesday, that idea turned into a potentially big partnership with Shire (NASDAQ: SHPG) to help conquer currently untouchable rare diseases.

Neither Shire nor Cambridge, MA-based Nimbus divulged the numbers surrounding the partnership, but the gist is this: Shire will tap into Nimbus’ unique approach to co-develop treatments for rare genetic diseases known as lysosomal storage disorders. This is the same category of rare diseases that companies like Sanofi’s Genzyme unit, and BioMarin Pharmaceuticals (NASDAQ: BMRN), have had big success in treating.

After about nine months of discussions analyzing potential disease targets, the two have identified one to pursue together at first. Though Nimbus declined to disclose the target, its vice president of business operations, Jonathan Montagu, says that there is no treatment for it currently. Nimbus will use its computer-based drug discovery programs to quickly and efficiently look at a vast number of molecules that could attack the disease. Once Nimbus identifies a drug, Shire will take the reins, run it through clinical trials, and eventually sell the product if it can win regulatory approval. Nimbus stands to collect milestone payments along the way for hitting preclinical, development, and commercial goals.

After Shire test-drives the process, there is the chance that the partnership can expand into more rare disease drugs.

Tackling rare diseases, and in particular lysosomal storage disorders—inherited diseases such as Fabry’s Disease, Gaucher Disease and Hunter Syndrome that occur due to malfunctioning lysosomes in the cells—is big business. Genzyme became a multi-billion dollar beast that way, leading to its $20 billion acquisition by Sanofi in 2011. BioMarin has seen its market value skyrocket to more than $9 billion based on its ability to target similar disorders. And Shire has gone to great lengths to establish such a presence in its own right. It fashioned its rare disease-focused Human Genetic Therapies arm when it bought Cambridge-based Transkaryotic Therapies in 2005 and has built it up through acquisitions and a partnership with Atlas Venture in 2011.

But Shire hopes to create an edge here by using Nimbus’ help to create small molecule, oral drugs for lysosomal storage disorders. These would have a leg up over the traditional approach, enzyme replacement therapy, which is done through an intravenous infusion.

“[Enzyme replacement therapies] work very well, but they can only treat one specific disease area,” Montagu says. “The cool thing about small molecules that is you can have one molecule that can literally treat three or four disease areas. So you can have a very valuable product coming out of this.”

Nimbus, co-founded by Atlas and Schrodinger in 2009, believes it can do this through its unorthodox approach to discovering drugs. The company essentially performs a more efficient, virtual form of “high throughput screening,” a common industry practice to test millions of known compounds, on a computer with Schrodinger software that has been boosted and tailored to the company’s needs, as well as with the help of a Schrodinger team of computational chemists that aid in the effort. Montagu says this allows Nimbus to not only be more efficient in discovering drug targets, but also have the chance to succeed “where traditional chemistry has failed.”

“Most people are kind of doing wet chemistry when it comes to designing molecules and there are real limitations when it comes to the amount of chemical space you can explore,” Montagu says.

He adds that this directly applies to the Shire partnership, where the two are attacking a disease where the underlying biological problem is understood, but no one has been able to create an effective drug to address it.

The Shire deal represents Nimbus’ biggest step forward since inception, but it’s not the only thing the startup has on its plate. It has two drugs targeting cancer and metabolic diseases in its pipeline, at the preclinical stage of development. Nimbus has structured itself as a limited liability company, or basically a holding company, to make it easy to sell off individual drug candidates to buyers who may be interested in one drug, but not the whole company. That structure also enables Nimbus immediately pay off shareholders if, for example, it hits a milestone in the Shire deal.

Nimbus plans to find partners for its programs when it gets to the clinical proof-of-concept stage, giving it several different methods to pay off the $24.5 million it raised in its Series A in June 2011. The Shire partnership is a good start.

“For us, it’s validating on the science side, but also, it’s the first deal that we’ve done under an LLC structure,” he says. “We’re very happy with how the value is going to accrue to investors.”

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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