NEA, Pfizer build a launch pad for a portfolio of orphan-drug biotechs

David Mott at New Enterprise Associates has spearheaded the development of an orphan drug accelerator that plans to hunt down the best in vivo-stage assets in academia, biopharma and the nonprofits, assemble them in a portfolio of rare disease therapies and mold them into pipeline programs that can be spun out into a slate of new biotech companies.

David Mott, general parter--Courtesy of NEA

NEA's high-profile biotech partner Mott--who helmed MedImmune until AstraZeneca ($AZN) came along and scooped it up for $15.6 billion--partnered with Pfizer Ventures and gained the support of Alexandria Real Estate Equities in piecing together the initial $16 million in startup funds for the newly coined Cambridge, MA-based Cydan. And he recruited Cristina Csimma, a researcher with a Big Pharma background, to head up a team that includes Chris Adams, a seasoned business development executive with stints at TKT and FoldRx; James McArthur, the new CSO who held positions at Synovex, Phylogix and Cell Genesys; and Deborah Geraghty, whose resume includes Aileron Therapeutics and Infinity Pharmaceuticals.

"It's useful having a corporate looking over our shoulders," Mott tells FierceBiotech about Pfizer's participation in the deal. A Big Pharma like Pfizer has insights on "markets and modalities" that will be instrumental in setting up companies that can be spun out with their financial support. 

Mott says the accelerator was inspired by years of work on various rare disease initiatives, dating back to his CEO days at MedImmune, when the company was researching orphan drugs for pediatric diseases. A stint on the Shire board deepened his understanding of the potential of orphan drugs. He's been closely following the work at Prosensa on Duchenne muscular dystrophy. Then a chat with Eric Topol at Scripps Research Institute about the fast pace of scientific understanding of these diseases helped crystallize his thoughts, assisted by his conversations with JC Gutierrez-Ramos about Pfizer's rare-disease initiative.

Mott came to the conclusion that the right preclinical rare disease drugs could be accelerated at a brisk pace, somewhere between the fast pace of medical devices and the much slower speed of general drug development work. In the hands of an experienced crew, with Csimma taking the lead on drug development, adding a resume that included the kind of marquee Big Pharma drug work that would be respected in the industry, the spinout class of biotechs could launch with a promising future.

Despite the industry's increasingly heated interest in rare diseases, which has already grabbed the attention of a number of Big Pharma companies as well as new and existing biotechs, Mott says there will be "more than enough" projects to review. "The pace of science is driving it."

NEA isn't the first venture group to set up a portfolio operation like this. CMEA, Versant and Atlas have set up their own fledgling greenhouse efforts. And Atlas and Shire signed a pact in late 2011 to partner on investing in early-stage rare disease research. Like his colleagues in VC, Mott is intrigued by the idea of an extremely capital-efficient approach that relies on a small team of professionals to identify assets and then take them through an initial development stage, de-risking the best of the bunch and building new companies around them. Mott is sticking strictly with orphan drugs that promise a relatively straight shot at the market.

"We're funded to look at a number of assets and de-risk them to establish the best for company formation," Csimma tells FierceBiotech, outlining plans to launch 5 new companies with the money now on hand. "We are in an extensive outreach mode right now, connecting with academic centers, foundations, tech transfer offices and potentially companies where there are assets that don't fit in their portfolio."

Csimma added that Cydan will have a global reach, seeking out projects not only in the U.S. and Europe but some other hotbeds of orphan drug research, like Israel and Australia. And the first new company should be ready to launch in 2014.

- here's the press release