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Thursday, March 9, 2017

Parkinson’s nonprofit founds virtual single-asset biotech

March 9, 2017

Nonprofit Parkinson’s UK has teamed up with the University of Sheffield to create a virtual biotech. The single-asset startup will work with service providers to advance an Nrf2 inhibitor toward the clinic.

Parkinson’s UK joined with the university to found the biotech Keapstone Therapeutics and commit £1 million ($1.2 million) to its activities to ensure research it sees as promising makes the leap from academia to commercial drug development.

Keapstone is built on the research of Richard Mead, Ph.D., a scientist at the Sheffield Institute for Translational Neuroscience who identified molecules with the potential to enter the central nervous system and act on the Nrf2-ARE pathway. That work led to orphan drug status for a Parkinson’s disease drug, apomorphine-S, in acute lateral sclerosis. And Parkinson’s UK now sees the potential for Mead’s work on oxidative stress to translate into advances in its field of interest.

“Activating Nrf2 signalling has the potential to be a much more pharmacologically efficient way to fight oxidative stress than simply flooding the body with antioxidant compounds, an approach which has been tried in the past with limited success. A small molecule which gets into the brain and switches on natural cellular antioxidant defenses could break the vicious circle that we believe underlies neurodegeneration in Parkinson’s,” Arthur Roach, Ph.D., director of research at Parkinson’s UK, said.

Parkinson’s UK and the University of Sheffield have put together a joint steering committee to run Keapstone. Drug discovery service provider Sygnature will handle the early heavy lifting and, if all goes to plan, help turn Keapstone into a clinical-phase biotech.

"Today this is essentially a hit-to-lead stage project, that we hope will progress to lead optimization and clinical candidate selection in the next few years. The ultimate goal is a compelling package of data and IP that will justify investment in clinical testing,” said Roach, a former senior director of neurodegenerative disease research at Serono.

Keapstone is still some way from realizing that goal. But, with the first tranche of funding covering the next 16 months, it is equipped to start finding out whether research-stage promise translates into a viable candidate.

Beyond that, Keapstone is open to looking to more traditional sources of investment to keep the project moving forward. Roach listed VCs and pharma companies as potential future backers of the company, but Parkinson’s UK is also willing to keep investing in Keapstone as it gets deeper into development.

“Parkinson’s UK is committed to ensuring that so long as the scientific results justify further investment, Keapstone will have the resources it needs to create new drug candidates for Parkinson’s. We plan to be investing several million pounds per year in projects such as this over the coming decade, and second and third round funding of Keapstone might be part of that,” Roach said.

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