Pfizer, NEA orphan drug project launches its first biotech on PhII/III threshold

Less than two years after New Enterprise Associates and Pfizer Ventures got together to launch Cydan, an incubator for new orphan disease drug developers, the group is spawning its first new biotech with a $25 million round and a program for Niemann-Pick disease type C ready to go straight into a Phase II/III study.

The venture backing provides enough money to get the pivotal data needed to know whether or not they have a product, says Chris Adams, who runs Cydan out of Cambridge, MA, and is on the board of the newly created Vtesse. The same syndicate that set up Cydan--NEA, Pfizer ($PFE), Lundbeckfond Ventures, Bay City Capital and Alexandria Venture Investments--is also backing the startup, he adds, which is being run by the experienced drug developer Ben Machielse and his small but knowledgeable team.

It's a virtual operation, notes Machielse, but there's also a wide group of investigators at the NIH and elsewhere who have pitched in to get VTS-270--a formulation of 2-hydroxypropyl-beta-cyclodextrin--to the threshold of a pivotal study.

Ben Machielse

"I actually got approached by Dave (Mott, NEA partner and former MedImmune CEO) in May to actually see if I could help out with this particular opportunity," says Machielse, a MedImmune veteran and former CEO of Omthera, which was acquired by AstraZeneca ($AZN). Vtesse licensed in the program but will continue to work with public investigators to take it the final step in the clinic.

"This public/private model is pretty cool," says Machielse, adding that this particular biotech business model should be something that can be replicated in other developers. Machielse is keeping the biotech close to home--and the NIH--in Gaithersburg, MD.

Their lead drug, VTS-270, is designed to clear away the cholesterol that builds up inside the cells of Niemann-Pick patients. But there are also plans to add to the pipeline. Vtesse is starting up with a Cooperative Research and Development Agreement, or CRADA, with the Eunice Kennedy Shriver National Institute of Child Health and Human Development and the National Center for Advancing Translational Sciences at NIH. Vtesse and NCATS forged a licensing agreement for the current rights held by NIH for the worldwide use of cyclodextrin, delta-tocopherol, and derivatives of tocopherol for lysosomal storage diseases, including NPC.

It wouldn't be unusual for a venture-backed company like this to be flipped in a buyout deal once the key study data are in. Machielse, though, believes that positive data will provide some good choices.

"At the end we have optionality," says the CEO. "The syndicate is well funded and we can take it to a commercial product and also look potentially for partners."

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