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Going small for broad potential
June 2013
by Kelsey Kaustinen  |  Email the author


CAMBRIDGE, Mass.Nimbus Discovery LLC, a biotechnology company discovering novel medicines against what it calls "exciting but previously inaccessible disease targets," announced May 8 a co-development agreement with Shire PLC focused on small-molecule treatments for several rare genetic diseases known as lysosomal storage disorders (LSDs).
Through the collaboration, the companies plan to use Nimbus' computational chemistry approach to discover and develop novel, disease-altering therapies for LSDs, many of which remain untreated because of challenges in creating drugs that can effectively reach the disease site.
"The significant potential of this partnership is the development of the first small-molecule agents designed to penetrate inaccessible tissues while offering the convenience of an orally administered pill," the companies say.  
"Our target is one that has never been drugged before and has proved to be quite challenging with regard to traditional approaches," Jonathan Montagu, Nimbus' vice president of business development and operations, tells DDNEWS. "So, this is ideal for us. It's a joint target that bring all the weight of our breakthrough computational approach to bear, while Shire brought us its skills around biological understanding, commercial potential and patents."  
Enzymes often have limitations in terms of treatment potential, he notes, while small molecules can get to places where enzymes are too large to go.  
"And in the case of enzyme replacement approaches," he continues, "you tend to end up with one drug for one disease. In the case of a small-molecule approach like we're pioneering with Shire, one molecule could potentially treat multiple diseases, giving much better market potential."
The Nimbus-Shire collaboration, the companies say, is the result of a joint assessment of a series of important rare disease targets with significant unmet medical need. One target was ultimately chosen to be the research focus, as a sort of "test drive" of Nimbus' methods, and the collaborative relationship could extend beyond it later. Under the terms of the agreement, Nimbus will use its research and development platform to extensively profile molecules against the agreed-upon disease target and will deliver a drug candidate that is ready to enter late preclinical studies.  
Up to the point the collaboration achieves an actual drug candidate, Nimbus will control and conduct all research related to the effort, and then Shire will have an exclusive option to acquire the program. Shire would then be responsible for all clinical development and future commercialization activities. Nimbus would be eligible to receive preclinical, development and commercial-stage milestones.  
As is Nimbus' style, the program will be set up as its own dedicated subsidiary under an LLC umbrella, so that its intellectual property and assets can be more easily acquired, such if Shire takes over the program, Montagu says. Also, as Nimbus notes on its website, this structure "solves the classic drug discovery illiquidity problem where it takes seven to 10 years to get a return on capital via M&A or an IPO. The LLC structure enables shareholders to cycle capital back to investors in a tax-efficient manner on a per-project basis."  
"This collaboration validates our computationally driven, structure-based drug discovery engine and innovative partnering model," said Dr. Rosana Kapeller, chief scientific officer of Nimbus, in the news release about the deal, adding that this is Nimbus' first alliance with Shire, "a company that shares our passion for uncovering breakthroughs in highly sought-after disease targets that have proven inaccessible to traditional industry approaches."
It is also worth noting that this is the first deal announced through Shire's strategic alliance with Atlas Venture, which identifies investments for early-stage venture creation around rare genetic diseases. That multiyear collaboration deal was announced in December 2011. The partnership leverages Shire's capabilities and knowledge in the research and development of rare diseases with Atlas' expertise in the formation and growth of early-stage companies.  
"As a leader in rare diseases, this partnership is another way for Shire to ensure that we expand into new disease areas and continue to apply cutting-edge technologies in this space," said Philip J. Vickers, Shire's senior vice president of research and development, when the collaboration was announced in 2011. "Working with an organization like Atlas provides us with a new source of external expertise that is complementary to our internal capabilities and has a clear focus on Shire's goal of bringing innovative therapies to patients suffering from rare diseases worldwide."
The alliance structure was designed to provide an opportunity early in the venture process to utilize all of Shire's capabilities in rare diseases from research to commercialization, while leveraging the extensive Atlas network and experience in company formation.  
"The partnership with Shire is truly synergistic and leverages our individual strengths to create and fund new startups around high potential medical science early in the R&D cycle," said Dr. Bruce Booth, an Atlas Venture partner.  
Looking to the more recent deal that brought Nimbus into the mix, Vickers says, "Nimbus is groundbreaking in their approach to drug discovery and, in a short period of time, have already assembled an impressive track record in delivering clinical candidates for challenging disease targets. "
Code: E061311



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